CHMP Grants Positive Opinion for Expanded Use of Darzalex (daratumumab) in Combination with Bortezomib, Thalidomide and Dexamethasone (VTd) for…

The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended broadening the existing marketing authorisation for Darzalex (daratumumab) to include the use of daratumumab in combination with bortezomib, thalidomide and dexamethasone (VTd) for the treatment of adult patients with newly diagnosed patients with multiple myeloma who are eligible for autologous stem cell transplant (ASCT).

The Positive Opinion is supported by data from Part 1 of the Phase 3 CASSIOPEIA (MMY3006) study, published in The Lancet3 in June 2019, and presented at the 2019 American Society of Clinical Oncology (ASCO) Meeting. Additional information about this study can be found at http://www.ClinicalTrials.gov (NCT02541383).

"Todays Opinion takes us a step closer to offering the first daratumumab combination regimen to transplant eligible patients, redefining treatment for those people newly diagnosed with multiple myeloma," said Dr Patrick Laroche, Haematology Therapy Area Lead, Europe, Middle East and Africa (EMEA), Janssen-Cilag. "We are committed to delivering advances in multiple myeloma care, including providing innovative treatment options that meet the evolving needs of people living with this disease."

Craig Tendler, M.D., Vice President, Clinical Development and Global Medical Affairs, Oncology at Janssen Research & Development, LLC., commented: "Our robust clinical development programme continues to demonstrate that daratumumab provides a foundation for the treatment of patients with multiple myeloma across the treatment continuum."

The CHMPs Positive Opinion comes after the US Food and Drug Administrations approval in September 2019. It will now be reviewed by the European Commission, which has the authority to grant marketing authorisation for medicines in the European Economic Area.

#ENDS#

In Europe, daratumumab is indicated:4

About the CASSIOPEIA Trial5

The randomised, open-label, multicentre, Phase 3 study is sponsored by the French Intergroupe Francophone du Myelome in collaboration with the Dutch-Belgian Cooperative Trial Group for Hematology Oncology and Janssen Research & Development, LLC. The study included 1,085 newly diagnosed patients with previously untreated, symptomatic multiple myeloma who were eligible for high-dose chemotherapy and stem cell transplant. In the first part of the study, patients were randomised to receive induction treatment with VTd alone or in combination with daratumumab, high-dose therapy and ASCT, and consolidation therapy with VTd alone or in combination with daratumumab. The primary endpoint in this part of the study is the proportion of patients who achieve an sCR 100 days after transplant. In the second part of the study, which is ongoing, patients who achieved a partial response or better in part one will undergo a second randomisation to receive maintenance treatment with daratumumab 16 mg/kg every eight weeks for up to two years or will be observed with no further treatment. The primary endpoint in this part of the study is progression-free survival (PFS).

About daratumumab

Daratumumab is a first-in-class6 biologic targeting CD38, a surface protein that is highly expressed across multiple myeloma cells, regardless of disease stage.7 Daratumumab is believed to induce tumour cell death through multiple immune-mediated mechanisms of action, including complement-dependent cytotoxicity (CDC), antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), as well as through apoptosis, in which a series of molecular steps in a cell lead to its death.4 A subset of myeloid derived suppressor cells (CD38+ MDSCs), CD38+ regulatory T cells (Tregs) and CD38+ B cells (Bregs) were decreased by daratumumab.4 Since launch, it is estimated that 100,000 patients have been treated with daratumumab worldwide.2 Daratumumab is being evaluated in a comprehensive clinical development programme across a range of treatment settings in multiple myeloma, such as in frontline and relapsed settings.5,8,9,10,11,12,13,14 Additional studies are ongoing or planned to assess its potential in other malignant and pre-malignant haematologic diseases in which CD38 is expressed, such as smouldering myeloma.15,16 For more information, please see https://www.clinicaltrials.gov/.

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For further information on daratumumab, please see the Summary of Product Characteristics at https://www.ema.europa.eu/en/medicines/human/EPAR/darzalex.

In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive licence to develop, manufacture and commercialise daratumumab.17

About Multiple Myeloma

Multiple myeloma (MM) is an incurable blood cancer that starts in the bone marrow and is characterised by an excessive proliferation of plasma cells.18 In Europe, more than 48,200 people were diagnosed with MM in 2018, and more than 30,800 patients died.19 Almost 60 percent of patients with MM do not survive more than five years after diagnosis.20

Although treatment may result in remission, unfortunately, patients will most likely relapse as there is currently no cure.21 Refractory MM is when a patients disease progresses within 60 days of their last therapy.22,23 Relapsed cancer is when the disease has returned after a period of initial, partial or complete remission.24 While some patients with MM have no symptoms at all, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.25 Patients who relapse after treatment with standard therapies, including proteasome inhibitors and immunomodulatory agents, have poor prognoses and few treatment options available.26

About the Janssen Pharmaceutical Companies of Johnson & Johnson

At Janssen, were creating a future where disease is a thing of the past. Were the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.

Learn more at http://www.janssen.com/emea. Follow us at http://www.twitter.com/janssenEMEA for our latest news. Janssen-Cilag, Janssen Research & Development, LLC and Janssen Biotech, Inc. are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.

Cautions Concerning Forward-Looking Statements

This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding the benefits of daratumumab for the treatment of patients with multiple myeloma. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialise, actual results could vary materially from the expectations of the Janssen Pharmaceutical Companies and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behaviour and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 30, 2018, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in the companys most recently filed Quarterly Report on Form 10-Q, and the companys subsequent filings with the Securities and Exchange Commission. Copies of these filings are available online at http://www.sec.gov, http://www.jnj.com or on request from Johnson & Johnson. Neither the Janssen Pharmaceutical Companies of Johnson & Johnson nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.

References

1 Johnson & Johnson. European Commission Approves VELCADE As A Frontline Induction Therapy Before Stem Cell Transplantation. Press release August 8, 2013. Available at: https://www.jnj.com/media-center/press-releases/european-commission-approves-velcade-as-a-frontline-induction-therapy-before-stem-cell-transplantation Last accessed December 2019.2 Janssen. Data on file: RF-82203. Daratumumab: New patient starts launch to date. November 20193 Moreau P, Attal M, Hulin C, et al. Bortezomib, thalidomide, and dexamethasone with or without daratumumab before and after autologous stem-cell transplantation for newly diagnosed multiple myeloma (CASSIOPEIA): a randomised, open-label, phase 3 study. Lancet. 2019;394:29-38.4 European Medicines Agency. DARZALEX summary of product characteristics, November 2019. Available at: https://ec.europa.eu/health/documents/community-register/2019/20191119146548/anx_146548_en.pdf Last accessed December 2019.5 ClinicalTrials.gov. A study to evaluate daratumumab in transplant eligible participants with previously untreated multiple myeloma (Cassiopeia). NCT02541383. Available at: https://clinicaltrials.gov/ct2/show/NCT02541383 Last accessed December 2019.6 Sanchez L, Wang Y, Siegel DS, Wang ML. Daratumumab: a first-in-class CD38 monoclonal antibody for the treatment of multiple myeloma. J Hematol Oncol. 2016;9:51.7Fedele G, di Girolamo M, Recine U, et al. CD38 ligation in peripheral blood mononuclear cells of myeloma patients induces release of protumorigenic IL-6 and impaired secretion of IFNgamma cytokines and proliferation. Mediat Inflamm. 2013;2013:564687.8ClinicalTrials.gov. A study comparing daratumumab, lenalidomide, and dexamethasone with lenalidomide and dexamethasone in relapsed or refractory multiple myeloma. NCT02076009. Available at: https://clinicaltrials.gov/ct2/show/NCT02076009 Last accessed December 2019.9 ClinicalTrials.gov. Addition of daratumumab to combination of bortezomib and dexamethasone in participants with relapsed or refractory multiple myeloma. NCT02136134. Available at: https://clinicaltrials.gov/ct2/show/NCT02136134 Last accessed December 2019.10 ClinicalTrials.gov. A study of combination of daratumumab and Velcade (bortezomib) melphalan-prednisone (DVMP) compared to Velcade melphalan-prednisone (VMP) in participants with previously untreated multiple myeloma. NCT02195479. Available at: https://clinicaltrials.gov/ct2/show/NCT02195479 Last accessed December 2019.11ClinicalTrials.gov. Study comparing daratumumab, lenalidomide, and dexamethasone with lenalidomide and dexamethasone in participants with previously untreated multiple myeloma. NCT02252172. Available at: https://clinicaltrials.gov/ct2/show/NCT02252172 Last accessed December 2019.12 ClinicalTrials.gov. A study of Velcade (bortezomib) melphalan-prednisone (VMP) compared to daratumumab in combination with VMP (D-VMP), in participants with previously untreated multiple myeloma who are ineligible for high-dose therapy (Asia Pacific region). NCT03217812. Available at: https://clinicaltrials.gov/ct2/show/NCT03217812 Last accessed December 2019.13ClinicalTrials.gov. Comparison of pomalidomide and dexamethasone with or without daratumumab in subjects with relapsed or refractory multiple myeloma previously treated with lenalidomide and a proteasome inhibitor daratumumab/pomalidomide/dexamethasone vs pomalidomide/dexamethasone (EMN14). NCT03180736. Available at: https://clinicaltrials.gov/ct2/show/NCT03180736 Last accessed December 2019.14ClinicalTrials.gov. Study of carfilzomib, daratumumab and dexamethasone for patients with relapsed and/or refractory multiple myeloma (CANDOR). NCT03158688. Available at: https://clinicaltrials.gov/ct2/show/NCT03158688 Last accessed December 2019.15 ClinicalTrials.gov. A study to evaluate 3 dose schedules of daratumumab in participants with smoldering multiple myeloma. NCT02316106. Available at: https://clinicaltrials.gov/ct2/show/NCT02316106 Last accessed December 2019.16ClinicalTrials.gov. An efficacy and safety proof of concept study of daratumumab in relapsed/refractory mantle cell lymphoma, diffuse large B-cell lymphoma, and follicular lymphoma. NCT02413489. Available at: https://clinicaltrials.gov/ct2/show/NCT02413489 Last accessed December 2019.17 Johnson & Johnson. Janssen Biotech announces global license and development agreement for investigational anti-cancer agent daratumumab. Press release August 30, 2012. Available at: https://www.jnj.com/media-center/press-releases/janssen-biotech-announces-global-license-and-development-agreement-for-investigational-anti-cancer-agent-daratumumab Last accessed December 2019.18 American Society of Clinical Oncology. Multiple myeloma: introduction. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/introduction Last accessed December 2019.19 GLOBOCAN 2018. Cancer Today Population Factsheets: Europe Region. Available at: https://gco.iarc.fr/today/data/factsheets/populations/908-europe-fact-sheets.pdf Last accessed December 2019.20De Angelis R, Minicozzi P, Sant M, et al. Survival variations by country and age for lymphoid and myeloid malignancies in Europe 2000-2007: results of EUROCARE-5 population-based study. Eur J Cancer. 2015;51:2254-68.21 Abdi J, Chen G, Chang H, et al. Drug resistance in multiple myeloma: latest findings and new concepts on molecular mechanisms. Oncotarget. 2013;4:2186207.22 National Cancer Institute. NCI dictionary of cancer terms: refractory. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=350245 Last accessed December 2019.23 Richardson P, Mitsiades C, Schlossman R, et al. The treatment of relapsed and refractory multiple myeloma. Hematology Am Soc Hematol Educ Program. 2007:317-23.24National Cancer Institute. NCI dictionary of cancer terms: relapsed. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=45866 Last accessed December 2019.25American Cancer Society. Multiple myeloma: early detection, diagnosis and staging. Available at: https://www.cancer.org/content/dam/CRC/PDF/Public/8740.00.pdf Last accessed December 2019.26 Kumar SK, Lee JH, Lahuerta JJ, et al. Risk of progression and survival in multiple myeloma relapsing after therapy with IMiDs and bortezomib: a multicenter international myeloma working group study. Leukemia. 2012;26:149-57.

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CHMP Grants Positive Opinion for Expanded Use of Darzalex (daratumumab) in Combination with Bortezomib, Thalidomide and Dexamethasone (VTd) for...

100 hospitals and health systems with great neurosurgery and spine programs | 2019 – Becker’s Hospital Review

Becker's Healthcare named the following organizations to the 2019 edition of its list "100 hospitals and health systems with great neurosurgery and spine programs."

The organizations featured on this year's list have extensive neurosurgery and spine programs, providing treatment and cutting edge research into brain and spine disorders. Many hospitals and health systems featured have earned top honors for medical excellence, outcomes and patient experience in their spine and brain surgery departments.

To develop this list, the Becker's Healthcare editorial team examined national rankings and awards for neurosurgery and spine care. The editorial team examined U.S. News & World Report national rankings for neurology and neurosurgery; CareChex national and regional rankings for neurological care and Blue Distinction Center for Spine Surgery designation to develop this list. Please contact Laura Dyrda at ldyrda@beckershealthcare.com with any questions about this list.

Note: Hospitals cannot pay for inclusion on this list. Organizations are presented in alphabetical order.

Abbott Northwestern Hospital (Minneapolis). At Abbott Northwestern Hospital's Spine Institute, physicians treat more than 4,000 patients annually. The hospital has been designated as a Blue Distinction Center for Spine Surgery by BlueCross BlueShield of Minnesota. With five area partners, Abbott Northwestern Hospital was ranked on U.S. News & World Report's list of 50 best hospitals for neurology and neurosurgery in 2019-20.

AdventHealth Orlando (Fla.). AdventHealth's Neuroscience Institute provides comprehensive care to patients with brain and spinal disorders. The health system's brain tumor team includes 12 physicians, and the spine team features 15 physicians that aim to stay at the forefront of spinal treatment. With an elite team, U.S. News & World Report ranked AdventHealth Orlando among the 50 best hospitals for neurology and neurosurgery for 2019-20.

Ascension Seton (Austin, Texas). Physicians at Ascension Seton's Brain & Spine Institute specialize in minimally invasive and complex surgery for patients suffering from problems with their brain, spine, cerebrovascular system and peripheral nerves. Spine, orthopedic and neurosurgeons at the hospital have been recognized nationally and internationally for pioneering new treatments and research. The Seton Brain & Spine Institute has five locations offering neurosurgery across Texas.

Atrium Health (Charlotte, N.C.). Atrium Health offers one of the region's largest neurosurgery specialty programs. The health system's spine institute has been recognized by Blue Cross Blue Shield as a Blue Distinction Center for its quality care and outcomes. An early adopter of minimally invasive and robotic surgery, Atrium Health's Neurosciences Institute has 20-plus years of groundbreaking investigator-initiated and industry-sponsored clinical trials.

Barnes-Jewish Hospital/Washington University School of Medicine (St. Louis). The Barnes-Jewish & Washington University Spine Center receives patient referrals from all around the world. As a result, it has grown into one of the largest clinical spine practices in the nation. U.S. News & World Report ranked Barnes-Jewish Hospital and Washington University School of Medicine among the top 50 hospitals in the nation for neurology and neurosurgery, and the top hospital in Missouri for those specialties in 2019-20.

Barrow Neurological Institute (Phoenix). Physicians at Barrow Neurological Institute oversee more than 8,000 admissions and perform more than 5,000 neurosurgeries annually more than anywhere else in the U.S. There are 26 neurosurgeons, nine neurosurgery fellows and 28 neurosurgery residents on staff at the hospital. As one of the leading spine organizations in the world, U.S. News & World Report named Barrow Neurological Institute among the top 50 hospitals in the nation for neurology and neurosurgery in 2019-20.

Baptist Medical Center Jacksonville (Fla.). Surgeons at Baptist Medical Center have teamed up with Baptist MD Anderson Cancer Center in Jacksonville to conduct clinical trials related to brain and spine tumors. Along with its extensive brain and spine tumors division, Baptist Medical Center Jacksonville has a robust stroke and cerebrovascular care team and four neurologic oncologists. Neurosurgeons at the hospital's Stroke & Cerebrovascular Center are participating in more than 20 ongoing clinical trials.

Baylor St. Luke's Medical Center (Houston). Baylor St. Luke's Medical Center created its Neurosciences Institute in 2013, and it has since has been accredited by DNV GL Healthcare as a certified comprehensive stroke center. Baylor St. Luke's Medical Center neurosurgeons have also helped the hospital receive the Gold Plus Quality Achievement Award from the American Stroke Association. Baylor St. Luke's Medical Center was recognized in 2019-20 by U.S. News & World Report as one of the best hospitals in the nation for neurology and neurosurgery.

Beaumont Health (Southfield, Mich.). Beaumont Health was the first hospital in Michigan to create a pediatric stereo-EEG epilepsy surgery program to pinpoint seizures and cure drug-resistant epilepsy. The hospital's neurosurgery team is researching stem cell regeneration and spine reconstruction. Additionally, as a leading hospital for orthopedic surgery, neurosurgery and neurology, Beaumont Health has a leading spine team with on-call surgeons who can be at the hospital in 15 minutes no matter the time of day.

Beth Israel Deaconess Medical Center (Boston). All physicians at Beth Israel's Spine Center are board-certified and faculty of Boston-based Harvard Medical School. In total, there are four neurosurgeons and three orthopedic spine surgeons. To make care more accessible, Beth Israel Deaconess Medical Center has opened six spine centers throughout Massachusetts that provide comprehensive care patients in Boston and the surrounding area.

Boston Children's Hospital. The spine division of Boston Children's Hospital collaborates with the hospital's department of neurosurgery to treat complex spine conditions. Boston Children's Hospital has a complex cervical spine program as well as a spine and sports program. As a leading hospital for orthopedics and spine in the U.S., Boston Children's has various study groups, including a spinal deformity group whose Scoliosis Outcomes Database is cited in more than 45 abstracts and 15 peer-reviewed publications annually. In 2019-20, U.S. News & World Report ranked Boston Children's No. 1 in the nation for pediatric neurology and neurosurgery.

Brigham and Women's Hospital (Boston). The Comprehensive Spine Center at Brigham and Women's Faulkner Hospital brings together neurosurgeons, orthopedic surgeons, and specialists in pain management, physical medicine and rehabilitation. Brigham and Women's Hospital has five other spine centers throughout Massachusetts. U.S. News & World Report ranked the hospital among the top 20 in the nation for neurology and neurosurgery in 2019-20.

Carilion Clinic (Roanoke, Va.). The 1,026-bed Carilion Clinic hospital system provided care to nearly 1 million residents of Virginia and West Virginia in 2018. Touting around 13,320 employees and nine hospitals, the health system has 732 physicians across 77 specialties. Surgeons at Carilion's Institute for Orthopaedics and Neurosciences perform approximately 300 minimally invasive spine procedures annually, and it has the region's only deformity correction program for both adult and pediatric patients.

Cedars-Sinai Medical Center (Los Angeles). Serving more than 1 million patients annually at more than 40 locations, Cedars-Sinai Medical Center has more than 4,500 physicians on staff. The Cedars-Sinai spine team has 26 spine surgeons, assisted by eight advanced care providers. The health system is active in research efforts, hosting an array of clinical trials. Cedars-Sinai splits its spine care between four locations, including the Cedars-Sinai Spine Center and the Cedars-Sinai Kerlan-Jobe Institute. U.S. News & World Report ranked Cedars-Sinai as the No. 12 hospital for neurology and neurosurgery for 2019-20.

Cincinnati Children's Hospital Medical Center. Established in 1883, Cincinnati Children's Hospital Medical Center has more than 600 beds and around 1.3 million patient encounters in fiscal year 2017. The hospital's Crawford Spine Center was recognized by U.S. News & World Report as among the best hospitals for pediatric neurology and neurosurgery for 2019-20. The hospital is also involved in spine research, focusing on endoscopic technology, scoliosis correction and guided spine growth.

Cleveland Clinic. Featuring both a robust clinical program and a comprehensive spine research lab, Cleveland Clinic's Center for Spine Health sees thousands of patients annually. The Center for Spine Health has three specialty departments to address lower back pain, spinal deformity and spine tumors. Cleveland Clinic has 14 spine surgeons on staff, 14 medical/interventional staff members and 13 advanced care providers in its Center for Spine Health. The center is also testing robotics and is in the midst of a cervical spondylotic myelopathy surgical trial. U.S. News & World Report ranked Cleveland Clinic No. 10 in the nation for neurology and neurosurgery in 2019-20.

Dartmouth-Hitchcock Medical Center (Lebanon, N.H.). Dartmouth-Hitchcock developed its Center for Pain and Spine to meet the needs of its patient population, which comprises about 1.9 million people across northern New England. Anthem BlueCross BlueShield named the center a Blue Distinction Center for Spine Surgery for its commitment to good patient outcomes. U.S. News & World Report recognized the hospital as the highest performing neurosurgery institute in New Hampshire in 2019-20.

Duke University Hospital (Durham, N.C.). Duke University Hospital's comprehensive spine institute has 106 physicians on hand to provide an array of spine-related treatments. The spine team at Duke performs more than 1,200 spine surgeries annually. The hospital equipped all its surgery centers with intraoperative imaging equipment to ensure all procedures and physicians have access to real-time imaging information. U.S. News & World Report named Duke its No. 1 hospital in North Carolina and among the top 25 hospitals in the nation for neurology and neurosurgery in 2019-20.

Emory University Hospital (Atlanta). With its six locations throughout Atlanta, Emory University Orthopaedics & Spine Hospital offers patients comprehensive treatment options. Emory's neuroscience program will soon be headlined by the Emory Musculoskeletal Institute in Brookhaven, Ga. The institute broke ground in October 2019 and will be a 180,000-square-foot center dedicated to spine care. Emory University Hospital planners incorporated several environmentally conscious features into the institute. U.S. News & World Report ranked Emory among the top 50 hospitals in the nation for neurology and neurosurgery in 2019-20.

Froedtert & the Medical College of Wisconsin (Milwaukee). Froedtert & the Medical College of Wisconsin is the region's only academic regional medical center. Froedtert physicians see patients across the greater Milwaukee suburbs. Froedtert has four locations that specialize in spine care, including the outpatient clinic SpineCare. In 2017, Froedtert physicians treated 8,606 patients through its neurosurgery program.

Geisinger (Danville, Pa.). Founded more than a century ago, Geisinger has provided care to central Pennsylvania residents for generations. At the forefront of its neuroscience program is the Geisinger Neuroscience Institute. Employing a combination of treatment methods including microsurgery, minimally invasive surgery, robotic surgery and image-guided surgery, Geisinger has 24 providers on its neurosurgery staff. The health system is also on the forefront of innovation in healthcare delivery, with its ProvenCare program offering refunds to spine patients dissatisfied with their care. Geisinger is also a Walmart spine center of excellence, meaning the retail giant sends patients from across the country to undergo spinal evaluation and surgery at the health system.

Hackensack (N.J.) University Medical Center. Hackensack University Medical Center offers a robust program for neurosurgery care, including the Orthopedic Institute, which has more than 50 physicians on staff. Healthgrades has recognized Hackensack University Medical Center with its Cranial Neurosurgery Excellence Award for the last four years, and U.S. News & World Report ranked Hackensack as high performing for neurology and neurosurgery in 2019-20.

Henry Ford Hospital (Detroit). With more than 35 physicians specializing in spine and related specialties on staff, Henry Ford draws patients in Detroit and its greater suburban locations to its Henry Ford Spine Centers. In 2017 alone, Henry Ford surgeons performed some 75,000 surgeries across its 200 care sites. U.S. News & World Report ranked Henry Ford among the top 50 hospitals in the nation for neurology and neurosurgery in 2019-20.

Hoag Health Network (Newport Beach, Calif.). Hoag Health Network offers numerous spine programs, including care at Hoag Orthopedic Institute. In 2018, the institute reported 3,246 hospital spine procedures, as well as 180 ambulatory procedures. Hoag has two acute care hospitals, 11 urgent care centers and eight health centers it staffs with a team of more than 1,700 physicians and 6,000 employees. U.S. News & World Report ranked Hoag Hospital among the top 50 hospitals in the nation for neurology and neurosurgery in 2019-20.

Hospital for Special Surgery (New York City). Holding the No. 1 U.S. News & World Report ranking in orthopedics for 10 consecutive years, HSS surgeons perform more than 32,000 procedures annually. In 2016, the hospital reported 469 non-cervical spine fusion cases, which was well above the 48-procedure average for New York state. The hospital also focuses on research, with a 300-plus member research department that has a $45 million grant portfolio and $25 million in industry funding. Current spine-focused projects include studying spine instability, developmental deformity and tissue degeneration.

Houston Methodist Hospital. For nearly 30 years, Houston Methodist Hospital has been on the forefront of spine and neurosurgical care. The hospital is home to the center for neurodegeneration, which is comprised of 11 labs staffed with researchers working on therapies for chronic paralysis and neurologic loss. The hospital has 14 neurosurgeons on staff and was among the top 50 hospitals in the nation for neurology and neurosurgery by U.S. News & World Report for 2019-20.

Huntington Hospital (Pasadena, Calif.). Spine surgeons at Huntington Hospital specialize in treating spinal degenerative diseases such as deformities, lumbar stenosis and traumatic disorders. The hospital is home to a 32-bed orthopedic and neurological nursing unit, a 24-bed rehabilitation unit, an outpatient neurophysiology lab as well as angiography suites. In 2019-20, U.S. News & World Report ranked Huntington Hospital among the top 50 hospitals in the nation for neurology and neurosurgery.

Inova Fairfax Hospital (Falls Church, Va.). Inova Fairfax hospital is home to the largest neurological practice in the Washington, D.C., area. Nine Inova neurosurgeons perform more than 3,000 cases a year. The Inova team was the first in Northern Virginia to conduct MRI-guided surgeries to treat Parkinson's disease and a brain tumor. The Inova Neuroscience and Spine Institute has 12 specialized treatment programs and was awarded The Joint Commission's Gold Seal of Approval for its cervical and lumbar spine surgery program.

IU Health (Indianapolis). IU Health's Neuroscience Center offers patients treatment across a variety of neurological specialties, including oncology, spine surgery, stereotactic and functional neurosurgery, trauma treatment and pediatric neurosurgery. IU Health's team of neurosurgeons are at the forefront of using new technology for improving outcomes. Researchers at IU Health are currently pioneering four clinical trials on epilepsy and hematoma evacuation.

Jefferson Health (Philadelphia). Jefferson Hospital for Neuroscience is the only hospital in the Philadelphia region dedicated to neuroscience, and is one of the busiest academic neurosurgical programs in the U.S. The neuroscience program has five surgeons on staff, and in 2015, was the first in the region to offer deep brain stimulation. In 2019-20, U.S. News & World Report ranked Jefferson Health-Thomas Jefferson University Hospital among the top 50 hospitals in the nation for neurology and neurosurgery. The health system also has a robust spine program, with its spine surgeons completing around 7,000 procedures each year at inpatient and ambulatory locations. It was also the first in the country to enroll a patient in the INSPIRE 2.0 clinical trial examining treatment for spinal cord injury.

Johns Hopkins Medicine (Baltimore). Physicians in the department of neurology and neurosurgery at Johns Hopkins Medicine have been treating patients since 1889, and now perform more than 4,000 operations and 30,000 outpatient consultations each year. The Johns Hopkins Carnegie Center for Surgical Innovation, a collaboration between the departments of neurosurgery and biomedical engineering, is working on new technology to make spine surgery safer through image-guided interventions. Johns Hopkins neurosurgeons are actively researching and conducting clinical trials on Parkinson's disease, dementia and brain cancer. The Johns Hopkins Hospital was ranked No. 1 in the in the nation for neurology and neurosurgery in 2019-20 by U.S. News & World Report.

Kaiser Permanente Los Angeles Medical Center. Kaiser Permanente's neurologists and neurosurgeons perform hundreds of complex procedures each year in one of the nation's busiest neurosurgical centers. The health system's 70-year history gives it a leg up in educating the next generation of specialists through neurology and neurosurgery residency programs, as well as a neuroanesthesia fellowship program. Kaiser's Los Angeles Medical Center houses the health system's comprehensive spine surgery department as well as a radiosurgery program dating back to 1989.

Keck Medicine of USC (Los Angeles). The USC Spine Center aims to deliver a coordinated, conservative approach to spine care at four locations in the greater Los Angeles area. Specialists at USC Spine Center are all fellowship-trained faculty members at the Keck School of Medicine of USC and have collectively gained an overall patient satisfaction rating of over 4.5 out of 5 stars. USC Spine Center, which is part of USC Orthopaedic Surgery and USC Neurological Surgery, is recognized as a Blue Distinction Center for spine surgery by Blue Shield of California. Additionally, Keck Medicine of USC was ranked No. 16 in the nation for neurology and neurosurgery by U.S. News & World Report.

Lehigh Valley Health Network (Allentown, Pa.). Lehigh Valley Health Network's spine and neurology services are provided through its Institute for Surgical Excellence, where surgeons perform over 35,000 surgeries annually 70 percent more than other centers in the area. The system boasts the region's only spine neuronavigation system, as well as a 14-bed neuroscience intensive care unit. With surgeons currently involved in at least five clinical trials focused on neurology, Lehigh Valley Health Network offers patients unique opportunities to undergo new treatments in addition to surgery.

Lifespan (Providence, R.I.). With six experts on its surgical team, Lifespan's Comprehensive Spine Center is affiliated with the Providence-based Warren Alpert Medical School of Brown University, giving patients access to advanced technology and treatments. Operating at both Rhode Island Hospital in Providence and the newly opened Newport (R.I.) Hospital, the Comprehensive Spine Center is housed within the Norman Prince Neurosciences Institute. Lifespan's 13 neurosurgeons perform about 2,000 procedures annually, and they're researching the use of microelectrode arrays in epilepsy, light treatments for neurological disease, and deep brain stimulation for Alzheimer's patients.

Loyola University Medical Center (Maywood, Ill.). As an academic medical center with researchers involved in nearly 200 clinical trials, Loyola University Medical Center leverages unique neurosurgical techniques such as deep brain stimulation and stereotactic radiotherapy. Each year, Loyola's highly experienced surgeons perform over 1,000 cranial surgeries and collaborate on about 150 cranial-base operations at the Center for Cranial Base Surgery, which features a fully equipped speech and swallowing laboratory. With a 13-bed neuro intensive care unit, Loyola was ranked No. 28 in the nation for neurology and neurosurgery by U.S. News & World Report for 2019-20.

Massachusetts General Hospital (Boston). Massachusetts General Hospital's neurosurgery department performs more than 4,000 procedures every year and was the first to use deep brain stimulation to reduce epileptic seizures. MGH's team of 20 faculty neurosurgeons and 20 residents in training oversees 86 dedicated beds and a 22-bed ICU. Home to the nation's largest hospital-based neuroscience research program, MGH is committed to studying rare disorders of the nervous system, neurodegenerative disorders and effective ALS therapies. U.S. News & World Report ranked Mass General among the top 20 hospitals in the nation for neurology and neurosurgery in 2019-20.

Mayo Clinic (Rochester, Minn.). Mayo Clinic is one of the premier institutions for neurosurgery in the nation, with its Rochester location ranked No. 2 for neurology and neurosurgery by U.S. News & World Report in 2019-20. Neurosurgeons annually perform 7,000 procedures at its three campuses in Arizona, Florida and Minnesota. The health system is also on the forefront of neurosurgical research and currently has 31 clinical trials open for participation as well as a registry for primary spinal tumor research.

Medical University of South Carolina (Charleston). The Medical University of South Carolina's spine center team is a designated Blue Distinction Center for spine surgery, a mark of demonstrated quality outcomes. The hospital has $9 million in technology development funding through an in-house innovation program called Zucker Institute for Applied Neurosciences, a technology accelerator embedded within the health system to move new neuroscience innovations into the clinical settings quickly. The Medical University of South Carolina's spine program was one of the first in the state to offer patients endoscopic spine surgery, and it plans to expand its endoscopic and minimally invasive spine outpatient offerings.

MedStar Georgetown University Hospital (Washington, D.C.). The 609-bed MedStar Georgetown University Hospital has a multidisciplinary spine center and offers comprehensive neurosurgery services, including a pediatric neurosurgery program. It includes 12 neurosurgeons and spine specialists, of which five are trained in minimally invasive spine care. Its minimally invasive spine specialists have performed thousands of procedures, from discectomies to decompressions. In 2019-20, U.S. News & World Report named MedStar Georgetown University Hospital high performing in neurology and neurosurgery, as well as one of the best regional hospitals in the nation.

Memorial Hermann-Texas Medical Center (Houston). Memorial Hermann-Texas Medical Center includes the Memorial Hermann Orthopedic & Spine Hospital to offer patients the latest in spine care. The Memorial Hermann Orthopedic & Spine Hospital features 64 private patient rooms, eight two-room suites and 10 surgical suites. Patients coming to the medical center can also receive care at the Mischer Spine Center, where neurosurgeons perform more than 3,000 spine surgeries annually. The Mischer Spine Center is affiliated with Memorial Hermann Mischer Neuroscience Institute at the Texas Medical Center, which offers opportunities for patients to receive the benefits of cutting-edge research. There are 25 spine and nerve research clinical trials in progress or recently completed at the Mischer Neuroscience Institute.

MemorialCare (Fountain Valley, Calif.). Spine care at MemorialCare is offered by a multidisciplinary team of neurosurgeons, orthopedic surgeons and nonoperative specialists. Patients seeking care have the option of going to one of three California-based locations, in Long Beach, Laguna Hills or Fountain Valley. Two MemorialCare hospitals are ranked high performing in adult neurology and neurosurgery by U.S. News & World Report. The Spine Center at MemorialCare Long Beach Medical Center recently acquired robotic navigation technology to enhance precision in spine surgery.

Michigan Medicine (Ann Arbor). Michigan Medicine's neurosurgery department celebrated 100 years of offering adult and pediatric neurosurgical care last year. It includes 24 clinical faculty and eight research faculty members. Michigan Medicine's neurosurgery department also has a mission to educate and train the next generation of neurosurgical and spine care specialists via its residencies and fellowships. Its neurosurgeons are involved in an outreach program, Project Shunt, that offers neurosurgical care to children in Guatemala. U.S. News & World Report ranked Michigan Medicine No. 19 on its list of the 50 best hospitals for adult neurology and neurosurgery in 2019-20.

Milton S. Hershey Medical Center (Hershey, Pa.). Milton S. Hershey Medical Center offers spine and neurosurgery services via its spine center, which includes a 25-person care team. The spine center has developed a "back coach" program, which offers information and resources to those suffering from chronic back and neck pain. The hospital also has a robust neurosurgery research faculty that includes 17 members focusing on several key research areas, including using 3D models to understand tumor growth.

Montefiore Health System (New York City). Montefiore Health System aims to be at the forefront of neurosurgical research and care with eight comprehensive care centers and 12 neurosurgeons on staff. Earlier this year, Montefiore held its first pediatric neurointerventional symposium, which included experts from the U.S. and Canada. Montefiore Medical Center, the health system's flagship, was ranked among the top 50 hospitals in the nation for adult neurology and neurosurgery by U.S. News & World Report for 2019-20.

Moses H. Cone Memorial Hospital (Greensboro, N.C.). The 517-bed Moses H. Cone Memorial Hospital has received several recognitions for its spine care services, including being ranked No. 1 in the state for medical excellence in spinal surgery and spinal fusion by CareChex in 2018. Blue Cross and Blue Shield of North Carolina also designated the facility a Blue Distinction Center + for spine surgery last year. The hospital offers major interventional neuroradiology and neurosurgery treatments with a multidisciplinary team of neurologists, neuroradiologists other nonoperative specialists to ensure comprehensive care. Cone Health includes 19 orthopedic spine surgeons and neurosurgeons.

Mount Sinai Hospital (New York City). The Spine Hospital at Mount Sinai offers the full spectrum of cervical, thoracic and lumbar spine care. It includes 16 orthopedic and neurospine surgeons who provide care along with a team of nonoperative spine specialists. The hospital is also a preferred spine care site for retired NFL players. The health system has a strong foundation in research through various programs, including the Friedman Brain Institute, an interdisciplinary clinical and research hub focused on brain and spinal cord disorders. U.S. News & World Report ranked Mount Sinai Hospital among the top 20 hospitals in the nation for neurology and neurosurgery in 2019-20.

NewYork-Presbyterian/Weill Cornell Medical Center (New York City). NewYork-Presbyterian/Weill Cornell Medical Center's Weill Cornell Brain and Spine Center includes 24 neurosurgeons, neuroradiologists, neuropsychologists and neuroendocrinologists, who offer clinical services, conduct research and train students, residents and fellows. Patients receiving care at the center have access to the latest research-based medicine, including access to 16 neurosurgery-focused clinical trials. NewYork-Presbyterian was ranked No. 4 on U.S. News & World Report's 2019-20 list of the 50 best hospitals for neurology and neurosurgery.

Northwell Health (New Hyde Park, N.Y.). Northwell Health's Institute for Neurology and Neurosurgery includes a multidisciplinary team that offers a wide array of clinical services, and in collaboration with the Feinstein Institutes for Medical Research, the physicians and scientists conduct research and clinical trials that advance the field. U.S. News & World Report ranked Northwell's North Shore University Hospital among the top 50 hospitals in the nation for neurology and neurosurgery, while also distinguishing Lenox Hill Hospital in New York City as high performing in the specialty for 2019-20.

Northwestern Memorial Hospital (Chicago). Northwestern Memorial Hospital includes 38 neurological surgery and spine surgery specialists. It also offers a combined orthopedic spine and neurosurgical spine fellowship to train the next generation of spine and neurosurgeons. Earlier this year, the hospital launched the Northwestern Medicine Hispanic Brain and Spine Tumor Program in Chicago, which aims to reduce barriers to specialized care for the Hispanic and Latino population. U.S. News & World Report ranked Northwestern Memorial Hospital No. 5 on its list of the 50 best hospitals for neurology and neurosurgery in 2019-20.

Norton Healthcare (Louisville, Ky.). Norton Healthcare is home to the Norton Leatherman Spine Center, serving patients in Louisville and southern Indiana. Specialists at Norton Leatherman Spine perform more than 4,000 surgeries annually, and patients see its providers there more than 30,000 times a year. The fellowship-trained specialists at Norton Leatherman Spine have an average of 20 years of experience and focus on research as well as training the next generation of spine surgeons. The hospital has trained more than 100 spine surgeons who are practicing across the country.

NYU Langone Health (New York City). NYU Langone Health's neurosurgery department consists of more than 20 full-time clinical and research faculty members who take on other physicians' most complex surgical cases. Combined with the system's orthopedic spine surgeons, NYU Langone supports about 2,700 spine procedures per year. Its spine center is equipped with robotic technology and a 3D platform for planning and performing surgeries and provides operative and nonoperative treatment for about 18,000 adults and children annually. NYU Langone Hospitals is ranked No. 9 among U.S. News & World Report's top 50 hospitals for neurology and neurosurgery.

Ochsner Medical Center (New Orleans). Ochsner Health System's neurosurgery program stands out for various reasons, including being the only such program in Louisiana and one of only five U.S. centers to offer in-utero surgery to repair spinal bifida in babies during pregnancy. Each year, neurosurgeons across Ochsner Health System perform more than 1,500 adult and pediatric surgeries. The health system's spine and back care program is also highly rated, earning five stars from Healthgrades. Ochsner Medical Center, part of Ochsner Health System, is among the nation's top 50 hospitals for neurology and neurosurgery, according to U.S. News & World Report's 2019-20 rankings.

Ohio State University Wexner Medical Center (Columbus). Ohio State University is the home of an 87-bed, 60,000-square-foot brain and spine hospital that includes specialized units for stroke care, neurotrauma and traumatic brain injuries and spinal cord injury. The university also houses one of the nation's only centers that pursue innovative projects and research through the Traumatic Brain Injury Model Systems program, which is sponsored by the National Institute on Disability and Rehabilitation Research. Additionally, the university has the only rehabilitation program in central Ohio certified to handle traumatic brain injury. In 2019-20, U.S. News & World Report recognized Wexner Medical Center as high performing in adult neurology and neurosurgery.

Oregon Health & Science University Hospital (Portland). Oregon Health & Science University Hospital has pioneered innovation in neurological surgery, including North America's first deep brain stimulation surgery, the world's first neuronal stem cell transplants and an intraoperative MRI facility. It also has a leading neurosurgery training program and advanced fellowships in skull base and vascular, functional and pediatric neurosurgery. The hospital is ranked No. 44 among U.S. News & World Report's top 50 hospitals for neurology and neurosurgery in 2019-20.

Penn Medicine (Philadelphia). Penn Medicine neurosurgeons perform more than 5,000 operations annually at the Hospital of the University of Pennsylvania, Pennsylvania Hospital, Penn Presbyterian Medical Center, Penn Medicine Virtua Neurosciences and the Children's Hospital of Philadelphia. Penn Medicine's neurosurgery department also includes a research program led by basic scientists in brain, spine and nervous system diseases and disorders. Penn Medicine researchers are working on a prognostic blood test that would detect and measure neuronal proteins in the cerebrospinal fluid. In 2019-20, U.S. News & World Report ranked the health system's Pennsylvania Hospital No. 31 in the nation for neurology and neurosurgery.

ProMedica (Toledo, Ohio). ProMedica is a health system with more than 794 hospital beds serving 27 counties in Ohio and southeast Michigan. The system includes the ProMedica Wildwood Orthopaedic and Spine Hospital, which is designated a Blue Distinction Center for spine care by the Blue Cross Blue Shield Association and earned the 2018 Press Ganey Guardian of Excellence Award for physician engagement. The system's ProMedica Toledo Hospital also offers advanced navigation and robotics for complex brain and spine surgeries; it became the first in Ohio to offer the 3D mapping technology in 2017.

Rush University Medical Center (Chicago). Rush University Medical Center is one of the top 10 hospitals in the nation for neurology and neurosurgery, according to U.S. News & World Report. Its neurosurgery program consistently reports the most neurosurgical discharges in the Chicago area. In 2018, the health system reported 4,334 neurological surgery outpatient visits focused on the brain and 6,498 outpatient neurological visits focused on the spine. The health system also has a robust spine and back care program, with 12 physicians and surgeons increasingly moving toward minimally invasive and outpatient procedures. The health systems surgeons aim to stay at the forefront of patient treatment and participate in clinical trials investigating degenerative disc disease treatment, registry data for metastatic spine tumors and spinal stenosis treatment with new technology.

Saint Barnabas Medical Center (Livingston, N.J.). Saint Barnabas Medical Center houses the 22-physician neurological team of RWJBarnabas Health, the largest healthcare system in New Jersey. The Saint Barnabas Institute of Neurology & Neurosurgery is a level 4 epilepsy center with nine physicians focused on excellence in clinical care as well as clinical research. The hospital also has an innovative spine surgery department that has used a microdiscectomy technique developed by a member of its team to treat more than 500 patients. Saint Barnabas is recognized as high performing in neurology and neurosurgery by U.S. News & World Report.

Saint Luke's Hospital of Kansas City (Mo.). Saint Luke's Marion Bloch Neuroscience Institute has an advanced comprehensive stroke center accredited by the Joint Commission that leads the region in endovascular interventions and outcomes. It provides advanced stroke care to more than 2,000 patients annually, and the integrated spine program earned designation as a Blue Distinction Center+ for Spine Surgery by Blue Cross Blue Shield Association. Saint Luke's Spine Surgery Program has earned the Joint Commission's Gold Seal of Approval of Spine Surgery Certification. The system also has a level 4 comprehensive epilepsy center and a seven-member neurosurgeon team. For 2019-20, Saint Luke's Hospital of Kansas City was named among U.S. News & World Report's top 50 hospitals for neurology and neurosurgery.

Scripps Health (San Diego). Scripps Health offers neurosurgery and follow-up care at five San Diego County locations. At Scripps Health locations, physicians offer advanced techniques and technology, including minimally invasive brain surgery treatments and a robotics platform. Programs of Scripps Green Hospital and Scripps Memorial Hospital La Jolla ranked among U.S. News & World Report's top 50 hospitals for neurology and neurosurgery in 2019-20.

Spectrum Health (Grand Rapids, Mich.). Spectrum Health's neurosurgery department specializes in disorders affecting the central nervous system and offers services at five centers across Michigan, including a level 4 epilepsy center. Spectrum Health is also home to Helen DeVos Children's Hospital, which has the only pediatric neurosurgery program in the region. As the largest hospital group in West Michigan, Spectrum Health has been ranked among America's 50 best hospitals by Healthgrades for four consecutive years and has 1,600 physicians focused on more than 110 specialties.

St. Luke's Boise (Idaho) Medical Center. St. Luke's Boise Medical Center is part of the nonprofit St. Luke's Health System, Idaho's largest, comprising 14 hospitals. St. Luke's Boise, which includes four neurosurgery centers and three spine clinics in Idaho, has been ranked as a top 100 hospital by IBM Watson Health. After doubling the number of referrals to its spine care clinic in 2018, the hospital plans to open another location in 2020.

Stanford (Calif.) Health Care. The Stanford department of neurosurgery is composed of 61 neurosurgeons who perform 4,000 neurosurgical operations annually. It was named the No. 9 hospital in the nation for neurology and neurosurgery by U.S. News & World Report in 2019-20 and its stroke center was the first in the nation to be certified as a comprehensive stroke center by the Joint Commission. The department has 30 active labs researching topics including brain injury, deep brain stimulation, brain tumors and epilepsy.

Stony Brook (N.Y.) University Hospital. Stony Brook University Hospital's Neurosurgery Spine Center is a tertiary care academic medical center that has been named one of America's 100 Best Hospitals for stroke care by Healthgrades for five consecutive years. Stony Brook has more than 70 labs researching topics including spine and brain trauma, stroke and multiple sclerosis. The Neurosurgery Spine Center is the only practice in Suffolk County with two full-time pediatric neurosurgeons, and the adult neurology center sees more than 18,000 patients per year.

Sutter Health (Sacramento, Calif.). Eleven hospitals within the Sutter Health network a 24-hospital, nonprofit health system with more than 12,000 physicians received recognition from the American Stroke Association for providing a high level of stroke care in 2019. Eden Medical Center in Castro Valley, Calif., was named one of America's 100 Best Hospitals for stroke care by Heathgrades in 2019. Sutter Medical Center in Sacramento, Calif., also earned recognition as high-performing in neurosurgery by U.S. News & World Report in 2019.

Swedish Medical Center (Englewood, Colo.). Swedish Medical Center serves as the Rocky Mountain region's neurotrauma provider and has spine experts who perform an average of 90 spine surgeries per month. Part of HCA's HealthONE, Blue Cross Blue Shield Association designated it a Blue Distinction Center for spine surgery, and UnitedHealth Group designated it a Center of Excellence for spine surgery. It serves as the region's referral center for the most advanced stroke treatment and was the state's first Joint Commission-certified comprehensive stroke center.

Texas Children's Hospital (Houston). Texas Children's Hospital was the first hospital in the world to use real-time MRI-guided thermal imaging and laser technology to treat epilepsy. Named the No. 3 best neurosurgery center on U.S. News & World Report's 2019-20 list, the hospital performs more than 950 neurosurgical operations every year. It was also the first hospital to use a device similar to a pacemaker in the brain, which recognizes oncoming seizures and prevents them.

The Christ Hospital (Cincinnati). The Christ Hospital Joint & Spine Center is a seven-story facility with 14 operating rooms, four of which are dedicated solely to spine surgery. The Joint & Spine Center also offers physical, occupational and speech therapy services and physicians dedicated solely to joint, spine and brain care. Founded more than 125 years ago, The Christ Hospital has performed more spine procedures than any other hospital in the Cincinnati area.

Tulsa (Okla.) Spine & Specialty Hospital. Founded in 2002, Tulsa Spine & Specialty Hospital has a national reputation top-level patient care. It was named one of America's 100 best hospitals for spine surgery by Healthgrades. The physician-owned hospital was also honored with the Cigna Center of Excellence designation in 2018 for orthopedic back surgery and earned five stars from Healthgrades for spinal fusion in 2018. The hospital has 13 dedicated spine surgeons who perform minimally invasive procedures.

University of California San Diego Health. UC San Diego's neurosurgery division was founded in 1971 and features four intraoperative MRI and CT suites, destination skull base tumor programs and neurocritical care units. The division collaborates regularly with the UC San Diego School of Medicine, San Diego-based Scripps Research Institute and the Salk Institute for Biological Studies in La Jolla, Calif., and received $30 million in funding from the National Institutes of Health in 2018. It was ranked among the top 50 hospitals in the nation for neurology and neurosurgery in 2019-20 according to U.S. News & World Report.

UCI Health (Irvine, Calif.). UCI Health is the only academic health system in Orange County, Calif., and UCI Medical Center's neurosurgery department was recognized as high performing by U.S. News & World Report in 2019-20. The department of neurological surgery includes Orange County's first comprehensive stroke center, granted certification by The Joint Commission, as well as active research in neuro-oncology and spinal cord injury. The department frequently collaborates with other research organizations, such as UC Irvine's Reeve-Irvine Research Center, which is working to find new treatments for spinal cord injury.

UCLA Medical Center (Los Angeles). As UCLA Health's flagship hospital, UCLA Medical Center's neurosurgery department has ranked as one of the top neurosurgery programs in the nation for over 20 consecutive years by U.S. News & World Report. The department has its own neurosurgery app designed for patients with information about their physicians, procedures and hospital amenities. UCLA's Spine Center is also designated a Blue Distinction Center for Spine Surgery by Blue Shield of California.

UCSF Medical Center (San Francisco). The department of neurological surgery at UCSF Medical Center has 14 specialties, including pediatric neurosurgery. In 2011, the department developed the Quality Improvement and Patient Safety initiative with the goal of becoming a national leader in neurological surgery quality. The hospital is piloting an enhanced recovery after surgery pathway for cranial surgery as well as an opioid stewardship program. UCSF's Spine Center is also one of the largest spine centers in the country and sees over 10,000 patients a year. The department of neurological surgery at UCSF was recognized in 2019-20 as one of the top three neurosurgery programs in the country by U.S. News & World Report, which also ranks the UCSF Benioff Children's Hospitals among the top hospitals for pediatric neurosurgery in the nation.

UF Health (Gainesville, Fla.). The UF Health Spine Program provides comprehensive outpatient and inpatient treatment options at one location that features 17 neurosurgeons, three neurosurgery ORs, two neurosurgery hybrid interventional ORs and 48 private ICU patient rooms. The hospital provides complete spine services including the treatment of degenerative spinal diseases, spinal tumors as well as craniocervical junction anomalies and performs more than 1,000 procedures annually. For its 2019-20 rankings, U.S. News & World Report named UF Health Shands Hospital in Gainesville the No. 2 hospital in Florida, and it was ranked among the top in the nation for neurology and neurosurgery.

UK HealthCare Albert B. Chandler Hospital (Lexington, Ky.). UK Neurosurgery features nine neurosurgeons providing care for complex conditions including spinal tumors and deformities, stroke, ALS, and epilepsy. UK HealthCare Albert B. Chandler Hospital was ranked the No. 1 hospital in the state for neurology and neurosurgery by U.S. News & World Report's best hospitals survey for 2019-20. UK Neurosurgery collaborates with the UK Kentucky Neuroscience Institute on several research initiatives and is currently enrolling participants in ALS and epilepsy clinical trials.

UNC REX Hospital (Raleigh, N.C.). REX Neurosurgery & Spine Specialists features a team of 12 orthopedic spine and neurosurgeons providing comprehensive neurosurgical care including spinal fusion, minimally invasive spine surgery and reconstructive spine surgery. The department performs thousands of procedures each year and has been certified as a comprehensive stroke care center by The Joint Commission since 2011. UNC REX bolstered the department in the past year with the addition of an on-site spine physical therapist and a spine navigator to determine whether patients need imaging, surgery or physical therapy.

University Hospitals (Cleveland). The staff University Hospitals includes 11 orthopedic spine and neurosurgeons focused on providing exceptional patient care. The University Hospitals Spine Institute collaborates with the UH Neurological Institute, which features 13 centers of excellence and provides innovative neurosurgical therapies including CyberKnife, Gamma Knife and the NeuroBlate System. University Hospitals Cleveland Medical Center was ranked among the best hospitals for neurology and neurosurgery in the nation by U.S. News & World Report in 2019-20.

University of Alabama Hospital at Birmingham. UAB Hospital at Birmingham is widely recognized for its spine care and brain cancer research, and its specialists treat more than 4,000 patients annually. UAB Neurology and Neurosurgery has eight comprehensive divisions and seven centers that care for 26,000 patients per year. The neurosurgery department is also responsible for around 5,000 procedures annually for both pediatric and adult patients. It features research faculty and physician scientists who collaborate to advance research in conditions such as Parkinson's disease, spinal cord injury and neurovascular disorders.

University of California Davis Medical Center (Sacramento). Spine and neurosurgeons at UC Davis Medical Center actively participate in research and clinical trials spanning a range of areas including lumbar fusion, traumatic brain injury and thoracic spinal cord injury. UC Davis Health's neurosurgery department features 13 physicians on its clinical faculty and its brain tumor program incorporates 19 physicians from several subspecialties to provide optimum care for adult and pediatric patients with tumors of the nervous system. The UC Davis Medical Center ranked among the best hospitals for neurology and neurosurgery in U.S. News & World Report's 2019-20 list.

University of Colorado Hospital (Aurora). UCHealth Spine Center at the Anschutz Medical Campus is staffed by renowned spine and neurosurgeons who have built a comprehensive and award-winning program. The hospital is certified by The Joint Commission as a comprehensive stroke center and its epilepsy program is rated as a Level 4 center by the National Association of Epilepsy Centers and earned the 2019 Get With the Guidelines Stroke Gold Plus Elite Plus award from the American Heart Association and American Stroke Association for outstanding care. The department of neurosurgery features 23 physicians and U.S. News & World Report ranked UCHealth University of Colorado Hospital among the best in the nation for neurology and neurosurgery for 2019-20.

University of Iowa Hospitals and Clinics (Iowa City). The UI Spine Center has a robust program with 10 orthopedic spine and neurosurgeons who participate in next-generation surgical technology investigations to stay on the forefront of spine care. The hospital has earned the Blue Cross Blue Distinction Center+ designation for spine surgery and the University of Iowa Carver College of Medicine is often ranked in the top 10 in National Institutes of Health funding for faculty members, including neurosurgery. The neurosurgery department specializes in the surgical treatment of degenerative spine pathology, epilepsy, brain and spinal cord tumors and was designated a center of excellence by the Parkinson's Foundation in 2018.

University of Kansas Hospital (Kansas City). The department of neurosurgery at the University of Kansas Medical Center is equipped with virtual reality systems and a 3D printer to assist neurosurgeons in planning procedures and training physicians in the latest technology. The hospital includes 11 neurosurgeons and a 14-physician neurosurgery residency program. It also has a robust spine center, the Marc A. Asher, MD, Comprehensive Spine Center, which opened in 2008 and includes 27 exam rooms, four diagnostic rooms and a 4,000-square-foot outpatient rehabilitation gym.

University of Miami (Fla.) Hospital and Clinics. Neurosurgeons at University of Miami Hospital and Clinics see more than 14,000 patients and perform over 4,000 procedures annually. The hospital was named among the best hospitals in Florida by U.S. News & World Report in 2019-20 and scored as a high-performing facility in the departments of neurology and neurosurgery. The hospital integrates the latest innovations into its neurosurgical research programs including robotics, 3D interoperative imaging and deep brain stimulation.

UW Health (Madison, Wis.). The neurosurgery residency program was founded at the University of Wisconsin's department of surgery in 1942 and has been in operation ever since. UW Health features 14 neurosurgeons, 12 research faculty and two fellows. Neurosurgeons in the department see more than 1,200 brain tumor patients per year in collaboration with the UW Carbone Cancer Center. The department focuses on both clinical and investigative aspects of care for neurological diseases and is currently participating in a range of clinical trials involving brain tumors, stroke, spinal cord injury and cervical spondylotic myelopathy.

University of Utah Hospitals and Clinics (Salt Lake City). University of Utah Hospitals and Clinics provides the full spectrum of neurosurgical care to patients with cranial and spinal diseases and disorders. The faculty includes 25 physicians who provide a range of services including cerebrovascular, spinal, functional, traumatic, tumor, and pediatric neurosurgery. Physicians at the hospital are actively involved in clinical trials with current projects including pediatric neurosurgery and venous thromboembolism.

University of Virginia Medical Center (Charlottesville). Spine specialists at the University of Virginia Medical Center, in partnership with colleagues from the neurosurgery department, perform more than 1,500 spine procedures each year. The medical center's neurosurgery department is led by Jeffrey Elias, MD, who was honored as the 2018 Edlich-Henderson Innovator of the Year by the UVA Licensing & Ventures Group. At the university, Dr. Elias pioneered the use of focused ultrasound to treat essential tremor and led a clinical trial that resulted in FDA approval of the treatment.

UW Medicine (Seattle). The department of neurological surgery at UW Medicine is the primary referral center for patients in Washington, Wyoming, Alaska, Montana and Idaho who have complex neurological conditions. Twenty neurosurgeons, 15 neuroscientists and 79 adjunct clinical research faculty staff the department, and an additional 20 physicians are in its neurological surgery residency program. The neurological surgery department's outreach initiatives include National Institutes of Health-sponsored brain injury research in five Latin American countries.

UW Health (Madison, Wis.). In 1993, spine specialists at UW Health developed a minimally invasive treatment for spinal fusion surgery, becoming one of the first institutions to perform the procedure in the world and solidifying the health system as a leader in minimally invasive spine surgery. In addition to spine, providers at UW Health care for more than 1,200 brain tumor patients each year, working with the UW Carbone Cancer Center when additional treatment is needed. For 2019-20, U.S. News & World Report listed UW Health among the top 50 hospitals in the nation for neurology and neurosurgery.

UPMC (Pittsburgh). Part of the UPMC Neurological Institute, the UPMC department of neurosurgery is one of the largest academic neurosurgical providers in the nation, with more than 11,000 procedures performed annually. Among its accolades, the department ranks as one of the highest in the country in National Institutes of Health funding, and the department's chair, Robert Friedlander, MD, was elected to the National Academy of Medicine in 2018. U.S. News & World Report ranked UPMC's Presbyterian Shadyside hospital in Pittsburgh among the nation's top 50 hospitals for neurology and neurosurgery for 2019-20.

UR Medicine (Rochester, N.Y.). Patients across New York's Finger Lakes, Southern Tier and Western New York regions are served by UR Medicine Neurosurgery. At UR Medicine's Spine Center, physicians see more than 19,000 patients annually. In 2019, researchers led by the director of the hospital's department of neurosurgery's Translation Pair Research Program were selected to help the National Institutes of Health create a nonaddictive treatment for pain through clinical trials.

UT Southwestern Medical Center (Dallas). The Peter O'Donnell Jr. Brain Institute, part of the department of neurological surgery at UT Southwestern Medical Center, ranks No. 15 in the nation for neurology and neurosurgery, according to U.S. News & World Report's 2019-20 list. The department's neuro-oncology program is supported by the Annette G. Strauss Center for Neuro-Oncology and collaborates with the Harold C. Simmons Comprehensive Cancer Center. In 2018, the Decherd Foundation awarded the hospital an endowment to create an annual award to recognize exceptional care for neurotrauma patients at UT Southwestern's teaching hospital, Dallas-based Parkland Memorial Hospital.

Vanderbilt University Medical Center (Nashville, Tenn.). In addition to 21 residents, the neurological surgery department at Vanderbilt University Medical Center has 16 physician faculty members who are part of its 32-member advance practice and research team. The department sees more than 5,000 surgeries and procedures each year. In 2019, neurological surgery researchers at Vanderbilt, supported by the National Institutes of Health, for the first time found improvements in brain networks after surgery in 15 people with temporal lobe epilepsy.

VCU Medical Center (Richmond, Va.). With a 13-physician faculty, the Virginia Commonwealth University department of neurosurgery at VCU Medical Center is home to a new concept of outpatient medicine for orthopedic and neurological conditions. The VCU Health Neuroscience, Orthopaedic and Wellness Center, dubbed the "N.O.W. Center," aims to offer patient-centered care, using new software to help providers manage patients' progress during visits. The neurosurgery department at VCU Medical Center has been recognized by U.S. News & World Report, which named the hospital among the top 50 in the country for neurology and neurosurgery for 2018-19.

Vidant Medical Center (Greenville, N.C.). Vidant Medical Center is home to the only neuroscience intensive care unit in eastern North Carolina, as well as a specialized neuroscience rehabilitation unit. The hospital's neurosurgery department has 19 physicians and healthcare professionals. In April 2019, one of Vidant's neurosurgery department members co-authored a 12-month study of the use of a new neuro-spinal scaffold to treat acute thoracic complete spinal cord injury.

Wake Forest Baptist Health (Winston-Salem, N.C.). Every year, physicians at Wake Forest Baptist Health's spine center perform more than 1,000 surgeries on patients in need of treatment for back and neck disorders. Wake Forest Baptist Health is home to one of the nation's leading Gamma Knife Centers and is one of the few centers in the country to use deep brain stimulation to treat movement disorders, brain tumors, depression and Tourette syndrome. Additionally, the hospital's neurosurgery department has two neurosurgeons who specialize in pediatric care.

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100 hospitals and health systems with great neurosurgery and spine programs | 2019 - Becker's Hospital Review

Automation in Cell Therapy Manufacturing Is Driving the Growth of the Cell Therapy Market over the Forecast Period (2019-2025) – Press Release -…

A recent report published by Infinium Global Research on cell therapy market provides in-depth analysis of segments and sub-segments in the global as well as regional cell therapy market.

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Pune, India -- (SBWIRE) -- 12/13/2019 -- The Infinium Global Research analyzes the "Cell Therapy Market (Cell Type - Stem Cell, and Differentiated Cell; Therapy Type - Allogenic Therapies, and Autologous Therapies; Application - Autoimmune Diseases, Oncology, Dermatology, Musculoskeletal Therapies, and Other Applications; End-users - Hospitals and Clinics, Biotechnology and Pharmaceutical Companies, and Research Institutes): Global Industry Analysis, Trends, Size, Share and Forecasts to 2025."

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Cell therapy is the transplantation of human cells to replace or repair damaged tissue or cells. With new technologies, products, innovative, and limitless imagination. Cells used for cell therapy often stem cells, cells that can mature into different types of specialized cells. The most common type of cell therapy has been the replacement of mature, functioning cells through blood and platelet transfusions. Cell therapies treat cancer, improving a weakened immune system, autoimmune disease, rebuilding damaged cartilage in joints, urinary problems, and infectious disease, repairing spinal cord injuries, and helping patients with neurological disorders.

Automation in Cell Therapy Manufacturing Providing Intensive Opportunities in the Cell Therapy Market

The rising occurrence of chronic diseases across the globe is the major driver for the growth of the global cell therapy market. Moreover, increasing the geriatric population, increase in cell therapy transplantation rate, and replacement of animal testing model are some of the key factors fueling the market growth. Furthermore, increasing awareness of stem cell therapy and the development of cell banking facilities contributing to the growth of the global cell therapy market.

Moreover, technological advancements and improvements in the regulatory framework continuously provide to the growing market. However, challenges in research and development, lack of proper and advanced healthcare in developing regions may hinder market growth. Furthermore, automation in cell therapy manufacturing providing intensive opportunities in the cell therapy market in the coming years.

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Stem Cell Therapy is Expected to Hold Maximum Share in the Cell Therapy Market

The global cell therapy market is segmented on the basis of type, therapy, application, and end-user. Types are further segmented into stem cells and differentiated cells. Stem cell therapy is expected to hold maximum share in the cell therapy market. Stem cell therapies having several advantages like regenerating the body organisms and other cells, which contributes to the growth of the stem cell therapies market.

By therapy, the cell therapy market is segmented into allogeneic therapies and autologous therapies. On the basis of application, the market is further divided into autoimmune diseases, oncology, dermatology, musculoskeletal therapies, and other applications. Based on end-users, the market is hospitals and clinics, biotechnology and pharmaceutical companies, and Research Institutes.

North America is Leading the Market Share in the Cell Therapy Market

The cell therapy market is segmented regionally into North America, Europe, Asia-Pacific, and the Rest of the World. North America is leading the market share in the cell therapy market due to the high rate of cancer and blood-related disorders coupled with high investments in the research and development of novel technologies. North America has a firm healthcare organization that acts as an added advantage for the growth of the market in this region.

Asia-Pacific region is anticipated to stimulate the growth of the cell therapy market due to a large number of surgeries performed in this region. Rising awareness about advanced medicinal therapies contributes to the growth of the market in the Asia-Pacific region.

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Cell Therapy Market: Competitive Analysis

The leading players in the cell therapy market are NuVasive, Inc., Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., Osiris Therapeutics, Inc., Stemedica Cell Technologies, Inc., MEDIPOST, Stemedica Cell Technologies, Inc., Celgene Corporation, ANTEROGEN.CO., LTD, Vericel Corporation. These companies are adopting strategic partnerships to enhance their product portfolio.

Reasons to Buy this Report:

=> Comprehensive analysis of global as well as regional markets of the cell therapy.

=> Complete coverage of all the product type and application segments to analyze the trends, developments, and forecast of market size up to 2025.

=> Comprehensive analysis of the companies operating in this market. The company profile includes analysis of product portfolio, revenue, SWOT analysis and the latest developments of the company.

=> Infinium Global Research- Growth Matrix presents an analysis of the product segments and geographies that market players should focus to invest, consolidate, expand and/or diversify.

For more information on this press release visit: http://www.sbwire.com/press-releases/automation-in-cell-therapy-manufacturing-is-driving-the-growth-of-the-cell-therapy-market-over-the-forecast-period-2019-2025-1267918.htm

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Automation in Cell Therapy Manufacturing Is Driving the Growth of the Cell Therapy Market over the Forecast Period (2019-2025) - Press Release -...

Aspen Neuroscience gets funding to pursue personalized cell therapy for Parkinsons disease – The San Diego Union-Tribune

Aspen Neuroscience, a new San Diego biotech company working on stem cell treatment for Parkinsons disease, has come out of stealth mode and raised $6.5 million to pursue clinical testing for its therapy.

Co-founded by well-known stem cell scientist Jeanne Loring, Aspen Neuroscience proposes creating stem cells from modified skin cells of Parkinsons patents via genetic engineering.

The stem cells, which can become any type of cell in the body, then would undergo a process that makes them specialize into dopamine-releasing neurons.

People with Parkinsons lose a large number up to 50 percent at diagnosis of specific brain cells that make the chemical dopamine.

Without dopamine, nerve cells cannot communicate with muscles and people are left with debilitating motor problems.

Once these modified skin cells have been engineered to specialize in producing dopamine, they can be transplanted into the Parkinsons patient to restore the types of neurons lost to the disease.

The reason we called it Aspen is because l was raised in the Rocky Mountain states, said Loring. When there is a forest fire in the Rockies, the evergreens are wiped out but the aspens are the fist that regenerate after the burn. So it is a metaphor for regeneration.

Aspen still has a long way to go before its proposed therapy would be available to Parkinsons patients. It has been meeting with the U.S. Food and Drug Administration to provide animal trial data and other information in hopes of getting permission to start human clinical trials.

But the company expects the earliest it would get the go-ahead from FDA to start human trials would be 2021.

Loring has been working on the therapy for eight years. She is professor emeritus and founding director of the Center for Regenerative Medicine at the Scripps Research Institute.

Loring co-founded the 20-employee company with Andres Bratt-Leal, a former post-doctoral researcher in Lorings lab at Scripps.

Joining them as Aspens Chief Executive is Dr. Howard Federoff, former vice chancellor for health affairs and chief executive of the University of California Irvine Health System.

Federoff said the company is the only one pursuing the use of Parkinsons patients own cells as part of neuron replacement therapy.

Aspens proprietary approach does not require the use of immuno-suppression drugs, which can be given when transplanted cells come from another person and perhaps limit the effectiveness of the treatment.

Aspens approach is a therapy that is likely to benefit from the fact that your own cells know how to make the best connections with their own target cells in the brain, even in the setting of Parkinsons disease, said Federoff. So when transplanted it is able to set back the clock on Parkinsons.

In addition to Aspens main therapy, it is researching a gene-editing treatment for forms of Parkinsons common in certain families.

Aspens research work up to now has been supported by Summit for Stem Cell, a non-profit on which provides a variety of services for people with Parkinsons disease.

The new seed funding round was led by Domain Associates and Axon Ventures, with additional participation from Alexandria Venture Investments, Arch Venture Partners, OrbiMed and Section 32.

Aspens financial backing, combined with its experienced and proven leadership team, positions it well for future success, said Kim Kamdar, a partner at Domain Associates. Domain prides itself on investing in companies that can translate scientific research into innovative medicines and therapies that make a difference in peoples lives. We clearly see Aspen as fitting into that category, as it is the only company using a patients own cells for replacement therapy in Parkinsons disease.

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Aspen Neuroscience gets funding to pursue personalized cell therapy for Parkinsons disease - The San Diego Union-Tribune

Acute Leukemia: 5 Things to Know – Medscape

In the past 2 years, a host of new targeted treatments for acute leukemia have become availablemost notably the FLT and IDH inhibitorsand yet the best way to treatthese patients remains a challenge.Hereare five things to know about the therapeutic landscape ofthis diseaseand emerging evidence surroundingitsrisk factors.

Experts do not agree on the best approach for treating older patients with AML. Patients aged 65 years and older tend to have more aggressive disease and a worse prognosis compared with their younger peers. The 5-year survival estimates for this older cohort is 5%, whereas the rate for patients younger than 20 years is 67%.

Chemotherapy, which is the first-line therapy for the majority of patients with AML, often fails in older patients because the agents are too toxic or simply not effective. In fact, more than half of older patients with AML receive no care.

A central question for clinicians treating older patients with AML is whether to provide more-intensive or less-intensive therapy. Oncologists can make a case for both. For example, data from the Swedish Acute Leukemia Registry show that intensive treatment is well tolerated and extends survival in most patients aged 60-75 years. However, support for a low-intensity strategy also exists. A 2019 phase 1b/2 clinical trial found that a combination of the chemotherapy agents venetoclax and low-dose cytarabine led 54% of patients aged 82 years and older to achieve complete remission or complete remission with incomplete blood count recovery.

Oncologists can turn to a patient's degree of frailty and tumor biology to provide clues about which therapeutic strategy to pursue. Frail patients, for instance, are less likely to tolerate aggressive chemotherapy. Understanding the tumor biology of AML, which is often more complex in older patients, can also indicate how responsive a patient will be to different approaches. Overall, experts are working toward a more standardized way of assessing these metrics and how each should inform treatment decisions.

In the past 2 years, a host of new targeted treatments for AML have become available. Most of these drugs inhibit key genetic mutations associated with AML. Midostaurin, which targets FLT3 mutations, was the first such inhibitor to receive approval from the US Food and Drug Administration in April 2017, but more potent agents soon followedgilteritinib for FLT3 mutations, ivosidenib for IDH1 mutations, and enasidenib for IDH2 mutations.

The new crop of targeted drugs show promise for improving outcomes.

A phase 3 randomized controlled trial evaluating oral gilteritinib versus salvage chemotherapy reported a median overall survival of 9.3 months in patients who received gilteritinib compared with 5.6 months for those receiving chemotherapy. About one third of patients receiving gilteritinib achieved complete remission with partial hematologic recovery compared with 15% of those receiving chemotherapy alone.

An alternative targeted approach is to disrupt key players along metabolic or cell maintenance pathways instead of genetic mutations. A phase 2 randomized controlled trial found that glasdegib, which targets the Hedgehog pathway, almost doubled overall survival when combined with low-dose cytarabine compared with cytarabine alone (8.8 months vs 4.9 months).

Immunotherapy represents another potential treatment option for patients with AML. The antibody-drug conjugate gemtuzumab ozogamicin is designed to deliver the antitumor antibiotic calicheamicin directly to leukemia cells expressing the CD33 transmembrane receptor. The CD33 receptor is only expressed by cells of myeloid origin, not normal stem cells, which allows the drug to pursue cancerous cells with greater specificity.

Gemtuzumab ozogamicin has an interesting history. It was withdrawn from the market in 2010 after concerns over its safety and efficacy surfaced, but it was then reapproved in 2017 after further research showed its value. Recent analyses have found that, when added to conventional induction therapy, gemtuzumab ozogamicin improves relapse rates and overall survival in patients with CD33-positive AML.

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Acute Leukemia: 5 Things to Know - Medscape

Global T-Cell Therapy (CAR T-Cell, Tumor Infiltrating Lymphocytes) Market Size, Share & Trends Analysis Report 2019-2026 – ResearchAndMarkets.com…

DUBLIN--(BUSINESS WIRE)--The "T-Cell Therapy Market Size, Share & Trends Analysis Report By Modality, By Therapy (CAR T-Cell, Tumor Infiltrating Lymphocytes), By Indication (Hematologic Malignancies, Solid Tumors), And Segment Forecasts, 2019 - 2026" report has been added to ResearchAndMarkets.com's offering.

The global T-cell therapy market size is expected to reach USD 7.51 billion, expanding at a CAGR of 15.4% by 2026

The landmark approvals of Yescarta and Kymriah have spurred unprecedented advancements in the market. The launch of these breakthrough therapies has bolstered cash inflow for innovation, thereby driving the growth.

Expansion of the market for T-cell therapy significantly relies on shifting preference from first-line stem cell transplants and chemotherapy to third-line CAR T-cell therapy. Moreover, the ever-expanding plethora of medical conditions for which the T cell therapies is projected to bode well for the market growth. Rise in oncological disorders is projected to drive interest as well as investments in the T-cell therapy market in near future.

In contrast with the small-molecule landscape, engineered T cells market landscape is distinguished by an extensive network that encompasses several entities marked by connections academically, financially, and via technology licensing. Research bodies, and manufacturers, and regulators engage in assessing the long-term efficacy and safety of therapies to ensure safe access to patients.

By far, the antigen challenge and linked toxicity concerns have impeded the development of CAR T therapies in non-hematological malignancies. Market players are applying a data-driven approach of exploring this space to mitigate the challenge and expand the usage of T-cell therapy in indication type such as brain cancer and melanoma.

Further key findings from the report suggest:

Competitive Landscape

For more information about this report visit https://www.researchandmarkets.com/r/jgs2h6

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Global T-Cell Therapy (CAR T-Cell, Tumor Infiltrating Lymphocytes) Market Size, Share & Trends Analysis Report 2019-2026 - ResearchAndMarkets.com...

CAMPUS: EGG DONATION – Artificial sperm cells to remove the genetic worries of sperm donation – ESHRE

A Campus meeting in November reviewed the arguments for and against donor conception, and the sometimes difficult ethical arguments raised by the prospect of a donor-conceived child. 'Artificial' sperm cells derived from testicular tissue or stem cells may resolve some of those arguments.

The problem is especially acute in cancers diagnosed in prepubertal boys in whom there are no sperm cells available for storage. Their only option for future fatherhood in the face of cancer treatment is adoption or donor sperm. And this, added Goossens, is not an exceptional problem. Incidence rates are around 17 cases per 100,000 population, with leukemia and CNS tumours the most commonly diagnosed. So the usual pathway to fertility preservation in these young cases is for the oncologist to warn of the risk to future fertility from the cancer treatments and refer to the fertility clinic. Biopsy of testicular tissue, of course, must be performed before any radio- or chemotherapy.

Goossens described two experimental techniques, spermatogonial stem cell retrieval and transplantation, and homotopic tissue grafting. The danger in the former procedure is a risk of introducing malignancy, so banked tissue must be free of malignant contamination. Experiments in mouse-to-mouse models have demonstrated spermatogenesis from tissue grafting, and most recently fully functional conception and delivery in a non-human primate (Grady). Similarly, experiments in mouse models with spermatogonial stem cell transplantation have so far proved efficient, with spontaneous pregnancy already possible.

Of course, the objective of this impressive experimental work is not merely a resolution to the question of genetic continuity in couples faced with third-party donation, but the future fertility and long-term quality of life of so many unfortunate young boys. Advances in cancer treatment have led to the increased survival of all children with cancer, and with it a new imperative for the restoration of their fertility. Not all cancer treatments cause complete testicular damage, but around one-third of children having treatment for pediatric cancers will end up infertile. Following the proof-of-concept study which saw the birth of Grady - in which testicular samples removed from prepubertal monkeys was frozen, thawed and regrafted under scrotal skin - the research group declared that their next logical step, with safety and feasibility apparent, is human trials.

1. Fayomi AP, Peters K, Sukhwani M, et al. Autologous grafting of cryopreserved prepubertal rhesus testis produces sperm and offspring. Science 2019; 363: 1314-1319.

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CAMPUS: EGG DONATION - Artificial sperm cells to remove the genetic worries of sperm donation - ESHRE

Stem Cell Therapy Market Forecasted To Surpass The Value Of US$ XX Mn/Bn By 2045 2017 – 2025 – Markets Gazette 24

TMRR, suggest in its latest market report, that the Stem Cell Therapy market report is about to exceed US$ xx Mn/Bn by 2029. The report finds that the Stem Cell Therapy market registered ~US$ xx Mn/Bn in 2018 and is projected to expand at a healthy CAGR over the foreseeable period.

The Stem Cell Therapy market research focuses on the market structure along with various factors (positive and negative) that influence the market growth. The study contains a precise evaluation of the Stem Cell Therapy market, including growth rate, current market scenario, and volume inflation prospects, on the basis of DROT and Porters Five Forces analyses.

Important players profiled in the Stem Cell Therapy market research include player 1, player 2, player 3 and player 4.

In this Stem Cell Therapy market study, the following years are considered to project the market footprint:

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Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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The content of the Stem Cell Therapy market report includes the following insights:

The Stem Cell Therapy market study answers critical questions including:

All the players running in the global Stem Cell Therapy market are elaborated thoroughly in the Stem Cell Therapy market report on the basis of R&D developments, distribution channels, industrial penetration, manufacturing processes, and revenue. In addition, the report examines, legal policies, and comparative analysis between the leading and emerging Stem Cell Therapy market players.

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Stem Cell Therapy Market Forecasted To Surpass The Value Of US$ XX Mn/Bn By 2045 2017 - 2025 - Markets Gazette 24

Drugs, Biologics, and Regenerative Medicine in 2019 – The National Law Review

Friday, December 13, 2019

Following up on our first post in this year-end series that discussed medical device regulatory activities at the Food and Drug Administration (FDA), the Mintz FDA teams second year-end post will provide an overview of 2019 with a focus on the drug, biologic, and regenerative medicine programs at the agency. In many ways, the past year could be called a business as usual year for the FDAs drugs and biologics centers in that they continued to make progress on all of large-scale programs and priorities initiated by former-Commissioner Scott Gottlieb, who left the agency in April. FDA has been under the leadership of an Acting Commissioner since that time, although Texas radiation oncologist Dr. Stephen Hahn will be taking the reins soon following his confirmation by the full Senate in a 72-18 vote on December 12, 2019. (The Senate HELP Committee advanced the nominee on December 3, 2019; see our blog post just prior to that committee vote here.)

At the same time, however, the final months of 2019 have exposed several challenges for various FDA programs that operate under the extensive drug and biologic authorities contained in the Food Drug & Cosmetic Act (FD&C Act) and the Public Health Service Act (PHS Act), respectively. The agency will be forced to grapple with many of these issues directly and deliberately in 2020as a result of deadlines of the agencys own making as well as external pressures coming from other parts of D.C. and from the rapidly changing nature of the U.S. health care system.

According to data presented by the Office of New Drugs in early December (see ONDs slides here), FDA had another extremely productive year when it comes to its approval of new molecular entities that address a unique blend of therapeutic areas. The agency approved 45 new molecular entities in FY 2019 (October1, 2018 to September30, 2019), of which 71%, or 32 products, received priority review status and 23 were designated as orphan drugs intended to treat rare diseases. As OND emphasized in the presentation, several of those new product approvals are notable for their uniqueness and therefore, in the agencys view, 2019 reflects not only quantity but [also] quality. The OND presentation also highlights a significant amount of other information on new molecular entity approvals and may be of interest to those readers who want to take a deeper dive into the data.

In addition to advancing important new drugs and biologics to market, former Commissioner Gottlieb is well-known for having spearheaded to development of a Drug Competition Action Plan (DCAP) and a Biosimilars Action Plan (BAP) during his nearly two-year tenure as head of the agency. Some of our prior coverage of the DCAP and BAP is available here. In general terms, the DCAP encourages market competition for generic drugs and helps to bring greater efficiency and transparency to the generic drug review process; the BAP aims to achieve similar goals for biosimilar products as the agency continues its implementation of the 2010 Biologics Price Competition and Innovation Act (BPCIA), including the critical drug-to-biologic transition that will occur by operation of law in March of 2020.

FDA continued to make progress on its various goals under these two initiatives during 2019. Some examples of this progress can be captured with these two data points:

FDA approved 1,171 generic drugs (935 full approvals and 236 tentative approvals) in the last fiscal year. Notably, 125 drug approvals were for first generics of medicines that had no direct generic competition. The agency released a report in October 2019 noting that generic drug approvals reached a record high in FY 2019 and also that several complex generic drug products had been approved for the first time. Under the DCAP, the agency has prioritized getting complex generic drug products to market with the goal of increasing competition and thereby reducing commercial prices for such products, which may be expensive due to difficult manufacturing processes.

On December 6, 2019, FDA licensed its 26th biosimilar product since the BPCIA was enacted and the 10th biosimilar this year (Amgens Avsola (infliximab-axxq), which is a biosimilar to Remicade and the fourth Remicade biosimilar approved by the agency to date). Although thirteen of the previous 25 approved biosimilars have yet to launch due to ongoing patent litigation or for business reasons, the agency has continued to focus on positive developments in the still-nascent U.S. biosimilars market, including with the issuance of a public statement on November 15 in conjunction with approval of the 25th biosimilar (see here).

With all of these wins, however, FDA still faces its fair share of challenges related to its expedited drug and biologic programs, especially as it appears to have accelerated its review of products intended for diseases with an unmet need to breakneck speed. Indeed, the OND presentation from early December also added that for FY 2020 and as of November 21, 2019, the agency had already approved 13 such drugs, suggesting that next year could be a record-breaking one in terms of innovative drug product approvals. A recent Bloomberg Law article (available here) used the phrase breakneck speed to describe the agencys actions in this space based on several recent FDA approvals of new molecular entities that have come months in advance of their assigned target dates. The Bloomberg Law article highlights that in response to FDAs speediness in reaching approval decisions on new drugs and biologics for diseases with unmet needs, patient advocates and, increasingly, insurers that have to pay for those treatments are starting to raise concerns that these products lack sufficient safety or effectiveness data.

Relatedly, there has been increasing pressure on FDA to remove certain accelerated approval drugs from the market following a failure by the drug product sponsor to confirm the efficacy or clinical benefits of the product in the required post-marketing confirmatory clinical trial. The most visible example of this regulatory challenge came in October 2019 when FDA convened an advisory committee to recommend whether it should withdraw accelerated approval from hydroxyprogesterone caproate injection (marketed under the brand name Makena for the prevention of preterm birth in pregnant women). FDAs Bone, Reproductive and Urologic Drugs Advisory Committee voted 9-7 to withdraw approval, with the dissenters favoring leaving Makena on the market while requiring the sponsor to conduct a new confirmatory trial. (Notably, no one voted for the option of leaving it on the market without requiring a new confirmatory trial.)Among the concerns of some members who voted to leave the product on the market with a new clinical trial obligation was that the drugs withdrawal would leave no safe treatment options for pregnant women at high risk of preterm birth. FDA will have to make a final decision regarding what to do about Makena in 2020, and it undoubtedly will face intense criticism (and potentially legal challenge) no matter what route it chooses to take for this public health quandary in which it finds itself.

In a similar vein, FDA official Dr. Richard Pazdur participated in a Senate briefing on December 10, 2019, in which he and other speakers defended the Breakthrough Therapy Designation program. FDA insisted again that the designation was intended to let the agency have earlier interactions with drug sponsorsand that it was not meant to be an early rating system for drugs or a signal of how they might do commercially. Given that the Breakthrough program was created in 2012 and is considered to be wildly successful, some speakers at the briefing expressed surprise that there was still any confusion about its purpose and function. Whether Congress picks up any of these emerging areas for consideration as part of FDAs 2022 user fee reauthorization packages remains to be seenas those negotiations will begin in earnest after the New Year, but the issues certainly are complex enough to allow for robust policy discussions to occur.

Finally, there are expected to be bumps in the road with the upcoming March 2020 transition of proteins previously approved under New Drug Applications (NDAs) to Biologics License Applications (BLAs)for which FDA only finalized its guidance for industry last year. The March 2020 transition date was established under the BPCIA and the agency does not have discretion in getting the transition done (only in how it handles the logistical and administrative issues created by transitioning approved products in this way). Check out our prior blog post on the final deemed to be a license transition guidance.

FDA guidance documents for all regulated product categories continued to be released on a regular basis this year, including several related to areas of agency priorities under the DCAP and BAP including the final biosimilar interchangeability guidance issued in May (see our blog post here) and a draft guidance on insulin interchangeability issued in November 2019. The latter also relates to the March 2020 NDA-to-BLA transition, as insulins are one of the largest class of products that will be transitioning into regulation as biologics, making them open to what is expected to be more efficient competition through the BPCIAs biosimilar pathway than what was possible in the past as insulin NDAs.

Despite the accelerated pace of the issuance of Agency guidance, however, FDA is beginning to face more challenges related to its decision-making and the scope of its exercise of agency discretion. In particular, a significant District Court for the District of Columbia ruling issued on December 6, 2019, Genus Medical Technologies, LLC v. FDA, provides hints of a potential shift in judicial deference to certain agency actions. The court vacated FDAs classification of a medical imaging liquid as a drug rather than as a device after determining that FDA did not have discretion to decide how to regulate a product merely because the definitions of drug and device overlap in the FD&C Act.

According to the district court judge, FDA was not interpreting the drug/device definitions in the statute properly, and Congress did not intend to allow the agency unfettered discretion to pick between the two categories. Rather, the court found that the text of the definitions are clear and do not create a gap or any ambiguity for FDA to fill with an exercise of agency discretion. This recently issued decision may indicate a potential shift in how courts are going to apply long-standing precedents related to judicial deference to agency decisions. If FDA decides to appeal the Genus ruling, it may end up at the Supreme Court as one of many expected challenges to the doctrines that established our current framework for judicial deference of an administrative agencys interpretation of an ambiguous statute.

One of the first FDA press releases for 2019 was co-authored by former Commissioner Gottlieb and Center for Biologics Evaluation and Research (CBER) Director Peter Marks and was focused on the agencys new policies aiming to advance the development of safe and effective cell and gene therapies. In the press release, the agency leaders predicted that by 2020, FDA would receive more than 200 Investigational New Drug Applications (INDs) for cell and gene therapies each year. The agency has continued to work diligently to increase its staff in CBER to conduct clinical reviews for such INDs and to try to keep pace with the industrys development of these innovative technologies.

As we discussed in our update on FDAs Comprehensive Regenerative Medicine Policy Framework earlier this year, the agency is prioritizing two parallel goals: (1) clarifying the regulatory criteria for product marketing and providing support and guidance to legitimate product developers; and (2) removing unapproved, unproven, and potentially unsafe products from the U.S. market. The second prong of this comprehensive plan for regenerative medicine products was the topic of one of Dr. Gottliebs very last statements as Commissioner before he left the agency, issued on April 3, 2019 in conjunction with CBER Director Dr. Marks, indicating how important this area is to the agencys current public health priorities.

In the April 2019 statement, Drs. Gottlieb and Marks acknowledged FDAs challenges and efforts to stop stem cell clinics and manufacturers from marketing unapproved products that put patients at risk, citing several Warning Letters issued to manufacturers that violated current good manufacturing practices (CGMPs) for human cells and tissue products. They noted that it was of particular concern given that the industry was nearly halfway through the period during which the FDA intends to exercise enforcement discretion for certain regenerative medicine products with respect to INDs and premarket approval requirements. Now that it is December 2019, that deadline is even closer with less than one year left. November 2020 is the end of the three-year period of enforcement discretion announced by FDA when it first articulated the policies and goals of this comprehensive framework in 2017. See our prior posts on the topic here and here.

Under the Comprehensive Regenerative Medicine Policy Framework, FDA appears to have stepped up the pace of issuing Warning and Untitled Letters to sellers of unapproved stem cell products during the second half of 2019. In conjunction with a Warning Letter issued on December 5, 2019 to two related companies for processing and marketing unapproved umbilical cord blood-derived cellular products, Dr. Marks of CBER reiterated the agencys concerns about safety and reminded the public of the upcoming compliance deadline: As evidenced by the number of actions that the agency has taken this month alone, there are still many companies that have failed to come into compliance with the [FD&C Act] and FDAs regulations.

Dr. Marks was referring to two Untitled Letters that were issued to stem cell product distributors on November 20 and November 25, 2019, respectively. The press release cited above also added that the agency had also recently sent 20 letters to manufacturers and health care providers noting that it has come to [FDAs] attention that they may be offering unapproved stem cell products, reiterating the FDAs compliance and enforcement policy.

FDA also prevailed this year in the U.S. District Court of the Southern District of Florida against a stem cell clinic charged with violating the FD&C Act and the PHS Act. In June 2019, the court held that the defendants adulterated and misbranded a stem cell drug product made from a patients adipose tissue without FDA approval and for significant deviations from CGMPs, issuing a permanent injunction as requested by the Department of Justice on FDAs behalf. The agencys statement on that important court win by the government is available here.

Lastly, on December 6, 2019, FDA issued a Public Safety Notification on Exosome Products. The safety notification informed the public of multiple recent reports of serious adverse events experienced by patients in Nebraska who were treated with unapproved products marketed as containing exosomes, which came to FDAs attention through the Centers for Disease Control and Prevention, the Nebraska Department of Health and Human Services, and others. There are currently no FDA-approved exosome products and, to be honest, we are not even sure what such a product would be since an exome consists of all the sequenced exons within a single human genome after the introns are removed. (So were the clinics administering complete exomes to patients? that seems unlikely.)But what we found noteworthy about this public safety notice is the forceful and direct language FDA used when describing the unscrupulous conduct of the sellers of these products:

Certain clinics across the country, including some that manufacture or market violative stem cell products, are now also offering exosome products to patients. They deceive patients with unsubstantiated claims about the potential for these products to prevent, treat or cure various diseases or conditions. They may claim that they these products do not fall under the regulatory provisions for drugs and biological products that is simply untrue. As a general matter, exosomes used to treat diseases and conditions in humans are regulated as drugs and biological products under the [PHS Act] and the [FD&C Act] and are subject to premarket review and approval requirements.

The clinics currently offering these products outside of FDAs review process are taking advantage of patients and flouting federal statutes and FDA regulations. This ultimately puts at risk the very patients that these clinics claim to want to help, by either delaying treatment with legitimate and scientifically sound treatment options, or worse, posing harm to patients, as evidenced by these recent reports of adverse events.

As we enter the final year of FDAs enforcement discretion period, perhaps these public notices and Warning/Untitled Letters will become even more frequent and the agency will become even more frustrated by the ongoing violations and medical practitioners who flout federal law. This area will see substantial activity in 2020 and we will be watching closely to see what changes, if anything, about FDAs approach in November when the deadline to come into compliance ends. Will there be widespread FBI raids on stem cell clinics engaged in this kind of bad behavior? Only time will tell.

Although we have highlighted what we view as some important challenges for the FDA to address in the coming months, other areas continue to be business as usual without anything very new to report. FDA continues to invest significant resources into improving the quality of compounded drugs and ensuring compliance with Sections 503A and 503B of the FD&C Act. Drug compounding was another topic of one of Dr. Gottliebs very last statements as Commissioner on April 3, 2019, in which he laid out the 2019 compounding priorities that included maintaining quality manufacturing and compliance and regulating compounding from bulk drug substances.

Notwithstanding all the efforts by FDA and State regulators in this area over the past several years, the agency continues to see concerning activity when it comes to compounded drugs, such as problems related to the condition under which compounded sterile medicines are made, which raisesignificant risks to patients. As a result, FDA has made it an intense area of focus to take enforcement actions against compounders who fail to produce sterile drugs in compliance with the law. During the past year, for example, FDA has won at least four permanent injunctions against various compounders after the agency identified behavior that posed a significant risk to public health and safety.

In addition, in 2019 FDA also increased its activities towards reducing and mitigating the impact of drug shortages on the health care system. See our prior blog post on Drug Shortages.

Finally, after a fairly slow year of enforcement in the prescription drug advertising space, the last two months of 2019, at least as of December 12th, have given us three (!) letters two untitled and one warning from the FDAs Office of Prescription Drug Promotion (OPDP). Most interestingly, the Warning Letter issued on December 2, 2019 for omitting warnings about the most serious risks associated with [a medication-assisted treatment] drug from promotional materials was announced to the public via FDA press release, which is not a typical action for normal-course OPDP letters to industry. The drug in question, approved for the prevention of relapse to opioid dependence following opioid detoxification, is associated with several significant risks including potential opioid overdose. Given the countrys public health emergency that is the opioid epidemic, FDA appears to have felt the need to make the deficiencies in the advertisement and those risks more widely publicized. So another thing we will be watching for in the New Year is whether this OPDP action represents the beginning of a new trend by the agency to publicize these Warning Letters more directly, or whether its advertising enforcement activities may be picking up due to industrys evolving approaches to promoting therapeutic products.

1994-2019 Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C. All Rights Reserved.

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Drugs, Biologics, and Regenerative Medicine in 2019 - The National Law Review

US Network of Specialized Angelman Clinics to Double Thanks to New Partnership – Angelman Syndrome News

As part of her medical training, North Carolina pediatrician Elizabeth Jalazo had studied a little about Angelman syndrome.

I knew enough about it to answer a board question, but I didnt know the complexity of the disorder, she said. Im sure most general pediatricians would share the same sentiment.

Then Jalazo gave birth to a daughter with Angelman and she quickly became an expert on the disease. Evelyn, now 5, is the second of three siblings; theres also Anna, 7, and Charlie, 2.

I didnt know immediately that she had Angelman, but I knew there was something different about Evelyn within five days of her being born, Jalazo said. As most families, we endured a yearlong odyssey of diagnosis. And that was only because I was a clinician at a hospital that had a developmental center where I could push for her to be evaluated.

At the time, Jalazo was a chief resident at Baltimores Kennedy Krieger Institute a division of Johns Hopkins University so I could pick up the phone and ask a professional favor. I basically skipped 9-12 months of wait time.

Jalazo now is a full-time clinician at the University of North Carolina (UNC) School of Medicine in Chapel Hill, where she sees kids with Angelman, Hunter syndrome, and other genetic disorders.

For the past six months, shes also been director of clinical integration at the Chicago-based Angelman Syndrome Foundation (ASF), a nonprofit group dedicated to finding a cure for the severe neurological disorder that affects Evelyn and about 25,000 other Americans.

The U.S. network of specialized clinics Jalazo oversees is now set to more than double in size from eight to 18 thanks to a new partnership between the ASF and the Dup15q Alliance. The network also includes four non-U.S. clinics, in Argentina, Canada, Israel, and the Netherlands.

The 15q Clinical Research Network, as the joint effort is known, will serve patients with Angelman and dup15q syndromes both rare conditions that stem from mutations in the same region of chromosome 15.

In Angelman, the mutation is most commonly a microdeletion on chromosome 15 encompassing theUBE3A gene. This prevents the production of the UBE3A enzyme in certain brain regions.

Partnering with the Dup15q Alliance will allow us to increase our reach to the AS community and provide the best care for our families, Amanda Moore,the foundations CEO, said in a press release. Comprehensive and specific care is critical for AS families throughout the stages of their journey by partnering with Dup15q Alliance, we are able to reach thousands more families with care and support by bringing AS clinics to their location.

The ASF operates on an annual budget of $2 million and uses half of that to fund research. It currently serves 3,500 patients in the database and has around 22,000 followers on social media.

The network Jalazo supervises began in 2011, when three specialized Angelman facilities at Massachusetts General Hospital in Boston, UNCs Carolina Institute for Developmental Disabilities in Chapel Hill, and the NYU Langone Medical Center in New York City all became ASF-supported clinics. Six more were added over the last eight years.

Patients travel to where they can receive this evidence-based care, said Jalazo, who spoke to Angelman Syndrome News at the 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit, organized by the National Organization for Rare Disorders.

When its challenging to travel with a child that has special needs especially one with a rare disorder that no ones ever heard of it becomes incredibly beneficial to see all the specialists you need to see in one or two days, she said.

Most of these clinics operate one day a month, though some sites such as Mass General see patients weekly. The current U.S. network treats around 700 Angelman patients throughout the United States; including the four foreign clinics, the ASF network serves close to 1,000 patients.

In December 2018, Israels Sheba Medical Center at Tel HaShomer joined the network. That specialized AS clinic, housed at Shebas Edmond and Lily Safra Childrens Hospital just outside Tel Aviv, was established seven years ago by pediatric neurologist Gali Heimer. Since then, its evolved into the fourth-largest specialized Angelman syndrome clinic in the world, treating 85 of Israels known 100 or so Angelman patients.

Thanks to the new agreement between ASF and Dup15q Alliance both based in the Chicago area 10 Angelman clinics will join the ASF network, including Seattle Childrens Hospital, MiamisNicklaus Childrens Hospital, and the Intermountain Primary Childrens Medical Center in Salt Lake City, Utah.

This isnt the first time ASF and Dup15q Alliance have worked together. Because the two syndromes are similar, the groups have co-hosted a research symposium every two years. Many researchers who study Angelman also study dup15q.

Our goal is to bring together all the providers caring for Angelman individuals across the globe, Jalazo said. We encourage our sites to be multidisciplinary, so that patients can receive comprehensive care, but we do recognize that some clinicians around the world are not seeing patients in a multidisciplinary setting.

As clinical director at the ASF, Jalazo oversees a monthly virtual meeting via Zoom where representatives of all the clinics talk about pressing issues.

One of the biggest challenges is harnessing the power of patient data, she said. Every time a patient shows up at a clinic, an incredible amount of data is collected thats valuable to patient care, to understanding the natural history of the disease, and to drug development. So we want to harness that and ensure were utilizing and formatting it in a way thats accessible.

Jalazo said that when it comes to her own daughter, an early diagnosis definitely helped. Evelyn has been on a modified ketogenic (low-carb, high-fat) diet from a very early age.

She eats whatevers put in front of her, said the pediatrician, adding that Evelyn generally eats eggs and avocados every morning. Shell go find the blender and if its 3 p.m. and I havent made a smoothie, shell bang the cup on the counter.

Indeed, though the little girl cant speak, she does communicate.

If she needs something, shell go get it and bring it to me. Shell force eye contact. Before she could even walk, shed scoot over to you and direct your gaze over to whatever she wanted you to see, Jalazo said. I think she understands most of what I tell her. There are certain tones she uses. Her term of endearment is a g sound which means I love you.

Evelyns sister, Anna, is a natural caretaker and knows intuitively what Evelyn needs, while Evelyn and Charlie have a very typical sibling relationship they fight all the time, Jalazo said.

Shes completely nonverbal and youd assume she cant read the cues of social situations, but she and Charlie communicate 100% effectively, she said.

As for a cure for AS, Jalazo said gene therapy is extremely promising but that short-term, the focus is on antisense oligonucleotides, or ASOs. These are small pieces of DNA or RNA that can bind to specific molecules of RNA and reduce, restore, or modify protein expression.

Its an incredibly exciting time for the Angelman community, in terms of promising therapeutics coming down the pipeline, one of which is gene therapy, she said. In the immediate future, well have the opportunity to see the impact of non-gene therapy approaches like ASOs. This will give us a little sneak peek into what gene therapy can do.

Total Posts: 11

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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US Network of Specialized Angelman Clinics to Double Thanks to New Partnership - Angelman Syndrome News