CHMP Grants Positive Opinion for DARZALEX (daratumumab) Subcutaneous Formulation for the Treatment of Patients with Multiple Myeloma – Yahoo Finance

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 issued a Positive Opinion recommending approval for DARZALEX (daratumumab) subcutaneous (SC) formulation for the treatment of adult patients with multiple myeloma in frontline and relapsed/refractory settings. The novel SC formulation of daratumumab is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20) [Halozyme's ENHANZE drug delivery technology] and reduces treatment time from hours to approximately three to five minutes, with similar efficacy, and fewer infusion-related reactions compared to intravenous (IV) administration.1,2 The CHMPs Positive Opinion for daratumumab SC formulation applies to all current daratumumab indications including newly diagnosed and transplant-ineligible patients, as well as relapsed or refractory patients.

"Despite therapeutic advances in the treatment of multiple myeloma, the time taken for administration of most intravenous treatments is relatively long and there have been few significant improvements over the years," said Maria-Victoria Mateos, M.D., Ph.D., COLUMBA primary investigator and Director of the Myeloma Unit at University Hospital of Salamanca-IBSAL, Salamanca, Spain. "The daratumumab subcutaneous formulation has the potential to transform the treatment experience for patients and physicians as it reduces time in the chair from hours to minutes, and, because it is administered as a fixed dose from the first treatment, it reduces preparation time and chances of error by eliminating the need for dose calculations."

The Positive Opinion is supported by data from the Phase 3 COLUMBA (MMY3012) and Phase 2 PLEIADES (MMY2040) studies presented at the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting and 62nd American Society of Hematology (ASH) Annual Meeting, respectively.1,2 The COLUMBA presentation included a non-inferiority comparison of daratumumab SC formulation to daratumumab IV formulation for co-primary endpoints of overall response rate and maximum Ctrough concentration.1 Furthermore, in a subsequent paper published in The Lancet Haematology, patient-reported treatment satisfaction scores with daratumumab SC versus daratumumab IV were reported using the modified-Cancer Therapy Satisfaction Questionnaire.3 The PLEIADES study evaluated the daratumumab SC formulation in different combination regimens in patients with newly diagnosed multiple myeloma or with relapsed/refractory disease.2

"The subcutaneous formulation of daratumumab showed similar efficacy and fewer infusion-related reactions compared to intravenous daratumumab, and, overall, patients expressed satisfaction with subcutaneous therapy. If approved, we are hopeful this new formulation could offer improved quality of life for patients with multiple myeloma," said Patrick Laroche, M.D., Haematology Therapy Area Lead, Europe, Middle East and Africa (EMEA), Janssen-Cilag. "Janssen is proud to have developed a new formulation to meet the needs of our patients and continue to make a meaningful difference to the lives of those living with multiple myeloma."

"Since its first European approval in 2016, intravenous daratumumab has been used in the treatment of more than 100,000 patients worldwide and, if approved, both new and existing patients with multiple myeloma will be able to start or switch to the subcutaneous formulation as part of their multiple myeloma daratumumab-based treatment regimens," adds Craig Tendler, M.D., Vice President, Clinical Development and Global Medical Affairs, Oncology at Janssen Research & Development, LLC. "Todays Positive Opinion represents Janssens commitment to continuing to improve the treatment experience for patients living with multiple myeloma."

#ENDS#

In Europe, daratumumab is indicated:4

About the COLUMBA Study (MMY3012)3,5The randomised, open-label, multicentre Phase 3 study included 522 patients with multiple myeloma who had received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory drug (IMiD), or whose disease was refractory to both a PI and an IMiD. In the arm that received the subcutaneously (SC) administered formulation of daratumumab (n=263), patients (median age of 65) received a fixed dose of daratumumab 1,800 milligrams (mg) co-formulated with recombinant human hyaluronidase PH20 (rHuPH20) 2,000 Units per millilitre (U/mL), SC weekly for cycles 1 2, every two weeks for cycles 3 6, and every four weeks for cycle 7 and thereafter. In the daratumumab IV arm (n=259), patients (median age of 67) received daratumumab for intravenous infusion 16 milligrams per kilogram (mg/kg) weekly for cycles 1 2, every two weeks for cycles 3 6, and every four weeks for cycle 7 and thereafter. Each cycle was 28 days. Patients in both treatment arms continued until disease progression or unacceptable toxicity. Co-primary endpoints were overall response rate (ORR) (non-inferiority = 60 percent retention of the lower bound [208%] of the 95% CI of the SIRIUS trial, with relative risk [RR] analysed by Farrington-Manning test) and pre-dose cycle 3, day 1 (C3D1) daratumumab Ctrough (non-inferiority = lower bound of 90 percent confidence interval (CI) for the ratio of the geometric means [GM] 80%).

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About the PLEIADES Study (MMY2040)6 The non-randomised, open-label, parallel assignment study Phase 2 PLEIADES trial included 240 adults either newly diagnosed or with relapsed or refractory multiple myeloma. Patients with newly diagnosed multiple myeloma were treated with 1,800 mg of the subcutaneous formulation in combination with either bortezomib, lenalidomide and dexamethasone (D-VRd) or bortezomib, melphalan and prednisone (D-VMP). Patients with relapsed or refractory disease were treated with 1,800 mg of the subcutaneous formulation plus lenalidomide and dexamethasone (D-Rd). The primary endpoint for the D-VMP and D-Rd cohorts was overall response rate. The primary endpoint for the D-VRd cohort was very good partial response or better rate. An additional cohort of patients with relapsed and refractory multiple myeloma treated with daratumumab plus carfilzomib and dexamethasone was subsequently added to the study.

About daratumumabDaratumumab is a first-in-class7 biologic targeting CD38, a surface protein that is highly expressed across multiple myeloma cells, regardless of disease stage.8 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.9 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.10,11,12,13,14,15,16,17 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.18,19 For more information, please see https://www.clinicaltrials.gov/.

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.20

About Multiple MyelomaMultiple myeloma (MM) is an incurable blood cancer that starts in the bone marrow and is characterised by an excessive proliferation of plasma cells.21 In Europe, more than 48,200 people were diagnosed with MM in 2018, and more than 30,800 patients died.22 Almost 60 percent of patients with MM do not survive more than five years after diagnosis.23

Although treatment may result in remission, unfortunately, patients will most likely relapse as there is currently no cure.24 Refractory MM is when a patients disease progresses within 60 days of their last therapy.25,26 Relapsed cancer is when the disease has returned after a period of initial, partial or complete remission.27 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.28 Patients who relapse after treatment with standard therapies, including proteasome inhibitors and immunomodulatory agents, have poor prognoses and few treatment options available.29

About the Janssen Pharmaceutical Companies of Johnson & JohnsonAt 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 StatementsThis 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 and projections of 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 29, 2019, 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. None of the Janssen Pharmaceutical Companies nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.

ENHANZE is a registered trademark of Halozyme.

# # #

References

1 Mateos MV, Nahi H, Legiec W, et al. Efficacy and safety of the randomized, open-label, non-inferiority, phase 3 study of subcutaneous (SC) versus intravenous (IV) daratumumab (DARA) administration in patients (pts) with relapsed or refractory multiple myeloma (RRMM): COLUMBA. J Clin Oncol. 2019;37(Suppl.): abstract 8005.2 Chari A, San-Miguel J, McCarthy H, et al. Subcutaneous daratumumab plus standard treatment regimens in patients with multiple myeloma across lines of therapy: Pleiades study update. Blood. 2019;134(Suppl.1):abstract 3152.3 Mateos MV, Nahi H, Legiec W, et al. Subcutaneous versus intravenous daratumumab in patients with relapsed or refractory multiple myeloma (COLUMBA): a multicentre, open-label, non-inferiority, randomised, phase 3 trial. Lancet Haematol. 2020 Mar 23 [epub ahead of print].4 European Medicines Agency. DARZALEX summary of product characteristics. Available at:https://www.ema.europa.eu/documents/product-information/darzalex-epar-productinformation_en.pdf Last accessed April 2020.5 ClinicalTrials.gov. A Study of Subcutaneous Versus (vs.) Intravenous Administration of Daratumumab in Participants With Relapsed or Refractory Multiple Myeloma. NCT03277105. Available at: https://clinicaltrials.gov/ct2/show/NCT03277105 Last accessed April 2020.6 ClinicalTrials.gov. A Study to Evaluate Subcutaneous Daratumumab in Combination With Standard Multiple Myeloma Treatment Regimens. NCT03412565. Available at: https://clinicaltrials.gov/ct2/show/NCT03412565 Last accessed April 2020.7 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;9:51.8 Fedele 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;2013:564687.9 [Data on file]. DARZALEX: New Patient Starts Launch to Date. RF-8220310 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 April 202011 ClinicalTrials.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 April 2020.12 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 April 2020.13 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 April 2020.14 ClinicalTrials.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 April 2020.15 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 April 2020.16 ClinicalTrials.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 April 2020.17 ClinicalTrials.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 April 2020.18 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 April 2020.19 ClinicalTrials.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 April 2020.20 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 April 2020.21 American Society of Clinical Oncology. Multiple myeloma: introduction. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/introduction Last accessed April 2020.22 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 April 2020.23 De 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;51:2254-68.24 Abdi J, Chen G, Chang H, et al. Drug resistance in multiple myeloma: latest findings and new concepts on molecular mechanisms. Oncotarget. 2013;4:2186207.25 National Cancer Institute. NCI dictionary of cancer terms: refractory. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=350245 Last accessed April 2020.26 Richardson P, Mitsiades C, Schlossman R, et al. The treatment of relapsed and refractory multiple myeloma. Hematology Am Soc Hematol Educ Program. 2007:317-23.27 National Cancer Institute. NCI dictionary of cancer terms: relapsed. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=45866 Last accessed April 2020.28 American 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 April 2020.29 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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More here:
CHMP Grants Positive Opinion for DARZALEX (daratumumab) Subcutaneous Formulation for the Treatment of Patients with Multiple Myeloma - Yahoo Finance

Verify the URL – InnovationMap

Since even the early days of COVID-19's existence, researchers all over the world were rallying to find a cure or potential vaccine which usually take years to make, test, and get approved.

Houston researchers were among this group to put their thinking caps on to come up with solutions to the many problems of the coronavirus. From the testing of existing drugs to tapping into tech to map the disease, here are some research projects that are happening in Houston and are emerging to fight the pandemic.

Baylor College of Medicine has identified a drug that could potentially help heal COVID-19 patients. Photo via bcm.edu

While Baylor College of Medicine has professionals attacking COVID-19 from all angles, one recent discovery at BCM includes a new drug for treating COVID-caused pneumonia.

BCM researchers are looking into Tocilizumab's (TCZ), an immunomodulator drug, effect on patients at Baylor St. Luke's Medical Center and Harris Health System's Ben Taub Hospital.

"The organ most commonly affected by COVID-19 is the lung, causing pneumonia for some patients and leading to difficulty breathing," says Dr. Ivan O. Rosas, chief of the pulmonary, critical care and sleep medicine section at BCM, in a news release.

TCZ, which has been used to successfully treat hyperimmune responses in cancer patients being treated with immunotherapy, targets the immune response to the coronavirus. It isn't expected to get rid of the virus, but hopefully will reduce the "cytokine storm," which is described as "the hyper-immune response triggered by the viral pneumonia" in the release.

The randomized clinical trial is looking to treat 330 participants and estimates completion of enrollment early next month and is sponsored by Genentech, a biotechnology company.

A Texas A&M University researcher is trying to figure out if an existing vaccine has an effect on COVID-19. Screenshot via youtube.com

A researcher from Texas A&M University is working with his colleagues on a short-term response to COVID-19. A vaccine, called BDG, has already been deemed safe and used for treatment for bladder cancer. BDG can work to strengthen the immune system.

"It's not going to prevent people from getting infected," says Dr. Jeffrey D. Cirillo, a Regent's Professor of Microbial Pathogenesis and Immunology at the Texas A&M Health Science Center, in a news release. "This vaccine has the very broad ability to strengthen your immune response. We call it 'trained immunity.'"

A&M leads the study in partnership with the University of Texas MD Anderson Cancer Center and Baylor College of Medicine in Houston, as well as Harvard University's School of Public Health and Cedars Sinai Medical Center in Los Angeles.

Texas A&M Chancellor John Sharp last week set aside $2.5 million from the Chancellor's Research Initiative for the study. This has freed up Cirillo's team's time that was previously being used to apply for grants.

"If there was ever a time to invest in medical research, it is now," Sharp says in the release. "Dr. Cirillo has a head start on a possible coronavirus treatment, and I want to make sure he has what he needs to protect the world from more of the horrible effects of this pandemic."

Currently, the research team is recruiting 1,800 volunteers for the trial that is already underway in College Station and Houston with the potential for expansion in Los Angeles and Boston. Medical professionals interested in the trial can contact Gabriel Neal, MD at gneal@tamu.edu or Jeffrey Cirillo, PhD at jdcirillo@tamu.edu or George Udeani, PharmD DSc at udeani@tamu.edu.

"This could make a huge difference in the next two to three years while the development of a specific vaccine is developed for COVID-19," Cirillo says in the release.

Researchers at Rice University's Center for Research Computing's Spatial Studies Lab have mapped out all cases of COVID-19 across Texas by tapping into public health data. The map, which is accessible at coronavirusintexas.org, also identifies the number of people tested across the state, hospital bed utilization rate, and more.

The project is led by Fars el-Dahdah, director of Rice's Humanities Research Center. El-Dahdah used open source code made available by ESRI and data from the Texas Department of State Health Services and Definitive Healthcare.

"Now that the Texas Division of Emergency Management released its own GIS hub, our dashboard will move away from duplicating information in order to correlate other numbers such as those of available beds and the potential for increasing the number of beds in relation to the location of available COVID providers," el-Dahdah says in a press release.

"We're now adding another layer, which is the number of available nurses," el-Dahdah continues. "Because if this explodes, as a doctor friend recently told me, we could be running out of nurses before running out of beds."

A new compound being developed at Texas Heart Institute could revolutionize the effect of vaccines. Photo via texasheart.org

Molecular technology coming out of the Texas Heart Institute and 7 HIlls Pharma could make vaccines like a potential coronavirus vaccine more effective. The oral integrin activator has been licensed to 7 Hills and is slated to a part of a Phase 1 healthy volunteer study to support solid tumor and infectious disease indications in the fall, according to a press release.

The program is led by Dr. Peter Vanderslice, director of biology at the Molecular Cardiology Research Laboratory at Texas Heart Institute. The compound was first envisioned to improve stem cell therapy for potential use as an immunotherapeutic for certain cancers.

"Our research and clinical colleagues are working diligently every day to advance promising discoveries for at risk patients," says Dr. Darren Woodside, co-inventor and vice president for research at the Texas Heart Institute, in the release. "This platform could be an important therapeutic agent for cardiac and cancer patients as well as older individuals at higher risk for infections."

UH researchers have developed a pliable, thin material that can monitor changes in temperature. Photo via uh.edu

While developed prior to the pandemic, nanotechnology out of the University of Houston could be useful in monitoring COVID patients' temperatures. The material, as described in a paper published by ACS Applied Nano Materials, is made up of carbon nanotubes and can indicate slight body temperature changes. It's thin and pliable, making it ideal for a wearable health tech device.

"Your body can tell you something is wrong before it becomes obvious," says Seamus Curran, a physics professor at the University of Houston and co-author on the paper, in a news release.

Curran's nanotechnology research with fellow researchers Kang-Shyang Liao and Alexander J. Wang, which also has applications in making particle-blocking face masks, began almost 10 years ago.

Read more:
Verify the URL - InnovationMap

Houston banking exec shares tips for keeping online information secure amid COVID-19 threats – InnovationMap

Since even the early days of COVID-19's existence, researchers all over the world were rallying to find a cure or potential vaccine which usually take years to make, test, and get approved.

Houston researchers were among this group to put their thinking caps on to come up with solutions to the many problems of the coronavirus. From the testing of existing drugs to tapping into tech to map the disease, here are some research projects that are happening in Houston and are emerging to fight the pandemic.

Baylor College of Medicine has identified a drug that could potentially help heal COVID-19 patients. Photo via bcm.edu

While Baylor College of Medicine has professionals attacking COVID-19 from all angles, one recent discovery at BCM includes a new drug for treating COVID-caused pneumonia.

BCM researchers are looking into Tocilizumab's (TCZ), an immunomodulator drug, effect on patients at Baylor St. Luke's Medical Center and Harris Health System's Ben Taub Hospital.

"The organ most commonly affected by COVID-19 is the lung, causing pneumonia for some patients and leading to difficulty breathing," says Dr. Ivan O. Rosas, chief of the pulmonary, critical care and sleep medicine section at BCM, in a news release.

TCZ, which has been used to successfully treat hyperimmune responses in cancer patients being treated with immunotherapy, targets the immune response to the coronavirus. It isn't expected to get rid of the virus, but hopefully will reduce the "cytokine storm," which is described as "the hyper-immune response triggered by the viral pneumonia" in the release.

The randomized clinical trial is looking to treat 330 participants and estimates completion of enrollment early next month and is sponsored by Genentech, a biotechnology company.

A Texas A&M University researcher is trying to figure out if an existing vaccine has an effect on COVID-19. Screenshot via youtube.com

A researcher from Texas A&M University is working with his colleagues on a short-term response to COVID-19. A vaccine, called BDG, has already been deemed safe and used for treatment for bladder cancer. BDG can work to strengthen the immune system.

"It's not going to prevent people from getting infected," says Dr. Jeffrey D. Cirillo, a Regent's Professor of Microbial Pathogenesis and Immunology at the Texas A&M Health Science Center, in a news release. "This vaccine has the very broad ability to strengthen your immune response. We call it 'trained immunity.'"

A&M leads the study in partnership with the University of Texas MD Anderson Cancer Center and Baylor College of Medicine in Houston, as well as Harvard University's School of Public Health and Cedars Sinai Medical Center in Los Angeles.

Texas A&M Chancellor John Sharp last week set aside $2.5 million from the Chancellor's Research Initiative for the study. This has freed up Cirillo's team's time that was previously being used to apply for grants.

"If there was ever a time to invest in medical research, it is now," Sharp says in the release. "Dr. Cirillo has a head start on a possible coronavirus treatment, and I want to make sure he has what he needs to protect the world from more of the horrible effects of this pandemic."

Currently, the research team is recruiting 1,800 volunteers for the trial that is already underway in College Station and Houston with the potential for expansion in Los Angeles and Boston. Medical professionals interested in the trial can contact Gabriel Neal, MD at gneal@tamu.edu or Jeffrey Cirillo, PhD at jdcirillo@tamu.edu or George Udeani, PharmD DSc at udeani@tamu.edu.

"This could make a huge difference in the next two to three years while the development of a specific vaccine is developed for COVID-19," Cirillo says in the release.

Researchers at Rice University's Center for Research Computing's Spatial Studies Lab have mapped out all cases of COVID-19 across Texas by tapping into public health data. The map, which is accessible at coronavirusintexas.org, also identifies the number of people tested across the state, hospital bed utilization rate, and more.

The project is led by Fars el-Dahdah, director of Rice's Humanities Research Center. El-Dahdah used open source code made available by ESRI and data from the Texas Department of State Health Services and Definitive Healthcare.

"Now that the Texas Division of Emergency Management released its own GIS hub, our dashboard will move away from duplicating information in order to correlate other numbers such as those of available beds and the potential for increasing the number of beds in relation to the location of available COVID providers," el-Dahdah says in a press release.

"We're now adding another layer, which is the number of available nurses," el-Dahdah continues. "Because if this explodes, as a doctor friend recently told me, we could be running out of nurses before running out of beds."

A new compound being developed at Texas Heart Institute could revolutionize the effect of vaccines. Photo via texasheart.org

Molecular technology coming out of the Texas Heart Institute and 7 HIlls Pharma could make vaccines like a potential coronavirus vaccine more effective. The oral integrin activator has been licensed to 7 Hills and is slated to a part of a Phase 1 healthy volunteer study to support solid tumor and infectious disease indications in the fall, according to a press release.

The program is led by Dr. Peter Vanderslice, director of biology at the Molecular Cardiology Research Laboratory at Texas Heart Institute. The compound was first envisioned to improve stem cell therapy for potential use as an immunotherapeutic for certain cancers.

"Our research and clinical colleagues are working diligently every day to advance promising discoveries for at risk patients," says Dr. Darren Woodside, co-inventor and vice president for research at the Texas Heart Institute, in the release. "This platform could be an important therapeutic agent for cardiac and cancer patients as well as older individuals at higher risk for infections."

UH researchers have developed a pliable, thin material that can monitor changes in temperature. Photo via uh.edu

While developed prior to the pandemic, nanotechnology out of the University of Houston could be useful in monitoring COVID patients' temperatures. The material, as described in a paper published by ACS Applied Nano Materials, is made up of carbon nanotubes and can indicate slight body temperature changes. It's thin and pliable, making it ideal for a wearable health tech device.

"Your body can tell you something is wrong before it becomes obvious," says Seamus Curran, a physics professor at the University of Houston and co-author on the paper, in a news release.

Curran's nanotechnology research with fellow researchers Kang-Shyang Liao and Alexander J. Wang, which also has applications in making particle-blocking face masks, began almost 10 years ago.

Read the original:
Houston banking exec shares tips for keeping online information secure amid COVID-19 threats - InnovationMap

Trials and Tribulations: Neurology Research During COVID-19 – Medscape

With some pivotal trials on hold, the COVID-19 pandemic is slowing the pace of research in Alzheimer's disease (AD), stroke, and multiple sclerosis (MS).

However, researchers remain determined to forge ahead with many redesigning their studies, at least in part to optimize the safety of their participants and research staff.

Keeping people engaged while protocols are on hold; expanding normal safety considerations; and re-enlisting statisticians to keep their findings as significant as possible are just some of study survival strategies underway.

The pandemic is having a significant impact on Alzheimer's research, and medical research in general, says Heather Snyder, PhD, vice president, Medical & Scientific Relations at theAlzheimer's Association.

"Many clinical trials worldwide are pausing, changing, or halting the testing of the drug or the intervention," she told Medscape Medical News. "How the teams have adapted depends on the study," she added. "As you can imagine, things are changing on a daily basis."

The US Study to Protect Brain Health Through Lifestyle Intervention to Reduce Risk (U.S. POINTER) trial, for example, is on hold until at least May 31. The Alzheimer's Association is helping to implement and fund the study along with Wake Forest University Medical Center.

"We're not randomizing participants at this point in time and the intervention which is based on a team meeting, and there is a social aspect to that has been paused," Snyder said.

Another pivotal study underway is the Anti-Amyloid Treatment in Asymptomatic Alzheimers study (the A4 Study). Investigators are evaluating if an anti-amyloid antibody, solanezumab (Eli Lilly and Co), can slow memory loss among people with amyloid on imaging but no symptoms of cognitive decline at baseline.

"The A4 Study is definitely continuing. However, in an effort tominimize risk to participants, site staff and study integrity, we have implemented an optional study hiatus for both the double-blind andopen-label extension phases," lead investigator Reisa Anne Sperling, MD, told Medscape Medical News.

"We wanted to prioritize the safety of our participants as well as theability of participants to remain in the studydespitedisruptions from the COVID-19 pandemic," said Sperling, who is professor of neurology at Harvard Medical School and director of the Center forAlzheimer Research andTreatment at Brigham and Women'sHospital andMassachusetts GeneralHospital in Boston.

The ultimate goal is for A4 participants to receive the full number of planned infusions and assessments, even if it takes longer, she added.

Many AD researchers outside the United States face similar challenges. "As you probably are well aware, Spain is now in a complete lockdown. This has affected research centers like ours, Barcelonaeta Brain Research Center, and the way we work," Jos Lus Molinuevo Guix, MD, PhD, told Medscape Medical News.

All participants in observational studies like the ALFA+ study and EPAD initiatives, as well as those in trials including PENSA and AB1601, "are not allowed, by law, to come in, hence from a safety perspective we are on good grounds," added Molinuevo Guix, who directs the Alzheimer's disease and other cognitive disorders unit at the Hospital Clinic de Barcelona.

The investigators are creating protocols for communicating with participants during the pandemic and for restarting visits safely after the lockdown has ended.

A similar situation is occurring in stroke trials. Stroke is "obviously an acute disease, as well as a disease that requires secondary prevention," Mitchell Elkind, MD, president-elect of the American Heart Association, told Medscape Medical News.

"One could argue that patients with stroke are going to be in the hospital anyway why not enroll them in a study? They're not incurring any additional risk," he said. "But the staff have to come in to see them, and we're really trying to avoid exposure."

One ongoing trial, the AtRial Cardiopathy and Antithrombotic Drugs In Prevention After Cryptogenic Stroke (ARCADIA), stopped randomly assigning new participants to secondary prevention with apixaban or aspirin because of COVID-19. However, Elkind and colleagues plan to provide medication to the 440 people already in the trial.

"Wherever possible, the study coordinators are shipping the drug to people and doing follow-up visits by phone or video," said Elkind, chief of the Division of Neurology Clinical Outcomes Research and Population Sciences at Columbia University in New York City.

Protecting patients, staff, and ultimately society is a "major driving force in stopping the randomizations," he stressed.

ARCADIA is part of the StrokeNet prevention trials network, run by the NIH's National Institute of Neurologic Disorders and Stroke (NINDS). Additional pivotal trials include the Carotid Revascularization Endarterectomy Versus Stenting Trial (CREST) and the Multi-arm Optimization of Stroke Thrombolysis (MOST) studies, he said.

Joseph Broderick, MD, director of the national NIH StrokeNet, agreed that safety comes first. "It was the decision of the StrokeNet leadership and the principal investigators of the trials that we needed to hold recruitment of new patients while we worked on adapting processes of enrollment to ensure the safety of both patients and researchers interacting with study patients," he told Medscape Medical News.

Potential risks vary based on the study intervention and the need for in-person interactions. Trials that include stimulation devices or physical therapy, for example, might be most affected, added Broderick, professor and director of the UC Gardner Neuroscience Institute at the University of Cincinnati in Ohio.

Nevertheless, "there are potential waysto move as much as possible toward telemedicine and digital interactions during this time."

At the national level, the COVID-19 pandemic has had an "unprecedented impact on almost all the clinical trials funded by NINDS," said Clinton Wright, MD, director of the Division of Clinical Research at NINDS. "Investigators have had to adapt quickly."

Supplementing existing grants with money to conduct research on COVID-19 and pursuing research opportunities from different institutes are "some of the creative approaches [that] have come from the NIH [National Institutes of Health] itself," Wright said. "Other creative approaches have come from investigators trying to keep their studies and trials going during the pandemic."

In clinical trials, "everything from electronic consent to in-home research drug delivery is being brought to bear."

"A few ongoing trials have been able to modify their protocols to obtain consent and carry out evaluations remotely by telephone or videoconferencing," Wright said. "This is especially critical for trials that involve medical management of specific risk factors or conditions, where suspension of the trial could itself have adverse consequences due to reduced engagement with research participants."

For participants already in MS studies, "each upcoming visit is assessed for whether it's critical or could be done virtually or just skipped. If a person needs a treatment that cannot be postponed or skipped, they come in," Jeffrey Cohen, MD, director of the Experimental Therapeutics Program at theMellen Center for Multiple Sclerosis Treatment and Researchat the Cleveland Clinic, Ohio, told Medscape Medical News.

New study enrollment is largely on hold and study visits for existing participants are limited, said Cohen, who is also president of the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS).

Some of the major ongoing trials in MS are "looking at very fundamental questions in the field," Cohen said. The Determining the Effectiveness of earLy Intensive Versus Escalation Approaches for RRMS (DELIVER-MS) and Traditional Versus Early Aggressive Therapy for Multiple Sclerosis (TREAT-MS) trials, for example, evaluate whether treatment should be initiated with one of the less efficacious agents with escalation as needed, or whether treatment should begin with a high-efficacy agent.

Both trials are currently on hold because of the pandemic, as is the Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS) study.

"There has been a lot of interest in hematopoietic stem cell transplants and where they fit into our overall treatment strategy, and this is intended to provide a more definitive answer," Cohen said.

"The pandemic has been challenging" in terms of ongoing MS research, said Benjamin M. Segal, MD, chair of the Department of Neurology and director of the Neuroscience Research Institute at The Ohio State University Wexner Medical Center, Columbus.

"With regard to the lab, our animal model experiments have been placed on hold.We have stopped collecting samples from clinical subjects for biomarker studies.

"However, my research team has been taking advantage of the time that has been freed up from bench work by analyzing data sets that had been placed aside, delving more deeply into the literature, and writing new grant proposals and articles," he added.

Two of Segal's traineesare writing review articles on the immunopathogenesis of MS and its treatment. Another postdoctoral candidate is writing a grant proposal to investigate how coinfection with a coronavirus modulates CNS pathology and the clinical course of an animal model of MS.

"I am asking my trainees to plan out experiments further in advance than they ever have before, so they are as prepared as possible to resume their research agendas once we are up and running again," Segal said.

Confronting current challenges while planning for a future less disrupted by the pandemic is a common theme that emerges.

"The duration of this [pandemic] will dictate how we analyze the data at the end [for the US POINTER study]. There is a large group of statisticians working on this," Snyder said.

Harvard Medical School's Sperling also remains undeterred.

"This is definitely a challenging time, as we must not allow the COVID-19 to interfere with our essential mission to find a successful treatment to prevent cognitive decline in AD. We do need, however, to be asflexible as possible to protect our participants and minimize the impact to our overall study integrity," she said.

Molinuevo Guix, of the Barcelonaeta Brain Research Center, is also determined to continue his AD research.

"I am aware that after the crisis, there will be less [risk] but still a COVID-19 infection risk, so apart from trying to generate part of our visits virtually, we want to make sure we have all necessary safety measures in place. We remain very active to preserve the work we have done to keep up the fight against Alzheimer's and dementia," he said

Such forward thinking also applies to major stroke trials, said University of Cincinnati's Broderick.

"As soon as we shut down enrollment in stroke trials, we immediately began to make plans about how and when we can restart our stroke trials," he explained. "One of our trials can do every step of the trial process remotely without direct in-person interactions and will be able to restart soon."

An individualized approach is needed, Broderick added.

"For trials involving necessary in-person and hands-on assessments, we will need to consider how best to use protective equipment and expanded testing that will likely match the ongoing clinical care and requirements at a given institution.

"Even if a trial officially reopens enrollment, the decision to enroll locally will need to follow local institutional environment and guidelines. Thus, restart of trial enrollment will not likely be uniform, similar to how trials often start in the first place," Broderick added.

The NIH published uniform standards for researchers across its institutes to help guide them during the pandemic.

Future contingency plans also are underway at the NINDS.

"As the pandemic wanes and in-person research activities restart, it will be important to have in place safety measures that prevent a resurgence of the virus, such as proper personal protective equipment for staff and research participants, said Wright, the clinical research director at NINDS.

For clinical trials, NINDS is prepared to provide supplemental funds to trial investigators to help support additional activities undertaken as a result of the pandemic.

"This has been an instructive experience.The pandemic will end, and we will resume much of our old patterns of behavior," said Ohio State's Segal."But some of the strategies that we have employed to get through this time will continue to influence the way we communicate information, plan experiments, and prioritize research activities in the future, to good effect."

Snyder, Sperling, Molinuevo Guix, Elkind, Broderick, Wright, Cohen, and Segal have disclosed no relevant disclosures.

Follow Damian McNamara on Twitter: @MedReporter. For more Medscape Neurology news, join us on Facebook and Twitter.

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Trials and Tribulations: Neurology Research During COVID-19 - Medscape

Into the Lungs and Beyond – Harvard Medical School

This article is part of Harvard Medical Schoolscontinuing coverageof medicine, biomedical research, medical education and policy related to the SARS-CoV-2 pandemic and the disease COVID-19.

What makes SARS-CoV-2, the virus behind COVID-19, such a threat?

A new study in Cell led by researchers at Harvard Medical School, Boston Children's Hospital and MIT pinpoints the likely cell types the virus infects.

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The study also unexpectedly showed that one of the bodys main defenses against viral infections may actually help the virus infect those very cells.

The study, published as a peer-reviewed pre-proof, will help focus efforts to understand what SARS-CoV-2 does in the body, why some people are more susceptible, and how best to search for treatments, the researchers say.

Multiple research models

When news broke about a new coronavirus in China, Jose Ordovas-Montanes, assistant professor of pediatrics at HMS and Boston Childrens, and colleague Alex Shalek at MIT had already been studying different cell types from throughout the human respiratory system and intestine. They also had gathered data from primates and mice.

In February, they began diving into these data.

We started to look at cells from tissues such as the lining of the nasal cavity, the lungs and gut, based on reported symptoms and where the virus has been detected, said Ordovas-Montanes, who is co-senior author of the new study along with Shalek. We wanted to provide the best information possible across our entire spectrum of research models.

COVID-19-susceptible cells

Recent research had found that SARS-CoV-2, like the closely related SARS-CoV that caused the SARS pandemic, uses a receptor called ACE2 to gain entry into human cells, aided by an enzyme called TMPRSS2.

That led Ordovas-Montanes, Shalek and colleagues to ask a simple question: Which cells in respiratory and intestinal tissue express both ACE2 and TMPRSS2?

To get the answer, the team turned to single-cell RNA sequencing. This identifies which of roughly 20,000 genes are on in individual cells.

They found that only a tiny percentage of human respiratory and intestinal cellsoften well below 10 percentmake both ACE2 and TMPRSS2.

Those cells fall into three types: goblet cells in the nose that secrete mucus; lung cells known as type II pneumocytes that help maintain the alveoli (the sacs where oxygen is taken in); and one type of so-called enterocytes that line the small intestine and are involved in nutrient absorption.

Sampling from non-human primates showed a similar pattern of susceptible cells.

Many existing respiratory cell lines may not contain the full mix of cell types, and may miss the types that are relevant, said Ordovas-Montanes. Once you understand which cells are infected, you can start to ask, How do these cells work? Is there anything within these cells that is critical for the viruss life cycle?

"With more refined cellular models, we can perform better screens to find what existing drugs target that biology, providing a stepping stone to go into mice or non-human primates.

Interferon: Helpful or harmful?

But it was the studys second finding that most intrigued the scientists.

They discovered that the ACE2 gene, which encodes the receptor SARS-CoV-2 uses to enter human cells, is stimulated by interferonone of the bodys main defenses when it detects a virus.

Interferon actually turned on the ACE2 gene at higher levels, potentially giving the virus new portals to get in.

ACE2 is also critical in protecting people during various types of lung injury, said Ordovas-Montanes. When ACE2 comes up, thats usually a productive response. But since the virus uses ACE2 as a target, we speculate that it might be exploiting that normal protective response.

Interferons, in fact, are being tested as a treatment for COVID-19. Whether they would help or do more harm than good is not yet clear.

It might be that in some patients, because of the timing or the dose, interferon can contain the virus, while in others, interferon promotes more infection, said Ordovas-Montanes. We want to better understand where the balance lies, and how we can maintain a productive antiviral response without producing more target cells for the virus to infect.

ACE inhibitors and cytokine storms

The findings may also raise new lines of inquiry around ACE inhibitors. These drugs are commonly used to treat hypertension, which has been linked to more severe COVID-19 disease. Are ACE inhibitors affecting peoples risk?

ACE and ACE2 work in the same pathway, but they actually have different biochemical properties, Ordovas-Montanes said. Its complex biology, but it will be important to understand the impact of ACE inhibitors on peoples physiological response to the virus.

Its also too soon to try to relate the study findings to the cytokine storm, a runaway inflammatory response that has been reported in very sick COVID-19 patients.

Cytokines are a family of chemicals that rally the bodys immune responses to fight infections. Interferon is part of the family.

It might be that were seeing a cytokine storm because of a failure of interferon to restrict the virus to begin with, so the lungs start calling for more help," he said. "Thats exactly what were trying to understand right now.

Future directions

In addition, the team wants to explore what SARS-CoV-2 is doing in the cells it targets and to study tissue samples from children and adults to understand why COVID-19 is typically less severe in younger people.

Carly Ziegler, Samuel Allon and Sarah Nyquist of MIT and Harvard and Ian Mbano of the Africa Health Research Institute were co-first authors on the paper. The study was done in collaboration with the Human Cell Atlas Lung Biological Network.

This has been an incredible community effort not just within Boston, but also with collaborators around the world who have shared their unpublished data to try and make potentially relevant information available as rapidly as possible, said Shalek. Its inspiring to see how much can be accomplished when everyone comes together to tackle a problem.

This work was supported in part by the National Institutes of Health (U24AI118672, AI201700104, R56AI139053, R01GM081871, T32GM007753, AI078908, HL111113, HL117945, R37AI052353, R01AI136041, R01HL136209, U19AI095219, U19HL129902, UM1AI126623, U19AI051731, R01HL095791, R33AI116184, U19AI117945, UM1AI126617), Bill and Melinda Gates Foundation, MIT Stem Cell Initiative through Foundation MIT, Aeras Foundation, Damon Runyon Cancer Research Foundation (DRG-2274-16), Richard and Susan Smith Family Foundation, UMass Center for Clinical and Translational Science Project Pilot Program, Office of the Assistant Secretary of Defense for Health Affairs (W81XWH-15-1-0317), P.B. Fondation pour la Recherche Medicale (DEQ20180339158) and Agence Nationale pour la Recherche (ANR-19-CE14-0027).

Adapted from a post in Discoveries, the Boston Children's clinical and research innovation portal.

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Into the Lungs and Beyond - Harvard Medical School

Five UC San Diego Professors Elected to National Academy of Sciences – UC San Diego Health

Clockwise from top left, Dmitri Basov, Lawrence Goldstein, Terence Hwa, Clifford Kubiak, Kimberly Prather

The National Academy of Sciences elected five professors affiliated with the University of California San Diego to membership in the prestigious National Academy of Sciences, one of the highest honors bestowed on U.S. scientists and engineers.

UC San Diego faculty members Dmitri Basov, Lawrence Goldstein, Terence Hwa, Clifford Kubiak, and Kimberly Prather whose work spans fields ranging from medicine and biological sciences to atmospheric chemistry and physics were recognized Monday in recognition of their distinguished and continuing achievements in original research, according to the Academy. They were among 120 American scientists and 26 international members named this year.

For a young institution such as ours, having five professors inducted into the National Academy of Sciences speaks volumes of the innovative and visionary nature of this university and our well-respected and accomplished faculty, said UC San Diego Chancellor Pradeep K. Khosla. I am proud to see the career accomplishments of these five professors recognized on such a distinguished national platform, alongside the countrys other leading researchers.

This brings the total number of National Academy of Sciences members from UC San Diego to 86.

Dmitri Basov is an affiliated UC San Diego professor in the Department of Physics, where he served as chair between 2010 and 2015. He is also a Higgins professor in the Department of Physics at Columbia University, where he is the principal investigator of the Basov Infrared Laboratory, the director of the DOE Energy Frontiers Research Center on Programmable Quantum Materials and co-director of the Max Planck Society New York Center for Nonequilibrium Quantum Phenomena. His research interests include physics of quantum materials, superconductivity, two-dimensional materials and infrared nano-optics. Basov has received numerous prizes and awards including a Sloan Fellowship (1999), the Genzel Prize (2014), a Humboldt research award (2009), the Frank Isakson Prize, American Physical Society (2012), Moore Investigator (2014), the K.J. Button Prize (2019) and the Vannevar Bush Faculty Fellowship (U.S. Department of Defense, 2019).

Basov earned his PhD at the Lebedev Physical Institute of the Russian Academy of Sciences (1991). He served as postdoctoral research associate at McMaster University (1992-96) and as an assistant physicist at Brookhaven National Laboratory (1996) before joining UC San Diego.

Lawrence Goldstein, PhD, is Distinguished Professor in the Department of Cellular and Molecular Medicine and Department of Neurosciences in the UC San Diego School of Medicine. He founded and directed the UC San Diego Stem Cell Program and the Sanford Stem Cell Clinical Center at UC San Diego Health and is founding scientific director of the Sanford Consortium for Regenerative Medicine. He was instrumental in the development and passage of Proposition 71 in 2004, which created an unprecedented $3 billion fund and infrastructure for stem cell medical research in California.

For more than 25 years, Goldsteins research focus has been to unravel how molecular motors interact with and control the behavior of axonal vesicles in neurons, and how defects in these processes underlie neurological conditions, such as Alzheimers disease (AD).In 2012, his lab was the first to create stem cell-derived in vitro neurons of sporadic and hereditary AD, giving researchers a much-needed method for studying the diseases causes and pathologies and a new tool for developing and testing drugs to treat a disorder that afflicts 5.4 million Americans.

More recently, this work has led to the identification of new cellular targets in AD drug development and a deeper understanding of AD genetics and disease progression. He is among the nations leading scientific figures in promoting AD research and evidence-based treatments.

Terence Hwa is the Presidential Chair and Distinguished Professor in the Department of Physics with a joint appointment in the Division of Biological Sciences. Trained in theoretical physics, Hwa launched a biology wet-lab 15 years ago and developed a unique quantitative approach to studying bacterial physiology. During this time, the Hwa Research Group established a number of bacterial growth laws and formulated a principle of proteomic resource allocation. This line of study culminated in a theory of bacterial growth control, accurately predicting bacterial behaviors and gene expression for a variety of environmental and genetic perturbations, and resolving a number of long-standing mysteries in microbiology. Hwas research team continues to extend its quantitative approaches to characterize bacterial species singly and in consortium, to uncover underlying principles governing the spatiotemporal dynamics of microbial communities.

Hwa is a champion of interdisciplinary research. In 2001, he launched an extended program at the Kavli Institute of Theoretical Physics in Santa Barbara, which has been regarded as a watershed event in bringing physicists to post-genome biology. He is also the founder and co-director of the Quantitative Biology specialization program at UC San Diego. Hwa received fellowships and awards from the Sloan, Beckman, Guggenheim and Burroughs-Wellcome Foundations, and is a Fellow of the American Physical Society and the American Academy of Microbiology. Hwa received his PhD in physics from MIT. After postdoctoral research at Harvard University in condensed-matter physics, he joined UC San Diegos physics faculty in 1995.

Clifford Kubiak is a Distinguished Professor and former chair of the Department of Chemistry and Biochemistry, who holds the Harold C. Urey Chair in Chemistry. His Kubiak Research Group at UC San Diego is especially known for its work on developing catalysts for the electrochemical reduction of carbon dioxide. Kubiak is also a fellow of the American Academy of Arts and Sciences and the American Chemical Society (ACS). He has received several awards including the prestigious ACS Award in Organometallic Chemistry (2018), the Tolman Medal (2018), the Basolo Medal for Outstanding Research in Inorganic Chemistry (2015), the Inter-American Photochemical Society, Award in Photochemistry (2013) and the ACS Award in Inorganic Chemistry (2012). Kubiak has held visiting appointments at Tohoku University, University of Chicago and University of Erlangen, and he was a visiting associate in chemistry at the Joint Center for Artificial Photosynthesis at Caltech. He has served on the Editorial Advisory Boards of Accounts of Chemical Research, Inorganic Chemistry and Materials Science in Semiconductor Processing. He is the author of more than 290 scientific articles.

Before joining UC San Diego in 1998, Kubiak was a faculty member at Purdue University (1982-98). Before that he was a postdoctoral associate with Mark S. Wrighton at MIT (1980-81). He received his PhD in chemistry from the University of Rochester (1980), where he worked with Richard Eisenberg.

Kimberly Prather is a Distinguished Professor who holds a joint appointment between UC San Diegos Scripps Institution of Oceanography and the Department of Chemistry and Biochemistry. Prathers research focuses on understanding the influence of atmospheric aerosols on clouds, human health, and climate. Early in her career, she developed a technique known as aerosol time-of-flight mass spectrometry that is widely used in atmospheric field studies around the world to determine the origin and chemistry of aerosols. She is the founding director of the National Science Foundation Center for Aerosol Impacts on Chemistry of the Environment (CAICE), the largest federally funded center in the history of UC San Diego. CAICE researchers replicate ocean/atmosphere interactions in a laboratory setting to study the influence of ocean biology on atmospheric chemistry, clouds, and climate.

Prather joined UC San Diego in 2001. She was elected as a member of the American Academy of Arts and Sciences and a fellow of the American Geophysical Union in 2010. In 2019, she became the first woman at UC San Diego to be elected as a member of the National Academy of Engineering. Previously this year, she won the 2020 Frank H. Field and Joe L. Franklin Award for Outstanding Achievement in Mass Spectrometry from the American Chemical Society. She received her PhD in chemistry from the University of California, Davis.

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Five UC San Diego Professors Elected to National Academy of Sciences - UC San Diego Health

Suffering from ED? Discover how our combined treatments of Hormone Balancing and FDA Approved Acoustic Wave Pulse Therapy Ends Your ED – Magazine of…

More than 30 million men are suffering from impotence or erectile dysfunction in the United States.Many men find it difficult to discuss a sexual health problem such as ED. However, its important to discuss your ED openly and honestly with your doctor.Complications resulting from erectile dysfunction can include: an unsatisfactory sex life, stress or anxiety, embarrassment or low self-esteem, relationship problems, the inability to get your partner pregnant.TreatmentsMany men prefer taking pills instead of surgery. Pills are the short-term solution.Valencia Medical Center offers new medical breakthrough procedure that leads to more long-term treatment for erectile dysfunction. Using acoustic wave therapy to repair blood vessels to the genitals.Treatment principals:Initial office visit evaluates your condition and order a comprehensive blood testing including hormone test. The result indicates if any hormonal imbalance such as testosterone exist.The first part of the treatment plan is to restore the hormone deficiency which is essential to recovery of sexual function. Hormone Pellet Therapy is the most effective restoration to last up to six months. Just one treatment every six months to keep balanced. The next step to treat your ED is three to several sessions of 20 to 30 minutes of new acoustic wave therapy, each a few days apart. It is mostly without discomfort, and non-surgical. Many patients show satisfactory improvement in their sexual functions. Patients upon each treatment can continue their normal activities.Stem Cell Therapy using your blood to use growth factor in your stem cells and injected. This procedure has been effective and restores normal functions. Valencia Medical Center offers Care Credit and in-house financing. For more information, please contact Valencia Medical Center at 661-222-9117. The office is located at 24159 Magic Mountain Parkway in Valencia.

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Suffering from ED? Discover how our combined treatments of Hormone Balancing and FDA Approved Acoustic Wave Pulse Therapy Ends Your ED - Magazine of...

BrainStorm-Cell Therapeutics to Announce First Quarter Financial Results and Provide a Corporate UpdateThursday, May 7, 2020, 8:30 am EDT – BioSpace

NEW YORK, April 29, 2020 (GLOBE NEWSWIRE) -- BrainStorm-Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in developing innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, announced today, that the Company will hold a conference call to update shareholders on financial results for the first quarter ended March 31, 2020, and provide a corporate update, at 8:30 a.m, Eastern Daylight Time, on Thursday, May 7, 2020.

BrainStorms CEO, Chaim Lebovits, will present a corporate update, after which, participant questions will be answered. Joining Mr. Lebovits to answer investment community questions will be Ralph Kern, MD, MHSc, President and Chief Medical Officer, David Setboun, PhD, MBA, Executive Vice President and Chief Operating Officer and Preetam Shah, PhD, MBA, Executive Vice President and Chief Financial Officer.

Participants are encouraged to submit their questions prior to the call by sending them to: q@brainstorm-cell.com. Questions should be submitted by 5:00 p.m. EDT, Tuesday, May 5, 2020.

Teleconference Details BRAINSTORM CELL THERAPEUTICS 1Q 2020

The investment community may participate in the conference call by dialing the following numbers:

Those interested in listening to the conference call live via the internet may do so by visiting the "Investors & Media" page of BrainStorm's website at http://www.ir.brainstorm-cell.com and clicking on the conference call link.

Those that wish to listen to the replay of the conference call can do so by dialing the numbers below. The replay will be available for 14 days.

ABOUT NUROWNNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

ABOUT BRAINSTORM CELL THERAPEUTICS INC.:BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement as well as through its own patents, patent applications and proprietary know-how. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

SAFE HARBOR STATEMENT:Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTSInvestor Relations:Preetam Shah, MBA, PhDChief Financial OfficerBrainStorm Cell Therapeutics Inc.Phone: + 1.862.397.1860pshah@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

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BrainStorm-Cell Therapeutics to Announce First Quarter Financial Results and Provide a Corporate UpdateThursday, May 7, 2020, 8:30 am EDT - BioSpace

Healing the heart by returning it to its infancy – FierceBiotech

Nearly a decade ago, researchers at UT Southwestern Medical Center discovered that when mouse hearts were damaged in the first seven days of life, they would regenerate. They reasoned that if they could find a way to recreate that regenerative ability later in life, it might provide a new way to treat heart damage.

Now, that same team has discovered that a protein called calcineurin plays a key role in blocking the ability of heart muscle to regenerate after the first week of life. The discovery could be used to develop treatments that reverse this process, in essence returning the heart to its developmental stage, they reported in the journal Nature.

The discovery builds on previous work at UT Southwestern that focused on the protein Meis1, a transcription factor that prevents heart cells from dividing. When the researchers deleted the gene in mice that makes that protein, their cardiomyocytes continued to divide after the first week of life. But the effect was transient.

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This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

RELATED: Stem cells don't repair injured hearts, but inflammation might, study finds

Then the researchers discovered that another protein called Hoxb13 was also key, because it shuttles Meis1 into the cell nucleus. So they deleted the genes for both Meis1 and Hoxb13 in adult mice to see what would happen after a heart attack.

It worked. The ability of the animals hearts to pump blood quickly returned to near-normal levels, they said. Even though the mice were adults, their hearts looked much like they would in animals that were still developing.

After a series of further experiments, the UT Southwestern scientists discovered that calcineurin regulates both Hoxb13 and Meis1. Inhibiting calcineurin prolongs the window of cardiomyocyte proliferation, they wrote in the study.

The idea of treating heart damage by turning back the clock isnt new. In fact, several research teams have tried using stem cells to repair damaged heart tissue. But those efforts have been disappointing so far.

Last year, a team from the Cincinnati Children's Hospital Medical Center tracked stem cells injected into the hearts of mice and concluded that it was not the cells themselves, but rather their ability to activate macrophage cells from the immune system that promoted healing. That led the researchers to suggest that efforts to regenerate the heart focus less on stem cells and more on other processes in the body that might promote healing.

The discovery of calcineurins role in regulating the regeneration of the heart is notable due to the fact that there are already drugs on the market that target the protein. Thats because calcineurin plays a role in a variety of diseases, including rheumatoid arthritis and diabetes. Testing these drugs, either individually or in combination, and developing new medicines that target calcineurin directly could offer new strategies for repairing hearts damaged by heart attacks, high blood pressure, viruses and more, suggested co-author Hesham Sadek, M.D., Ph.D., a professor of internal medicine, molecular biology and biophysics at UT Southwestern.

"By building up the story of the fundamental mechanisms of heart cell division and what blocks it, Sadek said in a statement, we are now significantly closer to being able to harness these pathways to save lives.

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Healing the heart by returning it to its infancy - FierceBiotech

What Is The Economic Value of Receiving CAR T-Cell Therapy in an Outpatient Setting? – AJMC.com Managed Markets Network

Chimeric antigen receptor (CAR) T-cell therapy has been shown to improve health-related quality of life in patients with relapsed/refractory diffuse large B-cell lymphoma (LBCL). Currently, CAR T-cell therapies are primarily administered in inpatient settings. In a study published in JAMA Network Open, researchers found CAR T-cell therapy administered to patients with relapsed or refractory LBCL in outpatient settings was associated with lower estimated overall costs.

In a study published in JAMA Network Open, researchers found CAR T-cell therapy administered to patients with relapsed or refractory LBCL in outpatient settings was associated with lower estimated overall costs. CAR-T cell therapies also hold promise for patients with hematologic malignant neoplasms that are unresponsive or resistant to standard treatments, researchers said.

The treatment involves harvesting and reengineering an individuals own cells to attack specific malignant cells. CAR-T cells were initially developed using knowledge gleaned from allogeneic stem cell transplantsthat donor mature immune cells can attack healthy cells in the recipient patient.

In an economic evaluation, researchers used a decision-tree model to document patient clinical outcomes and costs, using only hypothetical patients and facilities. Excluding the CAR T-cell acquisition cost, researchers found hospitalization and office visits comprised 65.3% of the costs in inpatient settings and 48.4% of the costs in outpatient settings. Specifically, outpatient administration of CAR T-cell therapy in nonacademic specialty oncology networks was associated with a $32,987 (40.4%) reduction in total costs. Sensitivity analyses were carried out to address assumptions made to build the model.

Before the approval of CAR T-cell therapy, the available treatments for patients with relapsed or refractory LBCL included high-dose chemotherapy, salvage chemotherapy, and autologous hematopoietic stem cell transplantation (auto-HSCT); however, the prognosis after these treatments is often poor, authors said.

Researchers analyzed a predefined period from lymphodepletion to 30 days after the receipt of CAR T-cell infusion, in order to account for potential incidences of adverse events. Data were collected from several sources including theHealthcare Cost and Utilization Project National Inpatient Sample and the Medicare Hospital Outpatient Prospective Payment System. Investigators used secondary literature to inform model inputs.

Total cost of therapy included any costs associated with lymphodepletion, acquisition and infusion of CAR T-cells and management of acute adverse events.

The model also showed:

In this scenario, the model found patients who received CAR T-cell therapy in a nonacademic specialty oncology network setting would save $27,294 compared with the inpatient setting. In addition, in the scenario analysis the decrease in incremental cost reductions was associated with a lower overall incidence of AEs, which reduced the consequences of associated AE management costs, while the monitoring required at baseline was held constant.

The results indicate CAR T-cell therapy with a better safety profile may be more economical and could further leverage the outpatient site of care, researchers said. They concluded, The potential availability of CAR T-cell therapies with lower AE rates that are suitable for outpatient administration may reduce the total costs of care.

One limit to the study highlighted by the authors is the fact that outpatient administration may not be an option for all patients with LBCL.Eligibility can be contingent on the patients health status, support system, or the availability of housing near hospitals.

However, some specialists predict a gradual shift of all CAR T-cell therapies to the outpatient setting. Part of the reason for that [shift] is the way that it's reimbursed as an outpatient therapy is slightly more favorableso, financially it is better to do that, said John Sweetenham, MD, professor in the Department of Internal Medicine at UT Southwestern Medical Center and the Associate Director for Clinical Affairs at UTSWs Harold C. Simmons Comprehensive Cancer Center.

He continued, The problem is that these are very toxic treatments, and that many of the patients are not going to be manageable in the outpatient setting; but I do see that as one of the factors which is influencing a slow transition to outpatient CAR-Ts.

Expanded access to novel therapies in immune-oncology (IO) like CAR T-cell therapy also remains a priority among community level oncologists. Reimbursement, operational, and medical challenges associated with cellular therapy inhibit widespread uptake of the therapy, explained Lee Schwartzberg, MD, FACP, chief medical officer and board member at OneOncology. Only a small number of patients, frankly, have been treated with the first-generation CAR-T cells. So we need to develop new technologies and new operational models to do this, he said in an interview with The American Journal of Managed Care.

Reference

Lyman GH, Nguyen A, Snyder S, et al. Economic evaluation of chimeric antigen receptor T-cell therapy by site of care among patients with relapsed or refractory large B-cell lymphoma [published online April 6, 2020]. JAMA Netw Open. doi: 10.1001/jamanetworkopen.2020.2072.

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What Is The Economic Value of Receiving CAR T-Cell Therapy in an Outpatient Setting? - AJMC.com Managed Markets Network