Category Archives: Stem Cell Medical Center


Cell Therapy Global Market Report 2021: COVID-19 Growth and Change to 2030 – ResearchAndMarkets.com – Business Wire

DUBLIN--(BUSINESS WIRE)--The "Cell Therapy Global Market Report 2021: COVID-19 Growth and Change to 2030" report has been added to ResearchAndMarkets.com's offering.

The global cell therapy market is expected to grow from $7.2 billion in 2020 to $7.82 billion in 2021 at a compound annual growth rate (CAGR) of 8.6%.

Major players in the cell therapy market are Fibrocell Science Inc., JCR Pharmaceuticals Co. Ltd., PHARMICELL Co. Ltd., Osiris Therapeutics Inc., MEDIPOST, Vericel Corporation, Anterogen Co. Ltd., Kolon TissueGene Inc., Stemedica Cell Technologies Inc., and AlloCure.

The growth is mainly due to the companies resuming their operations and adapting to the new normal while recovering from the COVID-19 impact, which had earlier led to restrictive containment measures involving social distancing, remote working, and the closure of commercial activities that resulted in operational challenges. The market is expected to reach $12.06 billion in 2025 at a CAGR of 11%.

The cell therapy market consists of sales of cell therapy and related services. Cell therapy (CT) helps repair or replace damaged tissues and cells. A variety of cells are used for the treatment of diseases includes skeletal muscle stem cells, hematopoietic (blood-forming) stem cells (HSC), lymphocytes, mesenchymal stem cells, pancreatic islet cells, and dendritic cells.

The cell therapy market covered in this report is segmented by technique into stem cell therapy; cell vaccine; adoptive cell transfer (ACT); fibroblast cell therapy; chondrocyte cell therapy. It is also segmented by therapy type into allogeneic therapies; autologous therapies, by application into oncology; cardiovascular disease (CVD); orthopedic; wound healing; others.

The high cost of cell therapy hindered the growth of the cell therapy market. Cell therapies have become a common choice of treatment in recent years as people are looking for the newest treatment options. Although there is a huge increase in demand for cell therapies, they are still very costly to try. Basic joint injections can cost about $1,000 and, based on the condition, more specialized procedures can cost up to $ 100,000. In 2020, the average cost of stem cell therapy can range from $4000 - $8,000 in the USA. Therefore, the high cost of cell therapy restraints the growth of the cell therapy market.

Key players in the market are strategically partnering and collaborating to expand the product portfolio and geographical presence of the company. For instance, in April 2018, Eli Lilly, an American pharmaceutical company entered into a collaboration agreement with Sigilon Therapeutics, a biopharmaceutical company that focused on the discovery and development of living therapeutics to develop cell therapies for type 1 diabetes treatment by using the Afibromer technology platform.

Similarly, in September 2018, CRISPR Therapeutics, a biotechnological company that develops transformative medicine using a gene-editing platform for serious diseases, and ViaCyte, a California-based regenerative medicine company, collaborated on the discovery, development, and commercialization of allogeneic stem cell therapy for diabetes treatment.

In August 2019, Bayer AG, a Germany-based pharmaceutical and life sciences company, acquired BlueRock Therapeutics, an engineered cell therapy company, for $1 billion. Through this transaction, Bayer AG will acquire complete BlueRock Therapeutics' CELL+GENE platform, including a broad intellectual property portfolio and associated technology platform including proprietary iPSC technology, gene engineering, and cell differentiation capabilities. BlueRock Therapeutics is a US-based biotechnology company focused on developing engineered cell therapies in the fields of neurology, cardiology, and immunology, using a proprietary induced pluripotent stem cell (iPSC) platform.

The rising prevalence of chronic diseases contributed to the growth of the cell therapy market. According to the US Centers for Disease Control and Prevention (CDC), chronic disease is a condition that lasts for one year or more and requires medical attention or limits daily activities or both and includes heart disease, cancer, diabetes, and Parkinson's disease.

Stem cells can benefit the patients suffering from spinal cord injuries, type 1 diabetes, Parkinson's disease (PD), heart disease, cancer, and osteoarthritis. According to Cancer Research UK, in 2018, 17 million cancer cases were added to the existing list, and according to the International Diabetes Federation, in 2019, 463 million were living with diabetes.

According to the Parkinson's Foundation, every year, 60,000 Americans are diagnosed with PD, and more than 10 million people are living with PD worldwide. The growing prevalence of chronic diseases increased the demand for cell therapies and contributed to the growth of the market.

Key Topics Covered:

1. Executive Summary

2. Cell Therapy Market Characteristics

3. Cell Therapy Market Trends And Strategies

4. Impact Of COVID-19 On Cell Therapy

5. Cell Therapy Market Size And Growth

5.1. Global Cell Therapy Historic Market, 2015-2020, $ Billion

5.2. Global Cell Therapy Forecast Market, 2020-2025F, 2030F, $ Billion

6. Cell Therapy Market Segmentation

6.1. Global Cell Therapy Market, Segmentation By Technique, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

6.2. Global Cell Therapy Market, Segmentation By Therapy Type, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

6.3. Global Cell Therapy Market, Segmentation By Application, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

7. Cell Therapy Market Regional And Country Analysis

7.1. Global Cell Therapy Market, Split By Region, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

7.2. Global Cell Therapy Market, Split By Country, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/719lux

Read the original here:
Cell Therapy Global Market Report 2021: COVID-19 Growth and Change to 2030 - ResearchAndMarkets.com - Business Wire

UM School Of Medicine Researchers Receive NIH Avant Garde Award For Out-Of-Box, Innovative Concept To Cure HIV And Treat Co-Existing Addiction -…

Newswise University of Maryland School of Medicine (UMSOM) Professor of Diagnostic Radiology & Nuclear Medicine, Linda Chang, MD, MS, received the National Institute on Drug Abuse (NIDA) 2021 Avant Garde Award (DP1) for HIV/AIDS and Substance Use Disorder Research a National Institutes of Health (NIH) Directors Pioneer Award. This prestigious award supports researchers with exceptional creativity, who propose high-impact research with the potential to be transformative to the field. Her proposed project will involve a team of experts in brain imaging, infectious diseases, addiction, animal research, and gene-editing technology with the goal to essentially eradicate all traces of HIV from the body, and treat commonly co-existing substance use disorders. 2021 Avant Garde Awardees are expected to receive more than $5 million over five years.

I am extremely pleased, and feel very fortunate to have received this award, says Dr. Chang, who has a secondary appointment in the Department of Neurology at UMSOM. This project takes my work in a new direction. I believe my track record of being able to work across multiple disciplines with various researchers to initiate new areas of research and getting good results, along with the outstanding collaborators and resources at UMB, gave the proposal reviewers confidence that my team and I can significantly advance this new project.

About 38 million people around the world live with HIV, according to the Centers for Disease Control and Prevention. Although antiretroviral therapies can treat HIV to the point of undetectable viral levels and lead to long, healthy lifespans, these medications must be taken for life to prevent a resurgence, as HIV can hide from these drugs by integrating copies of itself into a persons genome. Once the drugs are stopped, the virus can reemerge.

From start to finish, Dr. Changs plan is to remove HIV from the genome, even in tough to reach spots like the brain, get more of the antiretroviral therapies into the brain, and stimulate the reward system in the brain to reduce drug cravings. The work will start out in mice before it can be tested in people.

Dr. Chang plans to use the gene-editing technology known as CRISPR to cut out copies of the hidden HIV genes in the genomes of mice, so they can be eradicated by antiretroviral drugs.

However, getting the CRISPR therapy into the brain can be difficult because of the blood-brain barrier, which protects the brain from infectious bacteria and foreign substances. The blood-brain barrier also prevents antiretroviral drugs from reaching high enough concentrations in the brain and central nervous system to effectively destroy HIV.

To seek out HIV in the brain, Dr. Chang and her team will temporarily disrupt the blood-brain barrier to allow more of the antiretroviral drugs or the CRISPR compounds to cross over the blood-brain barrier using an unique resource at the University of Marylandthe MRI-guided focused ultrasound system. This technique uses the MRI scan to help guide 2,000 pinpointed beams of high energy sound waves, along with microscopic bubbles, to non-invasively and temporarily open an area of the brain with the goal of eliminating the hidden reservoirs of virus in the brains immune cells.

About half of the people with HIV use substances, like drugs or alcohol, or have substance use disorders. Even tobacco or cannabis use in people with HIV is at 2-3 times that of the general population. Together with Victor Frenkel, PhD, an Associate Professor in the Department of Radiology and the Director of Translational Focused Ultrasound, and Donna Calu, PhD, Assistant Professor in the Department of Anatomy and Neurobiology, Dr. Chang will use low energy MR-guided focused ultrasound to suppress brain activity in the reward center of the brain, the nucleus accumbens. They hope this approach will suppress drug cravings in people with HIV who have substance use disorders.

The different components of this project will first be tested in mouse or rat models before moving onto clinical studies. As HIV does not normally infect mice, researchers use humanized mice that have weak immune systems, which are replaced with human blood stem cells that become human immune cells that can be infected with HIV. Although these humanized mice make lots of T cells a main cell for HIV infectionthey dont make the immune cells that HIV uses to hide in the brain, known as microglia. Recently, Dr. Changs collaborator Howard E. Gendelman, MD, Margaret R. Larson Professor of Internal Medicine and Infectious Diseases Chair at University of Nebraska Medical Center, and his lab created a modified humanized mouse that has an extra human gene that allows the human blood stem cells to now make microglia.

These new mice mean that these experiments can be done in a fraction of the time and cost and without the other hurdles that come along with using non-human primates, which are the only other animal that a special strain of HIV can infect, says collaborator Alonso Heredia, PhD, Associate Professor of Medicine and scientist at UMSOMs Institute of Human Virology.

He adds, There have been many attempts to eradicate HIV in the body, and it is thought they have not been successful, in part because we cannot get to the HIV reservoirs in the brain. If this works, we will be much closer to a practical cure for HIV. Dr. Heredia will be collaborating with Dr. Chang on this project using HIV-infected humanized mice that he has developed for his other ongoing projects.

For the addiction studies, Dr. Changs team will use the expertise and rodent models of addiction developed and optimized by Mary Kay Lobo, PhD, Professor of Anatomy and Neurobiology, and Dr. Calu. The mice will self-administer fentanyl, a powerful, synthetic opioid.

Dr. Frenkel and Dheeraj Gandhi, MBBS, Professor of Diagnostic Radiology and Nuclear Medicine and Clinical Director of Center of Metabolic Imaging and Therapeutics at UMSOM, are the teams MRI-guided focused ultrasound and clinical research experts.

My hearty congratulations to Dr. Chang and her colleagues and collaborators. If anything is called cutting edge this work surely qualifies for that praise. We wish this group all the success possible, said Robert C. Gallo, MD, The Homer & Martha Gudelsky Distinguished Professor in Medicine, Co-Founder and Director, Institute of Human Virology (IHV), University of Maryland School of Medicine, a Global Virus Network (GVN) Center of Excellence, and GVN Co-Founder and International Scientific Advisor.

Dr. Chang is an expert in using brain imaging to study how HIV or drug use affect the brain in adults and during adolescence, and how exposure to drugs in the womb affects childhood development. She has also conducted clinical trials for treating HIV-associated cognitive disorders and substance use disorders.

Dr. Chang joined UMSOM in 2017 through the Deans initiative Special Trans-Disciplinary Recruitment Award Program (STRAP). The STRAP Initiative was part of UMSOM's multi-year research strategy ACCEL-Med (Accelerating Innovation and Discovery in Medicine) to increase the quality and reputation of clinical and basic science research bringing UMSOM among other top-tier medical research schools.

Dr. Changs arrival to UMSOM spurred the exact kind of collaborative efforts we had hoped to foster through our recruitment program in order to accelerate discoveries, treatments and cures for the worlds most pressing diseases, says E. Albert Reece, MD, PhD, MBA, Executive Vice President for Medical Affairs, UM Baltimore, and the John Z. and Akiko K. Bowers Distinguished Professor and Dean, UMSOM.I look forward to following her teams progress on this ambitious project in the hope that one day we can eradicate HIV.

Dr. Chang served on the National Advisory Council on Drug Abuse for NIDA and is a current member on the Council of Councils at the NIH.

Now in its third century, the University of Maryland School of Medicine was chartered in 1807 as the first public medical school in the United States. It continues today as one of the fastest-growing, top-tier biomedical research enterprises in the world -- with 45 academic departments, centers, institutes, and programs; and a faculty of more than 3,000 physicians, scientists, and allied health professionals, including members of the National Academy of Medicine and the National Academy of Sciences, and a distinguished two-time winner of the Albert E. Lasker Award in Medical Research.

With an operating budget of more than $1.2 billion, the School of Medicine works closely in partnership with the University of Maryland Medical Center and Medical System to provide research-intensive, academic and clinically-based care for nearly 2 million patients each year. The School of Medicine has more than $563 million in extramural funding, with most of its academic departments highly ranked among all medical schools in the nation in research funding. As one of the seven professional schools that make up the University of Maryland, Baltimore campus, the School of Medicine has a total population of nearly 9,000 faculty and staff, including 2,500 student trainees, residents, and fellows.

The combined School of Medicine and Medical System (University of Maryland Medicine) has an annual budget of nearly $6 billion and an economic impact of more than $15 billion on the state and local community. The School of Medicine, which ranks as the 8th highest among public medical schools in research productivity, is an innovator in translational medicine, with 600 active patents and 24 start-up companies. The School of Medicine works locally, nationally, and globally, with research and treatment facilities in 36 countries around the world. Visitmedschool.umaryland.edu

Continue reading here:
UM School Of Medicine Researchers Receive NIH Avant Garde Award For Out-Of-Box, Innovative Concept To Cure HIV And Treat Co-Existing Addiction -...

Antengene Announces Acceptance of IND Application in China for the Phase II Clinical Trial of Single-Agent Selinexor for the Treatment of…

SHANGHAI and HONG KONG, July 5, 2021 /PRNewswire/ -- Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in-class therapeutics in hematology and oncology, recently announced that China's National Medical Products Administration (NMPA) has accepted the Investigational New Drug (IND) application for single agent selinexor, a first-in-class orally available Exportin 1 (XPO1) inhibitor, for the treatment of patients with myelofibrosis (MF) in China.

MF is a clonal hematologic neoplasm which can emerge either as primary MF, polycythemia vera (PV) or essential thrombocythemia (ET)[1]. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is currently the only curative treatment for MF. However, such treatment is difficult to carry out and has a low rate of success. According to the National Comprehensive Cancer Network (NCCN) Guidelines for the Treatment of MF, patients with intermediate-2 or high-risk MF ineligible for allo-HSCT and with a platelet count of 50109/L should be treated with ruxolitinib or fedratinib, while there are few follow-on treatment alternatives for patients failed or resistant to ruxolitinib. At present, only ruxolitinib has been approved for clinical treatment in China, and as a result, MF remains a disease with limited treatment options, representing an urgent unmet medical need.

This randomized, open-label, multicenter Phase II study is designed to evaluate the safety and efficacy of selinexor versus physician's choice (PC) in patients with MF who had at least six months of treatment with a JAK1/2 inhibitor. Approximately 112 patients with MF from 75 trial centers across the world will be randomized in a 1:1 ratio into one of the two treatment arms.

"This acceptance by the NMPA of the IND application for the China study of selinexor in patients with MF marks another major step forward in our effort to develop selinexor into a novel cancer drug. It also paves the way for our on-going exploration of additional indications for Antengene's novel assets," said Dr. Jay Mei, Founder, Chairman and CEO of Antengene. "We are hopeful that, through this novel drug candidate with strong potential in this disease, coupled with our deep expertise in the field of hematologic malignancies, we will be able to bring renewed hope to patients with MF in China. Moving forward, we will work closely with the NMPA to advance this trial in China, and strive to bring this innovative therapeutic to patients in the region and beyond."

About Selinexor (XPOVIO)

Selinexor is a first-in-class oral selective inhibitor of nuclear export (SINE) compound discovered and developed by Karyopharm Therapeutics Inc. (NASDAQ: KPTI), Selinexor is currently being developed by Antengene, which has the exclusive development and commercial rights in certain Asia-Pacific markets, including Greater China, South Korea, Australia, New Zealand and the ASEAN countries.

In July 2019, the US Food and Drug Administration (FDA) approved selinexor in combination with low-dose dexamethasone for the treatment of relapsed/refractory multiple myeloma (RRMM) and in June 2020 approved selinexor as a single-agent for the treatment of relapsed/refractory diffuse large B-cell lymphoma (RR DLBCL). In December 2020, selinexor also received FDA approval as a combination treatment for multiple myeloma after at least one prior therapy. In February 2021, selinexor was approved by the Israeli Ministry of Health for the treatment of patients with RRMM or RR DLBCL and in March 2021, the European Commission (EC) has granted conditional marketing authorization for selinexor (NEXPOVIO) for the treatment of adult patients with RRMM.

Selinexor is so far the first and only oral SINE compound approved by the FDA and is the first drug approved for the treatment of both MM and DLBCL. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple solid tumor indications, including liposarcoma and endometrial cancer. In November 2020, at the Connective Tissue Oncology Society 2020 Annual Meeting (CTOS 2020), Antengene's partner, Karyopharm, presented positive results from the Phase III randomized, double blind, placebo controlled, cross-over SEAL trial evaluating single agent, oral selinexor versus matching placebo in patients with liposarcoma. Karyopharm also announced that the ongoing Phase III SIENDO trial of selinexor in patients with endometrial cancer passed the planned interim futility analysis and the Data and Safety Monitoring Board (DSMB) recommended the trial should proceed as planned without any modifications. Top-line SIENDO trial results are expected in the second half of 2021.

Antengene is currently conducting five late-stage clinical trials of selinexor for the treatment of MM, DLBCL, non-small cell lung cancer, and peripheral T and NK/T-cell lymphoma. Furthermore, Antengene has submitted New Drug Applications (NDAs) for selinexor in multiple Asia-Pacific markets including China, Australia, South Korea, and Singapore, and was granted the Priority Review status by China's NMPA and an Orphan Drug Designation by the Ministry of Food and Drug Safety of South Korea (MFDS).

About Antengene

Antengene Corporation Limited ("Antengene", SEHK: 6996.HK) is a leading clinical-stage R&D driven biopharmaceutical company focused on innovative medicines for oncology and other life threatening diseases. Antengene aims to provide the most advanced anti-cancer drugs to patients in the Asia-Pacific Region and around the world. Since its establishment in 2017, Antengene has built a broad and expanding pipeline of clinical and pre-clinical stage assets through partnerships as well as in-house drug discovery, and obtained 15 investigational new drug (IND) approvals and submitted 5 new drug applications (NDA) in multiple markets in Asia Pacific. Antengene's vision is to "Treat Patients Beyond Borders". Antengene is focused on and committed to addressing significant unmet medical needs by discovering, developing and commercializing first-in-class/best-in-class therapeutics.

Forward-looking statements

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.

[1] J. Mascarenhas, B.K. Marcellino, M. Lu, M. Kremyanskaya, F. Fabris, L. Sandy, M. Mehrotra, J. Houldsworth, V. Najfeld, S. El Jamal, B. Petersen, E. Moshier, R. Hoffman, A phase I study of panobinostat and ruxolitinib in patients with primary myelofibrosis (PMF) and post-polycythemia vera/essential thrombocythemia myelofibrosis (post-PV/ET MF).

View original content to download multimedia:https://www.prnewswire.com/news-releases/antengene-announces-acceptance-of-ind-application-in-china-for-the-phase-ii-clinical-trial-of-single-agent-selinexor-for-the-treatment-of-myelofibrosis-mf-301325442.html

SOURCE Antengene Corporation Limited

Company Codes: HongKong:6996

Read the original here:
Antengene Announces Acceptance of IND Application in China for the Phase II Clinical Trial of Single-Agent Selinexor for the Treatment of...

The Top Doctors in the Twin Cities, 2021 – Mpls.St.Paul Magazine

As medicine evolves with lightning speed, our medical community, built on a tradition of excellence, is taking a leading role in delivering transformative care. Local doctors are constantly shaping innovations that go on to become common practice and exploring new ideas about where care should be heading. The resultsimproved lives and outcomes, cutting-edge targeted treatments, and patient-centric care strategiesbenefit us all.

This year, as we celebrate the 25th edition of our Top Doctors list, we talked with professionals who have been on this list for decades and others newer to the ranks about the changes theyve seen and whats coming next in their fields. Read on to discover more than 800 local top doctors from 46 specialties who have been selected through a process involving extensive research and peer review.

See the complete list of doctors selected to this year's Top Doctors.

Theres much that is sobering about brain cancer: Glioblastoma (the most common type of brain tumor in adults) remains one of the deadliest cancers, and one of the most challenging to treat. Even with the most advanced care, it is a devastating disease. But patients today benefit from some major advancements of the past 15 years: Before 2005, there was no common standard for treating glioblastoma. Now, the protocol of surgery, radiation, and chemotherapy has extended life expectancyand much more is on the horizon. Im optimistic that before the end of my lifetime, we are going to see major changes for glioblastoma patients and achieve meaningful benefits, says Dr. Clark Chen. We asked him for his thoughts on the milestones in his field and whats giving him hope.

Surgically implanted radioactive seeds have the potential to improve life expectancy and quality of life for brain tumor patients.

Dr. Clark Chen

Why is brain cancer one of the most challenging/deadly cancers? The brain is a very different organ than the rest of the body. Our brain is so precious to us that our body has evolved barriers to protect it. For example, if you get food poisoning, your brain is protected from those toxins. And many chemotherapies are like toxins, so they cant get into the brain. Surgeons also have a very challenging task in figuring out how much of a tumor to remove, since one millimeter of difference could be the difference between the patient talking and understanding and not.

What improvements have been made in treatments over the past few decades? Surgically removing as much of a tumor as possible dictates how a patient will do. We have a number of tools to help with that, including MRI. But an MRI machine weighs tonsits not like you could easily bring it into the operating room. In the 1970s, the University of Minnesota started using one of the first intraoperative MRIs in the world, so we could do a surgery and bring in this state-of-the-art MRI and look in and know how much was removed during the surgery. It gives you real-time feedback. So surgeons could see when they could remove more and save a second surgery. That was a major, major milestone. We just installed the newest generation in 2019.

Another milestone allowing us to remove more of a tumor is a drug called 5-ALA. Its a drug that the patient drinks before surgery so that if you shine a blue light on the tumor, it will become fluorescent red. We were the first in the state, at the University of Minnesota, to have it during surgerywe started using it the moment the FDA approved it in 2017. Sometimes its very difficult to tell where the tumor ends, and this dye gives us that tool.

Im optimistic that before the end of my lifetime, we are going to see major changes for glioblastoma patients and achieve meaningful benefits.

Dr. Clark Chen

Also, surgically implanted radioactive seeds have the potential to improve life expectancy and quality of life for brain tumor patients. Picture cancer cells as unruly students, and the seeds as teachers. Having one teacher for 10,000 students is not going to work. But after a surgeon removes the majority of the cancer cells, the seed can be used to destroy the microscopic cells that are invisible to the naked eye. Because of its short range, which is five to eight millimeters, the radiation is concentrated in the region of the tumor and does not affect the rest of the brain. In the old days, the patient would have had to go back to the operating room if the tumor grew back after surgery.

When will immunotherapy be able to be used in brain cancer? Injection of a genetically engineered virus into glioblastomas will solicit immune responsesresponses that can be further enhanced with the immunotherapy that was awarded the 2018 Nobel Prize. Tumors that tend to benefit from immunotherapy are known as hot tumors. They are tumors with a lot of immune cells in there already; thats why theyre called hot. Cold tumors are ones that dont have a lot of immune cells. So a really exciting thing that we are doing is to surgically inject this virus into a cold glioblastoma that is devoid of immune cells and does not respond to immunotherapy and convert the tumor into a hot glioblastoma that responds to immunotherapy. In this way, we can engage the patients innate immune system to fight off the tumor cells. People have survived beyond expectation, but it doesnt work for everybody. Were trying to figure out why it works for some and not others. We are beginning to see extraordinary responses, and with each extraordinary response, we are one step closer to a cure for every patient. S.M.E.

Dr. Brian Swiglo, an endocrinologist with Allina Health, specializes in the treatment of conditions related to hormones and their effects on the bodys organs. Two-thirds of his practice is devoted to the treatment of people with diabetes.

Its been standard procedure for many people with type 1 diabetes to check their glucose levels with a finger stick about four times a day. However, modern technology is changing all of that. Dr. Brian Swiglo says that continuous glucose monitors are replacing finger sticksand theyre doing a much better job. Abbott, Dexcom, and Medtronic make small wearable devices (FreeStyle Libre, Dexcom G6, and Guardian Connect) that adhere to your arm or stomach and automatically read your blood sugar levels every 5 to 10 minutes. Then the information is sent to your phone or device. For people taking insulin, this is a huge boon. You really need that information in order to choose the right dose, Swiglo says. They can just look at their phone and know, Oh, Im 220. Well, now I need an extra three units on top of what I take for my food. It helps them dose their insulin a lot more accurately.

This knowledge is powerful, both for the patient, who gets a better understanding of how their glucose levels are affected by certain foods, activity, and insulin, and for the endocrinologist, who gets a better understanding of their patients meal and exercise habits. When were in clinic with them, we can download that information, look at it, and make more accurate adjustments in their insulin regimen, Swiglo says.

Insulin pumps have been around for about 30 years and are now being replaced with closed-loop insulin pumps, which talk to continuous glucose monitors. Its a huge step toward an artificial pancreas, Swiglo says. And its giving people greater freedomespecially at night. The biggest advantage is when someone goes to bed, they can fall asleep and sleep through the night while still having good control. Thats because the pump and the monitor are in constant communication and making any needed adjustments.

The newest medicines for type 2 diabetes can do more than lower a persons blood sugar. Theyre helping with weight loss and lowering the risk of cardiovascular disease. Most people with type 2 diabetes struggle with their weight, Swiglo says. So anything we can do to help them with weight loss is a benefit. J.J.

A cardiologist since the 1990s, Dr. Pamela Paulsen has worked at all three Level 1 trauma centers in the Twin Cities. After starting her career at Hennepin Healthcare, she moved to North Memorial Health Hospital for 17 years, and shes now with Regions Hospital. She also started the first womens heart clinic in Minnesota, which she ran for 10 years. Paulsen has cared for patients in acute emergency situations. Today, she practices all areas of general cardiology.

Dr. Pamela Paulsen completed her fellowship training in interventional cardiology at the University of Minnesota more than 25 years ago. We had reasonable treatment in 1994, but its unbelievably different now for patients, Paulsen says. Its our advancements in preventionbut also in heart attack carethat really revolutionized during the time I have been a cardiologist.

A combination of technological advancements and medications are helping to reduce heart disease and death from heart disease. Paulsen lists the three biggest game changers, the first of which is the use of stents. The ability to treat heart attack and chronic coronary artery disease with revascularizationor restoring blood flow predominantly through stentsreplaced a more invasive approach and had excellent outcomes, she says. Bonus: Todays stents come in all sizes and are drug-coated so that they are less likely to narrow as time goes on.

The second is the less invasive method now used to replace aortic heart valves. Transcatheter aortic valve replacement (TAVR) is performed by creating a small incision in the chest or using a blood vessel in the leg rather than through open-heart surgery. Advanced imaging guides a catheter to the aortic valve, where a new valve is positioned with the help of an inflated balloon. Its the preferred strategy for replacing aortic valves when theyre narrowed, Paulsen says.

Lastly, statins followed by injectable cholesterol drugs have significantly helped lower the risk of heart disease altogether. Since the 1970s, the risk of death from heart disease is down an average of 60 percent in the United States.

Paulsen is hopeful that the wave of the future will include incorporating personalized medicine in heart care that would pinpoint the best drugs and diet for an individual. She also is encouraged by the future potential of wearable technology for monitoring blood pressure, heart rhythms, and sleeping patterns and helping doctors care for patients. J.J.

In the 20 years that Dr. Gigi Chawla has been practicing as a pediatrician, advancements have fundamentally altered childrens health care experiences. If you still call a well-child visit a yearly checkup, heres a glimpse into the way todays parents and children experience the doctors office. First, new routine childhood immunizations for rotavirus, approved in 2006 and 2008, mean parents have one less thing to fret about. Hospitalizations from severe gastroenteritis, caused by rotavirus, have almost been eliminated. Kids would have serious morbidity, and some would die with the rotavirus, says Dr. Chawla, recalling the days when wards of kids would be hospitalized with the disease every winter.

With every scientific breakthrough, we have to think about how we apply those breakthroughs. This piece has got to be elevated.

Dr. Gigi Chawla

And COVID-19 vaccines may have a similar impact, she predicts. Even though most children dont experience serious illness from COVID-19, the technology used to create the Pfizer and Moderna vaccines could be applied to vaccine development for other illnesses, such as respiratory syncytial virus (RSV), a common respiratory virus that can hit infants hard, she says. That will also change the face of how pediatric illness is experienced, she says, explaining that RSV is the number one cause of childhood hospitalizations in winter.

But the COVID vaccines also revealed a negative side, highlighting disparities in health careand further accentuating them, Chawla says. Its not going to do us much good to have these breakthroughs if all it does is divide the haves and have-nots, she says.

Chawla has devoted the past three years to better understanding disparities in immunization rates between patients of color and white patients. With every scientific breakthrough, we have to think about how we apply those breakthroughs, she says. This piece has got to be elevated.

Its something Chawla thinks about constantly, she says, working to help health care overcome generations of institutional racism. Looking at vaccine disparity, for example, reveals a gap of 40 percent between Black and white patients in completion rates of the entire routine vaccination series before age 2, she found. For me, the first step is really understanding that vaccine disparity, which has likely been present during my entire career and weve just never been brave enough to look at it or willing as clinicians to understand what our role is in mitigating it, she says. To close the gap, Childrens Minnesota has implemented drive-up vaccination and mobile vaccination clinics and recognized other access hurdles, including the ability to make an appointment during clinic hours and to speak a language understood by front desk staff and providers, as well as the ability of clinicians to earn a patients trust.

Another important shift thats happened over Chawlas career, she says, is a recognition of just how important early childhood development is, as well as understanding the science behind it. Understanding how critical that bonding is with a trusted adult/caregiver/parent, and really engaging infants, toddlers, and young children in experiencing and exploring the worldincluding facilitating early language development, she says. There are 700 neural connections being made every second. Its immense. The time and energy we can all put into things like early language development pay off in multiple ways, including decreasing educational or opportunity gaps, which has inspired her to serve as Reach Out and Read Minnesotas Medical Director.

Childrens Minnesota and others have also integrated behavioral health into routine pediatric exams. Therapists, psychologists, and social workers are available during well-child and primary care visits should families need them. And given the high impact social determinants beyond the clinic have on kids healthwhere they live, learn, and playprimary care appointments at Childrens Minnesota now include screening for food insecurity, educational needs, housing concerns, and legal issues. After witnessing the value of this more comprehensive approach to caring for children, Chawla notes, Hopefully, every health care organization is working toward families experiences. S.M.E.

Dr. Mohamed Hassan has spent his nearly 32-year career specializing in gastroenterology and hepatology (diseases of the liver). In the last 17 years, he has also been practicing transplant hepatology. Today, he practices at M Health Fairview Clinics and Surgery Center in Minneapolis and M Health Fairview Clinics and Specialty Center in Edina.

More than 30 years ago, Drs. Harvey Alter, Michael Houghton, and Charles Rice identified the hepatitis C virus. In 2020, they received the Nobel Prize for their discovery. That was a big deal because hepatitis C went from not being known before 1989 to a disease that can easily be treated more than 93 percent of the time with direct-acting antiviral agents (DAAs), says Dr. Mohamed Hassan.

The field of liver transplantation has also made great strides since the first successful procedure in 1967. These days, a liver donor doesnt need to have a perfect bill of health. Patients in need of a liver transplant can even receive a good-functioning liver from a donor who has the hepatitis C virus. There are people who are very sick and cannot wait for a negative hep C liver or donor, Hassan says. After the transplantation is complete, the latest DAAssuch as sofosbuvirtreat the hep Cpositive liver. That is really remarkable and probably one of the best things thats happened. Hassan hopes in the near future there will be medications that can eliminate hepatitis B, a disease more likely to affect people born in Southeast Asian and African countries. It is now being tested, he says.

Hassan enjoys helping others, and his influence reaches beyond the clinic with the work he does in the local Somali community. Recently, to help build trust around the COVID-19 vaccine, Hassan was in a video with an imam at a mosque getting the vaccine. A lot of people gave me feedback and told me it did work, he says. Even family and friends back in Somalia saw the video in their community. J.J.

Back in 1990, Dr. John Wagner conducted the first-ever cord blood transplant for a leukemia patient. Now, he looks back on those early days of transplant medicine and exploration of the potential of cell therapies as a preamble to a future with endless possibilities for treating all types of diseases: manipulating cells to do what doctors and scientists want them to do. Whether you call it immunotherapy or precision or personalized medicine, Wagners work is on the fast track to curing some types of cancer.

What has been the biggest game changer in your specialty in the last few decades? Engineered cells, living drugs that can last in the body for days or a whole lifetime. One example is the use of genetically modified immune cells that specifically target a cancer. I became involved in the mid-80s during my training at Johns Hopkins School of Medicine, when we first started working with bone marrow for transplantation, but weve learned how to engineer cells more precisely since then.

Today, we can take cells from a sample of blood or skin biopsy and generate cells called induced pluripotent stem cellsreplacing the need for embryonic stem cells.

Dr. John Wagner

Has your work ever yielded any unintended results with a positive impact? A decade and a half ago, we thought that the embryonic stem cell was the only way you could make the various tissues of the body. Today, we can take cells from a sample of blood or skin biopsy and generate cells called induced pluripotent stem cells (IPSC)replacing the need for embryonic stem cellscells which can be used to test drugs and other treatments as well as repair damaged tissues. For example, our lab is working on using IPSC to make a nearly endless supply of blood-forming stem cells from a small sample of blood. You wont need any other donors, potentially.

What advancements are on deck? Even though the current approach is very effective, I cant tell when a patient with leukemia is going to come in the door and then everything stops to make it happen. Say you had leukemia. First, wed have to give chemotherapy to beat down the number of leukemia cells so that sufficient numbers of normal T cells can be collected for genetic engineering. It takes a month for manufacturing and testing the cell therapy before it is shipped back to the hospital where they give it to you. There are a couple of problems with that. One, you have to have chemo. Two, its an individual product for every single patient. That makes it logistically challenging and much more expensiveand you might not have a month. Many people are lost while waiting for the manufacturer. My guess is we may, for the first time, be able to cure cancer without using chemotherapy, radiation, or surgery. Thats the path were pursuing. So when youre diagnosed with leukemia, we will be able to forget about the traditional way of treating it and use cell therapy as the only treatment. Thats what we hope to move forward in the next one to two years. And we could move it into other fields . . . cardiology or multiple sclerosis or brain injury. All are in development with the same concept of taking a cell and modifying it to do what we want. This is going to be the game changer, and its sitting on the shoulders of the work of the prior 30 years.

How do these advancements impact previously overlooked or underserved communities? In the past, getting patients to take advantage of new advancements was more of a passive process. If you had a specific disease and you figured out how to get to the University of Minnesota and to me, I would check to see if you met the eligibility requirements and treat you. Today, we go to much greater lengths to find patients and make sure they know what is available, potentially, to them. We want to make sure the patients being enrolled into clinical trials appropriately reflect the makeup of our larger communitythat is, we want to ensure that we have people of all races and ethnicities.

One example: We want to develop off-the-shelf, cryopreserved products. Youd make them all in advance, and theyd be immediately available. Otherwise, some treatments, like immunotherapy, when manufactured as individual products, may never be available for the majority because of logistics and expense. But if I could take one sample of starting material and make 1,000 or more products and verify their potency in advance, we could send them anywhere in the world and people could get it the day theyre diagnosed.S.M.E.

Dr. Siobhan Flanagan is an interventional radiologist at University of Minnesota Health (M Physicians) who does minimally invasive procedures under imaging guidance. She treats liver cancer, vascular malformations, aortic aneurysms, peripheral arterial disease, and more.

University of Minnesota physician Kurt Amplatz helped invent interventional radiology more than three decades ago when he created a small plug-shaped medical device that could be passed through a catheter from the leg to the heart, thus repairing an atrial septal defect (hole in the heart) and avoiding open-heart surgery. Today, Dr. Siobhan Flanagan continues to move the field forward by providing targeted treatments to patients with serious medical problems, including liver cancer. If we can treat patients liver cancers effectively, we can then bridge them to transplant, Flanagan says.

With the help of a catheter and x-ray guidance, the treatment is injected directly into the artery that leads to the liver. As opposed to chemotherapy that circulates all throughout the body, were targeting the treatment just where it needs to go: the local blood supply to the tumor, Flanagan says. And thats especially great news for people with a liver tumor thats less than three centimeters. We can cure tumors less than three centimeters with some specific treatments and control tumors larger than three centimeters with a local therapy.

Compared to cancer, an abscess that develops in the abdomen after surgery might seem like an easy fix. But its not for a surgeon who has to navigate an area that was recently operated on and has a lot of inflammation. Thankfully, an interventional radiologist can come to the rescue. We can place an abscess drain under CT or ultrasound guidance to help drain the infected fluid so the patient doesnt have to have another operation, Flanagan says.

Vascular malformations, abnormal groupings and developments of blood vessels throughout the body, can also be treated with this technology. A lesion can be biopsied to test its genetics and determine which medication will help shrink it.

Flanagan is excited to see whats on the horizon in terms of personalized medicine, especially with tumor-specific therapy, determined by tissue receptors and genetics. Theres this continued opportunity for us to be involved with these treatments by delivering them directly to a tumor, she says. J.J.

Dr. Charles E. Crutchfield III practices dermatology at his clinic in Eagan, where he sees patients for the treatment of a variety of medical and cosmetic conditions. He also teaches dermatology to medical students, residents, and other clinical physicians at the University of Minnesota, Carleton College, and around the world.

Crutchfield has made it his mission to share the knowledge he has with others, including those in the medical field. Skin conditions in skin of color can look a lot different than what were trained on. So I coauthored a textbook of dermatology and made sure to include over 3,000 photographs, half of them in skin of color. He also covers the topic in lectures at the University of Minnesota and around the country. And he writes a weekly health column for the Minnesota Spokesman Recorder, the oldest Black newspaper in the state of Minnesota. Were doing our part to educate and further the knowledge in treating skin of color, he says.

Crutchfield is excited about the advances he sees in the treatment of various skin conditions. Were seeing treatments now for psoriasis, atopic eczema, and vitiligo that are extraordinarily effective for treating skin and inflammatory conditions in the human body, he says. They are changing the lives of our patients, he says.

And the advancements in aesthetic dermatology are also big game changers. Hyaluronic acid fillers and Botox injections rejuvenate the skin with natural-looking results. Crutchfield estimates he does 5 to 10 aesthetic treatments a day. And many of his patients will express to him that they wish they would have done the treatment years ago. I tell them, Well, we couldnt have done it years ago because we didnt have it, but we have it now. And well use it moving forward.

As everyone learned in 2020, COVID-19 created a paradigm shift in the delivery of care. And physicians had to pivot their practices to meet the needs of patients in a safe manner. These days, telehealth visits make up 30 to 40 percent of Crutchfields appointments. A year and a half ago, I had not done one telehealth visit, he says. And now Ive done 10,000-plus. Its surprising how much can be covered in a visit with a dermatologist via a webcam. Platforms such as Zoom, FaceTime, and Google Duo have great clarity of picture for medical evaluations and discussions. Crutchfield uses them for triage purposes, follow-up visits, and prescription refills. This will be part of our practice moving forward, he says. Patients love it, and I like it too.

After more than two decades of being the doctor, Crutchfield is now the patient. In early 2021, he was diagnosed with non-Hodgkins lymphoma, for which he is currently receiving treatments at the Mayo Clinic in Rochester. And the cancer treatment he has received has a medical legacy that ties back to early efforts by friends in the medical community. I have colleagues that were on the development team at Genentech 30 years ago that helped develop the [monoclonal antibody treatment] thats being used to treat me right now, Crutchfield says. My friends are so delighted that the medicine they helped develop is actually helping their friend. J.J.

Back when Dr. Elizabeth Arendt was playing sports in her first few years of college, the training room was only for male athletes. When Arendt or a female teammate got injured, they went to student health services, where ankle sprains were sometimes mistakenly treated with a hot-water bath.

Arendt, who played everything growing up, from Ping-Pong to horseshoes, competed in varsity volleyball and basketball in college in the early days of Title IX, the federal civil rights law aimed at preventing discrimination based on sex in education. As a premed student studying biology and anatomy, she was encouraged by administrators to fill a gap in the training room by treating female athletes. By her senior year, she was a student athletic trainer, challenging inequities in the newly coed training room. This experience not only motivated her to work to change the culture of athletics at the University of Rochester in New York; it also inspired her to pursue orthopedics and sports medicine instead of pathology, she says.

Research suggests that simple warm-up exercises can drastically lower the risk of ACL injuries.

Dr. Elizabeth Arendt

The world of orthopedic surgery is heavily male-dominated. In a 2015 survey, according to the Association for American Medical Colleges, 95 percent of orthopedic surgeons were male. The field is also lagging in diversity as a subspecialty field, but that is improving, Arendt says. But since graduating from medical school in 1979, shes seen some shift in the way athletic injuries are viewed in the field, placing less emphasis on gender.

When it was first discovered that females tear their anterior cruciate ligaments (ACL) more than males, some doctors theorized this was related to the hormonal environment due to menstrual cycles. While that hasnt been ruled out conclusively, theres much more evidence linking the risk to anatomic risk factors present in both sexes and body movement patterns, Arendt says. I think the focus has been less on the sex of the patient and more on what it is that characterizes knee injuries, says Arendt, who conducts research in the area.

This has led to a better understanding of acute injuries to the ACL and the patellofemoral joint, including an increased recognition of risk factors such as body movement patterns and anatomy in other musculoskeletal injuries. And thats helpful because smart training and coaching can reduce the risk of injury. Research suggests that simple warm-up exercises can drastically lower the risk of ACL injuries, for example.

The biggest change across knee surgeries, however, is probably the push toward outpatient surgeries. Ultimately, Arendt says, the shift comes with pros and cons, but to optimize the experience, patients should understand their insurance plans more than most do. For example, even patients who stay in the hospital overnight may be counted as outpatient surgeries, she says, meaning they dont qualify for a skilled nursing facility and would be expected to complete physical therapy with in-home physical therapy or on an outpatient basis.

And, love it or hate it, the biggest change in medicine in the past 30 years is the advancement of electronic medical records (EMR), she says, recalling the days of taking charts home to work on at night. From the patients perspective, there are clear benefits.

As much as it burdens us, theres no doubt the ability to document a patients pertinent information electronically and share it across medical systems is a huge improvement, she says. And its greatly improved our ability to deliver service at all hours. S.M.E.

Dr. BJ Harris specializes in gynecology and urogynecology at Womens Health Consultants and the Pelvic Floor Center.

In her 21-year career, Dr. BJ Harris has witnessed many gynecological and medical game changers. One of the big ones is the reduction in the number of hysterectomies, which used to be much more common and not always a medical necessity. Today, she says, minimally invasive hysteroscopies allow doctors to manage many issues in the uterus, such as abnormal bleeding and the removal of polyps and some fibroids. Also, some types of IUDstypically used for contraceptionhave been FDA-approved to help mitigate heavy bleeding. And tranexamic acid, originally prescribed to women to help make their periods less heavy, is now being used to help reduce blood loss from C-sections or other big surgeries, she says.

Other discoveries, such as the fact that some of the more aggressive types of ovarian cancers can begin in the fimbriae (fingers) of the fallopian tubes, help women make decisions about how to manage their long-term health. So, if one of Harriss patients is having a procedure, like removing an ovarian cyst, and this person is not planning on having (more) children, Harris also recommends removing the patients fallopian tubes at the same time. The fallopian tubes have never provided any hormone in the past, and they never will in the future, she says. All they provide is a cancer risk once women are done childbearing.

The majority of her practice focuses on patients with pelvic floor issues, such as vaginal prolapse or urinary incontinence. She helps them assess treatment options. For those with incontinence, she says, I do quality-of-life surgeries. One womans quality of life versus another womans is very different. I have some people that leak urine like a sieve, and they dont want surgery. And I have other people that say, I leaked twice at my aerobics class last week and I want surgery yesterday. She helps these individuals weigh risks and find healthy solutions and alternatives if they dont want surgery. J.J.

Dr. Mumtaz Kazim grew up watching her parents take care of patients in their 17-bed hospital on the island of Trinidad. She went to college in Canada and med school in India. Shes seen plenty of changes in health care in her career as a physician in the field of family medicine and as president of Edina Family Physicians.

What has been the biggest game changer in your specialty in the last 20 to 25 years? You said game changer, but maybe youll allow me three! The first everyone will agree with: EMR. Its increased access and ability to share information with specialists, allowing for better communication for multidisciplinary care. Also, the concept of what has happened with outpatient clinics. Edina Family Physicians used to be an independent family practice; most of these are now being purchased by larger systemsEdina Family Physicians is now affiliated with Allina. Its a big change. Then theres the concept of virtual visits. With COVID, our clinic was closed for a while, except for emergency care and phone calls, and you cant imagine the expressions of patients who were so lonely. When you see each other face-to-face, there is such delight to talk to somebody. And you can call back to check up on them. It was very helpful. It shouldnt be used for all medical care, but when they cannot come in, seeing them face-to-face in their own home gives you a whole different perspective.

I believe the increased number of women and minorities in all medical fields has had a positive impact on patient care.

Dr. Mumtaz Kazim

Whats the most exciting advancement just around the corner in family medicine? Personalized medicine, including genomic sequencing to target drugs for certain diseases, immunologic approaches to treat cancer, and CRISPR, which is a technology that adds or deletes genes to improve and cure medical conditions. It will help to improve the medical health of the community from sickle cell disease to diabetes and various cancers. Think about it, how great it would be if we could cure diabetes.

How has the diversity of our medical community changed things, and how have advances impacted previously overlooked or underserved communities? I believe the increased number of women and minorities in all medical fields has had a positive impact on patient care. Providers are so much more sensitive to disparities in the medical services available nowso much more than 30 years ago. I think that will eventually have a positive effect on delivery of care to underserved communities. And with the continuity of care now with EMR, we can provide better tracking of patients. Even though EMR is far, far from perfect (this is not an ad for it!), it provides us a tool to have continuity of care. And access to previous medical records is a tremendous help. For example, take the case of a patient who has mental health issues, in addition to some dementia, and no family in the Twin Cities. Hes gone to different emergency rooms and been seen by different providers across all of the systems. We have the EMR, and we can then get access to some of those records, which will then educate me on what has been happening.

Patients dont always offer a detailed medical history, and many patients, especially with cardiac disease, get shipped from one hospital to the next. So we may have patients with records from Abbott and then Southdale, and by the time they come back to me, I can check all of it. Thats very important because doctors shuffle around medications and dosages all the time, and cardiac patients are often on 6 to 10 medications.S.M.E.

Dr. Marjorie Hogan is a pediatrician at Hennepin Healthcare whos been caring for the needs of infants, children, adolescents, and young adults for 41 years.

When Dr. Marjorie Hogan started her practice in 1980, office visits typically involved the usual litany of questions regarding nutrition, sleep, and vaccines. Since then, pediatric careand the worldhas become much more complicated. We used to think brain development stopped sort of abruptly in childhood, but thats not the case, she notes. The brain continues evolving and maturing and arborizing and getting more complex and wonderful in many kids up to their 20s.

One of the dramatic shifts she has seen recently is in the number of school-aged children and teens expressing feelings about anxiety, fear, and stress, Hogan says. Thankfully, during these unprecedented times, providers like Hogan can put patients and their families in touch with a team of resourcesincluding nurses, social workers, educators, community health workers, and morethat will support the whole health of the child. However, she says, We need more mental health providers for children and teens. Its a crisis. It impacts every part of their lives. I am seeing more teensboth male and femalegrapple with eating disorders, with the risk of self-harm, [and] with substance abuse.

One of the complicating factors in the struggle: media. Back in the 80s, Hogan authored some of the first American Academy of Pediatrics (AAP) media statements that recommended parents limit childrens TV watching to less than 2 hours a day. Today, young people have access to more media than ever. In this past year, theres not one family Ive seen that has not had concerns about their childrens screen time and media use. Hogan recommends that parents be good media role models and develop a family plan. That means no electronic devices at the dinner table and certain shows and games should be off-limits. Be aware of what your children are watching or using as much as you can.

In recent years, the medical community has become more aware of the role that social determinants and inequities play in a childs overall health. Hogan is pleased that todays medical students and residents take courses on the topic so they can be better prepared to help children in a changing world. J.J.

See the complete list of doctors selected to this year's Top Doctors.

The 25thedition of our Top Doctors list includes 816doctors in 46specialties. Heres how we put it together. When compiling a list thats as relied upon as our annual Top Doctors list, research is essential. We asked physicians to nominate one or more doctors (excluding themselves) to whom they would go if they or a loved one were seeking medical care. From there, candidates were grouped into 46specialties and evaluated on myriad factors, including (but not limited to) peer recognition, professional achievement, extensive research, and disciplinary history. Doctors who had the highest scores from each grouping were invited to serve on a blue-ribbon panel that evaluated the other candidates. It should be noted that doctors cannot pay to be included on this list, nor are they paid to provide input. Only doctors who acquired the highest total points from the surveys, the research, and the expert physician review panel were selected to this list. Of course, no list is perfect. Many qualified doctors providing excellent care are not included on this years list. However, if youre looking for exceptional physicians who have earned the confidence and high regard of their peers, you can start your search here.In addition, this years crop of Top Doctors will join a prestigious group of doctors from more than 20 cities around the country who have been selected to Super Doctors, the full list of which you can find at superdoctors.com.

Editors Note: Many of our Top Doctors have specialty certification recognized by the American Board of Medical Specialties. This board certification requires substantial additional training in a doctors area of practice. We encourage you to discuss this board certification with your doctor to determine its relevance to your medical needs. More information about board certification is available at abms.org.

2021MSP Communications. All rights reserved. Super Doctors is a registered trademark of MSP Communications. Disclaimer: The information presented is not medical advice, nor is Super Doctors a physician referral service. We strive to maintain a high degree of accuracy in the information provided. We make no claim, promise, or guarantee about the accuracy, completeness, or adequacy of the information contained in the directory. Selecting a physician is an important decision that should not be based solely on advertising. Super Doctors is the name of a publication, not a title or moniker conferred upon individual physicians. No representation is made that the quality of services provided by the physicians listed will be greater than that of other licensed physicians, and past results do not guarantee future success. Super Doctors is an independent publisher that has developed its own selection methodology; it is not affiliated with any federal, state, or regulatory body. Self-designated practice specialties listed in Super Doctors do not imply recognition or endorsement of any field of medical practice, nor do they imply certification by a Member Medical Specialty Board of the American Board of Medical Specialties (ABMS) or that the physician has competence to practice the specialty. List research concluded April 19, 2021.

Sign up for our Be Well newsletterto get the latest health and wellness coverage.

See the original post here:
The Top Doctors in the Twin Cities, 2021 - Mpls.St.Paul Magazine

Innovative Regenerative Medicine Therapies Safety Comes First – FDA.gov

Caption

By: Peter Marks, M.D., Director, Center for Biologics Evaluation and Research

The U.S. Food and Drug Administration continues to facilitate the development and availability of innovative medical products, such as regenerative medicine therapies, that have the potential to treat or even cure diseases or conditions for which few effective treatment options exist. For example, the agency has recently licensed (approved) its first product that received Regenerative Medicine Advanced Therapy (RMAT) designation, underscoring our ongoing commitment to work with sponsors and manufacturers to bring these products to market.

Unapproved products marketed as regenerative medicine therapies may cause serious harm to patients. Cellular therapies, including stem-cell products, are often marketed by clinics as being safe and effective for the treatment of a wide range of diseases or conditions, even though they havent been adequately or appropriately studied in clinical trials.

In 2017, the FDA issued guidance on the regulatory framework for regenerative medicine therapies and announced its intent to exercise enforcement discretion with respect to the FDAs investigational new drug (IND) and premarket approval requirements for certain regenerative medicine products. This policy gave manufacturers three-and-a-half-years to determine the appropriate regulatory pathway for their products, and if an application is needed, ample time to prepare and submit the appropriate application to the FDA.

Now that we have reached the end of the compliance and enforcement discretion policy period, we are once again reminding manufacturers, clinics, and health care practitioners and providers that the compliance and enforcement discretion policy for certain human cells, tissues, and cellular and tissue-based products (HCT/Ps), including regenerative medicine therapies, ended on May 31, 2021. If manufacturers continue to illegally market unapproved HCT/Ps, they do so at their own risk and may be subject to an enforcement action.

The FDA continues to receive consumer complaints and has warned consumers about unapproved regenerative medicine products and the unfounded claims made in advertisements and direct-to-patient marketing. Despite the FDAs warnings that an IND may be required for these products, many entities still ignore such warnings and offer these unapproved and unproven products, with some consumers subsequently experiencing serious adverse effects.

The compliance and enforcement discretion policy was never intended to excuse the violations of manufacturers or health care providers who are offering unapproved regenerative medicine products that have the potential to put patients at significant risk. The policy did not apply to products that have been associated with reported safety concerns or have the potential to cause significant safety concerns to patients.

Indeed, while the policy was in place, the FDA took swift and aggressive action in the face of serious violations of the law, including some involving patient harm. Since November 2017, the FDA has pursued two enforcement actions for injunction against manufacturers of such violative HCT/Ps.

The FDA prevailed in one of those cases, United States v. US Stem Cell Inc. et al., in June 2019, before the United States District Court for the Southern District of Florida. Earlier this week, the United States Court of Appeals for the Eleventh Circuit affirmed the lower courts judgment. The US Stem Cell decision is a victory for public health and an endorsement of the FDAs work to stop stem cell clinics that place patients at risk by marketing products that violate the law.

The other case for injunction, United States v. Cell Surgical Network et al., is currently being litigated in the United States District Court for the Central District of California. A third enforcement action pursued by the FDA was resolved in March 2018. That case involved the seizure of vials of Vaccinia Virus Vaccine, Live, used to create an unapproved and dangerous stem cell product (a combination of excess amounts of live virus and stromal vascular fraction a stem cell mixture derived from body fat).

The FDA also has taken numerous actions since the compliance and enforcement policy was announced. During this period, the agency issued 14 Warning Letters and 24 Untitled Letters involving violative HCT/Ps regulated under Section 351 of the Public Health Service Act and the Federal Food, Drug, and Cosmetic Act and applicable FDA regulations. Additionally, since December 2018, the FDA has issued 400 letters to manufacturers and health care providers who may be offering violative stem cell or related products since December 2018.

The FDA reminds all stakeholders that the agencys acceptance of an establishment registration and HCT/P listing does not constitute a determination that an establishment is in compliance with applicable rules and regulations or that the HCT/P is licensed or approved by the FDA. It is inappropriate and misleading to advertise establishment registration and product listing in any manner that may imply product approval or compliance with the law.

If manufacturers, clinics, and health care providers offering regenerative medicine products to patients did not contact the FDA about the need for an IND during the period the Tissue Reference Group Rapid Inquiry Program was offered, there remain three options that have been available for many years and these options continue to be available. We want to remind stakeholders that a product that requires but lacks premarket approval may not be lawfully marketed or offered for sale, including when a sponsor has an IND or is pursuing an IND or BLA for its HCT/P.

The FDA is committed to helping advance the development of clinical trials for regenerative medicine products with the shared goal of safe and effective products for patients. We look forward to working with those who share this goal.

For more information: Important Patient and Consumer Information About Regenerative Medicine Therapies

Continue reading here:
Innovative Regenerative Medicine Therapies Safety Comes First - FDA.gov

The stem cell market was valued at USD 14.7 billion in 2020, and it is expected – GlobeNewswire

New York, June 01, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Stem Cell Market - Growth, Trends, COVID-19 Impact, and Forecasts (2021 - 2026)" - https://www.reportlinker.com/p06079777/?utm_source=GNW According to a 2020 research article published in the scientific journal Aging and Disease (2020), mesenchymal stem cells are a safe and effective approach to the treatment of COVID-19. At least 10 projects have been registered in the official international registry for clinical trials, implicating the use of mesenchymal stem cells to patients with coronavirus pneumonia. However, it is still at an initial stage of study in relation to the market studied.

Stem cells are majorly used in regenerative medicine, especially in the field of dermatology. However, oncology is expected to grow at the highest rate due to a large number of pipeline products present for the treatment of tumors or cancers. With the increase in the number of regenerative medicine centers, the stem cell market is also expected to increase in the future.

One of the richest sources of stem cells is the umbilical cord, which possesses unique qualities and has greater advantages over embryonic stem cells or adult stem cells. There are an increasing number of stem cell banks, which collaborate with hospitals and increase awareness about the storage of cord blood units in families, particularly in the emerging markets. The support is increasing with the rising number of medical communities and government initiatives active in promoting the use of stem cells for the treatment of more than 100 diseases. Currently, there is an increase in the number of clinical trials for testing future treatment possibilities of cord blood. Over 200 National Institutes of Health (NIH)-funded clinical trials with cord blood are currently being conducted in the United States alone.

Key Market Trends The Oncology Disorders Segment is Expected to Exhibit the Fastest Growth Rate Over the Forecast Period

The global cancer burden has been increasing, and thus, cancer therapies must be modified according to regional and national priorities. According to the World Cancer Research Fund, in 2018, there were an estimated 18 million cancer cases around the world. According to the World Health Organization (WHO), cancer is the second-leading cause of death across the world, with an estimated number of 9.6 million deaths in 2018, accounting for nearly one in six deaths.

Bone marrow transplant or stem cell transplant is a treatment for some types of cancer, like leukemia, multiple myeloma, neuroblastoma, or some types of lymphoma. For cancer treatments, both autologous and allogeneic stem cell transplants are done. Autologous transplants are preferred in the case of leukemias, lymphomas, multiple myeloma, testicular cancer, and neuroblastoma.

The major disadvantage associated with autologous stem cell transplants in cancer therapy is that cancer cells sometimes also get collected, along with stem cells, which may further put it back into the body during the therapy.

In case of allogeneic stem cell transplants, the donor can often be asked to donate more stem cells or even white blood cells, as per the requirement, and stem cells from healthy donors are free of cancer cells. However, the transplanted donor stem cells could die or be destroyed by the patients body before settling in the bone marrow.

Moreover, due to the growing focus of stem cell-based research and the rising demand for novel treatments, several companies, such as Stemline Therapeutics, have been focusing on developing technologies and treatments to attack cancer cells, which may help the market grow. However, owing to the COVID-19 pandemic, the detection and treatment of new cancer cases are impended, which may slightly impact the segment growth in the year.

North America Captured The Largest Market Share and is Expected to Retain its Dominance

North America dominated the overall stem cell market, with the United States contributing to the largest share in the market. The United States and Canada have developed and well-structured healthcare systems. These systems also encourage research and development. The increasing number of cancer cases is providing opportunities for market players. The major market players are focusing on R&D activities to introduce new stem cell therapies in the market.

For instance, the National Cancer Institute (NCI) had stated that the national expenditure on cancer care was expected to reach USD 156 billion by 2020. This factor is expected to boost the growth of the market in the future. In December 2019, the researchers at the National Eye Institute (NEI) launched a clinical trial to test the safety of a novel patient-specific stem cell-based therapy to treat geographic atrophy, the advanced dry form of age-related macular degeneration (AMD), a leading cause of vision loss among people aged 65 years and above.

In addition, the current situation of COVID-19 is another factor driving the growth of the market in the country, as research activities are undergoing for the treatment of COVID-19. Stem cell therapy can also be a promising approach for the treatment of COVID-19 in the future. For instance, on May 6, 2020, Lineage Cell Therapeutics received a grant of USD 5 million from the California Institute for Regenerative Medicine (CIRM) to support the use of VAC, Lineages allogeneic dendritic cell therapy for the development of a potential vaccine against SARS-CoV-2, the virus that causes COVID-19.

Competitive Landscape The stem cell market is highly competitive and consists of several major players. In terms of market share, few of the major players currently dominate the market. The presence of major market players, such as Thermo Fisher Scientific (Qiagen NV), Sigma Aldrich (a subsidiary of Merck KGaA), Becton, Dickinson and Company, and Stem Cell Technologies, is in turn, increasing the overall competitive rivalry in the market. The product advancements and improvement in stem cell technology by the major players are also increasing the competitive rivalry.

Reasons to Purchase this report: - The market estimate (ME) sheet in Excel format - 3 months of analyst support Read the full report: https://www.reportlinker.com/p06079777/?utm_source=GNW

About Reportlinker ReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

__________________________

Originally posted here:
The stem cell market was valued at USD 14.7 billion in 2020, and it is expected - GlobeNewswire

Early Promise of AntiCLL-1 CAR T-Cell Therapy Reported in Pediatric AML – Cancer Network

Feasibility of chimeric antigen receptor (CAR) T-cell therapy targeting C-type lectin-like molecule-1 (CLL-1) for pediatric patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) was validated in a small patient cohort in a phase 1/2 trial (ChiCTR1900027684), results of which were presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.1

At an interim analysis, 10 of 11 patients completely responded to anti-CLL1based CAR-T cell therapy, with CLL-1positive AML blasts eliminated within 1 month. Six patients achieved complete remission with minimal residual disease (MRD) negativity, added lead investigator Hui Zhang, MD, PhD, an assistant professor at Shanghai Childrens Medical Center and director of Guangzhou Women and Childrens Medical Center at Chinas Guangzhou Medical University.

From all the research shown, we can say that anti-CLL1 based CAR T-cells is a safe therapeutic candidate with manageable CAR T-cellassociated toxicity for children with R/R AML, he said. It is highly effective in targeting CLL1-positive AML cells with superior overall response rate (ORR) relative to conventional/novel targeting compounds.

In this study, 11 pediatric R/R AML patients aged 2 to 16 years were infused between October 2019 and January 2021 with a second-generation CLL1 CAR-T created in Zhangs laboratory. Investigators administered a single dose of CLL1 or CLL1-CD33 dual CAR-T cells (target dose: 0.3-1 x 106/kg) following lymphodepleting conditioning with a cyclophosphamide/fludarabine combination.

Zhang said all 11 patients experienced CAR T-cell expansion in vivo during the first month. Five patients demonstrated persistence of T-cell expansion.

All patients experienced grade 1 to 3 cytokine release syndrome (CRS) but there were no lethal events, Zhang said. All patients experienced myelosuppression, which he said might be due to chemotherapy. Three patients experienced a grade 1/2 hepatic event. No patient experienced cardiac, renal, or gastrointestinal adverse events.

Investigators have suggested that CLL-1 is a promising target because it not expressed on normal hematopoietic stem cells (HSCs), but is expressed on 85% to 92% of AML blasts cells and leukemia stem cells.2 In a humanized mouse model, investigators demonstrated that CAR T-cell therapy specific for CLL-1 exhibit potent cytokine production and cytotoxicity against CLL-1-expressing AML cell lines without disrupting normal HSCs.

Investigators theorized that developing an anti-CLL1 CAR T therapy would help patients avoid the need for HSC transplant.

In 2020, Zhang published a case study of a 10-year-old girl who presented with an elevated peripheral blood blast percentage while undergoing maintenance treatment for a B-cell ALL relapse. Investigators developed a CAR containing a CLL1-specific single-chain variable fragment.3

The patient received lymphodepleting chemotherapy for 4 days before CAR T-cell transfer to enhance in vivo expansion of CAR T-cells. This was followed by a single dose anti-CLL1 CAR-T cells infusion. She experienced Grade 1 to CRS.

After completing CAR T-cell therapy, the patient achieved a complete response and was negative for MRD (<0.1%) on day 29. But the CLL1+ cells were not completely eliminated until 6 months after CAR T-cell therapy. The patient achieved a 10-month response using 1 dose of anti-CLL1 CAR-T monotherapy.

References

1. Zhang H, Bu C, Pen Z, et al. The efficacy and safety of anti-CLL1 based CAR-T cells in children with relapsed or refractory acute myeloid leukemia: A multicenter interim analysis. J Clin Oncol. 2021;39(suppl 15):10000. doi:10.1200/JCO.2021.39.15_suppl.10000

2. Tashiro H, Sauer T, Shum T, et al. Treatment of acute myeloid leukemia with T cells expressing chimeric antigen receptors directed to C-type lectin-like molecule 1. Mol Ther. 2017;25(9):2202-2213. doi:10.1016/j.ymthe.2017.05.024

3. Zhang H, Gan WT, Hao, WG, et al. Successful ant-CLL1 CAR T-cell therapy in secondary acute myeloid leukemia. Front Oncol. 2020;10:685. Doi:10.3389/fonc.2020.00685

Read this article:
Early Promise of AntiCLL-1 CAR T-Cell Therapy Reported in Pediatric AML - Cancer Network

Part 3: Moving Forward and Keeping Stem Cell Treatments Safe – MedShadow

Times up, said the Food and Drug Administration (FDA). A three-and-a-half-year grace period during which companies manufacturing and providing regenerative medicine procedures were instructed to get their operations in line with new, clarified rules announced in 2017, ended May 31, 2021. Those procedures had previously fallen into a gray area of regulation because they rely on harvesting live stem cells or related products rather than traditionally manufactured drugs to repair damaged tissues and organs. Starting June 1, the agency expects all such companies to be in compliance or risk a variety of enforcement actions from warning letters to pursuing criminal prosecution.

For many years, the regenerative medicine industry operated with limited FDA oversight. The field exploded in the 2010s, with nearly a thousand clinics popping up throughout the US. In 2017, the FDA announced stricter, clearer regulations for those treatments. Knowing that a multibillion-dollar industry already existed and wanting to support research and innovation in what most researchers believe is a promising field of medicine, the agency didnt penalize all clinics that werent following the rules. Instead, the FDA offered a grace period, during which companies could file regulatory paperwork and design trials in line with the agencys requirements.

Traditional clinical trials can take years and sometimes even decades to complete, so the FDA has offered several types of expedited approval pathways for therapies expected to provide valuable treatment to patients who have few options. In 2016, it created the Regenerative Medicine Advance Therapy Designation (RMAT) as a part of the 21st Century Cures Act. If the FDA grants the designation to a treatment, the researchers conducting its trials get special support from the agency that can streamline the approval process. The company may be permitted to submit real world data and patient registries in lieu of data from a standard clinical trial, in which some patients are randomized to receive a placebo, to be considered for approval.

But in June 2019, Ned Sharpless, the acting FDA commissioner, announced that the agency had received far fewer approval requests RMAT or otherwise than it had anticipated during the grace period. Were more than halfway through the enforcement discretion period, and the pace of progress of those offering these human cells, tissues and cellular and tissue-based products, including stem cell treatments, to come into compliance with the requirements has been slower than expected. Its possible some stakeholders have questions about the requirements or the length of the process, he said in a statement announcing the Tissue Reference Groups Rapid Inquiry Program (TRIP). Essentially, companies that were unsure which level of regulation their products required could have submitted a request to the Tissue Regerence Group, which would answer within three days, outlining the steps needed to meet the FDAs requirements for compliance. That program ended on March 31, 2021.

While the FDA offered to help companies willing to seek regulatory approval, the agency continued to penalize those companies it believes are conducting the riskiest procedures and making the boldest claims, which had fallen outside of even the previous, cloudier regulations.

For instance, Vibrant Health Care received a warning letter from the FDA in November 2020, after marketing umbilical-cord-derived stem cell treatments designed to boost the immune system and protect patients against COVID-19.

A banner on the companys homepage now reads: Vibrant Health Care does not offer any products or treatments that can mitigate, prevent, treat, diagnose or cure COVID-19. If you are experiencing COVID 19 symptoms, please contact your primary care physician or local hospital.

The letter also cited patient testimonials on the website that claimed that Vibrants treatments had cured their asthma overnight, for example, or led to other dramatic improvements. The testimonials page no longer includes references to specific treatments other than Botox. Instead, patients make broad statements like, Dr. Farrell has been keeping me functional for many years. She always finds some way to alleviate my pain.

An FDA spokesperson told MedShadow in an email, Clinics currently offering 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.

While some providers are working to get their products in line with FDA recommendations, others continue to claim that their products should not be subject to FDA review. Some companies may be toeing a fine line, registering clinical trials as a way to offer treatments to patients, but not designing those trials in ways that are likely to bring the therapies to market.

A search for stem cell and COVID on clinicaltrials.gov, a government website that lists clinical trial information, yields over 100 results. It could be a sign that researchers are working on new therapies and developing them through traditional clinical trial pathways sanctioned by the FDA. But, trials listed here are not always what they seem and the listings are subject to limited oversight. Thousands of trials registered on clinicaltrials.gov are not completed and the results never published. Its possible that some companies dont intend to send their results to the FDA for review and instead have set up sham trials for the appearance of legitimacy.

Leigh Turner, PhD, a bioethicist at the University of Minnesota published an analysis in 2017 that found 18 US-based clinical trials testing stem cells listed on clinicaltrials.gov required the patients to pay for their own treatments. In most clinical trials, patients are responsible for little to none of the cost of treatment or are paid a stipend and compensated for some travel costs to and from the medical facility. Moreover, Wired reported that patients paid $5,000 to $15,000 per treatment, a fact that was not disclosed in any of the clinicaltrials.gov listings themselves. None of the 18 studies were randomized or blinded, conditions usually required in studies intended for FDA review, because they minimize bias in results.

In 2019, Google banned advertisements for treatments that have no established biomedical or scientific basis. The companys announcement also stated, The new policy also includes treatments that are rooted in basic scientific findings and preliminary clinical experience, but currently have insufficient formal clinical testing to justify widespread clinical use. Some companies, Turner suggests in his article, may be using clinicaltrials.gov as an advertising tool to recruit patients willing to pay for the treatments, without conducting scientifically sound trials.

[Disclosure: The MedShadow Foundation advocated against the Right to Try Act.]

Even if you might benefit from an experimental drug, you might not be eligible for a trial. Maybe youre not the right age, youve been prescribed drugs in the past or have a comorbidity that interferes with the treatment being tested. Those conditions could cloud the data for scientists, even if the treatment is still helpful to you. For these situations, the FDA created the Expanded Access Pathway.

The expanded access pathway has been around for a long time. It tries to acknowledge that there may be circumstances where its justifiable to provide access to investigational new drugs outside of a clinical trial context, says Turner. But with the expanded access route, there is a fair degree of oversight.

The FDA evaluates each application for a patient who has exhausted other options to receive a drug through the expanded access pathway. According to a 2017 study, initiating the process requires paperwork that takes about 45 minutes to fill out. On average, the FDA issues a decision within four days. In emergency situations, it usually responds in less than 24 hours. The overwhelming majority of requests are approved, though about 11% require adjustments like a change in dosing or an informed consent form before approval.

The Right to Try Act allows patients, physicians and sponsors to bypass this FDA review. It really means that decision-making devolves onto patients, their physicians and a sponsor, says Turner. If everyone is being careful and cautious and doing everything they can to be compliant, it may be an approach that works in an acceptable fashion.

When Congress passed the Right to Try act in 2018, Matthew Feshbach, who had previously run a company that provided stem cell treatments in the Bahamas, saw an opportunity to return to the US and open Ambrose Cell Therapy, which now offers stem cell treatments for patients with a wide range of diseases who have exhausted conventional therapeutic options.

On the companys website, the tagline under the Ambrose Cell Therapy logo reads your right to try, and the site has a page dedicated to explaining the legislation. The company uses a system made by another company, Cytori Therapeutics, to process cells collected from a patients fat and reintroduce them into the patients body. The system has been tested for safety in nine Phase I and Phase II trials, but Ambrose is not currently pursuing any clinical trials of the treatment to bring it to market for specific diseases under FDA approval. Rather, the company is offering the stem cell treatment exclusively on a Right to Try basis. Feshbach says, There are very few large-scale clinical trials that have been done with adult stem cells. They usually dont make it past Phase II, primarily because of funding. Additionally, he says, he is not a big believer in randomized controlled trials, because in the real world, [treatments] dont work out the way they did in a trial.

He explains that there is a growing body of peer-reviewed literature to support the cells that Ambrose uses (and encourages patients to ask for such literature when searching for stem cell treatment options). The company is collecting data on patients and plans to publish a series of case reports.

Turner worries that the offering treatment in this context is never going to bring a safe and efficacious stem cell product to market. Its a way of sitting out there for years, [technically] complying with regulations.

Prices for different products and procedures arent readily available, and Feshbach declined to discuss the cost of care at Ambrose. A 2017 study showed that the average price quoted to a patient seeking stem cell injections for osteoarthritis in the knee is about $5,000. Its important to review all costs you can expect before beginning treatment, especially considering that, in most cases, insurance wont cover it.

During our conversation, Turner also mentions that there is a line in the Right to Try act that seemed to suggest that companies, like Ambrose Cell Therapy, couldnt profit solely from offering treatments on a Right to Try basis. He admitted that while it had caught his eye, he wasnt yet positive if he was interpreting the law accurately.

The Right to Try states that eligible investigational drugs must be in compliance with 312.6, 312.7 and 312.8 d (1). Of Title 21 Code of Federal Regulations. 312.8d states that, A sponsor may recover only the direct costs of making its investigational drug available.

To investigate, I reached out to a retired biotech executive who was involved with several expanded access requests, which are also required to conform to 312.8d, prior to the approval of the Right to Try act. She explains that her companies were only allowed to charge patients what it cost the company to make and send the treatments to a patients doctors, and that her companies never charged patients for anything.

I also emailed the FDA spokesperson, who responded, FDA does not review or approve requests for use under the Right to Try Act. FDAs role is limited to receipt and posting of certain information submitted under the Right to Try Act. Section 561B (C)(b) of the Right to Try Act (Public Law 115-176), Investigational Drugs For Use By Eligible Patients, describes the requirement to be in compliance with the applicable regulations set forth in section 312.8(d)(1) of the CFR [Code of Federal Regulations].

At this point, it seems clear to me that a company cant profit from selling its unapproved treatments to patients outside of clinical trials, but that its unlikely the rule would be enforced because as Turner put it, The FDA is not actively involved in scrutinizing any of this. The Right to Try law stops the agency from overseeing requests.

When I present this information to Feshbach, however, he explains that I am missing a key detail. The price of the treatment itself cannot exceed the companys cost of providing access to it, but the law does not address additional costs like having a doctor administer the treatment on-site.

While the cost of knee injections average $5,000, some stem cell treatments cost tens of thousands of dollars. In 2018, one company said it may even charge several hundreds of thousands to patients who requested their Right to Try a treatment that had demonstrated little efficacy even in the companys own trials. The company later announced it would offer the treatment to only a limited number of patients through expanded access, and that it would do so for free. One for-profit cancer treatment company currently offers Right to Try treatments alongside other options.

Some types of minimally manipulated regenerative medicine are still exempt from much FDA oversight, requiring only that their facilities keep up manufacturing standards that limit contamination. Even in these instances, there is a movement among some researchers to collect better data on patient outcomes, in hopes of better understanding who benefits from the treatments and when.

At the Center for Regenerative Orthopedic Medicine at the Feinstein Institutes for Medical Research, where Daniel Grande, PhD is the scientific director, he and others provide, for a fee, platelet-rich plasma and stem cell injections derived from a patients own bone marrow or fat, with techniques that fall under the FDAs lowest-risk tier and are thus not subject to the clinical trial process.

But he laments the lack of consistent data reporting in the field. He says you can do a literature search and find thousands of papers on a particular procedure only to realize theyre mostly individual case studies or lack a control group. We want to bring a standardization to the clinic, he says. For example, when Grande gives a patient a platelet-rich plasma treatment, he takes a sample of the blood and conducts a complete blood count, which analyzes the concentration of different cells and biomarkers in your blood to evaluate overall health and diagnose certain diseases. Next, he takes a sample of just the plasma. Both are stored in freezers for continued analysis. Then [I] follow these patients from zero to one year to see how they actually do, he adds.

Grande is not alone. His group has teamed up with several other institutions, including the Cedars-Sinai Medical Center, Northwell Health, Hospital for Special Surgery, Cleveland Clinic, Mayo Institute, Stanford University and the University of Colorado Denver, to form the Biological Alliance of Regenerative Medicine and Biorepository. He says its members have committed to measuring the same variables through treatment and sharing data in hopes of answering questions about who the treatments are most likely to help and how many stem cells are actually needed for best results. In the first year, their goal is to enroll 1,400 patients. Grande also hopes the effort may lead to insurance companies eventually reimbursing for the procedures.

Theres a movement underway nationally to better characterize these regenerative therapies in a way that everybody can either figure out whats going on [with them], Grande adds. Theres a call to action for trying to better characterize these things and to provide information to not only clinicians, but also [to] the public about what works and what doesnt, so that people can be informed.

Continued here:
Part 3: Moving Forward and Keeping Stem Cell Treatments Safe - MedShadow

Innovative research refines the treatment of patients with advanced cancers and the use of immunotherapy – Network News, Press Releases – Hackensack…

June 4, 2021

John Theurer Cancer Center investigators report new findings at American Society of Clinical Oncology 2021 Annual Meeting

Researchers from Hackensack University Medical Centers John Theurer Cancer Center, a part of Georgetown Lombardi Comprehensive Cancer Center, are presenting data from 25 studies at the Annual Meeting of the American Society of Clinical Oncology, the largest gathering of cancer professionals in the country. This years meeting is being held virtually June 4-8, 2021. Abstracts of the studies can be viewed at abstracts.asco.org.

At John Theurer Cancer Center patients have access to the latest cancer treatments and technologies, including those being evaluated in clinical trials. People with all types and stages of cancer are treated by world-renown experts. The cancer center is especially well-known for its research that drives treatment guidelines, and expertise in the management of hematologic cancers, having pioneered more effective therapies for leukemia, lymphoma, and multiple myeloma. John Theurer Cancer Center was the first center in New Jersey to offer CAR T-cell therapy, a revolutionary immunotherapy for patients with select leukemias and lymphomas. The center is home to one of the nations largest bone marrow transplant programs, with more than 7,500 completed.

Many of the studies being presented at ASCO report on novel treatments for patients with recurrent or persistent multiple myeloma or non-Hodgkin lymphomas (including mantle cell lymphoma, an especially challenging type), as well as acute myeloid leukemia and chronic lymphocytic leukemia. John Theurer Cancer Center investigators are also leaders in the development of immunotherapy regimens, and several of the studies being presented at ASCO evaluated its effectiveness and side effects, including real-world data in unique patient populations. Other studies report provocative findings on targeted therapies and other treatments for kidney and bladder cancers, brain cancer, and other solid tumors. Data from the following studies by John Theurer Cancer Center researchers are being presented at ASCOs 2021 virtual meeting:

Blood Cancers and Stem Cell Transplantation

Developmental Therapeutics and Immunotherapy

Targeted Therapies and Tumor Biology

Central Nervous System Tumors

The COVID-19 pandemic challenged health care in ways we have never been challenged before. Despite the obstacles it presented, however, investigators at John Theurer Cancer Center continued to expand our understanding of cancer, refine its treatment, and develop innovative approaches to improve patient outcomes," asserted Andre Goy, M.D., M.S., chairman and executive director of John Theurer Cancer Center.

Read more here:
Innovative research refines the treatment of patients with advanced cancers and the use of immunotherapy - Network News, Press Releases - Hackensack...

In Some Heavily Pretreated Patients with R/R MM Ide-Cel Continues to Show Deep and Durable Responses – Targeted Oncology

Long-term follow-up data from the KarMMa trial found that treatment with the chimeric antigen receptor (CAR) T-cell therapy, idecabtagene vicleucel (ide-cel; formerly bb2121; Abecma), continues to demonstrate improved survival among heavily pretreated patients with relapsed/refractory multiple myeloma, according to a presentation presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.1

The favorable benefit risk profile of ide-cel, regardless of the number of prior lines of therapy, supports its role as a treatment option for heavily pretreated relapse refractory multiple myeloma, Larry D. Anderson, MD, PhD, associate professor, UT Southwestern Medical Center, said during a presentation of the poster.

At the December 21, 2020, data cutoff, the median follow-up was 24.8 months (range, 1.7-33.6).

Overall response rate (ORR) was 73% in the overall population, including a 33% complete response rate (CRR; complete response [CR] or stringent complete response [sCR]), 20% with a very good partial response (VGPR), and 20% who had a partial response (PR). ORR rates were 50%, 69%, and 81%, respectively, across the 150, 300, and 450 million CAR T cell-dose arms, including CR/sCR rates of 25%, 29%, and 39%.

Of note, ORR did not vary by the number of prior lines of therapy received. For those who received 3 prior lines of therapy (n = 15), the ORR was 73%, including a CRR of 53% and VGPR of 20%, compared with an ORR of 73% in those who received 4 (n = 112) lines of therapy, including a CRR of 30%, VGPR of 23%, and PR of 20%.

Median duration of response (DOR) was 10.9 months (95% CI, 9.0-11.4), including 9.9 months for the 300 million CAR T cells-dose arm and 11.3 months for the 450 million CAR T cells-dose arm -dose arm. Median DOR was 21.5 months in patients who experienced a CR or sCR. Median DOR by response were 21.5 months (95% CI, 12.5 to not estimable [NE]) among those who experienced a CR, 10.4 months (95% CI, 5.1-12.2) for those with VGPR, and 4.5 months (95% CI, 2.9-6.7) in those with PRs.

Moreover, the rate of event-free 24-month DOR appeared to be similar in patients who received 3 or 4 or more lines of therapy. For those who received 3 lines of prior therapy, median DOR was 8.0 months (95% CI, 3.3-11.4), compared with 10.9 months (95% CI, 9.2-13.5) in those who received 4 or more lines of therapy.

Median progression-free survival (PFS) was 8.6 months (95% CI, 5.6-11.6) across all target doses, including 5.8 months for the 300 million CAR T cells-dose arm and 12.2 months for the 450 million CAR T cells-dose arm -dose arm. Similarly, median PFS was similar among those who previously received 3 lines of therapy, compared with 4 or more prior lines of therapy (8.6 months (95% CI, 2.9-12.1) vs 8.9 months (95% CI, 5.4-11.6)]

The median time to first response was 1 month (range, 0.5-8.8), with a median time to CR of 2.8 months (range, 1.0-15.8).

Median overall survival (OS) was 24.8 months (95% CI, 19.9-31.2), including a median OS of 22.0 months (95% CI, 10.-NE) in those who received 3 lines of prior therapy and 25.2 months (95% CI, 19.9-NE) in those who received 4 or more lines of prior therapy. Moreover, OS was 20 months or longer across several key high-risk subgroups, including those aged 65 or older (21.7 months; 95% CI, 17.1-31.2), those with extramedullary disease (20.2 months; 95% CI, 15.5-28.3), and those with triple refractory disease (21.7 months; 95% CI, 18.2-NE).

In regards to safety, cytokine release syndrome (CRS) and neurotoxicity were similar, regardless of prior lines of therapy received, and were mostly low grade. In total, 85% and 18% of the overall population experienced at least 1 CRS or neurotoxicity event, respectively.

The safety profile of ide-cel was consistent with long-term follow-up, with similar rates of infections and secondary primary malignancies, and no unexpected gene therapy related toxicities were observed. The most common grade 3 to 4 adverse events (AEs) in the overall population were neutropenia (89%), anemia (61%), thrombocytopenia (52%), leukopenia (39%), lymphopenia (27%), and infections (27%).

Long-term results from the KarMMA trial continue to demonstrate frequent, deep, and durable responses in heavily pretreated patients with [relapsed/refractory multiple myeloma], the study authors write in the poster. ORR, CRR, DOR, and PFS were consistent with previous reports and patients received similar benefit regardless of the number of prior lines of therapy.

In his presentation, Anderson presented data on long-term efficacy and safety following treatment with ide-cel in the pivotal phase 2 KarMMa trial (NCT03361748)-including overall data and by prior line of therapy that patients had received (3 vs 4), since the FDA label is requiring at least 4 prior lines, and this study only required 3, he added.

In total, 140 patients who had received at least 3 prior lines of therapy for multiple myeloma including an IMiD, a PI, and an anti-CD38 antibody and were refractory to their last treatment regimen, were enrolled in the study. However, only 128 patients received infusion with ide-cel.

Patients were treated with ide-cel across the target dose range of 150 (n = 4), 300 (n = 70), and 450 (n = 54) million CAR T cells.

ORR served as the primary end point of the study. Secondary end points included CRR, safety, DOR, PFS, OS, pharmacokinetics, minimal residual disease, quality of life, and health economics and outcomes research.

At baseline, the median patient age was 61 years (range, 33-78) and patients had a median of 6 years (range, 1-18) since their diagnosis. A majority of the patients were male (59%), had high tumor burden (51%), B-cell maturation antigen (BCMA) expression 50% at screening (85%), ECOG performance status of 1 (53%), and Revised International Staging System disease stage of II (70%). Thirty-five percent of patients had high-risk features.2

The median number of prior therapies was 6 (range, 3-16) and 94% had previously undergone at least 1 autologous hematopoietic stem cell transplant (94%). Eighty-eight percent of patients required bridging therapy. Eighty-nine percent of patients had double-refractory disease, 84% were triple-refractory, and 26% were penta-refractory.

Patients who had received 3 prior lines of therapy had similar baseline characteristics, compared with those who received 4 prior lines, including differences in extramedullary disease, high-risk cytogenetics, prior refractoriness, and time since the initial diagnosis to screening.

Patients with relapsed/refractory multiple myeloma previously exposed to immunomodulatory agents, protease inhibitors, and anti-CD38 antibodies have poor outcomes with subsequent therapy using previously approved regimens, with expected response rates in the 26% to 31% range, PFS in the 2- to 4-month range, and overall survival less than 9 months, Anderson explained.

However, the BCMA-directed CAR T-cell therapy previously demonstrated favorable tolerability with deep, durable responses in patients who were heavily pretreated with relapsed/refractory multiple myeloma.2 As a result, the FDA approved the agent for the treatment of adult patients with relapsed or refractory multiple myeloma after 4 or more prior therapies, including an immunomodulatory drug, a proteasome inhibitor, and an anti-CD38 antibody, representing the first BCMAdirected CAR T-cell therapy approved.3

The study authors noted that ide-cel is being explored in ongoing clinical trials, including the following:

See more here:
In Some Heavily Pretreated Patients with R/R MM Ide-Cel Continues to Show Deep and Durable Responses - Targeted Oncology