Category Archives: New York Stem Cells

NEW YORK, Jan. 13, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium"), announced today that it has entered into an agreement with the University of California, Davis (UC Davis) to utilize Actinium's Antibody Radiation-Conjugate or ARC apamistamab-I-131 for targeted conditioning and replace the chemotherapy conditioning being used in an ongoing Phase 1/2 stem cell gene therapy clinical trial. In the trial, patients with relapsed or refractory HIV-related lymphoma are being treated with autologous stem cell gene therapy. This is the first gene therapy clinical trial that will utilize ARC based conditioning. The clinical trial will be conducted at UC Davis and may be expanded to additional sites in the future.

Dr. Mehrdad Abedi, Professor, Hematology and Oncology at UC Davis and study lead, said, "This collaboration represents an exciting combination of revolutionary technologies that could further our ability to treat patients with HIV and other life-threatening diseases with gene therapy. Despite the advances made in the field of gene therapy, the reliance on non-targeted chemotherapy and external radiation as conditioning regimens is less than optimal and poses a problem that we hope to reduce or eliminate as part of this collaboration by replacing our conditioning regimen in this study with Actinium's ARC based targeted conditioning. Advances in HIV therapies have dramatically improved patient survival, but current therapies require life-long daily use to keep the HIV virus at bay, can have severe side effects, may be overcome by HIV resistance and do not address the needs of all patients like those in this study with HIV-related lymphomas. We envision a future where a single treatment of our stem cell gene therapy can cure patients of their lymphoma and HIV leaving the patient with a new immune system that can fight, be resistant to and prevent the mutation of HIV. Apamistamab-I-131's demonstrated antitumor effect against lymphoma and ability to condition patients in a targeted manner with a demonstrated tolerable safety profile in the bone marrow transplant setting makes it an ideal conditioning agent for this patient population. Based on these factors and extensive supporting clinical data in the Iomab-B program, we selected this ARC as the conditioning agent for the next phase of our trial as we believe antibody radiation-conjugates are more advanced and hold distinct advantages over novel but unproven conditioning technologies such as Antibody Drug Conjugates and naked antibodies that are beginning to be developed albeit at the preclinical stage."

In the current clinical trial, the anti-HIV stem cell gene therapy is produced by taking a patient's own or autologous, blood forming stem cells and genetically modifying them via gene therapy with a combination of three anti-HIV genes. The intended result is for the gene modified bone marrow stem cells to produce a new immune system and newly arising immune cells that are resistant to HIV via a single treatment. Conditioning is necessary prior to adoptive cell therapies such as gene therapy to eliminate certain cell types such as immune cells and stem cells in the bone marrow so the transplanted cells can engraft. Until now, conditioning in this trial, as is typical, used a multi-drug chemotherapy regimen administered over several days. This approach is non-targeted, associated with toxicities that impairs patients and restricts the use and efficacy of cellular therapy. Apamistamab-I-131, which requires just one therapeutic administration, will displace the non-targeted chemotherapy to condition patients in a targeted manner with the goal of reducing conditioning related toxicities and improving patient outcomes. Actinium and UC David will cross-reference their respective Investigational New Drug applications and will work collaboratively to obtain necessary regulatory and institutional approvals. In this clinical collaboration, Actinium will provide drug product, support for its administration and certain trial costs. UC Davis will be responsible for the production of the anti-HIV stem cell gene therapy and overall conduct of the study and its cost.

Dr. Dale Ludwig, Actinium's Chief Scientific Officer, said, "We are excited to be working with Dr. Abedi on this clinical study and we appreciate his recognition of the value of our Iomab-ACT targeted conditioning program may provide in support of gene stem cell therapy. This targeted approach using our CD45 ARC, enables both anti-tumor activity and effective conditioning with the potential for reduced toxicity compared to non-targeted chemotherapy and external radiation in the bone marrow transplant setting. Supported by extensive clinical investigation in 12 trials and over 300 patients, a single therapeutic dose of apamistamab-I-131 is sufficient for conditioning and, due to its dual activity, even a patient with active disease could expect to receive therapy within two weeks, which is anticipated to lead to better outcomes compared to chemotherapy, external beam radiation, or exploratory approaches such as naked antibodies or Antibody Drug Conjugates. In addition, CD45, the target of apamistamab-I-131, is ideal for targeted conditioning, as it is not expressed outside of the haemopoietic system and, because it is a poorly internalizing receptor. An ARC approach which does not require internalization of its radionuclide warhead for target cell killing, is anticipated to be more viable and more effective than Antibody Drug Conjugate approaches which need to internalize their payloads. Given the potential of this ARC targeted conditioning technology for bone marrow transplant, we are grateful to Dr. Abedi for the opportunity to advance the Iomab-ACT program into the promising field of gene stem cell therapy."

Sandesh Seth, Actinium's Chairman and Chief Executive Officer, said, "Actinium is thrilled to be working with UC Davis and honored to now be part of this important trial. It has become evident that better conditioning regimens are needed for cell and gene therapies to reach their full potential. Our team is proud to be the first company to establish a clinical stage targeted conditioning portfolio for both cell and gene therapy. We are pleased to extend our ARC technology for targeted conditioning into these rapidly advancing fields and we are committed to establishing a strong leadership position in enabling these adoptive cell therapies fully realize their great potential for improving patients' lives."

Apamistamab-I-131's demonstrated conditioning and antitumor effect in lymphoma1

Actinium's apamistamab-I-131 ARC has been studied as a targeted conditioning agent in over 300 patients in the bone marrow transplant setting in the Iomab-B Program and is currently being studied in a pivotal Phase 3 clinical (SIERRA) trial in patients with relapsed or refractory acute myeloid leukemia. Clinical proof of concept has been established with Iomab-B for targeted conditioning in high-risk, relapsed or refractory lymphoma patients prior to an autologous stem cell transplant where a favorable safety profile with no dose limiting toxicities and minimal non-hematologic toxicities observed and promising efficacy with median overall survival not reached (range: 29 months to infinity) and 31% of patients in prolonged remission at a median of 36 months follow up (range: 25 41 months)1.

1) Cassaday et al. Phase I Study of a CD45-Targeted AntibodyRadionuclide Conjugate for High-Risk Lymphoma. AACR Clin Cancer Res Published OnlineFirst September 3, 2019

About Actinium Pharmaceuticals, Inc.

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, apamistamab-I-131 (Iomab-B) is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and promising single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. Apatmistamamb-I-131 will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc.

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Hans VitzthumLifeSci Advisors, LLCHans@LifeSciAdvisors.com(617) 535-7743

Media:Alisa Steinberg, Director, IR & Corp Commsasteinberg@actiniumpharma.com(646) 237-4087

SOURCE Actinium Pharmaceuticals, Inc.

http://www.actiniumpharma.com/

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Actinium Pharmaceuticals Announces Iomab-ACT Program Gene Therapy Collaboration with UC Davis in Ongoing Clinical Trial for Patients with HIV-Related...

The passage of time holds few changes for the Ginkgo biloba tree, commonly known as the maidenhair. For tens of millions of years and through multiple mass extinctions, this "botanical oddity" has stood unwavering, an ever-fixed "living fossil", as the world ages around it.

The species is practically immortal - and that's not an exaggeration. As it turns out, individual trees can live over 1,000 years(some accounts suggest 3,000 even), and now, the most detailed study to date suggests their lifespan is theoretically unlimited.

While ageing and death are a natural part of being alive in this world, some plants like the ginkgo show few signs of growing old.

Even though these trees grow thinner annual rings as the years go on, researchers have found little difference in their ability to photosynthesise, germinate seeds, grow leaves, or resist disease compared to younger trees.

In fact, examining tissue samples from nine ginkgo trees aged up to over 600 years old,the team was unable to find any evidence of senescence, or deterioration, at all.

"In humans, as we age, our immune system begins to start to not be so good," biologist Richard Dixon from the University of North Texas told The New York Times, adding that "the immune system in these trees, even though they're 1,000 years old, looks like that of a 20-year-old."

Unlike previous studies, which have focused mainly on the ginkgo's leaves, the new research hones in on the tree's vascular cambium - a thin layer of tissue in the trunk that produces new bark and wood.

This region contains meristem cells, which are similar to stem cells in animals, although far less researched at a molecular level.

To figure out how the cambrium changes with age, researchers examined each individual's cambrium activity, hormone levels, and resistance-associated genes, as well as transcription factors connected to cell death.

In all the tree ages, they found no significant difference in gene activity or disease resistance. In fact, theonly thing that really changed was the width of the tree's rings, which appeared to decrease sharply during the first 100 and 200 years, before continuing at a slower decline over the next few hundred years.

But this doesn't mean all growth was hindered. Interestingly enough, secondary tree growth (measured by the tree's basal area increment, or BAI), did not show any decline from 10- to 600-year-old ginkgo trees.

"Since BAI is a reliable indicator of tree growth," the authors write, "it seems that the vascular cambium in G. biloba can retain the capacity for continuous growth for hundreds of years or even millennia."

It's this feature, theythink, that allows the tree to "escape senescence at the whole-plant level".

To be clear, this doesn't mean that ginkgo trees will never die, just that they probably won't die of old age. Instead, ginkgos usually fall from other external factors like wind, fire, lightning, disease, or overlogging, which has, incidentally, brought the species to the brink of extinction in modern times.

"Ageing is not a problem for this species," plant physiologist Sergi Munn-Bosch, who was not involved in the study, told Science. "The most important problem that they have to deal with is stress."

What happens to the ginkgo tree after 600 years of life, however, is still up for debate. There's still a chance this ancient tree species will begin to show signs of molecular ageing in the upper reaches of its lifespan, but the scope of this study was just too small to say.

Further research will be needed before we can know for sure what happens to this special tree over the course of its long, long life.

The study was published in PNAS.

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This Ancient Tree Species Is Virtually Immortal, And We Finally Understand How - ScienceAlert

In the months after the gene therapy infusion at Boston Childrens, her symptoms disappeared. But doctors had given her blood transfusions while she regrew her own red blood cells, so it was not clear if the absence of symptoms was because of the gene therapy or the transfusions.

As she recovered, Helen returned to her passion: dancing. One day, she came back from her school dance group and told her mother, My legs hurt. It feels funny. Ms. Cintron smiled. Thats soreness, she explained. Helen laughed. She had only known pain from sickle cell.

Helen was scheduled for her six-month checkup on Dec. 16. By then, all the transfused cells were gone, leaving only blood made by stem cells in her own marrow. The doctors would finally tell her whether the therapy was working.

The day before, she and her parents visited the New England Aquarium in Boston. She was able to stay outside on a cold, blustery day, watching one seal bully the others, barking and fighting. When Helen mentioned that her hands were cold, Ms. Cintrons stomach clenched in fear. But it was just a normal thing to feel on a winter day.

The next morning, Dr. Esrick delivered the news. Helens total hemoglobin level was so high it was nearly normal a level she had never before achieved even with blood transfusions. She had no signs of sickle cell disease.

Now you are like me, her father told her. I jump in the pool, I run. Now you can do it, too!

Her family, accustomed to constant vigilance, is only now getting used to normal life.

On Dec. 23, Helen and her mother flew to the familys new home in Arizona.

Helen recently described her transformed outlook on Facebook.

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At 16, Shes a Pioneer in the Fight to Cure Sickle Cell Disease - The New York Times

NEW YORK, Jan. 9, 2020 /PRNewswire/ -- Tacitus Therapeutics, a clinical-stage company, has launched in collaboration with the Mount Sinai Health System to develop stem cell therapies initially targeting blood cancers and related clotting disorders. Their first therapy, HSC100, currently is being investigated in a Phase I clinical trial1.

Tacitus is building upon technology developed by and exclusively licensed from Mount Sinai. Based on research by scientific co-founders Ronald Hoffman, M.D., and Camelia Iancu-Rubin, Ph.D., the technology includes proprietary cell expansion, differentiation and engineering methods. Together, these methods manufacture healthy cells that overcome the limitations of traditional allogeneic, or donor, cell transplantations.

Blood cancers comprise about 10% of new cancer cases in the U.S. each year, and almost 60,000 people die from blood cancer complications annually. Most blood cancers start in the bone marrow, where blood is produced. A common therapy for such blood cancers is a hematopoietic stem cell (HSC) treatment or, as more commonly referred to, bone marrow transplantation. In this process, doctors infuse healthy HSCs into the patient's bloodstream, where they migrate to the bone marrow to grow or engraft.

HSCs for this process can be collected from bone marrow, circulating blood, or umbilical cord blood (CB) of healthy donors. While HSC transplants are common, significant barriers to success exist, including high levels of graft-versus-host disease, low numbers of healthy cells obtained from CB, and increased risk of bleeding due to delayed megakaryocyte, or platelet, engraftment.

Hoffman and Iancu-Rubin are pioneers of bone marrow cell therapy treatments, and development of this technology was enabled by the New York State Stem Cell Science program, NYSTEM. As a New York State Department of Health initiative, NYSTEM awarded a $1 million grant to Hoffman in 2010 that supported the original research underpinning this platform technology. In 2015, NYSTEM awarded Hoffman and Iancu-Rubin an $8 million grant to translate the technology from the laboratory into the clinic, where it is currently in clinical trial1.

Hoffman also serves as Director of the Myeloproliferative Disorders Research Program and Professor of Medicine (Hematology and Medical Oncology) and Iancu-Rubin is Associate Professor of Pathology at the Icahn School of Medicine and Director of the Cellular Therapy Laboratory at Mount Sinai Hospital.

"Promising discoveries by Mount Sinai scientific thought leaders may lead to new, essential cell-based therapies that will broadly benefit patients," said Erik Lium, Executive Vice President and Chief Commercial Innovation Officer, Mount Sinai Innovation Partners. "We're pleased to be collaborating with Tacitus to launch the next stage of development for these technologies."

"Tacitus is committed in its mission to advance next-generation cell therapies with curative potential," said Carter Cliff, CEO of Tacitus. "Based on our founders' solid foundation of research, we are translating these discoveries into broad clinical practice as we look to dramatically improve the standard of care for patients with life-threatening conditions."

About HSC100

HSC100 is an investigational therapy based on allogeneic hematopoietic stem cells (HSC) expanded from umbilical cord blood. HSC100 is being investigated currently in an open-label Phase I clinical trial1 in the United States for treatment of hematological malignancies. The success of unmanipulated cord blood as a source of stem cells has been hampered by the small number of stem cells present in a single cord, leading to delayed engraftment and frequent graft failure. Our proprietary technology includes the use of an epigenetic modifier, valproic acid, to expand the number and the quality of HSCs found in cord blood collections. For more information on HSC100 clinical trials, please visit http://www.clinicaltrials.gov.

1ClinicalTrials.gov identifier NCT03885947.

About Tacitus Therapeutics

Tacitus Therapeutics is a clinical-stage biotechnology company developing advanced medicines for treatment of blood cancers, immune disorders and other intractable disease conditions. Our mission is to pioneer best-in-class therapies using proprietary cell expansion, differentiation and engineering platform technologies that overcome the limitations of traditional cell transplantation. Initial targets include a lead clinical program (HSC100) investigating the treatment of blood cancers, followed by preclinical programs to address clotting disorders and other serious unmet medical needs. For additional information, please visit http://www.tacitustherapeutics.com.

About Mount Sinai Health System

The Mount Sinai Health System is New York City's largest integrated delivery system, encompassing eight hospitals, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region. Mount Sinai's vision is to produce the safest care, the highest quality, the highest satisfaction, the best access and the best value of any health system in the nation. The Health System includes approximately 7,480 primary and specialty care physicians; 11 joint-venture ambulatory surgery centers; more than 410 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. The Icahn School of Medicine is one of three medical schools that have earned distinction by multiple indicators: ranked in the top 20 by U.S. News & World Report's "Best Medical Schools", aligned with a U.S. News & World Report's "Honor Roll" Hospital, No. 12 in the nation for National Institutes of Health funding, and among the top 10 most innovative research institutions as ranked by the journal Nature in its Nature Innovation Index. This reflects a special level of excellence in education, clinical practice, and research. The Mount Sinai Hospital is ranked No. 14 on U.S. News & World Report's "Honor Roll" of top U.S. hospitals; it is one of the nation's top 20 hospitals in Cardiology/Heart Surgery, Diabetes/Endocrinology, Gastroenterology/GI Surgery, Geriatrics, Gynecology, Nephrology, Neurology/Neurosurgery, and Orthopedics in the 2019-2020 "Best Hospitals" issue. Mount Sinai's Kravis Children's Hospital also is ranked nationally in five out of ten pediatric specialties by U.S. News & World Report. The New York Eye and Ear Infirmary of Mount Sinai is ranked 12th nationally for Ophthalmology, Mount Sinai St. Luke's and Mount Sinai West are ranked 23rd nationally for Nephrology and 25th for Diabetes/Endocrinology, and Mount Sinai South Nassau is ranked 35th nationally for Urology. Mount Sinai Beth Israel, Mount Sinai St. Luke's, Mount Sinai West, and Mount Sinai South Nassau are ranked regionally. For more information, visit http://www.mountsinai.org or find Mount Sinai on Facebook, Twitter and YouTube.

About Mount Sinai Innovation Partners (MSIP)

MSIP is responsible for driving the real-world application and commercialization of Mount Sinai discoveries and inventions and the development of research partnerships with industry. Our aim is to translate discoveries and inventions into health care products and services that benefit patients and society. MSIP is accountable for the full spectrum of commercialization activities required to bring Mount Sinai inventions to life. These activities include evaluating, patenting, marketing and licensing new technologies building research, collaborations and partnerships with commercial and nonprofit entities, material transfer and confidentiality, coaching innovators to advance commercially relevant translational discoveries, and actively fostering an ecosystem of entrepreneurship within the Mount Sinai research and health system communities. For more information, please visit http://www.ip.mountsinai.orgor find MSIP onLinkedIn, Twitter, Facebook,Medium, and YouTube.

Media Contacts:

Mount Sinai Cynthia Cleto Mount Sinai Innovation Partners (646) 605-7359 cynthia.cleto@mmsm.edu

Tacitus TherapeuticsJoleen RauRau Communications(608) 209-0792232130@email4pr.com

SOURCE Tacitus Therapeutics

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Tacitus Therapeutics Launches in Collaboration with Mount Sinai to Develop Stem Cell Therapies for Life-Threatening Diseases - PRNewswire

NEW YORK, Jan. 07, 2020 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, announced today that Chaim Lebovits, President and Chief Executive Officer, will provide a corporate overview at the 2020 Biotech Showcase, being held on January 13-15, 2020 at the Hilton San Francisco Union Square in San Francisco, California.

Mr. Lebovits will also present at the 3rd Annual Neuroscience Innovation Forum, taking place on January 12, 2020, at the Marines Memorial Club in San Francisco. Additionally, Ralph Kern M.D., MHSc, BrainStorms Chief Operating Officer and Chief Medical Officer, will participate on aRare & Orphan Diseases Panel.

Meetings

BrainStorms senior management will also be hosting institutional investor and partnering meetings at the 2020 Biotech Showcase conference (https://goo.gl/SGFm62). Please use the Investor contact information provided below to schedule a meeting.

About NurOwn

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

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. For more information, visit BrainStorm's website at http://www.brainstorm-cell.com.

Safe-Harbor Statement

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

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BrainStorm Cell Therapeutics to Present at the 2020 Biotech Showcase and 3rd Annual Neuroscience Innovation Forum at JPM Week - Yahoo Finance

A Chinese scientist who set off an ethical debate with claims that he had made the worlds first genetically edited babies three, according to a Chinese court was sentenced to three years in prison because of his research, state media, the Associated Press and the New York Times have reported.

He Jiankui, who was convicted of practicing medicine without a license, was also fined 3 million yuan ($430,000) by a court in the southern city of Shenzhen, Chinas official Xinhua News Agency reported on Monday. Two other researchers involved in the project received lesser sentences and fines.

The verdict said the three defendants had not obtained qualification as doctors, pursued fame and profits, deliberately violated Chinese regulations on scientific research, and crossed an ethical line in both scientific research and medicine, according to Xinhua. It also said they had fabricated ethical review documents.

The court also confirmed a third birth, saying the researchers were involved in the births of three gene-edited babies to two women. It said all three scientists pleaded guilty during the trial, which Xinhua reported was closed to the public because of privacy concerns.

Hes declaration made him a pariah among scientists, cast a harsh light on Chinas scientific ambitions and embroiled other U.S. scientists who were connected to He. Although He offered no proof and did not share any evidence or data that definitively proved he had done it, his colleagues had said it was possible that he had succeeded, The New York Times reported.

U.S. scientists who knew of Hes plans came under scrutiny. Hes former academic adviser, Stephen Quake, a star Stanford University bioengineer and inventor, was cleared of any wrongdoing after an investigation into his interaction with his former student. Rice University has been investigating Michael Deem, Hes doctoral adviser, because of allegations that he was actively involved in the project.

Duke engineers improve CRISPR genome editing with biomedical tails

He, the lead researcher, shocked the scientific world when he announced in November 2018 that he had altered the embryos of twin girls who had been born the same month. He described his work in exclusive interviews with The Associated Press.

The announcement sparked a global debate over the ethics of gene editing. He said he had used a tool called CRISPR to try to disable a gene that allows the AIDS virus to enter a cell, in a bid to give the girls the ability to resist the infection. The identity of the children has not been released, and it isnt clear if the experiment succeeded.

The CRISPR tool has been tested elsewhere in adults to treat diseases, but many in the scientific community denounced Hes work as medically unnecessary and unethical, because any genetic changes could be passed down to future generations. The U.S. forbids editing embryos except for lab research.

He, who is known as JK, told the AP in 2018 that he felt a strong responsibility to make an example, and that society would decide whether to allow the practice to go forward. He disappeared from public view shortly after he announced his research at a conference in Hong Kong 13 months ago, apparently detained by authorities, initially in an apartment in Shenzhen, a city in Guangdong province that borders Hong Kong.

It wasnt clear if the three-year prison term includes any of the time he has already spent in Chinese custody.

A Chinese scientist said the sentence should have been harsher to deter others. Kehkooi Kee, a Tsinghua University researcher who conducts gene-editing research on stem cells, also said that He should be held responsible for any fallout from the experiment on the lives of the babies and their families.

Dr. William Hurlbut, a Stanford University bioethicist whose advice He sought for more than a year before his experiment, said he felt sorry for the scientist, his wife and two young daughters.

I warned him things could end this way, but it was just too late, Hurlbut wrote in an email addressed to the AP; the director of the U.S. National Institutes of Health, Dr. Francis Collins; and gene-editing pioneer Jennifer Doudna at the University of California, Berkeley.

Sad story everyone lost in this (JK, his family, his colleagues, and his country), but the one gain is that the world is awakened to the seriousness of our advancing genetic technologies, Hurlbut wrote.

Dr. Eric Topol, who heads the Scripps Research Translational Institute in California, noted its almost unheard of for a scientist to get imprisoned but in this case the sheer recklessness and unethical behavior warranted it. Topol praised China for standing up for proper medical research conduct.

Doudna told the AP she was concerned about the mysterious legal process in China, but she said the sentences are a step toward bringing this case to closure and send a strong message to discourage other such work. (Doudna is paid by the Howard Hughes Medical Institute, which also supports APs Health and Science Department.)

As a scientist, one does not like to see scientists going to jail, but this was an unusual case, Doudna said. Hes work was clearly wrong in many ways.

Before setting up a lab at the Southern University of Science and Technology of China in Shenzhen, He studied in the U.S. The verdict accused him of colluding with Zhang Renli and Qin Jinzhou, who worked at medical institutes in the same province.

Zhang was sentenced to two years in prison and fined 1 million yuan, Xinhua said. Qin received an 18-month prison sentence, but with a two-year reprieve, and a 500,000 yuan fine.

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China confirms third birth of gene-edited baby; scientists involved get prison terms - WRAL Tech Wire

Looking good, lady!Sarah Fergusonis gearing up to celebrate her upcoming milestone birthday, and the beloved royal is doing whatever it takes to make sure she looks her best! During a recent interview with the Daily Mail, the Duchess of York opened up about experimenting with cosmetic procedures.

Ive had a lot of help to look like this at 60! Sarah, who is celebrating her upcoming 60th birthday on October 15, candidly shared with the U.K. outlet. Ive started the laser treatment, but its not finished yet. The collagen needs to rebuild. I hope it will all be done by my birthday.

The red-haired royal, who has long been known to fans as Fergie, referred to the same treatment she received ahead of her daughter Prince Eugenies royal wedding in November 2018. Fergie explained that she prefers non-invasive laser treatments, like the 6-Dimension Platinum Laser Lift by her friend Dr. Gabriela Mercik, opposed to more complicated procedures.

I dont like the frozen look, she confessed. Im so animated and I like to be myself. I dont like the thought of needles and am very glad if I look well and happy Im really happy to be open about what Ive had done.

Although Fergie admitted to previously trying botox, she ultimately decided to only undergo less invasive treatments in pursuit of a more youthful appearance. The former wife of Prince Andrewrevealed shes even been experimenting with mesotherapy since 2013 in hopes to help clear up years of sun damage.

According toHealthline, mesotherapy uses injections of vitamins, enzymes, hormones and plant extracts to rejuvenate and tighten the skin on your face. I need to repair the damage that was done on the beach when I was a child, Fergie explained. Its why I had the mesotherapy, the vitamin cocktail to hydrate and boost the skin.

Besides mesotherapy, Fergie has also had a facial thread lift, which involves temporary sutures placed under the skin to lift sagging areas and smooth away fine lines. Its like garden trellising for sweet peas. You insert the threads under the skin with a fine needle and they hold everything up, she said. They also encourage collagen production. It takes a couple of months, then the sweet peas bloom!

Although Fergie anticipated an extremely painful procedure, the royal beauty dished that it actually wasnt as bad as she thought. My skin responded well, she said. I think if you look at photos of me after I had it done, I look much better.

While Fergies hoping to have a more youthful look at her upcoming birthday bash, the duchess explained that the procedures dont stop at her face! Earlier this year, Fergie traveled from her home in London to the Bahamas for a new therapy treatment for her feet.

I think my toes were ruined by all the riding I did when I was young. They shaved the bone here, she said, pointing to her big toe, and implanted stem cells, 20 million of them taken from my midriff, into my feet to make new cartilage. It takes about six months to heal but now I can walk in heels!

We bet Fergie is going to look and feel better than ever at her upcoming birthday party!

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Sarah Ferguson Talks Botox and Facelifts: 'I've Had a Lot of Help' - Closer Weekly

GettyBrooke Nevils pictured on her Twitter profile page.

Brooke Nevils is an American television producer and former NBC News employee who alleges that Matt Lauer anally raped her in his hotel room at the 2014 Sochi Olympics.

Variety obtained a copy of Ronan Farrows new book Catch and Kill, in which Farrow interviews the 35-year-old Nevils, who describes the incident in detail. Nevils complaint was the catalyst to Lauer being fired as co-anchor of the Today show in 2017. NBC had kept her identity and the details of the allegations private until now.

Per Variety:

In Sochi, Nevils was tasked with working with former Today co-anchor Meredith Vieira, whod been brought back to the show to do Olympics coverage. In her account, one night over drinks with Vieira at the hotel bar where the NBC News team was staying, they ran into Lauer, who joined them. At the end of the night, Nevils, whod had six shots of vodka, ended up going to Lauers hotel room twice once to retrieve her press credential, which Lauer had taken as a joke, and the second time because he invited her back. Nevils, Farrow writes, had no reason to suspect Lauer would be anything but friendly based on prior experience.

Once she was back in his hotel room, Lauer who was wearing a T-shirt and boxers pushed her against the door and kissed her. He then pushed her onto the bed, flipping her over, asking if she liked anal sex, Farrow writes. She said that she declined several times.'

According to Farrows book, she allegedly was in the midst of telling him she wasnt interested again when he just did it, Farrow adds, Lauer, she said, didnt use lubricant. The encounter was excruciatingly painful. It hurt so bad. I remember thinking, Is this normal? She told me she stopped saying no, but wept silently into a pillow.

When Lauer asked if she liked it, she told him yes, according to Farrows book. She then tells Farrow that she bled for days, following the incident.

Nevils grew up in Missouri and graduated from Johns Hopkins University with a double major in political science and the Writing Seminars. She was a TV producer at NBC working at 30 Rockefeller Plaza until 2018 when she went on medical leave after leaving NBC and allegedly receiving a seven-figure payout from them, according to Varietys reporting.

Shes billed as a producer on several high-profile shows for NBC including Headliners, 90 Day Fianc: Happily Ever After?, and Royal Wedding Watch.

Lauer wrote a lengthy open letter denying any wrongdoing and saying that everything with Nevils was part of a consensual affair.

I had an extramarital affair with Brooke Nevils in 2014. It began when she came to my hotel room very late one night in Sochi, Russia. We engaged in a variety of sexual acts. We performed oral sex on each other, we had vaginal sex, and we had anal sex. Each act was mutual and completely consensual, Lauer wrote. The story Brooke tells is filled with false details intended only to create the impression this was an abusive encounter. Nothing could be further from the truth. There was absolutely nothing aggressive about that encounter. Brooke did not do or say anything to object. She certainly did not cry. She was a fully enthusiastic and willing partner. At no time did she behave in a way that made it appear she was incapable of consent. She seemed to know exactly what she wanted to do. The only concern she expressed was that someone might see her leaving my room. She embraced me at the door as she left.

Lauers full letter can be read here.

Nevils responded in a statement published by NBC News later on October 9, saying, Theres the Matt Lauer that millions of Americans watched on TV every morning for two decades, and there is the Matt Lauer who this morning attempted to bully a former colleague into silence, she said, in part. His open letter was a case study in victim blaming I am not afraid of him now, regardless of his threats, bullying, and the shaming and predatory tactics I knew he would (and now has) tried to use against me.

NBC News issued a statement saying, Matt Lauers conduct was appalling, horrific and reprehensible, as we said at the time. Thats why he was fired within 24 hours of us first learning of the complaint. Our hearts break again for our colleague.

Heres what you need to know:

GettyMatt Lauer attends The Rolling Stones celebrate the North American debut of Exhibitionism at Industria in the West Village on November 15, 2016 in New York City.

After the incident at the 2014 Sochi Olympics, Nevils told Ronan Farrow that she continued her relationship with Lauer. Sources close to Lauer emphasized that she sometimes initiated contact, Farrow writes, according to Variety. What is not in dispute is that Nevils, like several of the women Id spoken to, had further sexual encounters with the man she said assaulted her.

This is what I blame myself most for, Nevils tells Farrow in an interview It was completely transactional. It was not a relationship.

Nevils says in Farrows book that she continued her relationship with Lauer because she was intimidated by the power he held over her career. She also claims she told like a million people about her encounter with Lauer including her colleagues and superiors at NBC.

Nothing was done about the incident until 2017 when the #metoo movement led her Today show colleagues to ask about Lauer and urge her to report the incident to NBC.

After colleagues urged her to report the 2014 incident, she went to NBC Universal human resources with a lawyer, Farrows book reports. Her report caused NBC Universal to fire Lauer. In Farrows book, Nevils alleges that President of NBC News Noah Oppenheim, and Chairman of NBC News and MSNBC Andrew Lack were emphasizing that the incident hadnt been criminal or an assault. She claims that when she heard this she threw up.

She was promised anonymity by NBC Universal but the company said internally that the incident happened at the Sochi Olympics. This detail limited the number of possible complainants and her colleagues eventually figured out it was her.

Nevils work life became torture, Farrow writes. She was made to sit in the same meetings as everyone else, discussing the news, and in all of them colleagues loyal to Lauer cast doubt on the claims, and judgment on her.

Nevils says she never wanted money but NBC still paid her seven figures after she went on medical leave in 2018.

The network proposed a script she would have to read, suggesting that she had left to pursue other endeavors, that she was treated well, and that NBC News was a positive example of sexual harassment, according to Farrow.

On October 9, after Varietys article on Farrows book was posted, NBC Today show hosts Hoda Kotb and Savannah Guthrie addressed the news on the show formerly hosted by Lauer.

I feel like we owe it to our viewers to pause for a moment, Guthrie said. This is shocking and appalling. I honestly dont even know what to say about it. I want to say I know it wasnt easy for our colleague Brooke to come forward then, its not easy now and we support her and any women who have come forward with claims. And its just very painful for all of us at NBC and who are at the Today show. Its very, very, very difficult.

Kotb added, We dont know all the facts on all of this, but there are not allegations of an affair, there are allegations of a crime. I think thats shocking to all of us here who have sat with Matt for many, many years. So I think were going to just sort of continue to process this part of this horrific story and as you said, our thoughts are with Brooke. Its not easy what she did, to come forward. Its not easy at all.

NBC News chairman Andy Lack issued a statement saying, Our highest priority is to ensure we have a workplace environment where everyone feels safe and protected. We are absolutely committed to making this a reality there can be no exception.

Farrow tweeted, Nevils did an incredibly brave thing coming forward the way she did. She upended her life to ensure accountability and protect others.

Ann Curry also showed Nevils support, tweeting, Brooke Nevils is a credible young woman of good character. She came to NBC News an eager and guileless 20-something, brimming with talent.I believe she is telling the truth. And that breaks my heart.

Nevils took to Twitter on October 9 to thank those who have supported her.

I want to thank the many survivors who shared their stories with me today and offered their support. It takes courage, and I am truly grateful, Nevils wrote.

Brooke Nevils is originally from Chesterfield, Missouri, and graduated from Parkway West High School in 2003. After graduating from Johns Hopkins in 2007, Nevils moved to New York City and got a job as a page for NBC at 30 Rockefeller Plaza. She started out greeting guests and getting them to the set in time for their interviews.

In an interview with Arts & Sciences Magazine, Nevils says she had injured her leg in a water skiing accident right before starting and was forced to work injured her first week on the job.

My first week at The Today Show, I was taking Vicodin for my leg and getting up at 3:30 a.m. to get to work. She told Arts & Sciences, It was certainly the last place in the world I saw myself getting a job. But it worked out.

One of the first fires she had to put out was finding actor Robert De Niros wallet after he lost it in a New York City cab. Shortly after Robert De Niro arrived at the studio, his manager told Nevils he had left his wallet in their cab

I cant leave Bobby, De Niros manager told her. You gotta get it back for me. Nevils eventually found the cab, found the wallet and returned it to the Academy Award-winning actor.

After her tenure as a page ended, Nevils became a personal assistant to Today Show host Meredith Vieira for 10 months. Her relationship led to her becoming an assistant producer for A Leap of Faith: A Meredith Vieira Special in 2014 where she was nominated for an Emmy for Outstanding Feature Story in a News Magazine as a member of the production team.

According to IMDB, in the special Vieira interviews thoracic surgeon Paolo Macchiarini. This controversial surgeon has performed trachea transplant surgeries using patients stem cells as well as transplanting synthetic tracheas.

Macchiarini went on to have an affair with Benita Alexander-Noel, the producer of the documentary, and was later exposed in this Vanity Fair article. He proposed to Alexander-Noel and promising her a lavish wedding with celebrity guests before it was later found out that the wedding was a big hoax and he was still married to his wife of 30 years.

According to The Guardian, most of the 17 patients he gave regenerating windpipes to are now dead.

Along with her work with Vieira, Nevils was a talent assistant at the Today show, an associate producer on Rock Center With Brian Williams, and a researcher for NBC Nightly News, among other roles at the news network.

In addition to her work as a Producer at NBC, Nevils occasionally writes for Womans Day Magazine. She contributes self-help articles and how-to articles on things related to money and womens issues.

Her content varies wildly in subject matter from Could the Tragedy at UVA Happen to Anyone? to How to Pack Like a Travel Pro.

She wrote most of her content in 2010 and 2011 where she published an article every few months but her work slowed down drastically, presumably as her producer work intensified. Her last article, 8 Clever Strategies for Saving Big With Coupons was published in February 2016.

Nevils has also worked as an editorial assistant at Chicago Magazine and was an editorial board intern at the Chicago Tribune and the Baltimore Sun and an editorial intern at The Atlantic Monthly. She now lives in New York, according to her Twitter profile.

READNEXT: Robert De Niros Former Assistant Accuses Him of Sexual Harassment

More:
Brooke Nevils: 5 Fast Facts You Need to Know - Heavy.com

See video of emotional encounter, via Gift of Life Marrow Registry, HERE

(Starts at 15:37) Video credit: Gift of Life Marrow Registry

NEW YORK, Sept. 20 A life-saving Baltimore, Md., blood stem cell donor met his transplant recipient, a Philadelphia, Pa., boy, for the very first time in an emotional ceremony at Gift of Life Marrow Registry's One Huge Night Gala in New York City on September 17.

Gregory Mitchell, 30, a financial advisor, donated stem cells last year to Marcus Haggins, 9, who was battling Severe Aplastic Anemia, until receiving his transplant. Mitchell joined Gift of Life's registry during a 2012 donor recruitment drive at an Atlantic City, N.J. cultural festival.

"My life became more meaningful the day I donated," Mitchell said, calling his joining the registry a once-in-a-lifetime opportunity. "By joining Gift of Life, you might get lucky enough to have the opportunity to step up to the plate for someone who needs you the most. You might be lucky enough to know how great it feels to donate to someone. You might even get lucky enough to know how great it feels to meet such a brave person."

Haggins' mother, Kim Kennedy, said that "you don't know how much this means to me to give me my son back. Thank God we didn't lose Marcus." Referring to Gift of Life and Mitchell, said added that "they saved my son's life. You are my angel. There is no greater gift than life. None."

Due to medical anonymity laws, donors and recipients must wait at least one year before meeting.

Since its start in 1991, Gift of Life Marrow Registry has grown to more than 347,000 individuals who have volunteered to donate blood stem cells or bone marrow to save a life. To date, Gift of Life has facilitated over 16,600 matches for those with leukemia, lymphoma, sickle cell, and nearly 100 other diseases, resulting in more than 3,475 transplants

See original here:
Life-saving Baltimore stem cell donor meets 9-year old recipient - Patch.com

FDA ought to promote stem cell therapy by easing up on regulation and its aggressive enforcement.

On International Rare Disease Day 2017, one month after being sworn in as President, Donald Trump gave his 2017 Joint Address to Congress. During his speech, he took particular note of the slow and burdensome approval process at the Food and Drug Administration (FDA) that keeps too many advances from reaching those in need. With a specific emphasis on the health of sick children, President Trump argued that if we slash the restraints at FDA, then we will be blessed with far more miracles.

In attendance that night was Sarah Hughes, a young woman who was forced to travel to Mexico for stem cell therapy (SCT) to treat her systemic idiopathic juvenile diabetes. In 2014, Hughes had her own cells extracted, processed and then infused back into her in a process known as adult autologous stem cell therapy. The results were life-changing.

Before the SCT, Hughes was taking 23 medications a day. After nearly two dozen stem cell infusions over a two-year period, Hughes was down to eight medications a day, and at lower doses. SCT alleviated Hughes chronic pain, allowed her to eat normally and absorb nutrients from food, and gave her choices in life she never had before. Despite her progress, she lamented the fact that that other Americans in her position could not avail themselves of SCT.

Since delivering his address to Congress, President Trump has in fact made progress in modernizing FDA, most notably by signing into law the Right to Try Act of 2017, which allows terminally ill patients increased access to experimental drugs that have completed Phase I of the clinical trial process but have not been approved by FDA.

President Trumps actions continue a broader trend in easing patients access to emerging medical treatments. In December 2016, for example, President Obama signed the 21st Century Cures Act into law, which contains special provisions for the accelerated approval for advanced regenerative therapies like SCT.

Despite the clear trend toward FDA modernization and the easing of restrictions by Presidents Obama and Trump, the U.S. House of Representatives Energy and Commerce Committee has recently signaled that it wants to see enhanced FDA regulatory enforcement over SCT. Leaders of the committee sent a letter to Acting FDA Commissioner Ned Sharpless voicing its concern about FDAs seemingly permissive use of its discretionary regulatory enforcement authority against potentially violative clinics.

The Committee is seeking more information about FDAs long-term enforcement strategy, including: financial resources dedicated to approving legitimate SCT products; human resources dedicated to the reporting of adverse events; and the possibility of state-federal partnerships to revoke the medical licenses of SCT clinicians.

To be sure, concerns over the safety of patients receiving SCT are reasonable and necessary. But any call for increased regulatory enforcement against clinics offering SCT is premature and will likely disadvantage far more Americans than it helps. At a time when an increasing number of Americans suffer from debilitating chronic medical conditions, we need more medical choice, not less.

The unspoken truth is that despite the constant invocation of the threat of harm from SCT, the actual number of reported cases of adverse harm is remarkably few. Conversely, success stories are numerous.

These SCT successes are built upon robust scientific literature and clinical practice that demonstrate the safety and efficacy of SCT for certain medical indications. The scientific and anecdotal evidence on SCTs efficacy in treating orthopedic conditions is substantial. Research has shown that it can facilitate the healing of bone fractures, stimulate cartilage regeneration, treat meniscus repair, and decrease lower back painthe greatest contributor to global disability according to 2010 Global Burden of Disease data.

Stem cell therapy has also been shown to treat both the chronic pain caused by opioid abuse and the effects of opioid tolerance. Likewise, SCTs ability to treat the symptoms of certain autoimmune conditions is well established. Perhaps most noteworthy is the virtual absence of adverse events that can be directly ascribed to adult SCT.

Although officials such as former FDA Commissioner Scott Gottlieb and current Commissioner Sharpless acknowledge the power and uniqueness of SCT, federal policy on stem cell research and rulemaking has a Janusian quality. On the one hand, it has expanded significantly in the past decade as a result of the relaxing of rules restricting embryonic and other types of stem cell research and the passage of federal laws aimed at expediting regenerative medicine therapies to market. On the other hand, FDA regulations that define the standards for determining which therapies can be offered without FDA approval and those that require approvallegally deemed drugstend to operate to slow down medical innovation.

In fulfillment of its obligations under the Cures Act, FDA released a guidance document in November 2017 with a new framework that is intended to balance the agencys commitment to safety with mechanisms to drive further advances in regenerative medicine so innovators can bring new, effective therapies to patients as quickly and safely as possible. Importantly, the guidance purports to clarify the terms minimally manipulated and homologous use, key standards that determine the availability of stem cell therapies to patients. Stem cells that are minimally manipulated and used for homologous purposes do not need to undergo clinical trials. However, by all accounts, the guidance document interprets these terms quite narrowly, effectively proscribing the therapies altogether.

In an effort to facilitate compliance for clinics that offer unapproved SCT services, FDA has stated that for the first 36 months following issuance of the guidance, it will adopt a risk-based approach to enforcement of the new rules. So far, FDA has indeed exercised its enforcement authority judiciously, targeting clinics that it deems flagrant in their marketing or medical practices. Enforcement has generally taken the form of either warning letters or federal lawsuits. The combination of FDAs narrow interpretations with increasing public demand for alternative medical therapies, however, has meant that the number of clinics offering unapproved SCT products has grown.

The Energy and Commerce Committee should consider the following four factors in determining how to proceed. First, it must recognize that SCT is a unique and unprecedented medical modality that requires a unique regulatory enforcement approach that balances the interest of regulators, scientists, clinician and patients.

Second, FDAs stated three-year grace period has not yet run. Any evaluation of enhanced enforcement should be deferred until that time comes.

Third, the reporting of adverse events resulting from the use of SCT in clinics is astonishingly small. Of course, any adverse events should be meticulously documented, investigated, and taken seriously. That said, to indict an entire practice because of the negligence or recklessness of a few is a step too far, especially given how many Americans have benefitted from SCT.

Fourth, it is precisely because so many people with no viable medical alternatives have benefitted from the therapy that the Committee should re-think its aggressive posture toward enforcement. When taken together, these four factors weigh against enhanced federal enforcement at this time.

Furthermore, states have played a critical role in SCT regulation. In 2017, Texas Governor Greg Abbott signed a law that makes Texas the first state to authorize the use of SCT for patients with certain severe chronic condition or terminal illnesses. Arkansas is on course to be the first state to require medical insurance companies to cover stem cell therapy.

States are also actively participating in enforcement against clinics that they believe fraudulently market SCT. New York State Attorney General Letitia James recently filed a lawsuit against a clinic offering SCT, maintaining that it misled patients with deceptive marketing practices. The Illinois Department of Financial and Professional Regulation is also investigating patient complaints. These two cases show that states are more than capable of weeding out alleged SCT bad actors without enhanced efforts by FDA.

Like Sarah Hughes, I was forced to travel abroad for SCT. In 2010, I traveled to Nanjing, China for SCT to treat a progressive neuromuscular condition. Although my time in China was wonderful, no Americans should have to travel for SCTa safe and inexpensive therapyto save or improve their lives. Stem cell therapy is a paradigm-shifting medical modality that allows persons to use cells from their own bodies to heal themselves. As such, it embodies the democratization of medicine. To unduly stifle, impair, or otherwise restrict the availability and affordability of SCT would not only injure the constituents that members of Congress fight for, but also wound our democratic ideals.

Despite disagreements over policy, all interested parties agree that SCT represents a revolution in medicine. Americans have recognized this shift, and the desire for SCT has reached a tipping point. Thoughtful, judicious, and balanced regulatory enforcement that targets the most flagrant bad actors and allows states to take the lead is the proper way forward at this point.

Read more here:
When it Comes to Federal Stem Cell Regulation, Less is More - The Regulatory Review