Stem cell therapy trial at Sanford first of its kind in U.S. for shoulder injuries

FDA-approved study explores utility of fat-derived stem cells SIOUX FALLS, S.D. – The first FDA-approved clinical trial of its kind in the United States using a person’s own fat-derived adult stem cells to treat shoulder injuries is available at Sanford Health. The trial opened in December and explores if adipose stem cells, which are taken from a participant’s own abdominal fat, can safely repair small and partial thickness tears in the rotator cuff by activating the body’s natural healing processes when injected into the injury area. The cell therapy technique may accelerate healing or regenerate tissue. According to clinicaltrials.gov, it’s the only trial the FDA has approved that uses adipose stems cells for this type of injury. Sanford’s Jason Hurd, M.D., who is based in Sioux Falls, and Mark Lundeen, M.D., of Fargo are the study’s principal investigators. Both are orthopedic surgeons. “Sanford Health physicians and scientists are the first in the country to work with the FDA on a trial using adipose stem cells in rotator cuff tears, which are quite common,” said Kelby Krabbenhoft, president and CEO of Sanford Health. “We’ve been monitoring the potential of these types of stem cells for quite some time. In Europe, adipose stem cells have been used as a therapy option for damaged tissues and are approved to carry the CE mark, which signifies that a product has been assessed by and meets certain safety, health and environmental protection requirements in the European Union.” Cell therapy, according to Sanford experts, uses the body’s own cells as therapy. Stem cells, in particular, have the ability to repair or regenerate cells that are damaged or killed as the result of injury or disease. Sanford’s cell therapy techniques focus on [...]

Landmark Alzheimer’s prevention trial to evaluate third drug

Effort to study drug's ability to prevent, delay the disease By Tamara Bhandari December 19, 2016 Washington University School of Medicine's Randall J. Bateman, MD, talks with DIAN-TU trial participant Natalie Shriver, of Omaha, about a study to test drugs that may prevent or delay Alzheimer's disease. (Photo: Robert Boston/Washington University School of Medicine) An international team led by Washington University School of Medicine in St. Louis has selected a third investigational drug to be tested in a worldwide clinical trial — already underway — aimed at finding treatments to prevent Alzheimer’s disease. The third drug is being developed by Janssen Research & Development, LLC, in New Jersey. It is designed to lower production of amyloid beta, a protein that clumps together into plaques and damages neurons in the brain, leading to memory loss, cognitive problems and confusion. The drug is designed to block the enzyme beta secretase — which produces amyloid beta — with a goal of reducing the amount of amyloid beta available to clump and cause neurodegeneration. This investigational drug joins two others already being evaluated in the Dominantly Inherited Alzheimer’s Network Trial Unit (DIAN-TU) study, which involves people with an inherited predisposition to develop Alzheimer’s at a young age, usually in their 30s, 40s or 50s. Participants already enrolled will continue on their existing drug regimens, and additional volunteers with no or mild symptoms of cognitive impairment will be enrolled to evaluate the third drug. “We are delighted with the new collaboration with Janssen Research & Development to expand the number of novel therapeutic targets we are testing,” said Washington University Alzheimer’s specialist Randall J. Bateman, MD, director of the DIAN-TU, a public-private-philanthropic research partnership. “Testing a beta secretase inhibitor in [...]

Researchers launch first clinical trial for Wolfram syndrome

Many people with rare genetic disease die prematurely by Jim Dryden•November 10, 2016 The drug dantrolene is a muscle relaxant approved to treat patients with cerebral palsy, multiple sclerosis and muscle spasticity. Recent research also suggests it can prevent the destruction of insulin-secreting beta cells in animal models of Wolfram syndrome. Researchers at Washington University School of Medicine in St. Louis are launching a new clinical trial to assess the safety of a drug treatment for patients with the rare disease Wolfram syndrome. Wolfram syndrome affects about one in every 500,000 people worldwide. Many of those patients die prematurely from the disease. Patients with Wolfram syndrome typically develop diabetes at a very young age and require insulin injections several times each day. The disorder also causes hearing loss, vision problems and difficulty with balance. Although doctors treat patients’ symptoms, there have not been any therapies that slow the syndrome’s progress. However, researchers at Washington University School of Medicine soon will test a drug treatment in 24 patients who have the genetic disorder. The scientists previously reported in the Proceedings of the National Academy of Sciences that the drug, dantrolene — a muscle relaxant approved to treat patients with cerebral palsy, multiple sclerosis and muscle spasticity — prevents the destruction of insulin-secreting beta cells in animal models of Wolfram syndrome and in brain cells differentiated from skin samples taken from patients with the illness. “Nobody has ever tested dantrolene in patients with Wolfram syndrome, so our first and most important objective is to make sure it’s safe,” said principal investigator Fumihiko Urano, MD, PhD, the Samuel E. Schechter Professor of Medicine. “I am very hopeful, however. The major question that I get from every patient I [...]

Innovative Regenerative Approach to Healing Diabetic Foot Ulcers Now Being Trialed at Two Additional UK sites

November 8, 2016 LONDON, UNITED KINGDOM and PERTH, AUSTRALIA and NORTHRIDGE, CA--(Marketwired - Nov 8, 2016) - Avita Medical (ASX: AVH), (OTCQX: AVMXY) Two London hospitals join the study to evaluate safety and effectiveness of new approach using ReGenerCell™ Innovative regenerative technology uses patients' own skin cells to close wounds caused by chronic Diabetic Foot Ulcers (DFUs) Diabetes costs the NHS £8.8bn pa (almost 10% of the NHS budget) with 140 diabetic patients a week having an amputation Treatment is now underway at three UK hospitals to trial an innovative approach of using a patient's own cells to close foot ulcers amongst diabetics, an affliction that leads to some 140 amputations per week across Britain. Avita Medical (ASX: AVH), (OTCQX: AVMXY), a regenerative medicine company specialized in wound treatment, said two new sites have been added to the study on the safety and effectiveness of its treatment of Diabetic Foot Ulcers (DFUs), and that ReGenerCell™ device is now deployed at London's King's College and Northwick Park Hospitals. The DFU clinical trial began earlier in 2016 at the Manchester Royal Infirmary, with seven patients already enrolled and being treated at that site. Avita said that with three sites now enlisted, enrollment can proceed apace to recruit up to 24 patients with DFUs, who will be followed over a 26-week evaluation period. Full enrollment of the trial is anticipated to be completed by early 2017. The treatment will be assessed as an adjunct to standard of care treatments, such as debridement, cleansing, dressings, and offloading. As well as the key outcome measures of incidence of healing and rate of wound closure, the study will also explore patient and physician satisfaction. ReGenerCell™, which is CE-marked and approved for sale [...]

CIRM-Funded Clinical Trial for ALS Given Go Ahead to Treat Patients

October 24, 2016 Oakland, CA – An ingenious method that uses a patient as their own control has been given clearance to start a clinical trial to treat amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease. The California Institute for Regenerative Medicine (CIRM) is funding the trial. ALS is a devastating disease that destroys nerve cells in the brain or spinal cord. There is no treatment or cure, and the average life expectancy for someone with ALS is 3 - 4 years.  Every 90 minutes someone in the US is diagnosed with ALS. Every 90 minutes someone in the US dies of ALS. The clinical trial is led by Clive Svendsen, PhD, director of the Board of Governors Regenerative Medicine Institute at Cedars-Sinai, who has spent a dozen years developing the new approach: “Any time you’re trying to treat an incurable disease, it is a long shot, but we believe the rationale behind our new approach is strong.” The new approach targets motor neurons, cells which help control movement in the body and that are killed by ALS. Svendsen and his team are using specially engineered stem cells to deliver a protein – GDNF – that is essential to the health of motor neurons. The hope is that by providing the motor neurons with GDNF they can keep them alive. How they intend to prove that is the ingenious part. Previous studies have shown that patients with ALS experience the same rate of deterioration of movement in both legs simultaneously. Using a new device developed specifically for this trial, Svendsen intends to inject the GDNF-producing stem cells in one side of the spinal cord, into an area that controls movement in just one [...]

Stem Cell Agency Spinal Cord Injury Clinical Trial Passes Safety Hurdles

Posted: August 31, 2016 Oakland, CA – A clinical trial using stem cells to treat people with recent spinal cord injuries has cleared two key safety hurdles, and been given approval to expand the therapy to a larger group of patients with a much higher dose of cells. Asterias Biotherapeutics announced that its Data Monitoring Committee (DMC) has reviewed the safety data from the first two groups of patients treated and found no problems or adverse side effects. One group of three patients was given 2 million cells. The second group of five patients received 10 million cells. Asterias is now cleared to enroll another 5-8 patients with 20 million cells. The SciStar study, funded in part by the California Institute for Regenerative Medicine (CIRM) is a Phase 1/2a clinical trial that is designed to test first the safety and then the effectiveness of Asterias’ AST-OPC1 cells. These are a form of cells called oligodendrocyte progenitors, which are capable of becoming several different kinds of cells some of which play a supporting role and help protect nerve cells in the central nervous system, including areas of the spinal cord that are damaged in spinal cord injury. “Our focus is always on the patient, so making sure a potential therapy is safe is an important first step,” says C. Randal Mills, Ph.D., the President and CEO of CIRM. “I recently met with Jake Javier, a young man who was treated in this trial, and heard first-hand what he and his family are going through in the aftermath of his injury. But I also saw a young man with remarkable courage and determination. It is because of Jake, and the others who volunteer to take part in [...]

From labs to lives: Self-replicating cells help treat neuro disorders

July 27, 2016 Scientists estimate that human bodies contain anywhere from 75 to 100 trillion cells. And of these cells, there are hundreds of different types. Yet, one cell type in particular has captured the fascination of assistant professor David Brafman: the human pluripotent stem cell (hPSC).Assistant professor David Brafman mentoring biomedical engineering junior Lexi Bounds, who plans to pursue a career in stem cell research. Photographer: Jessica Hochreiter/ASU As self-replicating cells — capable of dividing and forming new cells — hPSCs offer immense research potential. They are able to provide the raw material needed to generate the hundreds of different cell types that comprise the human body. Think of it as a reverse e pluribus unum. Something like out of one, come many. Brafman has received a $420,000 grant from the National Institutes of Health to take discoveries related to hPSCs out of the research lab and into the clinical setting where they can transform, even save, lives. In particular, his research focuses on using the remarkable qualities of hPSCs to generate large quantities of hPSC-derived neurons, which are instrumental in advances toward the study and treatment of Alzheimer’s disease, ALS, spinal cord injuries and other neurodegenerative disorders. “Neurodegenerative diseases and disorders remain some of the leading causes of mortality and morbidity in the United States,” said Brafman, a biomedical engineering faculty member in ASU’s Ira A. Fulton Schools of Engineering. According to the Alzheimer’s Association, the disease affects more than 130,000 individuals statewide and is the fifth leading cause of death in Arizona. “Several bottlenecks limit the translation of hPSCs and their derivatives from bench to bedside,” said Brafman, referring to the need to take this research from the laboratory bench to the clinical [...]

Napster Co-Founder Funds Immunotherapy Research Project

Wed, 04/13/2016 - 10:00am Ryan Bushey, Digital Editor, R&D Parker (left) made a generous donation to cancer research. (Credit: The Parker Institute)Sean Parker, the entrepreneur behind music-sharing service Napster, is providing a $250 million grant to support a consortium aimed at development of immunotherapy research.This venture, a non-profit named the Parker Institute for Cancer Immunotherapy, will bring together more than 300 scientists from six major academic cancer research centers, according to The Washington Post. Stanford, Memorial Sloan Kettering Cancer Center, and the University of Pennsylvania are some of the members taking part in this endeavor. One member from each of the participating institutions will constitute a steering committee, which will establish the group’s research agenda as well as enable better collection of data and clinical trials across all of the involved labs. It’s a similar strategy being implemented in Vice President Joe Biden’s Cancer Moonshot. A key tenet of this initiative is to “break down silos” regarding communication between the public and private sectors as a way to find a cure for these diseases. Plus, The Washington Post notes another unique innovation of Parker’s project is that the team will be able to retain ownership of the intellectual property while taking charge on licensing and negotiating potential market opportunities with drug-makers. Parker provided this money because he felt immunotherapy research, “is at a turning point and would benefit from research that is done without regard for its costs,” reported USA Today. This class of drugs essentially activates the body’s immune system in order to combat foreign pathogens. Early studies have shown these treatments have promise, but more research needs to be done in order to ensure these drugs can work on a large group of patients and [...]

Stem cell therapy ‘halves number of heart failure deaths’

April 11, 2016 Spectator Health reporter Injecting adult stem cells into the heart could potentially halve the number of deaths from heart failure, a study has suggested. The study, published in the Lancet, found that patients who received the treatment had a 37 per cent lower rate of death and hospitalisation for heart failure-related problems. The study involved 126 patients from hospitals across America. Sixty patients were injected with stem cells while 66 were injected with a placebo. The procedure took two hours and most patients were discharged a day after surgery. In the non-placebo group, stem cells were taken from the patients’ bone marrow, grown in culture, and then injected directly into the patients’ hearts. In the stem cell therapy group 3.4 per cent of the patients died and 51.7 per cent were hospitalised, compared to 13.7 per cent and 82.4 per cent respectively in the group that received a placebo. Dr Amit Patel, director of Cardiovascular Regenerative Medicine at the University of Utah, said: ‘For the last 15 years everyone has been talking about cell therapy and what it can do. These results suggest that it really works.’ He said it works either because it ‘slows down or reverses the rate of progression of disease’. Rather than increasing the number of muscle cells or blood vessels in the heart, he said, it likely makes the existing cells work better. However, the study authors say they found only a small overall improvement in heart function. The researchers are hoping to start a larger phase 3 clinical trial involving more heart failure patients. About 900,000 people in Britain live with heart failure, which blocks blood vessels and reduces blood supply to the body. Instant analysis [...]

Potential Huntington’s Treatment Successful in Animals, Moves to Clinical Testing

Tue, 03/01/2016 - 9:50am Bevin Fletcher, Associate Editor A new drug that treats the underlying cause of Huntington’s disease has been shown to be effective and safe in mice and monkeys, and a Phase 1/2a clinical human trial has begun.The findings will be presented at the American Academy of Neurology’s 68th Annual Meeting in Vancouver, Canada, in April. The drug, called IONIS-HTTRx, was developed by Ionis Pharmaceuticals, together with CHDI Foundation, Roche Pharmaceuticals and researchers at the University of California San Diego. It acts as a “gene silencer” that prevents the production of Huntington protein in people with the hereditary disease. This type of drug is known as an antisense treatment. Huntington’s disease is caused by genetically programmed degeneration of neurons in certain areas of the brain, which leads to uncontrolled movements, loss of intellectual abilities, emotional problems and eventually death.  Each child of a parent with Huntington’s has a 50/50 chance of inheriting the gene.  If a child does not inherit the gene, he or she cannot pass it on to subsequent generations and will not develop the disease.  Anyone who inherits the mutated gene will sooner or later develop the disease. Currently there are no drugs to stop or reverse Huntington’s, though there are a number of medications prescribed to help control emotional and movement problems associated with the disease. “It is very exciting to have the possibility of a treatment that could alter the course of this devastating disease,” said clinical study principal investigator Blair R. Leavitt, M.D. of the University of British Columbia in Vancouver, in a prepared statement. “Right now we only have treatments that work on the symptoms of the disease.” According to Leavitt, it will still be years before [...]