The Florida-based company Bioheart announced today results from Part 1 of its FDA-approved Phase II/III clinical trial in which its proprietary adult stem cell product is being tested in the treatment of congestive heart failure (CHF). Known as the "MARVEL Clinical Program", the trial is designed to assess cardiac function and quality of life in patients with CHF after they are treated with their own adult stem cells. Specifically, the study utilizes a proprietary therapeutic product known as MyoCell, which is developed from autologous skeletal myoblasts (ASMs) derived from each patient’s own leg muscles. After patient biopsies were shipped to Bioheart laboratories, the ASMs were isolated, purified, expanded and returned to the physician for injection back into the patient. During a six-month observation period, the patients who were treated with their own ASMs were found to have increased their walking distance by 91 meters during a six-minute period of exercise, whereas those patients in the control group who were treated only with a placebo were found to have decreased their walking distance by 4 meters. As stated in Bioheart’s official press release, "This may suggest that patients with heart failure could return to a more active lifestyle after receiving Bioheart’s treatment."
Although this was a small pilot study, with an enrollment of 20 patients from whom 3 and 6 month follow-up data were obtained, it was an important step in the lengthy and expensive process of obtaining FDA marketing approval. Now that the product has met safety requirements, it will move on to Part 2 of the Phase II/III clinical trials, just as soon as the company achieves additional funding.
Thomas Povsic, M.D., Ph.D., principal investigator of the study and assistant professor at the Duke University Clinical Research Institute, which coordinated the clinical trial, presented the efficacy data from Part 1 of the MARVEL study at a meeting of the Heart Failure Society of America in Boston. Also participating in the presentation were Dr. Chris O’Connor, head of the heart failure department at Duke University, and Dr. Warren Sherman, director of Stem Cell Research and Regenerative Medicine at the Center for Interventional Vascular Therapy at Columbia University Medical Center.
According to Dr. Sherman, "For years it has been very clear that certain patients with CHF derive considerable benefit from myoblast implantation. However, our clinical impressions have not been substantiated, and won’t be until we have data available from a large, Phase II/III randomized, controlled study, powered to detect the effects of these cells on meaningful clinical endpoints. Part 2 of MARVEL will help answer this question and become a benchmark study. The field is replete with ‘next steps’ and other studies are waiting in the wings for the ice to be broken. MARVEL will do that."
As Karl Groth, Ph.D., president and CEO of Bioheart, adds, "The Part 1 results of the MARVEL trial show positive signs during treatment and an improvement in chronic heart failure patient’s ability to walk distances and be active. This means that a patient may well return to a more active lifestyle. We are committed to moving this study forward and to seeing our technology impact on the population affected by chronic heart failure."
As Dr. Povsic further added, "We are pleased to be able to present this data to our heart failure colleagues. The MARVEL-1 trial was the first truly blinded study of intramyocardial stem cell delivery via a percutaneous approach. The results have immense implications for the design of MARVEL-2 as well as other similar trials. The improvements we observed in the 6-minute walk are clinically meaningful, and this is the first time improvements have been demonstrated in relation to a blinded placebo control group. MARVEL-1 is a pilot study, but the findings are encouraging and suggest that additional trials are warranted."
The American Heart Association estimates that there are over 5 million people suffering with CHF in the U.S. alone, with direct and indirect costs exceeding $30 billion per year. An additional 500,000 patients are diagnosed with CHF in the U.S. each year. Since conventional medicine currently offers no known cure for CHF, an autologous adult stem cell therapy would be able to fill an ever-increasing and unmet need.
As described on their website, "Bioheart is a biotechnology company focused on the delivery, development and, subject to regulatory approval, commercialization of autologous cell therapies for the treatment of chronic, acute heart damage, and peripheral vascular disease." The company develops its proprietary therapeutic products exclusively from autologous adult stem cells. In no case are embryonic stem cells ever used, since embryonic stem cells pose a number of technical problems and medical dangers, not the least of which is the formation of teratomas (tumors). By sharp contrast, adult stem cells do not pose any such dangers, and of all the various types of adult stem cells, autologous (in which the donor and recipient are the same person) adult stem cells are among the most easily attainable, with no risk of immune rejection and therefore no need for dangerous immunosuppressive drugs.
As further described on Bioheart’s website, the company’s lead product, MyoCell, "is an innovative clinical muscle-derived stem cell therapy designed to populate regions of scar tissue within a patient’s heart with new living cells for the purpose of improving cardiac function in chronic heart failure patients." In addition to MyoCell, other products in Bioheart’s pipeline include multiple candidates based upon autologous, adipose-derived adult stem cells, and MyoCell SDF-1, a therapy developed from autologous adult stem cells that are genetically modified to express additional growth proteins of potential therapeutic value.
Shares of Bioheart rose 22%, or 42 cents, at the news of the clinical trial data, closing today at $2.30.