Cell Therapy Foundation analysis calls for funding in medical research

Aug 31 2010

Some of the most promising medical research conducted to treat diseases of the brain, heart, and circulatory system is moving too slowly to help millions of patients, according to the Cell Therapy Foundation, which advocates the use of adult stem cells to dramatically impact and improve the condition of patients who suffer from a variety of conditions.

A Cell Therapy Foundation analysis of current clinical trials points to a critical need: more federal, private, and philanthropic funding for adult stem cell clinical trials.   Key gaps exist between breakthrough developments in basic research and translation to patients, revealing the essential need for funding in this area.

One gap is in the transition from pre-clinical work to early clinical trials.  Another is in the progression from early clinical trials to larger, definitive clinical studies.  There is a clear need for development of new concepts and funding strategies to bridge these gaps, building on already established platforms or establishing new centers dedicated to translation of stem cell and regenerative medicine research from the bench to the bedside.  

"Remarkable work is being done, but we must find more funding to get the critical research out of the laboratory and into practice, to help people with new therapies that utilize adult stem cells as a catalyst for healing," said Dr. Sotirios Karathanasis, Vice President and Head of Bioscience in Astra-Zeneca, and member of the advisory board of the Cell Therapy Foundation.

Headquartered in Indianapolis, IN, the Cell Therapy Foundation is a not-for-profit organization committed to advancing research and the treatment of disease using adult stem cells.  To bolster awareness, the Cell Therapy Foundation is highlighting clinical trials and centers that are dedicated to translating new adult stem cell advances into patient therapies, particularly focusing on disease areas that have not been previously addressed by standard bone marrow approaches, or for which there is currently no adequate therapy via more traditional therapeutic approaches.

In the USA, only a handful of such clinical trials have reached Phase III, the stage prior to submission for regulatory approval.  Phase III cell therapy studies for heart disease, peripheral vascular disease and multiple sclerosis have been initiated and are recruiting patients.

"Researchers need more support to move clinical trials along, addressing these and many other critical diseases.  Before a treatment can be approved, a definitive clinical ("Phase III") trial must show a positive impact to fight a disease.  The very limited number of Phase III trials, and even earlier ("Phase II") trials, illustrates the critical need for much more funding to be dedicated to the area of adult stem cell therapeutics. The recent breakthroughs in our understanding of stem cell and regenerative biology, and exciting early clinical data, point to a future of a very different medicine, involving rescue or replacement of damaged tissues by regenerated functional healthy tissues, opening the way for true disease cures. These developments may revolutionize the way medicine is practiced in the not-too-distant future, and such novel approaches will provide new hope for many serious diseases that impact millions of people," added Dr. Karathanasis.  

Among these Phase III trials currently underway in the United States:

Heart Disease: This heart disease trial is focused on testing whether transplantation of muscle-derived stem cells will benefit patients with severe and symptomatic heart failure.  Initial results from this trial were presented at the Heart Failure Society of America meeting and early indications from this trial appear encouraging.  It is sponsored by Bioheart, Inc., and its lead investigator is Dr. Warren Sherman, Director, Stem Cell Research and Regenerative Medicine, Columbia University Medical Center. Relevant to this, a recently-reported breakthrough in this area is the demonstration that three genetic factors alone are sufficient to reprogram non-embryonic cells from skin into beating ventricular cardiomyocytes.  This finding, if translated to humans, will have important implications due to its potential for cell transplantation from one part of the body to another part and even individualized therapy.

Vascular Disease:  Biomet Biologics is sponsoring a pivotal study on peripheral vascular disease using bone marrow aspirate concentrated at the point-of-care, for patients with threatened amputation.  The lead investigator is Dr. Michael P. Murphy, Associate Professor of Vascular Surgery at the Indiana University School of Medicine, and Clinical Director of the Vascular and Cardiac Center for Adult Stem Cell Research.

Multiple Sclerosis:  A trial addressing multiple sclerosis, sponsored by Northwestern University, is evaluating the use of peripheral blood stem cell transplants.  The lead investigator is Dr. Richard Burt, Associate Professor, Division of Immunotherapy at Northwestern University.