Program Emphasizes Clinical Applications
The National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health has awarded the first three grants in a new research program on cell-based therapy of heart, lung, and blood diseases.
The program, Specialized Centers for Cell-Based Therapy for Heart, Lung, and Blood Diseases, involves both basic and clinical research but is heavily focused on clinical applications of cell-based therapy.
"Recent advances in stem cell biology and transplantation have set the stage for the next level of research emphasis: a program that emphasizes the translation of knowledge about cell-based therapy into clinical practice," said NHLBI Director Elizabeth G. Nabel, M.D.
According to Dr. Nabel, the $6.5 million program is anticipated to stimulate clinical research efforts on important public health problems for those with heart, lung, and blood diseases. The program will attempt to solve some of the problems and challenges of cell-based therapy including repair of damaged heart muscle, reducing immune complications due to graft versus host disease, and enhancing the interaction of adult stem cells and their tissue environment. Many of the 5-year studies will begin with preclinical animal or laboratory research to support an Investigational New Drug Application submission to the FDA followed by Phase I and II clinical studies for safety and effectiveness, respectively.
The centers awarded the grants and the principal investigators are:
- Baylor College of Medicine, Houston, TX (Malcolm Brenner, M.D., Ph.D.) This group will focus on two clinical studies. In one study, the scientists will genetically modify donor immune cells used in stem cell therapy for treatment of patients with cancer and other diseases. The donor cells will be enhanced to speed recovery and reduce the effects of graft versus host disease, a potential complication of stem cell transplantation. The other clinical study involves modification of immune cells used in stem cell transplants to protect the recipients from the viral infections that are so common in immune compromised patients. A basic research study will investigate potential therapies using stem cells in the heart.
- Johns Hopkins University, Baltimore, MD (Joshua Hare, M.D.) This research team will study the development of stem cell-based therapies to regenerate the heart and to reverse heart failure in patients with ischemic heart disease. The team will initially study the use of bone-marrow derived mesenchymal stem cells as a treatment for ischemia (reduced blood flow to the heart). Mesenchymal stem cells have the potential to develop into mature cells that produce fat, cartilage, bone, tendons, and muscle and have been shown to reduce heart damage in animals following a heart attack. In future years, the team will also study the use of human cardiac stem cells grown from small pieces of human heart tissue with the goal of delivering them back to patients following a heart attack.
- Massachusetts General Hospital, Boston, MA (David Scadden, M.D.) This project team will focus on the specialized microenvironments where stem cells reside, targeting them to achieve stem cell therapies and tissue regeneration. Emphasis will be placed on the blood stem cell with laboratory and clinical studies using proteins to alter the stem cell microenvironment in the bone marrow. Treatment trials of stem cell therapies for individuals with hematologic cancers such as lymphoma, Hodgkin's disease and multiple myeloma will be the initial clinical studies performed.
- The EMMES Corporation, Rockville, MD (Shelly Carter, Sc.D.) EMMES will serve as the coordinating center for the program and will work with the Specialized Centers on establishing useful standard protocols for the emerging new field of cell-based therapies.
To interview an NHLBI spokesperson about this program, contact the NHLBI Communications Office at 301-496-4236. To interview Dr. Brenner, contact Ross Tomlin in the Office of Public Affairs at Baylor College of Medicine at (713) 798-7973; to interview Dr. Hare, contact David March at Johns Hopkins at 410-955-1534; to interview Dr. Scadden, contact Sue McGreevey at Massachusetts General Hospital Public Affairs at 617-724-2764; to contact Dr. Carter, call Sandi Sykes at 301-251-1161.