Institute researchers develop cell-based treatments for a broad array of injuries and conditions, with many different approaches and cell types. Because of limitations on embryonic stem cell research, the Institute is committed to exploring the full potential of adult stem and progenitor cells. The McGowan Institute hosts over two dozen faculty isolating and characterizing a variety of cell types, and investigating how to drive them into new, clinically relevant lineages.
| First Heart Failure Patients Ever to Receive Stem Cells in Minimally Invasive Procedure Have Improved Heart Function |
| Patients with severe congestive
heart failure who had exhausted all other treatment options
showed markedly improved heart function following a procedure
in which their own stem cells were deployed directly into the
heart by way of four tiny incisions in the chest, according
to results of a trial presented on January 25, 2005 at the
41st Annual Meeting of the Society for Thoracic Surgery.
The study, led by Amit N. Patel, M.D., M.S., of the McGowan Institute for Regenerative Medicine, is the first to use a minimally invasive surgical technique. While preliminary, the results of the prospective randomized trial indicate that a minimally invasive approach to cell therapy is feasible for the estimated 40 percent of heart failure patients whose disease is unrelated to coronary blockages and who therefore cannot benefit from bypass procedures. Moreover, the experience so far suggests the novel stem-cell approach may be a viable treatment for these and other heart failure patients, reported Dr. Patel, director of clinical cardiac cell therapies at the McGowan Institute. All 15 of the patients who received stem cell injections had some degree of improvement, some with dramatic results, while the conditions essentially remained unchanged in the 15 randomized to receive injections of their own blood serum. "It is remarkable the level of improvement we’ve seen in these patients, who came to us with no other medical or surgical options available to them. However, we don’t yet fully understand how these cells work, whether they differentiate to become heart muscle cells or cells that promote vessel growth, or whether they serve as homing signals to other cells and substances that help with repair," explained Dr. Patel.
The study took place at centers in South America. The research team obtained the necessary institutional and government health agency approval and each patient provided informed consent. The McGowan Institute now has approval for this country’s first FDA-approved phase I trial of a surgical cardiac stem cell therapy, injecting autologous bone marrow-derived CD34+ stem cells into the cardiac muscle of heart failure patients who have been implanted with VAD devices. As donor hearts become available to these patients, McGowan Institute researchers will have an opportunity to examine the original hearts for signs of stem cell proliferation and differentiation. MORE |
| Stem Cell Treatment for Urinary Incontinence |
| McGowan Institute researchers
Johnny Huard, PhD and Michael Chancellor, MD have developed
a treatment for stress urinary incontinence using autologous
muscle-derived stem cells. These are isolated from a small
biopsy in the patient’s calf,
replicated in culture over several weeks, then injected into
the bladder sphincter. The stem cells persist in the bladder
for up to six months and differentiate into smooth muscle tissue,
improving contractility. This technology has been licensed
to Cook MyoSite, Inc., and is being evaluated in a phase II
trial. MORE |
Duchenne Muscular Dystrophy
| Duchenne muscular dystrophy is an X-linked genetic disorder afflicting one in 3,500 boys, most of whom will die in adolescence or young adulthood as their muscles – including their hearts – weaken from lack of dystrophin. McGowan Institute researchers have identified and isolated a novel, uniquely robust line of myogenic-derived stem cells that offers new hope for Duchenne patients. These stem cells can express certain myogenic proteins on their own. When they are genetically engineered to contain a gene encoding for dystrophin, they actually produce dystrophin in Duchenne-affected muscles. This work, under the leadership of Dr. Johnny Huard may produce a minimally invasive treatment in which the patient's own stem cells are isolated, transfected in vitro to carry a functioning dystrophin gene, and reinjected into dystrophin-lacking muscle. MORE |
| A revolution in cancer research is stirring: the real culprit in cancer may not be its hallmark rapidly-dividing cells, but their parent stem cells. McGowan Institute researchers are in search of stem cells at the root of cancers of solid organs, such as the liver. Basic research is underway to identify a human liver-derived progenitor cell, to understand how hematopoietic cells differentiate into a hepatic lineage, and to develop a humanized mouse model of hepatitis viruses. These studies will ultimately contribute to new cell-based therapies for liver disease and cancer. |
Bruno Péault, Ph.D. who is internationally recognized for his stem cell research has joined the McGowan Institute for Regenerative Medicine. Professor Péault’s research includes identification, characterization and purification of several categories of human stem cells: hematopoietic stem cells, mesenchymal stem cells, endothelial stem cells, pancreas and respiratory epithelium stem cells. He serves as Professor in the Departments of Pediatrics and Cell Biology. His research has been focused on the characterization of human hematopoietic stem cells, a novel marker was identified, and he has been responsible for the development of new assays for human stem cells in immunodeficient mice. Besides normal, malignant and transgenic hematopoiesis, a novel xenochimeric system was developed that faithfully models the differentiation of human respiratory tissues in SCID mice. Most recently this assay system has been used to identify the first population of stem cells in the human respiratory epithelium. Thus model has also been modified to offer the first system in which human secondary lymphoid tissues (lymph nodes) can be maintained intact and functional for extended periods of time, thus offering the first ex vivo model of human immunity. However, the main breakthrough of this group in the recent years has been the discovery and characterization of the very territory where hematopoietic stem cells emerge and proliferate in the human embryo. Since he joined the Pittsburgh community, Bruno Peault has extended his interest to multipotent stem cells present in adult tissues. Whereas it was still recently assumed that such broadly committed stem cells are restricted to the embryo, converging reports in the past years have suggested that multipotent stem cells persist till adult stages. Bruno Peault has started investigating the lineage, filiation and anatomic distribution of these elusive adult multipotent stem cells, which remained so far completely obscure. In a close collaboration with Johnny Huard (see above), who had himself indirectly identified such multipotent stem cells in skeletal muscle, Peault has demonstrated that these cells find their origin in the walls of blood vessels. This would guarantee a usable reserve of stem cells to be available throughout the body. MORE |
Other Faculty with Similar Research Interests Includes: