Spotlight on Research - Dr. Bunnell

Dr. Bruce A. Bunnell is an Assistant Professor in the Department of Pharmacology and the Center for Gene Therapy at the Tulane University Health Sciences Center. His research project is “Marrow Stromal Cell-Mediated Therapy of Krabbe’s Disease in a Nonhuman Primate Model.” Dr. Bunnell’s research program involves the development of novel genetic and cellular therapy approaches for the treatment of Krabbe Disease in the rhesus monkey model. Dr. Bunnell has received funding from the Hunter’s Hope Foundation to isolate, characterize, and develop therapeutic approaches using mesenchymal stem cells for the treatment of Krabbe Disease. It is important work like this that helps not only a specific disease, but will help to advance the field of gene therapy for human application.

The following are some highlights from a recent interview with Dr. Bunnell about this research.

What is the long-term goal for your research project?
To apply stem cells for the treatment of Krabbe Disease in the rhesus monkey model.

How do you see your research benefiting the future of children born with Krabbe and other related diseases? When fully developed, our research program will provide novel therapeutic interventions for Krabbe Disease and other metabolic diseases that affect the Central Nervous System. It is our hope that stem cell based therapeutics will ameliorate the disease in children born with Krabbe in the future.

Are there other diseases potentially benefiting from the research you are doing with funding support from Hunter's Hope? The stem cell based therapeutic interventions we are exploring may be beneficial if applied to other lysosomal storage diseases, such as Tay-Sach and Sandhoff’s disease.

What is gene therapy? Could you tell us a little bit about it in general? Gene therapy refers to the use of genes or genetic element to provide cells with a new or altered function. In most cases, gene therapy refers to the addition of a functional copy of a defective gene to correct a genetic disease (i.e. Krabbe Disease). In Krabbe Disease, both alleles of the galactocerebrosidase (GC) gene are defective (cannot produce protein) as such the delivery a functional copy of the GC gene to cells within these patients may permit us to overcome this genetic deficiency. There are two basic gene delivery approaches used in gene therapy. To perform in vitro gene therapy, cells are removed from the patient, grown in the lab, the functional gene is delivered, and the cells are then returned to the patient, often in the peripheral blood stream. For in vivo gene therapy, the vectors carrying the functional gene are administered directly to the patient either in a specific organ (brain) or through the peripheral circulation. At the present time there is no good universal vector for gene delivery. The delivery of the functional copy of the defective gene is most often accomplished using vectors (vehicles of gene delivery) derived from viruses. Viruses are the moist efficient gene delivery tools that gene therapy has to offer at the present time. Gene therapy vectors have been derived from retroviruses, adenovirus, adeno-associated virus and herpes virus. Each of these virus vectors is presently under investigation for their safety and effectiveness in human gene therapy.

How does collaboration with other scientists benefit the advance of research? Collaboration is an integral part of research. It most often brings together a group of researchers with unique skill sets and expertise to work together to address complex problems. These diverse perspectives are often highly beneficial to resolve issues in complex disorders, such as Krabbe Disease. Collaborators can often bring a fresh perspective to other scientists who are intensely focused on a research problem.

Thank you Dr. Bruce Bunnell.

Dr. Bunnell is Chairman of the Division of Gene Therapy at the Tulane National Primate Research Center. His work includes the maintenance and expansion of a colony of rhesus macaques affected with Krabbe Disease.

If you’d like to learn more about Dr. Bunnell, his extensive background and his additional research projects, check out this Tulane University website: Dr. Bunnell

We are grateful to Dr. Bunnell for his work, “Marrow Stromal Cell-Mediated Therapy of Krabbe’s Disease in a Nonhuman Primate Model.” In May 2003, Dr. Bunnell presented his latest research findings at the Hunter’s Hope annual Scientific and Family Symposium, which provided him and other researchers additional time for collaboration. The symposium is important to help further advance the pace leukodystrophy research. Opportunities like these will help save the lives of future generations. We are also grateful to all our supporters that have made it possible for Hunter’s Hope to provide funding for this important research. We thank you all!