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| Vol. 21, No. 15 |
| August 15, 1999 |
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From the Lab to the Clinical Setting: by KRISTINA VAN ARSDEL Texas Medical Center News
Dr. Brenner hopes the International Center for Cell and Gene Therapy will do just that. A partnership between Baylor College of Medicine, Texas Children's Hospital and The Methodist Hospital, the Center will bring scientists and clinicians together under Dr. Brenner's direction to develop cell and gene therapy strategies for the treatment of disease. Cell and gene therapy could play a role in the treatment of a host of diseases, from cancer to HIV, and eventually cardiovascular disease and neurogenerative diseases like Alzheimer's and Parkinson's. "There are centers that do cell therapy with bone marrow transplantation and there are centers that do gene therapy," says Dr. Brenner, who came to the Texas Medical Center from St. Jude Children's Research Hospital in 1998. "As far as I know, this is a fairly unique combination in terms of having dedicated facilities from the most basic scientific to the most directly applied clinical." The first clinical application for cell therapy - the process of genetically modifying cells in vitro and then infusing them into a patient to optimize their function - came in the early 1970s with bone marrow transplantation. Today, says Dr. Brenner, with an increased understanding of the immune system and cell biology, the potential applications for cell therapy are vast. "Gradually, as we have learned more and more about the immune system, we have been able to extend bone marrow transplant beyond the original application in identical twins or genetically similar siblings to using unrelated donors and now donors that are quite genetically disparate," he says. "We've also been able to increase the safety of the procedures so that now a much higher proportion of patients survive. "The first application is the one that is currently predominant in the fields of cancer and infection, but I think what will happen is we will expand into regenerative therapies where you are trying to repair decayed tissue," he says. Part of Dr. Brenner's own research is currently focused on ways to make bone marrow transplantation safer. "We've developed monoclonal antibodies which will help to destroy, in a very specific way, the marrow-forming cells and spare all the other tissue. That will allow us, we hope, to get safer transplantation," he says. The Center will also work to develop clinical applications for gene therapy, a treatment which involves replacing or repairing a damaged gene. Dr. Brenner notes that the second and third phases of the Human Genome Project, which will identify the functions of genes and the polymorphisms (or variations) that exist, will be a great source of information. The Human Genome Project is the international effort to identify and sequence the 80,000 genes in the human body. "Once we have that information, we can incorporate it into gene therapy either by replacing a gene or adding in a new gene that hopefully will have an improved effect," he says. "That's going to be a very slow process because we are dealing with the interaction of the gene and understanding how each one influences the output of the other. It's not something that's going to be done over a short period of time. But, I think gradually, over the next 20 to 30 years, these things will emerge." Dr. Brenner envisions cell and gene therapy becoming two additional modalities that will augment current treatments in the future. "It has been clear for awhile that you get the best treatment for disease if you combine the available therapies," he says. "I think that's going to be true for almost every disease. What cell and gene therapy will do is give you two more modalities to add in, providing options that maybe weren't previously available."
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