FDA panel recommends 2 US gene therapy trials proceed on limited basis.A US government health panel has recommended that 2 US gene therapy trials involving children with X-linked severe combined immunodeficiency Severe Combined Immunodeficiency Definition Severe combined immunodeficiency (SCID) is the most serious human immunodeficiency disorder(s). It is a group of congenital disorders in which both the humoral part of the patient's immune system and the cells (X-SCID Noun 1. X-SCID - SCID in male children resulting from mutation of a gene that codes for a protein on the surface of T cells that allows them to develop a growth factor receptor X-linked SCID ) be allowed to resume but only on a limited basis. The Food and Drug Administration (FDA's) Cellular, Tissue and Gene Therapies Advisory Committee made the recommendation following a March 4 hearing called in response to a recent report a third child with X-SCID being treated with gene therapy had developed leukemia in a similar French study. The committee said the 2 experiments - at the National Institutes of Health and the University of Southern California The U.S. News & World Report ranked USC 27th among all universities in the United States in its 2008 ranking of "America's Best Colleges", also designating it as one of the "most selective universities" for admitting 8,634 of the almost 34,000 who applied for freshman admission - should be allowed to proceed only if all other treatments for the patients have been exhausted by using other treatments, including bone marrow transplantation Bone Marrow Transplantation Definition The bone marrow—the sponge-like tissue found in the center of certain bones—contains stem cells that are the precursors of white blood cells, red blood cells, and platelets. . Until this is assured, the advisory panel said the trials, which were halted when the French results were announced on January 24, should remain suspended. Dr. Mahendra Rao, chairman of the advisory committee, explained the committee felt extra caution must be taken. "The additional data hasn't suggested that there's a heightened risk, but we have to be careful," Rao said, the Baltimore Sun reported. "What's happening here today-the big picture-is that it shows the difficulty in developing any new class of therapy," said Dr. Daniel Salomon, a committee member and a professor in the Scripps Research Institute. "There was a period of time that there was a tendency to say gene therapy had been safe. What's clear now is that (problems can develop in) some gene therapy for some diseases." Dr. Theodore Friedman, director of the Program in Human Gene Therapy at the University of California, San Diego UCSD is consistently ranked among the top ten public universities for undergraduate education in the United States by U.S. News & World Report.[3] It is a Public Ivy. [1] For graduate studies, most of UCSD's Ph.D. , provided a different perspective. "These are normal problems with a difficult new therapy. The field has obviously been hampered and complicated with some missteps, reversals and, in fact, disasters. But that also comes on the background of hype and exaggeration and failure to deliver very quickly on clinical promises," Friedman said, HealthDay News reported. "At least a handful of participants (in the French trials) with SCID SCID severe combined immunodeficiency (disease); see under immunodeficiency. SCID abbr. severe combined immunodeficiency SCID severe combined immunodeficiency disease. are leading normal lives. These are kids who have an alternative fate and that fate is dying of infection, being isolated and not leading any kind of normal childhood life." Friedman noted many now successful therapies started out with mixed results including chemotherapy, organ transplantation The transfer of organs such as the kidneys, heart, or liver from one body to another. The transplantation of human organs has become a common medical procedure. Typical organs transplanted are the kidneys, heart, liver, pancreas, cornea, skin, bones, and lungs. , and bone marrow transplants bone marrow transplant: see bone marrow. . |
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