FDA panel recommends curtailing selected gene therapy trials.The US Food and Drug Administration (FDA FDA abbr. Food and Drug Administration FDA, n.pr See Food and Drug Administration. FDA, n.pr the abbreviation for the Food and Drug Administration. ) Biological Response Advisory Committee (BRMAC) has recommended that gene therapy trials involving the use of retroviruses to insert new genes in blood stem cells stem cells, unspecialized human or animal cells that can produce mature specialized body cells and at the same time replicate themselves. Embryonic stem cells are derived from a blastocyst (the blastula typical of placental mammals; see embryo), which is very young be severely curtailed. The FDA had asked the committee for direction following the report in mid-January that a second child in a clinical trial in France had developed leukemia, allegedly the result of otherwise successful treatment with gene therapy for 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 syndrome (x-SCID). (Transplant News, January 31, 2003) The committee recommended that the gene therapy technique be allowed if the therapy represents the only hope to stay alive for children with x-SCID, also known as "bubble baby syndrome." The FDA had asked the BRMAC to explore the risks and potential benefits of x-SCID trials and other trials using retroviral vectors, and to focus on what new safety measures safety measures, n.pl actions (e.g., use of glasses, face masks) taken to protect patients and office personnel from such known hazards as particles and aerosols from high-speed rotary instruments, mercury vapor, radiation exposure, anesthetic and might be employed to minimize the risk of continued and future trials utilizing related technologies. After considerable debate, however, the committee decided the experimental gene therapy technique should be severely restricted. "We need about two more years before I can say how safe this therapy is," said committee chairman Daniel Salomon, the Washington Post reported. Salomon, of the Scripps Research Institute in La Jolla, CA, expressed concern about the apparent lag time between treatment and the onset of cancer. "Until then, regardless of its incredible benefits, I'm not comfortable supporting it anymore unless there is no alternative." The committee advised the FDA not to allow patients into either of the 2 pending studies in the US similar to the French study unless patients had no access to, or had failed to respond to, alternative treatments such as potentially helpful bone marrow transplants bone marrow transplant: see bone marrow. . The BRMAC did not call for restricting all gene therapy trials, however. It recommended less stringent criteria to allow resumption of the single gene therapy study aimed at a related immune system immune system Cells, cell products, organs, and structures of the body involved in the detection and destruction of foreign invaders, such as bacteria, viruses, and cancer cells. Immunity is based on the system's ability to launch a defense against such invaders. disorder called ADA-SCID Noun 1. ADA-SCID - SCID resulting from mutation of a gene that codes for adenosine deaminase SCID, severe combined immunodeficiency, severe combined immunodeficiency disease - a congenital disease affecting T cells that can result from a mutation in any one of , and also that the agency allow 27 distantly related gene therapy experiments to proceed under strict informed consent guidelines and closely followed for signs of leukemia. |
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