Biotech Tracker News - Gene Therapy Hits the SCIDs Again
January 17, 2003 The FDA has halted approximately 30 gene therapy trials that are similar to a French trial in which a second child has developed leukemia. As we reported with the first such case in our October 7, 2002 report, the focus remains on evaluating the specific therapy’s risk/benefit profile.  A second case in a small patient cohort does not bode well for this particular treatment for x-linked severe combined immunodeficiency (X-SCID). Both study subjects who developed leukemia were children who lacked residual post-procedural evidence of X-SCID before leukemic symptoms arose. They were two of 10 X-SCID gene therapy study subjects in France who had significant therapeutic responses to the gene therapy.  Reportedly, the children remain in stable condition. Principal investigator, Dr. Alain Fischer of Hôpital Necker Enfants Malade (Paris), has implied that the cases have a common pathological development process. Dr. Harry Malech of the National Institute of Allergies and Infectious Diseases said, “I think we shouldn’t view this as death knell to gene therapy.… We need more good science to sort out what went wrong.” The Office of Biotechnology Activities at the National Institutes of Health has called an emergency meeting for Friday to discuss the issue.  The type of gene therapy correlated with the development of leukemia involves the use of retroviral vectors that cause insertion of genetic material into the patient’s genome. It is likely that the viral vector integrated near an oncogene in such a manner as to promote uncontrolled cell development and proliferation thus the formation of tumor cells. This mimics the processes of cancer-promoting viruses. According to the American Society of Gene Therapy, the risk of cancer as an adverse effect in the current trial, “is stated explicitly in the informed consent statements that describe the risks and potential benefits to patients and their families.” Investigation is ongoing into the precise nature, extent, and cause of the adversity, and there remains no evidence of the sort of gross misconduct that had plagued earlier trials such as the one in which Jesse Gelsinger died.  Dr. Philip Noguchi, head of gene therapy issues at the FDA, said at this Friday’s meeting that the FDA will consider continuing administration of the implicated gene therapy for those most definitely at risk for impending death. As many forms of childhood leukemia are manageable and X-SCID is uniformly fatal, it remains possible that the implicated therapy has an acceptable risk/benefit profile for X-SCID patients earlier in the course of their disease. However, this conclusion requires painstaking longer-term investigation.  X-SCID is the most severe form of SCID. All are disorders of a single gene. SCID affects one in 75,000 live births, and leaves carriers with severely impaired immunity. X-SCID affects one in 100,000 live male births. SCID gained popular culture recognition in 1984 when media attention began to focus on an affected boy, David, who lived in a “bubble” designed to protect him from exposure to pathogens.
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