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FDA Advisory Committee Discusses Steps for Potentially Continuing Certain Gene Therapy Trials That Were Recently Placed on Hold

FDA's Biological Response Modifiers Advisory Committee (BRMAC) met today to discuss possible measures that could potentially allow a number of gene therapy trials that involve the use of retroviruses to insert new genes in blood stem cells for treatment of life threatening diseases to proceed with appropriate safeguards. This meeting is one of the steps in the continuing process outlined by the agency last month that is intended to enhance the safety of participants in these trials, while supporting the development of new therapies.

These studies were placed on hold on Jan 14, 2003 following the report that a second child in a clinical trial in France had developed leukemia, potentially as a result of otherwise successful treatment with gene therapy for a condition which is frequently otherwise fatal, X-linked severe combined immunodeficiency syndrome (X-SCID, also known as "bubble baby syndrome"). While FDA currently believes that the two leukemia cases reported from that clinical trial were likely related to the gene therapy, a continuing review of adverse events from all U.S. studies involving similar retroviral vectors has so far found no evidence of leukemia believed to be due to the gene therapy.

Moreover, the agency continues to consider the potential risks of any experimental therapy within the context of its potential benefits. In the case of trials for children with X-linked severe combined immunodeficiency disease and many other gene therapy trials, the disease being treated can be devastating and fatal and for some patients, no other options exist.

Today's meeting dealt with the risks and potential benefits of X-SCID trials and other trials using retroviral vectors, and focused on what new safety measures might be taken to minimize the risk of continued and future trials utilizing related technologies.

FDA will review the discussions and advice of the BRMAC and work closely with sponsors to help them continue to develop innovative new treatments while working to do everything possible to better understand and prevent any adverse events.

While this process continues, FDA will actively consider specific requests for retroviral gene therapy trials involving fatal or life-threatening disorders for which there currently are no viable alternative treatments.

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