Subject:   Research on Gene Transfer Therapy
Date:   2002-10-18

An emergency meeting by the FDA was held regarding gene transfer by the Biological Response Modifiers Advisory Commmittee on October 10, 2002. As many of you know, one of the hopes of the research community is to turn viral infection agents into healing agents by carrying corrective genes into selected organs or tissue. Most of this special meeting was dedicated to dealing with a serious adverse event that occurred in the only documented successful gene therapy, a French study involving the treatment of children with severe combined immune deficiency (SCID). One of the initial eleven patients treated has developed a leukemia-like condition, and the likely cause is the retrovirus used for the gene transfer. The French researchers and the FDA are to be commended for their prompt reporting and handling of this serious side effect. The French were under no obligation to report to the FDA, but did so in an effort to get a broader perspective from the field. It is CIRCARE's opinion that, in order to best protect and inform the public, this is precisely how serious adverse event reporting should be handled.

Please read the related articles as reported in the Washington Post and NY Times:

Resumption of Gene Therapy Urged. Rick Weiss. The Washington Post. 2002-10-11.
URL: http://www.washingtonpost.com/wp-dyn/articles/A9649-2002Oct10.html

Panel Advises Resuming Gene Studies. Sheryl Gay Stolberg. The New York Times. 2002-10-11.
URL: http://www.nytimes.com/2002/10/11/health/11FDA.html

CIRCARE recommends that adverse events be reported in plain language to prospective research subjects and that the essential unknown nature of the experiment be revealed. In particular subjects should know not only the adverse effects to date but also:

• The esentially random nature of gene insertion
• The unknown level of risk for cancer
• The unknown level of long term risk
• A sense of the complexity and fragility of gene expression which impacts and is impacted by multiple biological levels.

CIRCARE has additional concerns:

1. An unknown amount of negative data with animal studies has been collected by industry but not revealed.
2. The lack of independent testing of data in any double blind study
3. In a field where many dogmas have been overturned and the genome has been found to be surprisingly plastic, there is also the possibility, expressed by some European scientists, that changes in the somatic gene line might alter the germ line

Michael Susko (President of CIRCARE) and Paul Gelsinger (Vice President of CIRCARE) expressed some of these concerns in public testimony before the advisory group.

Michael A. Susko MS, CIRCARE President
(410) 499-4895

Paul Gelsinger, Vice President, CIRCARE

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Last Updated: 2006-03-04

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