US Panel Recommends Reopening Gene Therapy Trials

AFX Press

NEW YORK (AFX) - The US Food and Drug Administration should resume gene therapy trials for a fatal immune disorder, a scientific advisory panel has recommended, saying the potential benefits of the controversial treatments outweighed the risks, an FDA spokeswoman said Friday.

The FDA halted gene therapy studies in September after a 3-year-old boy in France suffering from a genetic defect called severe combined immunodeficiency (SCID), commonly known as "bubble boy disease," developed a leukemia-like disease that seemed to have resulted from the treatment.

"There was fairly convincing evidence presented at the meeting that showed the adverse event in the French trial was directly related to the gene therapy treatment," said FDA spokeswoman Lenore Gelb.

Gene therapy involves the replacement of a person's faulty genetic material with normal genetic material to treat a disease or abnormal medical condition.

According to FDA, SCID is a particularly attractive target for gene therapy approaches because the genetic defect causing the disease is known and can be potentially corrected by providing a normal copy of the gene.

The French toddler was only one out of 11 SCID patients treated with gene therapy who suffered a cancerous side effect, said Gelb. "The committee felt the risk was too small to not allow gene therapy for cases where there are no alternatives and the patients would die without the treatment," said Gelb.

The FDA called the emergency meeting of its Biological Response Modifiers Advisory Committee on Thursday to advise the agency on whether to resume three US gene therapy trials similar to the case in France.

"We're going to carefully consider the discussions at the meeting and decide how to best proceed on the clinical trials we suspended," said Gelb. Proponents of gene therapy argue the treatment holds promise for fighting genetic disorders and illnesses including cancer, diabetes, high blood
pressure and heart disease.

The experimental treatment has suffered serious setbacks since 1999 when a teenager died while undergoing gene therapy for a rare liver disorder at the University of Pennsylvania.

Bioethics groups have called on the federal government to declare a moratorium on gene therapy trials until further research ensuring their safety has been adequately demonstrated.

The Council for Responsible Genetics (CRG) has warned of the dangers of gene
therapy since 1987.

"FDA monitoring of gene therapy's potential risks has been woefully inadequate. The agency has yet to set up a mechanism to document long-term side effects," said CRG spokesman Peter Shorett.


Printed by the AFX Press, 11 October 2002

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