Panel Urges Follow-Up to Gene Therapy

Participants in some gene therapy trials should be closely followed for up to 20 years after a study’s completion to guard against future complications, a U.S. advisory group said Friday.

Currently only organ transplant recipients are monitored that long, but Food and Drug Administration advisors said so little was known about the long-term consequences of gene therapy that it was better to err on the side of caution.

The 1999 death of Jesse Gelsinger, 18, during treatment for an inherited liver disorder sent shock waves through the genetic research world and led the FDA to tighten its oversight of the area.

The FDA asked the advisory panel to review whether new reporting and monitoring requirements set by the agency were adequate to protect patients.

In a second and final day of discussions, the panel also said that giving the public more information about ongoing trials involving gene therapy was a good idea, although a balance was needed between the public’s need for reassurance and industry’s desire to protect proprietary information.

On Thursday, the panel supported independent monitors for gene therapy studies. The FDA usually follows the advice of its advisory panels.

Panel chairman Daniel Salomon, a professor at the La Jolla-based Scripps Research Institute, acknowledged the public’s grave concerns about safety of gene therapy or any genetic engineering and said the panel should act to reassure Americans by insisting on long-term follow up.

The main concerns with gene therapy are:

* The genetic material could make a patient sick by altering their DNA in a harmful way.

* Viruses used to deliver gene therapy, called vectors, have the potential to replicate or become active years or decades later, which could lead to a chronic infection, cancer, autoimmune disease, or a neurological or blood disorder.