February 2, 2000

Mr. Chairman: I would like to begin by expressing my sympathy to Mr. Gelsinger and his family for the loss of his son. I appreciate his willingness to share his story with us.

I would also like to thank you, Mr. Chairman, for convening this hearing to review the troubling issue of patient safety in gene therapy research.

Over the past decade the science of gene therapy has developed at an astonishing pace. In laboratories across the country researchers are working hard to turn science fiction into reality– attempting to engage a patient?s own genes in the battle against diseases like cancer, AIDS, cystic fibrosis and rheumatoid arthritis. While we have yet to see the potential benefits of gene therapy realized, the promise that this research holds has ignited the hope of millions of individuals suffering from deadly and debilitating illnesses.

That is why it is so unfortunate that recent revelations about critical safety information from gene therapy trials being withheld from federal regulators and from patients is threatening to shatter the public?s confidence in gene therapy research.

Certainly all experimental treatments, by their very nature, contain some risk, and gene therapy is no exception. Yet, despite the risks, each year thousands of patients agree to participate in clinical trials, either in hopes of improving their own health or the health of future generations. In doing so, they place their trust in the researchers and in their assurances, backed by the federal government, that patient safety is paramount.

To maintain that trust, the actions of the researchers and the vigilance of federal regulators must be above reproach. That is why even the appearance of impropriety – of researchers putting business ahead of science -- is so troubling.

We should all be outraged when one of the most basic tenets of ethical research – informed consent – is violated. And we should all be alarmed by attempts to shroud information about adverse reactions in secrecy.

I deeply regret that it took a tragedy for us to focus our attention on this issue, but I am pleased that the Chairman has so quickly responded to calls for a thorough review of recent events and of the adequacy of existing mechanisms for ensuring patient safety.

I would like to thank our witnesses in advance for their contribution to our understanding of the issue of patient safety in gene therapy and I look forward to their testimony.