Put Light on Gene Therapy
Gene therapy, a procedure in which defective copies of a gene are replaced with new working copies, is the most promising frontier in medicine, offering hope of a cure for diseases like cancer and AIDS.
The death of 18-year-old Jesse Gelsinger following gene therapy last year, however, serves as a reminder that the therapy’s promise is still far from assured. Despite 300 clinical trials on about 6,000 patients since 1990, gene therapy has so far failed to cure a single one.
Earlier this week, Congress held a hearing to explore whether researchers are fully informing patients like Gelsinger of the hard facts. Testimony from medical ethicists and genetic researchers made clear that many researchers are falling short in their duty to explain the technique’s speculative nature and medical risks. At the hearing, Jesse’s father poignantly described an ordeal that medical ethicists say has become all too common. Gelsinger said he was not told that the therapy, wherein a common cold virus bearing normal genes was injected into his son’s liver, could lead to hepatitis, liver damage and even death. Nor, Gelsinger said, was he informed that the therapy stood no chance of curing his son’s condition, a rare genetically related liver disorder that is not fatal.
At the root of the problem are rules putting gene therapy oversight solely in the hands of the Food and Drug Administration, which keeps all gene therapy rules and records confidential. The secrecy deprives the public, scientists and gene experts outside the FDA of information they need to assess the therapy’s adverse effects.
Competitive pressures have left some researchers and biotechnology industry executives reluctant to disclose evidence of patient harm to government regulators, especially regulators outside the FDA who are legally obliged to make the evidence public. But for a field as new and uncertain and potentially perilous as gene therapy, there is simply no call for such secrecy.
That’s why the Clinton administration should require gene researchers to promptly and fully report serious effects, especially patient deaths, not only to the FDA but to the agency that houses the government’s top gene therapy experts--the National Institutes of Health. At a recent meeting, scientists of the NIH’s Recombinant DNA Advisory Committee said they were aware of only 5% or 6% of 691 adverse effects that gene therapy researchers had confidentially reported to the FDA in the past decade.
Meanwhile, Wall Street investors are lavishing money on the gene researchers. Billions of dollars now flow each year to gene researchers who appear ready to leapfrog past basic research and develop profitable, blockbuster therapies. Investment pressure like this is encouraging some researchers to hype their gene therapy’s benefits and to downplay risks that could lead patients to drop out of their clinical trials.
By compelling broader public disclosure of gene therapy risks, the Clinton administration could help ensure that patients are put ahead of potential profits.
