GENE THERAPY: NIH Will Order Researchers to Open Files
Hoping to uncover "problematic trends that might have been overlooked, missed or ignored," the NIH yesterday confirmed it will soon order gene therapy researchers to reopen files from old experiments and report "all past instances of side effects" during clinical trials, the Los Angeles Times reports. NIH officials will examine records from more than 400 trials involving over 4,000 patients during the past decade. NIH Office of Science Policy Director Lana Skirboll said, "This is a major effort and it won't be an easy task. ... We want to make sure our message is clear: that the past degree of noncompliance was unacceptable and ... we won't tolerate it." The agency also plans to visit some NIH-funded institutions to "ensure that researchers understand what is required and to identify any problems with their compliance" (Cimons, 2/24).
Waxman Writes NIH
Meanwhile, in a "strongly worded" letter to NIH Acting Director Ruth Kirschstein, Rep. Henry Waxman (D-Calif.) yesterday blasted the agency for providing "inadequate" oversight for gene therapy trials. Citing a 1996 death of a cystic fibrosis patient who participated in a 1993 UPenn trial, as well as a Houston cancer experiment in which 38 out of 48 participants died, Waxman wrote, "The most recent reports raise new questions about the conduct of gene therapy trials, the lapses of compliance and the adequacy of federal oversight. ... At a minimum, NIH and FDA must aggressively enforce the law." According to the letter, NIH officials did not follow up reports of the deaths of 38 patients during a trial at an M.D. Anderson Cancer Center in Houston, Texas. "It would seem obvious that there would be vigorous and immediate oversight of any protocol that results in the deaths of 75% of the participating patients," he said. Waxman also accused officials of failing to properly investigate the 1996 UPenn death and another incident at Georgetown University last year, where six patients were given the wrong medicine (Vedantam, Philadelphia Inquirer, 2/24).
On the Defense
The NIH defended its actions, contending it has reviewed hundreds of adverse-event reports from the trials "in great detail" and has "found nothing to indicate these deaths were related to gene therapy." The institutions associated with the cited experiments also took issue with Waxman's charges. A spokesperson for Introgen Therapeutics, which conducted the Houston study, said the patients died due to their "underlying disease, not the gene therapy itself." James Merritt, Introgen's vice president for clinical affairs, noted, "They had end-stage cancer" (Adams, Wall Street Journal, 2/24). UPenn spokesperson Ken Wildes called Waxman's letter "outrageous." Pointing out that the cystic fibrosis patient died over two years after undergoing gene therapy and shortly after receiving a lung transplant, he said, "This is craziness ... There is nothing whatsoever that indicates this has anything to do with gene transfer. ... [T]he death was due to complications from lung transplant." Still, Waxman said he will push to have the Recombinant DNA Advisory Committee (RAC), which oversees gene therapy trials, moved from NIH supervision to direct administration by the HHS secretary. "I believe moving the RAC to the Office of the Secretary may be the best way of ensuring effective public oversight of this important research," he wrote (Philadelphia Inquirer, 2/24).