GENE THERAPY: Two New Successes Boost Ailing Field

      Doctors have successfully used gene therapy to treat hemophilia B, the New York Times reports. Researchers reported yesterday that they have been able to "inject patients with a blood-clotting protein without adverse side effects." Scientists at Alameda, CA-based Avigen Inc., the Children's Hospital of Philadelphia and Stanford University Medical Center injected three patients with a genetically engineered virus used as a vector for the gene that "prompts cells to produce Factor IX" -- a protein that promotes clotting. Avigen's President and CEO John Monahan said, "What we have observed is that the gene has been successfully delivered and is producing biologically active Factor IX." Although the trial used low doses to test for safety, the three patients injected with the gene carrier began producing Factor IX and have continued to do so for six months. The researchers plan to move into the next phase of the trial, testing additional patients to confirm treatment efficacy.

Heart Failure Treatment
      In another, more preliminary trial, researchers reported that gene therapy also shows great promise in combating heart failure. The study, which appears in today's issue of the journal Circulation, notes that "delivering additional copies of the gene Serca2a to muscles cells" from faltering hearts caused faster and more powerful heart contractions. The study's lead physician, Roger Hajjar of the Massachusetts General Hospital Cardiovascular Research Center, stated, "This ability to modify the contraction of failing human cardiac cells represents an important step in gene therapy for heart failure."

Shot in the Arm
      The studies offer a morale boost for the downtrodden field of gene therapy, which has been hit with bad publicity after the September death of 18-year-old Jesse Gelsinger in a University of Pennsylvania experiment. The Recombinant DNA Advisory Committee (RAC) -- an NIH panel that oversees gene therapy research -- is meeting this week to evaluate the UPenn death and the safety of adenovirus, the virus used to administer genes to Gelsinger. The virus in question was used in the heart research announced today but not in the hemophilia trials (Fisher, 12/7). The Wall Street Journal reports, however, that the treatment successes will not keep gene therapy critics at bay for long. Secrecy in gene therapy clinical trials likely will receive scrutiny again, as several key researchers have declined to present trial findings at the NIH advisory committee meetings. Researchers at Berlex Laboratories and Collateral Therapeutics refused to present data about gene therapy research seeking to grow new heart vessels for patients with clogged arteries. In their trials, the firms injected patients with adenovirus. Many are questioning the decision not to present, especially in light of the recent failure of gene therapy researchers to fully disclose adverse effects in clinical trials. But the Wall Street Journal notes that a "culture clash" between university researchers and for-profit pharmaceutical and biotech firms has "started to erode" the field's openness. While Collateral researchers -- who are partnering with Schering AG subsidiary Berlex -- have submitted information to the RAC concerning adverse events, they have requested that it remain confidential. Currently, the RAC is considering rule changes that would prohibit confidentiality of adverse event data reported from gene therapy trials (Adams, 12/7).