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Researchers from the Universities of Pennsylvania and Florida have reported that vision improvement for three individuals treated with gene therapy in a Phase I clinical trial has persisted for one year. The trial participants, all in their early 20s and with severe vision loss from Leber congenital amaurosis (LCA), continue to experience significant increases in night and day vision. Most importantly, the safety of the treatment, which is the primary goal of this and all Phase I clinical trials, is now extended to one year. “This is a critical milestone for gene therapy,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “It is essential that we establish long-term safety and effectiveness before moving this innovative treatment forward through clinical trials. We congratulate the Pennsylvania and Florida team for doing just that.” Study investigators noted that one clinical trial participant was able to read an illuminated clock on a car dashboard for the first time in her life. Artur V. Cideciyan, Ph.D., co-investigator of the trial from the University of Pennsylvania, determined that the young woman had shifted her focus to use the treated portion of her retina to see the clock. He added that it took the patient 12 months to be able to see the clock. Samuel G. Jacobson M.D., Ph.D., principal investigator of the trial, commented that such visual gains might be accelerated in the future through visual training. Two other Phase I clinical trials of similar gene therapies for LCA (caused by variations in the gene RPE65) are also continuing at the Children’s Hospital of Philadelphia and Moorfields Eye Hospital in the U.K. Both of these studies are funded by the Foundation Fighting Blindness. Investigators in all three clinical trials are extending their enrollment to younger and older patients and altering parameters of dose and location of injection. The goal is to refine the treatment strategy to make it more valuable to patients with this form of LCA. Collectively, approximately 20 people have been treated thus far. One-year results for the Universities of Pennsylvania and Florida study were reported in the September 2009 Human Gene Therapy (online edition) and the August 13, 2009 New England Journal of Medicine (online edition). |
