Statement by Dr. Bennett on upcoming clinical trials

It is real! It is really going to happen. Human clinical trials on gene therapy for LCA caused by mutations in the RPE65 gene are scheduled to begin next year! Two trials are planned, one enrolling adults and one for children. What follows is a statement by Dr. Jean Bennett, a leading researcher in LCA gene therapy, which was sent directly to the Foundation for Retinal Research. If you have any question just email them to me and, if necessary, I’ll forward them to FRR.

Enjoy!

Fran

On Tuesday December 13, 2005, the Recombinant DNA Advisory Committee (the RAC) at the National Institutes of Health (NIH) unanimously approved a pediatric clinical trial for Leber congenital amaurosis (LCA). This approval was a landmark event because it endorsed the idea that the risk/benefit ratio of a clinical trial in a pediatric population with this disease is acceptable. Traditionally, investigators have avoided studies in pediatric subjects until safety had been established in studies of adults.
The Principal Investigator of this trial, Dr. Albert Maguire (Scheie Eye Institute, University of Pennsylvania) explained to the RAC that the first human studies in this trial, which will involve 8-18 year old subjects with mutations in the RPE65 gene, will emphasize safety of the treatment but that there is a potential for benefit with respect to visual function. He explained that, due to characteristics of the disease and of the biology of visual development, there is a higher likelihood of restoration of vision in a pediatric population than in an adult population. If the treatment is shown to be safe in these phase I studies and additional regulatory approvals are given, younger subjects could be enrolled. At the RAC meeting, Dr. Jean Bennett, scientific director of the study and professor at the F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, showed data demonstrating rescue of vision in dogs and mice with this form of the disease with minimal evidence of toxicity. The investigators hope that the studies approved by the RAC will ultimately lead to a similar effect in children. This study is sponsored by the Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia. The team is completing additional safety studies relating to this clinical trial and seeking additional funds to support the studies, and anticipates that the first subjects may be enrolled in late 2006.
This pediatric clinical trial will be complemented by results from an adult clinical trial for the same disease (PI, Dr. Samuel Jacobson, University of Pennsylvania). Updates on the timelines of these two trials will be available from the Foundation for Retinal Research.

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Jean Bennett, M.D., Ph.D.
Ophthalmology; Cell and Developmental Biology
University of Pennsylvania
Philadelphia, PA 19104