Results of Clinical Trial Supported by the Foundation Fighting Blindness

are Published in the New England Journal of Medicine

    OWINGS MILLS, Md., April 27 /PRNewswire/ -- Scientists employing a gene
 therapy have provided partial vision to patients who were nearly blind from
 a condition known as Leber congenital amaurosis (LCA) -- a severe form of
 retinitis pigmentosa. Initial results from the clinical trial, which was
 funded in part by the Foundation Fighting Blindness, were published today
 in the New England Journal of Medicine.
 
     All three patients, who had severely abnormal vision before entering
 the study, can now read several lines on an eye chart and are able to see
 better in dimly lit settings. One was also able to navigate better after
 the injection.
 
     "This breakthrough is the greatest advancement in the 37-year history
 of the Foundation Fighting Blindness and the entire history of retinal
 degenerative disease research. We have achieved a critical milestone in
 curing a form of childhood blindness," says Gordon Gund, Co-Founder and
 Chairman of the Foundation Fighting Blindness, which is the largest
 non-governmental source of funding for this research.
 
     "Our clinical trial results represent an important first step in
 developing therapies and treatments that will reverse blindness in people
 with a variety of retinal degenerative diseases," says Jean Bennett, M.D.,
 Ph.D., who is the study's lead researcher at The Children's Hospital of
 Philadelphia.
 
     "The three participants in the Foundation-supported study at The
 Children's Hospital of Philadelphia are ages 19-26. Though the trial's main
 goal was to evaluate safety of the treatment, the research team is very
 excited about the participants' improvements in vision," says Stephen Rose,
 Ph.D., Chief Research Officer, Foundation Fighting Blindness.
 
     This Phase I study will continue through its planned enrollment of nine
 individuals between the ages of 8 and 27. The success in the first three
 patients, however, will position the researchers well to plan Phase II
 clinical studies to evaluate the treatment's potential effectiveness in
 younger children who were born blind from LCA. The investigators believe
 the treatment has the potential to give near-normal vision to these
 children.
 
     The first step toward the development of this treatment began with the
 discovery of the RPE65 gene in 1993. In 2000, the first dog born blind from
 LCA, a Briard named Lancelot, was successfully treated with gene therapy,
 and has been seeing well since then with just a single treatment. More than
 50 dogs have now been treated successfully and are all seeing well.
 Clinical trials of the procedure began in October 2007 at the
 Foundation-funded Children's Hospital of Philadelphia (CHOP)-Penn Pediatric
 Center for Retinal Degenerations in Philadelphia.
 
     More than 10 million people across the United States are affected by
 retinal degenerative diseases which include: macular degeneration,
 retinitis pigmentosa, and Usher syndrome.
 
     About the Foundation Fighting Blindness
 
     The Foundation Fighting Blindness (www.FightBlindness.org) is the
 largest source of non-governmental funding for retinal degenerative disease
 research in the world. The urgent mission of the Foundation Fighting
 Blindness is to drive the research that will provide preventions,
 treatments and cures for people affected by retinitis pigmentosa, macular
 degeneration, Usher syndrome, and the entire spectrum of retinal
 degenerative diseases.
 
 
 

SOURCE Foundation Fighting Blindness