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All three patients, who had severely abnormal vision before entering thestudy, can now read several lines on an eye chart and are able to see betterin dimly lit settings. One was also able to navigate better after theinjection.
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"This breakthrough is the greatest advancement in the 37-year history ofthe Foundation Fighting Blindness and the entire history of retinaldegenerative disease research. We have achieved a critical milestone in curinga form of childhood blindness," says Gordon Gund, Co-Founder and Chairman ofthe Foundation Fighting Blindness, which is the largest non-governmentalsource of funding for this research.
"Our clinical trial results represent an important first step indeveloping therapies and treatments that will reverse blindness in people witha 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 TheChildren's Hospital of Philadelphia are ages 19-26. Though the trial's maingoal was to evaluate safety of the treatment, the research team is veryexcited 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 nineindividuals between the ages of 8 and 27. The success in the first threepatients, however, will position the researchers well to plan Phase IIclinical studies to evaluate the treatment's potential effectiveness inyounger children who were born blind from LCA. The investigators believe thetreatment has the potential to give near-normal vision to these children.
The first step toward the development of this treatment began with thediscovery of the RPE65 gene in 1993. In 2000, the first dog born blind fromLCA, a Briard named Lancelot, was successfully treated with gene therapy, andhas been seeing well since then with just a single treatment. More than 50dogs have now been treated successfully and are all seeing well. Clinicaltrials of the procedure began in October 2007 at the Foundation-fundedChildren's Hospital of Philadelphia (CHOP)-Penn Pediatric Center for RetinalDegenerations in Philadelphia.
More than 10 million people across the United States are affected byretinal degenerative diseases which include: macular degeneration, retinitispigmentosa, and Usher syndrome.
About the Foundation Fighting Blindness
The Foundation Fighting Blindness (www.FightBlindness.org) is the largestsource of non-governmental funding for retinal degenerative disease researchin the world. The urgent mission of the Foundation Fighting Blindness is todrive the research that will provide preventions, treatments and cures forpeople affected by retinitis pigmentosa, macular degeneration, Usher syndrome,and the entire spectrum of retinal degenerative diseases.
SOURCE Foundation Fighting Blindness
