Gene Therapy Provides Vision to People who Were Nearly Blind

Sun Apr 27, 2008 5:00pm EDT

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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

Allie Laban-Baker, Office, +1-410-568-0126, Cell, +1-443-895-0157,
alaban-baker@FightBlindness.org; David Harrison, Office, +1-410-568-0124,
Cell, +1-410-804-1728, dharrison@FightBlindness.org, both of Foundation
Fighting Blindness
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