Navigation Links
Gene Therapy Provides Vision to People who Were Nearly Blind
Date:4/27/2008

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 (http://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
Copyright©2008 PR Newswire.
All rights reserved

Related medicine technology :

1. Dr. Calvin Grant Presents Results from Xibrom(TM) Combination Therapy with Lucentis(R) at the 2008 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO)
2. FTY720, a novel oral therapy in development for MS, shows sustained benefits for the majority of patients after three years of treatment
3. In Blood Vessel Stents, Innovative Materials Allow Better Control, Delivery of Gene Therapy
4. Karmanos Researchers Find Non-Toxic Agents Added to Conventional Chemotherapy Decrease Survival of Colon Cancer Cells
5. ALS Therapy Development Institute Signs Collaboration Agreement with the Allen Institute for Brain Science
6. MAP Pharmaceuticals Reports Positive Results From Phase 2a Clinical Trial of Combination Therapy in Adult Asthmatics
7. CV Therapeutics Anti-Ischemic Therapy Ranexa(R) Significantly Reduces Cardiac Chest Pain Symptoms and Recurrent Ischemia in MERLIN-TIMI 36 Patients With Angina
8. Final Data From the Bioheart Seismic Trial Suggest Safety, Efficacy of Autologous Stem-Cell Therapy for Treating Congestive Heart Failure
9. Final Data From the SEISMIC Trial Suggest Safety, Efficacy of Autologous Stem- Cell Therapy for Treating Congestive Heart Failure
10. NeoVista Presents One Year Data on Novel Wet AMD Therapy at Macula Society
11. Early Research Suggests Umbilical Cord Blood Cell Therapy May Reduce Progression of Alzheimers Disease
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:11/30/2016)... Nov 30, 2016 Research and Markets has ... Monitoring Devices 2017 - MedCore" report to their offering. ... , , ... the skull. In healthy individuals, it is circulated though the brain and ... where the amount of CSF surrounding the brain changes significantly. ...
(Date:11/30/2016)... Research and Markets has announced the addition of the ... their offering. ... Detachable coil embolization is a minimally invasive method ... embolization treatment of cerebral aneurysms is less invasive and requires less recovery ... wall of an artery in the brain. This area bulges in the ...
(Date:11/30/2016)... Calif. , Nov. 30, 2016 Varian ... it was named America,s Most JUST Company in the ... and Forbes magazine,s inaugural "JUST 100 List." ... of the largest surveys ever conducted on attitudes towards ... months. This inaugural list ranks U.S companies against their ...
Breaking Medicine Technology:
(Date:12/2/2016)... (PRWEB) , ... December 02, 2016 , ... ... proudly announces the launch of its 60-day free trial program for all of ... shipping make the offer a truly hassle free experience. , FlexiSpot’s unique desktop ...
(Date:12/2/2016)... ... 02, 2016 , ... With the number of pain management programs available for ... find the one that works for them. When an inventor from Suisun City, Calif., ... and decided to share it with others. , He developed a prototype for PRO ...
(Date:12/2/2016)... ... December 02, 2016 , ... Mediaplanet is proud to ... which covers the innovative treatments, therapeutic technologies, and revolutionized nutrition that are helping ... prolonging life 6 years in the last 3 decades,” says Dr. Valentine Fuster, ...
(Date:12/2/2016)... ... , ... Sourced from the Isbre Springs beneath the 5,000 year old Hardanger ... of just 6 ppm TDS (Total Dissolved Solids) in addition to its excellent taste ... several ShopRite and FoodTown stores in NJ and received rave comments from consumers. , ...
(Date:12/2/2016)... Francisco, CA (PRWEB) , ... December 02, 2016 ... ... digitally-enabled care journeys, announced today that it has raised $6.0 million in an ... inspired by Clarify Health’s conviction that patients and their caregivers can receive far ...
Breaking Medicine News(10 mins):