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:2/10/2016)... Association, in partnership with ALS Finding a Cure, is pleased ... track TDP43 aggregation. The successful team(s) with the most developed ... --> --> ALS is ... brain and the spinal cord. Eventually, people with ALS lose ... leads to total paralysis and death within two to five ...
(Date:2/10/2016)... Feb. 10, 2016 Convergence scenarios ... of various technologies that results in diverse ... These opportunities create a cyclical system where ... drives the development of new technologies. ... by technology convergences, which are constantly fostering ...
(Date:2/10/2016)... 10, 2016 Antibacterial Drugs Market - ... - 2023 ", reveals that the global antibacterial drugs market ... to 2023 owing to patent expiries of blockbuster drugs. Even ... during the forecast period, the value is anticipated to decline ... --> Antibacterial Drugs Market - Global Industry Analysis, ...
Breaking Medicine Technology:
(Date:2/10/2016)... ... February 10, 2016 , ... As part of its ongoing ... February 2016. Each webinar features a dynamic expert and thoughtful presentation to give ... patients and facilities. Both events are free to attend, but registration is ...
(Date:2/10/2016)... (PRWEB) , ... February 10, 2016 , ... ... American Academy of Emergency Medicine , an emergency medicine professional association, ... practice management services . , The American Academy of Emergency Medicine, or AAEM, ...
(Date:2/10/2016)... ... February 10, 2016 , ... The Wickman Agency in ... assist the local community. Pledging to select a new beneficiary every 60 days, ... area. Their goal is to bring community awareness to important local causes by ...
(Date:2/10/2016)... , ... February 10, 2016 , ... 10 Best ... There were three leading bottled water brand owners that topped the list as a ... connectivity and optimize conversion. The premier brand was Tibet 5100, a top notch water ...
(Date:2/10/2016)... ... February 10, 2016 , ... Dr. Jessica Barron, of ... is now accepting new dental patients and families in the North Metro Denver area. ... services from cleanings to cosmetic dentistry, and all in the most relaxing environment. , ...
Breaking Medicine News(10 mins):