Drs. Albert Maguire and Jean Bennett lead the research team conducting the study.
I am very pleased to tell you that a Foundation-funded team at The Children’s Hospital of Philadelphia (CHOP) released positive results today regarding the treatment of the second eyes of three adult patients in an ongoing gene therapy trial for Leber congenital amaurosis (LCA). The results: After six months, it proved safe and improved light sensitivity, visual acuity and visual field.
So, what does this mean, exactly? Well, it’s the first time that patients in any retinal gene therapy clinical trials have received treatment in both eyes. And thanks to these successful results, the other nine patients in the CHOP study can now be invited to have their second eyes treated – which, I suspect, they’ve been looking forward to very much. This milestone, achieved by Drs. Jean Bennett and Albert Maguire (wife and husband, in case you didn’t know), is a big boost for the whole retinal gene therapy field, because it establishes a precedent for treating both eyes in human studies.
In fact, one other CHOP participant who has already received some second-eye treatment is Corey Haas, a boy whose first-eye success the Foundation reported on a few years ago. “Corey has had his second eye treated, and is doing well,” Dr. Bennett reports. “We are waiting to measure the results.”
All of this also bodes well for the eventual development of a treatment that can be made available to the public. And, by the way, there are four other LCA clinical trials out there. Collectively, more than 40 people have been successfully treated. In fact, researchers from the Universities of Pennsylvania and Florida recently published very nice three-year results for their clinical study.
Steve is highly respected for his expertise and tireless commitment to finding treatments and cures for vision-robbing retinal diseases. 
As the Foundation's senior science writer, Ben writes science and research articles for the Foundation’s website, newsletters and Eye on the Cure blog. 
As the Foundation's senior writer, Rich writes and edits content for all of the Foundation’s print and online publications, including its blog, Eye on the Cure. 
Bravo! This news will surely bring many tears of joy. Congratulations to everyone involved. I believe this kind of treatment has the most promise IMO.
Mr. Rose, do you have any information about how this can relate to forms of retinitis pigmentosa? Does this mean one day this type of therapy may be possible for RP patients? Being almost 30 years old now, diagnosed at the age of 12, it would be awesome if there was any positive gene therapy information out there for RP. Restoring vision type therapies. Again, any information is greatly appreciated. Love the blog by the way. It seems it’s going to be the best way for us impacted by a disease to get solid information. Keep it up!
Thank you for providing funding, advocating and leading the way to finding cures for retinal diseases. The current research is very exciting and as a mom of a child born blind with LCA, the news is very encouraging.
My 8 year old son has a defective GUCY2D gene. To my knowledge current studies are focused on the RPE65 gene. Are additional genes being studied and if not, is there a projection as to when they might?
Sincerely,
Suzanne D. Carver
I am sure I read there is already FFB funded research is going on for GUCY2D, it was one of the LCA genes that they are trying to get clinical trial for, just check the past articles on the FFB page.
Suzanne,
I also have an 8 year old son with GUCY2D, I know this message is from last year but please e-mail me at kellrock@verizon.net, I have information for you.
Suzanne, I’d love you to join our group of GUCY2D families – http://www.lca-gucy2d.com/
The GUCY2D is being studied currently at Penn
http://www.lca-gucy2d.com/what-is-lca-gucy2d/
Kate
Thank you for sharing this fantastic news. We can’t wait to hear more of the details.
Granddaughter of almost 5 months is being seen for vision problems. As yet, there is no confirmed reason for the blindness. Optic nerves are said to be pink and healthy. Now the consideration is in the retina area. Where do I look for the experts??
Are they going to check for LCA mutations or other retinal disease mutations if the optic nerves are okay
Diagnosing eye diseases in young children can be challenging. Obviously, kids have a harder time communicating their visual experiences. It is important to find a pediatric retinal specialist or ophthalmologist. Sometimes, it makes sense to consult with a neurologist, as well. Each case is different. Some research centers may have the expertise and resources to diagnose rarer conditions such as LCA or RP.
I suggest you contact the Foundation at info@FightBlindness.org and we can try to help locate a doctor or facility in your area.
Hi. My 5 years old son has LCA. I would love to hear
More about this research.
Could you send me more links?
Thanks.
Oran
Hi Oran, is there information in particular you are looking for?
Please can you let us know if the gene therapy can cure Stargardt macular dystrophy eye disease also.
Thank you
Hi Richard, a clinical trial is currently underway to answer that question. Please see this article on our website, http://goo.gl/Rwclp. It includes the latest information on that trial and the research behind it.
I hope that Dr. Stephen Rose will shed some light on the current Clinical Trials of Stargen at The University of Oregon.
Fantastic news!!! – I believe its only a matter of time before we unlock the potential of gene therapy for many congenital conditions …hopefully for patients with CXLRS also
Well done – keep the faith people!
Thanks for the support. Glad that you found the post useful!
Will Dr. Rose write about the Stargen Clinical Trials at Oregon University ?
Thanks for your comment! Steve may discuss Stargen down the road, but he has not written a post on it yet. The latest news on this trial can be found on our website here: (http://www.blindness.org/usher-syndrome/2925-fda-approves-start-of-usher-syndrome-gene-therapy-clinical-trial)
Anytime you see flashes of light you need to go see your eye doctor because it could be a retinal detachment. Also if you loose vision or it looks like a curtain is falling those are other symptoms of a retinal detachment. Please see your doctor right away.
Dear Sir
I am from Saudi Arabia had sent earlier medical reports you
I have two children girl the age of 4 years, and I’m two years old
To have the disease in the cells of the retina of the cones
Is there a cure?
thank you.
i hope this will work for my 12 year old daughter.
wish you all the best &happy holydays