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The Foundation’s Center Grants Foster Essential Retinal Research Collaborations

Question: How many researchers does it take to develop a retinal-disease treatment? (No, this isn’t a politically incorrect joke.) The answer is near the end of this article, but no looking ahead!

Clearly, no scientist can develop a sight-saving treatment alone. While an individual investigator tucked away in his or her lab might come up with a breakthrough concept for saving vision, that person needs lots of help from a diverse group of scientific collaborators to move a therapy into human studies and out to the people who need it. In many cases, the researcher with the great idea will need dozens of collaborators to assist with the following essential activities:

  1. Develop a delivery system for the treatment (e.g., eye drops or a pill)
  2. Develop and/or identify animal models of relevant retinal disease(s)
  3. Conduct imaging, vision, and tissue studies in animal models
  4. Find and characterize patients with relevant retinal disease(s)
  5. Produce the therapy so that it’s safe for human studies
  6. Develop a plan to obtain FDA authorization to launch a clinical trial
  7. Conduct the clinical trial (including imaging and vision studies)

 

This is why fostering collaboration among researchers has been a major strategy for the Foundation since its inception in 1971. In the coming year, the Foundation will invest more than $5 million in 14 collaborative research projects, which we call Center Grants.

Center Grants typically last five years. Three were renewed in July: the Berman-Gund Laboratory at Harvard Medical School; the Children’s Hospital of Philadelphia-University of Pennsylvania (CHOP-PENN) Center for Pediatric Retinal Degenerations; and the University of Pennsylvania Large Animal Model Translation and Research Center.

For an example of the collaborative nature of these groups, consider the CHOP-PENN Center led by gene therapy pioneer Jean Bennett, M.D., Ph.D. Her ultimate goal is to develop new gene therapies for Stargardt disease and two forms of Leber congenital amaurosis: LCA5 (lebercillin mutations) and LCA10 (CEP290 mutations).

In support of that goal, her team of more than 30 investigators and technicians is conducting a wide variety of synergistic research efforts, including: natural history studies of people with retinal degenerations; development of human models of retinal disease, using induced pluripotent stem cells; and imaging studies for tracking disease progression and treatment effects. In addition, she is eliciting the help of 17 researchers outside of her institution, virtually all of whom are also funded by the Foundation.

So, if you do the math, Jean is getting help from nearly 50 scientific personnel to advance three new gene therapies into clinical trials.

I am sure if you asked Jean how many people helped in developing her first gene therapy — the landmark gene therapy for Leber congenital amaurosis (RPE65 mutations) — she would say hundreds. Just don’t ask her to name them all.

Visit the Foundation’s online annual report to learn about all of our Center Grants and individual investigator awards.

Visit our recent blog post from VISIONS 2013 to learn the latest on six gene therapy clinical trials currently underway.

Pictured above:
Top left, Researchers from the lab of Eric Pierce, M.D., Ph.D., at Massachusetts Eye and Ear Infirmary, Harvard Medical School.
Bottom Right, Researchers from the lab of Gus Aguirre, V.M.D.,Ph.D., and William Beltran, V.M.D., Ph.D., at the University of Pennsylvania.

 

 

 


11 Responses to 'The Foundation’s Center Grants Foster Essential Retinal Research Collaborations'

  1. Kevin Lawless says:

    I am very interested in learning more about clinical trials. As an individual with Stargardts Macular Degeneration it is difficult to find information about what trials are ongoing , where the are and how to qualify. Just looking for information as to what might be avilible.

    • EyeOnTheCure says:

      Kevin, thanks for messaging us. Have you visited http://www.ClinicalTrials.gov?

      On this website, which is maintained by the National Health Institute, you are able to search for Stargardt clinical trials by searching based on the disease. Each clinical trial listing will tell you more about what the study is about and how you can contact them to see if they are recruiting and what the participant criteria may be.

      We hope this information is helpful.

  2. esther says:

    May God continue to show the researchers the right treatment for the cure of this disease iam a patient of the disease and I have not being finding it easy. i Want to know if they is any foundation in nigeria that is assisting the retinas patients in area of treatment and counselling here in nigeria.

  3. Allie says:

    I was recently diagnosed with RP and I would like to know more about clinical trails and cure for this disease.

    • EyeOnTheCure says:

      Allie, Thanks for your comment.

      We would like to point you to a few places on our website that will provide some useful information. Be sure to visit our section on RP:
      http://www.blindness.org/index.php?option=com_content&view=article&id=50&Itemid=67

      There is a section on this page that discusses research advancements. While there is no cure for RP, there is a lot of promising research being done.

      We often report on new research advancements and so we encourage you to check out our RP written articles information every so often for the latest news on this disease.
      http://www.blindness.org/index.php?option=com_content&view=category&id=65&Itemid=121

      As for getting involved with clinical trials, We would recommend that you visit http://www.ClinicalTrials.gov.

      On this website, which is maintained by the National Health Institute, you are able to search for RP clinical trials by searching based on the disease name. Each clinical trial listing will tell you more about the details of the study and how you can contact them to see if they are recruiting and what the participant criteria may be.

      We hope this information is helpful and encourage you to contact us for additional help or further information if needed.

  4. I pray that God gives the wisdom to Retina Researchers to cure RP. I am a RP patient and the disease is frightening not to mention humiliating! I am; however, grateful to have what vision I do have, unlike my brother Bryan. His has progressed further. I watched the movie “The Titanic” the other night with my 15 year old daughter. I thought about it… All those people just panicking and the anticipation of plunging into a deep dark place. No where to go, no way out. Knowing the end result; an ocean full of darkness and death! That is how I feel on the bad days. Things are slowly getting dim and life as I SEE it will be gone. My family keeps me going, especially my two children. I don’t talk about it much because nobody likes a “negative Nancy”! With all the progress the medical field has made up to date, I really hope Retinitis Pigmentosis will soon be a disease of the past!

    • EyeOnTheCure says:

      Sherri, thanks for commenting – as i’m sure you know, because you are part of the FFB family, some of the best support can come from those who are going through the same thing as you and your family are. Are you a member of one of our chapters? And if you are not already a fan – be sure to visit our Facebook page. There is a lot of conversation going on there about what its like to live with vision loss and what can be done to cope with losing sight.

  5. Tio Cabrera says:

    This was just post recently but as far aa technology goes, it seems very likely that they will find a cure. I really hope they do i want to live like a normal human and not somebody that they have to watch over, i have dreams to.It’s just a matter of principles that scientists keep investigating new treatments for the deseased I wouldn’t mind to be their experiment i got nothing to lose by that decision. I have stargardts decease by the way

  6. mary says:

    I have friend who has stargardts,one of her eyes has a vision of 6/30 and another one just started to shows the symptom of disease.she is really desperate and wants to be cured and willing to know about clinical trails and cure let us know how to be involve please email me … you are not only researchers or scientists, you are angels…I love you all,god bless you <3

    • EyeOnTheCure says:

      Mary, thanks for your support

      We would recommend that you visit http://www.ClinicalTrials.gov. We fund many clinical trials but do not operate the studies ourselves and so reaching out them directly may be the best way for you to find out more about where they are happening and if participating is an option.

      On http://www.ClinicalTrials.gov, which is maintained by the National Health Institute, you are able to search for Stargardt clinical trials by searching based on your disease. Each clinical trial listing will tell you more about what the study is about and how you can contact them to see if they are recruiting and what the participant criteria may be.

      We hope this information is helpful and encourage you to contact us for additional help at info@fightblindness.org for further information if needed.

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