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Several New Stem Cell Clinical Trials Poised to Begin in Two to Three Years

Microscopic Stem CellsOne of the more exciting aspects of FFB’s recent annual conference, VISIONS 2013, was the news on the clinical development of several emerging stem cell therapies. The field has picked up a lot of steam, with many new human studies of stem-cell treatments anticipated to begin in 2014 and 2015.

What’s particularly noteworthy is that some of these trials will be for people with retinitis pigmentosa (RP) — a population not yet included in human stem-cell studies. Also, investigators will evaluate new types of cells and new, innovative approaches to implanting them.

Here’s a review of three of the stem-cell clinical studies on the horizon:

Riken Institute: Sheets of RPE Derived from Induced Pluripotent Stem Cells

In June, the Japanese Health Ministry gave approval to the Riken Institute to launch a clinical trial of sheets of retinal pigment epithelial (RPE) cells for the treatment of wet age-related macular degeneration (AMD). The study is planned for 2014.

The sheets will replace native RPE cells damaged by leaky blood vessels, which are the hallmark of wet AMD. RPE cells play a critical role in supporting photoreceptors, the cells that make vision possible.

For the first time in a human study, the researchers will produce RPE cells from induced pluripotent stem cells (iPSC) — stem cells derived by turning back the development clock on a small sample of skin taken from the patient. The stem cells are then coaxed forward to become RPE. Because the cells come from the patient, there’s less chance of immune rejection.

David Gamm, M.D., Ph.D., of the University of Wisconsin-Madison, said that transplanting sheets of RPE cells — rather than injecting a bolus or a clump of cells — was likely to produce better results, because RPE cells naturally occur as a layer in the retina. He believes that sheets of cells have a better chance to integrate and function normally.

By the way, Dr. Gamm, in collaboration with Dennis Clegg, Ph.D., of the University of California, Santa Barbara, is receiving Foundation funding to develop a patch of cells — comprised of photoreceptors and RPE cells — for treating retinal diseases. While his work is still a few years away from the clinic, it is very promising, because so many retinal diseases affect both of these cell types.

UC Irvine: Retinal Progenitor Therapy for Reactivating the Retina

Henry Klassen, M.D., Ph.D., of the University of California, Irvine, and a former recipient of a Foundation Career Development Award, is planning to launch a clinical trial of a stem-cell therapy for people with RP. He hopes to begin the study in late 2014 or early 2015.

His treatment involves the transplantation of progenitor stem cells — cells that have partially developed into retinal cells. Based on results from his lab studies, he believes the progenitors will rescue and reactivate dormant cones in the retinas of people with early- and late-stage disease.

Not only is Dr. Klassen an outstanding scientist, he’s resourceful and collaborative — qualities that will undoubtedly enhance his chances of success. He obtained a $17.3 million grant from the California Institute of Regenerative Medicine, a legislative initiative providing $3 billion in funding over 10 years for stem cell research in facilities throughout California. Dr. Klassen’s project was also accepted into the Therapeutics for Rare and Neglected Diseases (TRND) program established by the National Institutes of Health to speed the development of new treatments for rare and neglected diseases. TRND will provide him with specialized expertise and resources to help advance his efforts.

ReNeuron: Retinal Progenitor Therapy for Replacing Lost Photoreceptors

One of the more ambitious stem-cell treatments nearing human study is being developed by ReNeuron, a company from the United Kingdom. Its retinal progenitor treatment replaces photoreceptors lost to retinitis pigmentosa. When transplanted in the retina, ReNeuron researchers believe that the partially developed cells will mature into fully functional photoreceptors. The company hopes to launch a clinical trial, probably in the U.K., in the next year or two.

Previously funded by the Foundation, Michael Young, Ph.D., of Schepens Eye Institute, Massachusetts Eye and Ear Infirmary, conducted much of the research making this treatment approach possible. His work included the development of a biodegradable scaffold for growing and organizing the cells prior to transplantation. The structure increases the chances of survival and integration of the therapeutic cells.

But Wait — There’s More

Fortunately, there are far too many promising stem-cell research efforts to cover in one post, so here are links to other retinal-disease projects:

Pictured, above: partially developed retinal tissue derived from induced pluripotent stem cells. Courtesy of Dr. David Gamm.

 


48 Responses to 'Several New Stem Cell Clinical Trials Poised to Begin in Two to Three Years'

  1. Andy says:

    Like your list, but why does an active clinical trials with a patient 20/400 to 20/40 not get top billing at your foundations. Do you guys talk to ACT? If not Why?

  2. Katey Bader says:

    Thank you for the hard work and God bless you all, please hurry up and get us treatment or cure every day count with rp!!!

  3. sandy young says:

    Thank you for all the research you are doing. Will there be any stem cell research done on optic nerves that have died?

    • EyeOnTheCure says:

      Sandy,

      Thanks for your comment.

      Unfortunately, Optic Nerve Damage is not a disease that we specialize in funding research for and so we ar enot the best organization to comment on its research progress. We would encourage you contact or to check out the site of the National Eye Institute.

      At the link below, the eye institute provides contact information for other resources and organizations that can assist you and tell you more about Optic Nerve Damage specifically.

      http://www.nei.nih.gov/health/resourceSearch.asp?Disp=1&strKey=Optic+Nerve+Disease

      Please let us know if there is anything else we can help you with.

    • raye anne wade says:

      my son was hit by a car and lost his vision when he was 5. doctors told us it was the optic nerves that were damaged. he is 12 now and still prays that somehow he will be able to see again someday. would love to hear more about any new science. i feel blessed that he is so young, and with science being so advanced now, i know he WILL see again. we appreciate all that is being done to help and pray god guides these hands that are testing different areas. thank u.

      • EyeOnTheCure says:

        Raye, thank you so much for your comment and for your support. As the blog post states, we have yet to see what we will find out as a result of these trials, but like you, we are hopeful that this research could provide a possible treatment for a variety of conditions. We wish you and your family the best during this time.

    • Jim says:

      Sandy, I have the same question. We’re you ever able to find out any information? My son Michael suffered a TBI and his optic nerve has died as a result. He is 20 years old and completely blind. We have been to china for stem cell therapies but no results. Please email me at jtoto322@yahoo.com to share your findings. I would love to hear your progress and share ours.

      Thank you,
      Jimmy

    • Brian Butler says:

      Hi, I was wondering if you ever got an answer to your question about trials for dead optic nerve treatment. My brother lost his sight 10 years ago due to traumatic brain trauma. His optic nerves are in tact but not functional. We are hoping trials would start soon, his last doctor told us treatments were being worked on but haven’t heard much more. Could u let me know if something comes along. Thanks-Brian butler

    • Gretchen Steele says:

      My husband participated in a NIH clinical trial in Margate, Florida. He has had optic nerve ischaemia in both eyes, and had limited vision. He had the procedure on July 1, 2014. His vision is improving, but slowly. However, it is better than before and appears that it will continue to improve. This was done by using his own stem cells, to see if they would repair damage to the optic nerves. We are hopeful. A friend of his had the same procedure in October, and is noticing improvement in his vision. Their optic nerves were not dead…..they were compressed, which limited the blood flow in the optic nerve.

  4. Rick Jones says:

    I recently spoke to the Stem Cell Center at UC Berkeley. They also said clinical trials might start within 3 years but they couldn’t say where. I called on behalf of my good friend, Joe, whom I’ve known for 40 years. Joe has RP and has indicated a willingness to participate in clinical trials. Will the RP Foundation be following this and providing info on how to register or what criteria the researchers will be looking for in volunteers? A solution seems tantalizingly close!

    • EyeOnTheCure says:

      Thanks for your comment Rick.

      There is a company in the United Kingdom called ReNeuron (http://www.reneuron.com/our-products) which is planning a clinical trial of a stem cell therapy for people with RP. They are planning to launch their study within a year, perhaps sooner. It is not clear if that study will take place in the U.S. and/or the U.K. Aside from the ReNeuron trial, the first stem cell clinical trials for people in the U.S. with RP are likely a few years away. The Foundation will announce details of these trials – including participation criteria – on our Web site when it becomes available.

  5. Philomena Milbourne says:

    May the good Lord guide ye all, to bring a cure for R.P. and all other macular diseases. The.difference this would make to so many peoples lives.

  6. Anthony Steele says:

    This article brings me so much hope.

  7. abuturi says:

    Thank you for the hard work and God bless you all, please hurry up and get us treatment or cure every day count with rp!!!

  8. Rachel says:

    I want to thank all those who are working hard to bring a cure to RP.. I know it’s coming! God bless

  9. kimberlina Hernandez Krieger says:

    Thank you for the hard work.very exciting …

  10. Mary Lou Johnson Evans says:

    Thank you so much for pushing ahead and not giving up on the light at the end of the tunnel. I pray everyday that a cure happens soon for my teenage son with
    x-linked RP. It is through research and doctors like Samuel Jacobson at Scheie Eye Institute that give us hope. God Bless~

  11. Kaushik says:

    Hope this method of retinal treatment reaches out to the affected people sooner than later.

  12. sky seace says:

    where do I sign up for a trial ?

    • EyeOnTheCure says:

      Sky, thanks for your comment. These trials are at different stages and are each focused in different areas. If there is one that you are interested in learning more about, or even participating in, we would recommend visiting http://www.clinicaltrials.gov and doing a search for the particular study. this website provides additional details and contact information for those conducting the study.

  13. Shelley says:

    Thank you for trying, I hope there is a cure before I become blind because I’ve got very bad depression because of it and I really want to be able to see my kids and there kids, I know how bad my grandpa feels about not being able to see us :(

  14. Warren Aaronson says:

    Our problem (my wife Carole) is her optic nerve went ischemic after a stroke which lodged an embolus in the artery feeding her optic nerve. Since she had only one good eye, she became totally blind six years ago.
    Stem cell research to regenerate the optic nerve would be overwhelming for us!!

  15. Herb Fuller says:

    God Bless and God speed to save my sight

  16. Billie Young says:

    I am so happily excited that the research you are doing gives such hope. My two kids ages 16 and 18 have RP. They have had it passed to them through their fathers side of the family. I my self have 20/15 in both eyes, so I do not know how they are seeing things exactly. My daughter wanted to be a police officer until she found out this year she has signs of RT. With this research are you looking to completely restore the the cones back to “20/20″ or can you predict? where can i go for more info. if possible may you please send me up-dates on the latest information concerning RP. Thank you for the hope my kids may have with this research. Blessings to your reach and understanding. THANK YOU

    • EyeOnTheCure says:

      Thanks, Billie, for your inquiry. Here are a few comments/replies to your questions.

      Most forms of retinitis pigmentosa (RP) affect peripheral and night vision first. So, children and young adults have more issues with seeing in dark settings or objects off to the side. They may not have trouble reading a book – or other tasks that require the ability to perceive details –
      until later in life. Your kids may still have 20/20 vision (or relatively good acuity), and instead have trouble seeing at night. The severity and rate of progression of vision loss can vary from person to person, even within the same family.

      There are many emerging treatments for RP, some of which may slow disease progression or others that may restore vision. We recommend you visit http://www.fightblindess.org and this blog regularly for information on the research. At fightblindness.org, you can also register to receive our newsletters.

      It is also important that your family get care from a retinal specialist who is familiar with RP. If you call 1-800-683-5555, we can try to find an appropriate retinal specialist in your area.

      Good luck and thanks for your inquiry.

  17. Ruth says:

    I’m glad that there will be a stem cell for the eyes, I think its really needed considering the number of people having eye problems.

  18. Sylvester A. Pringle says:

    My twin brother and I have been dealing with RP Since 3 years old. Now 36. This is a Godsend. Our heavenly Father ,Jesus our Lord and savior have the last say so. We will be cured. Our prayers have been answered. My RP people i know the struggle and the pain. The tears the running into things. Soon, very soon, you”re be driving, seeing stars in the night sky, seeing love ones and telling them thank you for guiding and helping me all these years sincerely appreciated every moment. But now I got this!!!

  19. Dilshad says:

    I very much appreciate the amount of hard work each team is putting in research. My husband is suffering from RP, his vision is deteriorating with his age but I am not giving up any hope and believe he will be able to see one day. Please keep me updated on your research results.

  20. Abbbessi abdelbaki says:

    bonjour,
    je suis tunisien et je suis trés heureux d’entendre parler que vous etes sur le point de lancer votre test clinique en vue d’essayer de traiter la retine pigmentaire. en fait j’ai deux fils qui souffrent de l’RP. je suis vraiment heureux d’apprendre votre succés.
    que dieu vous aide à accomplir cette tache noble et donner de l’espoir à tous ceux qui souffrent de cette maladie.

  21. Marina Makinen says:

    My husband has ischemic optic Neuropathy in both eyes & can only see shadows he is 49. Is there any research being done?

    • Eye on the Cure says:

      Thanks for your message, Marina.

      Unfortunately, Optic Nerve Damage is not a disease that we specialize in funding research for. We would encourage you contact or to check out the site of the National Eye Institute.

      At the link below, the eye institute provides contact information for other resources and organizations that can assist you and tell you more about Optic Nerve Damage specifically.

      http://www.nei.nih.gov/health/resourceSearch.asp?Disp=1&strKey=Optic+Nerve+Disease

      Please let us know if there is anything else we can help you with.

  22. Alex says:

    For those posting here: It seems these clinical trials target RPE and photoreceptor cells. From what I’ve read, RPE cell clinical trials are already underway. ACT has had some success implanting between 50,000 and 100,000 cells or so under the retina and restoring some vision or at least better visual acuity. RPE cells feed the other retina cells including photoreceptors. Photoreceptor stem/progenitor cell trials seem close with dr. klassen in Irvine CA, but I haven’t heard much about progress with ganglion cells. They’re the ones that carry the signal to and through the optic nerve and are important for already severely damaged retinas and optic nerves. I wonder how close they are to trials. I’d also like to hear about how we can help. Donations, awareness events, etc…

  23. Hi to every , because I am actually eager of reading this weblog’s post to be updated daily.
    It carries nice material.

  24. muhammad adeel iqbal says:

    Hi every one .
    My heartily prays and best wishes for all the people !
    In Pakistan , I’ve been hearing since my childhood that there is no cure for RPE in the world and every time I came out crying from the hospital . I’m 30 of age now and have a very low tunnel vision . It seems that the m gonna be blind very soon . I have been facing too much pain and problems and wanna die now . Please tell me how can I be a part of three trials .

  25. ahmad says:

    Ihave rp .I would like to be in any trial .

  26. yonathan says:

    I have a problem with optic nerve and a retina. Do you think that trial fits for me, and if not- when do you think it will be?

    • EyeOnTheCure says:

      The first step is to obtain a specific diagnosis. You mentioned you had a problem with the optic nerve and retina. This could be any number of retinal diseases such as LCA, LHON, glaucoma, RP, etc. You should ask your doctor for the name of the disease. He or She should be able to provide you with this information. Once you have the specific diagnosis, I would be happy to help you try and find potential treatments. Thank you for your support.

  27. caren says:

    GOD BLESS YOU FOR DOING THIS RESEARCH I HOPE A CURE IS IN SIGHT AS MY HUSBAND HAS WET MACULAR DEGENERATION IN BOTH EYES NOW PLEASE CONTINUE YOUR GOOD WORK FOR ALL OF THE PATIENTS

  28. Richard says:

    I realy hope you find a cure for Glaucoma too. I’m only 51 and canot function anymore due to this terrible disease. I want to live a normal life and see things as they were.I have to stay home to be familiar with my surroundings. I’m very depressed. Once a fully member of society, now a wreck. Pls. speed up, because I don’t know how long I can stand this misserable life anymore.

    • EyeOnTheCure says:

      Richard, please be assured that researchers are working hard to try and unravel the mysteries surrounding these diseases. The good news is that what scientists are learning from one disease is helping them with the design of treatments for other diseases. The drug Brimonidine was first approved for use in glaucoma but is now in human clinical trials for retinitis pigmentosa. You should see your ophthalmologist regularly to make sure your IOP is not too high.

  29. stella morangi says:

    i had a retinal detachment some years ago.i had surgery and after a year i got a cataract.so i had another surgery but the retina detached again.by the time i went in for another surgery the retina had crumpled,and after the surgery i could only see light and nothing else.are u able to restore my retina through stem cell surgery?

    • EyeOnTheCure says:

      It is possible that stem cell therapy could help but you should seek the advice of a trained vitreo-retinal specialist. He or she can provide you with detailed information regarding the physical state of your retina and which, if any restorative treatments such as stem cells, visual prosthetics or optogenetics may be of value.

  30. Ram says:

    Please please let me know how my brother can enrol for trials, who is suffering from rp from past 10yrs. Can’t see my brother sulkin over d disease. Please reply whatever it is. Crossing my fingers n hoping to be one among them.

  31. Carol says:

    I have a father n law who has had his retna die almost completely. We are very interrested in finding out how to get excepted for the human trials. To see my father nlaw not be able to continue working due to this is very hard. He does what he loves to do and needs his eyes to do it. He is a veteran and we want him to be able to continue to work. Please let me know more about this and help him. Thank u.

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