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Stem-Cell Research: Moving at the Speed of Light

Dr. Dennis CleggWhen it comes to designing stem-cell-based treatments for retinal diseases, Dennis Clegg, Ph.D., is one of the go-to researchers. He not only heads his own lab at the University of California at Santa Barbara; he’s the recipient of a CIRM grant for a treatment of age-related macular degeneration, and can turn complicated science into a compelling narrative, as this TED Talk demonstrates.

It’s thus no surprise that Dr. Clegg is part of an all-star research team designing a “retinal patch” project funded by FFB. He and I recently spoke about his work as well as the great promise of stem-cell research.

 

Please describe the “retinal patch” project and your role in it.

The idea is to generate a patch that would consist of a scaffold and two different retinal cell types that are sometimes missing in people with retinal diseases—photoreceptors and retinal pigment epithelium (RPE) cells. Both would be derived from induced pluripotent stem (iPS) cells, where you can take a skin biopsy and convert it into whatever kind of cell you need.

My group was the first to show you could make functional RPE cells from iPS cells. Now, we’re teaming up with Dr. David Gamm’s group, which will make the photoreceptors. Then we’ll try to combine them and basically rebuild the outer retina.

And you’re supervising another project, targeting AMD?

The FFB-funded project is really building on four years of another project using human embryonic stem cells to make RPE for the treatment of age-related macular degeneration. That’s a $20 million project funded by the California Institute for Regenerative Medicine, our state-funded stem-cell-research program. That project involves five universities, and our role, again, is to make the RPE cells.

We’re targeting the dry form of AMD, for which there is no treatment. We’ve been successful in a rat model, and we’re now carrying out crucial studies the FDA needs to see for an application for a Phase I clinical trial. We’re hoping to launch that at the end of 2014, beginning of 2015.

What motivates you to do this work? 

There’s incredible potential in stem-cell research to treat blinding eye diseases. The eye is a really good place to develop cellular therapies, as compared to other tissues. Refined surgical techniques have already been developed, and the eye is easily accessible. Thanks to sophisticated imaging technology, there are also many ways to measure visual acuity to see if a treatment is working.

That’s compared to treating, say, a spinal cord injury, where you inject millions of cells into a spinal cord, and then it’s hard to know the results. The eye is a really good place to start.

It also seems as if stem-cell research is advancing relatively quickly.

It is. The first human embryonic stem cells were isolated in 1998, but these iPS cells were first described in humans in 2007, so that’s just a few years ago. And already we’re pushing toward clinical trials. In fact, in Japan, they’re going forward with a trial for the wet version of AMD. That’s the speed of light when it comes to scientific research, when you move so quickly from the basic level, discovery in a laboratory, to application in a clinical trial.

In developing retinal-disease treatments, what role does FFB play?

The Foundation’s role is crucial. It supports a lot of what’s called translational, or pre-clinical, research—what goes on before projects arrive in clinical trials. And to go from the basic discovery of making a cell in a lab to assembling all the data you need to present to the FDA for a clinical trial is an area where FFB provides support and basically moves the needle forward in very significant ways.

What do you foresee happening with potential treatments in the next decade?

It’s a very exciting time. Not only our group but a number of other groups are going forward with clinical trials using RPE cells for macular dystrophies. If you think about it, stem-cell research is similar to the space program in the 1960s—it’s the very early days, very experimental. We’re making these constructs and trying them out, and we don’t know which ones are going to be successful.

Different scaffolds are being tried, different cell lines, different types of applications. I think it’s a great way to go at it. It’s not going to happen overnight, but when we look back 10 years from now, we’re going to see at least one treatment getting into practice.

Pictured, above: Dr. Dennis Clegg


36 Responses to 'Stem-Cell Research: Moving at the Speed of Light'

  1. Linda K, Fuller says:

    I am dignoised with Dry Macular Degeneration.
    It wsv made known to me at lest 10 or 12 years
    ago. I am now 64 yrs old and each check up shows
    a little more increase. I would be so happy to be
    continuely enlightened on the progress of this
    Stem Cell study. If you please You may send
    any new shared information to my email posted
    below. Thank You
    Mrs. Linda Fuller

    shileo49@me.com

  2. We’re targeting the dry form of AMD, for which there is no treatment. We’ve been successful in a rat model, and we’re now carrying out crucial studies the FDA needs to see for an application for a Phase I clinical trial. We’re hoping to launch that at the end of 2014, beginning of 2015. – See more at: http://www.blindness.org/blog/index.php/stem-cell-research-moving-at-the-speed-of-light/#more-3045

    are we really going to spend money in this area when Advanced Cell Technology has been in clinical Trials now for 2 years, and have astounding results curing AMD and Stargardts?

    The Published results will be out in the next 30 days. I hope this money is spent solving some other problem because ACT has solved the AMD Stargardts issue.

    Thanks,

    Dave Anderson, Lincoln, CA

  3. Sharon Hynes says:

    Dr. Clegg,
    I am a 59-year old active woman, recently Dx with early Dry Macular. I am unsure of the future and everyday I wake, afraid to open my eyes for fear of discovering blind spots.
    I am hopeful for this stem cell research, although I believe most trials presently involve patients with the late stage of this terrible desease. I am still in the early stage. Can you tell me if there are trials pertaining to early intervention?
    Thank you for your time,
    Sharon Hynes

    • Eye on the Cure says:

      Dry AMD usually progresses slowly, so you shouldn’t worry too much. With that said, if you experience a sudden change in vision, it could be related to the onset of wet AMD. In any case, you should get to a doctor immediately if your vision changes quickly. You should also look at an Amsler grid everyday to help detect changes in vision. If you have early-stage dry AMD, you should take the AREDS supplement to reduce your risk of advanced AMD – talk to your doctor about prescribing that. It is over-the-counter, but you should still get your doc’s ok.

      Yes, many clinical trials of potential treatments are for people with advanced disease. One reason is that researchers don’t want to jeopardize the vision of people who still see well by giving them a drug that is still experimental. Keep in mind that clinical trials are experiments and have risks.

      With that said, you can visit http://www.clinicaltrials.gov and search for trials of potential therapies. The listings will include participant criteria (e.g., if people with early stage AMD are eligible).

  4. John Barron says:

    I was unaware of the process using iPS. That is amazing. I know for those with vision impairments like RP, the wait for a breakthrough is almost agonizing. My brother with RP constantly asks his doctor for some stem cells. I did order him the ORcam to help him while he waits.
    Keep up the great work.

  5. Katey Bader says:

    My son is diagnosed with RP and his 32 years old please please help him

  6. Kelly McGlaun-Fields says:

    I have a macular hole and fading vision in the R. Please consider me if you do trials.
    I am a very active 74 year old.

    • Eye on the Cure says:

      Kelly, thanks for your comment.

      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 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.

  7. Katy says:

    Are there any clinical trials currently that are focusing on cone dystrophy? Would the results of the trials mentioned above be able to be applied to cones dystrophy? Thank you for posting!

    • Eye on the Cure says:

      Katy, thanks for your comment.

      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 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.

  8. Robin M says:

    I was diagnosed with RP 2 years ago I am 54 years old if the stem cell study would help me I would love to be a part of it. Thank you

  9. Ila says:

    I have Usher Syndrome for those who do not know what this means it is a genetic disease that you are born deaf or hearing impaired and devoloped RP as you get older. I am hearing impaired and start losing my sight at the age of 18. Much to my regret it has prevent me from many of my favorite things. I am 53 years old and would like to know what it take to become a canidate for being used as a “guinea pig” to take this knowledge to the next level?

    • Eye on the Cure says:

      Thanks for your comment. 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 to 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 Usher syndome 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.

  10. Ann says:

    Dear Dr. Clegg,

    Thank you so much for your work with the “retinal patch” and using skin cells to make iPS cells for future use to treat Stargardt’s and other retinal diseases. By far this is the most exciting research I’ve read about…using one’s own cells is so much more sensible from a tissue-rejection standpoint, plus the moral dilemma is eliminated.

    Thank you and God bless you…my son began losing his vision due to Stargardt’s disease at age 6. He is now 14, with approx 20/400 vision. He’s such a brave soul, but it really pains us to see his activities more limited as he gets older…but he wants to do everything!

    Praying Dr. Clegg’s iPS research is supported and successful. Thank you so much for giving us hope!

    Ann

  11. Pam says:

    My 18 year old was just diag. with Stargardth. Any news on Advance Cell Technology that has been working on this?

    Thanks.

  12. rosa says:

    Like Katy. I am also interested in finding out if u can provide any input cone dystrophy disease. Do u believe the work u are doing can also be applied here.

    • EyeOnTheCure says:

      Hi Rosa,

      Yes, there is a lot of research that may benefit someone with cone-rod, including gene therapies, stem cell treatments and drugs. It may be helpful at some point to have the affected individual genetically screened to try and determine which gene mutation is causing his or vision loss. That information will help you understand which emerging treatments may be of the most benefit. We suggest that you visit our Web site and download or genetic testing information to learn more about the process. You can also call us at 1-800-683-5555 for more information.

  13. Patricia Berube says:

    Hi there I am a diabetic and the blood vessels had erupted in my eyes and caused me to go blind and the dr. said that he blood vessel is too weak and that there not enough blood flowing to my eyes.

    I was hoping someone out there could help me. I asked my Dr. about stem-cell and he said no. Could you tell more about stem-cell and if you think I have a chance to regain my sight?

    Feel free to call or text me 613-400-4754, thank you for the time you took to read this message.

    • Eye on the Cure says:

      Patricia, thank you for your message. Unfortunately, we can only speak about research for retinal degenerative diseases that affect the retina in the eye. Your doctors and other eye specialists will be best suited to answer your questions about what treatments are available for your own condition.

      You may want to visit the website of the National Eye Institute (http://www.nei.nih.gov/) or reach out to someone at the institute – they may be able to point you in the correct direction on other resources or organizations who specialize in your condition.

      We wish you all the best as you try to find out more.

  14. Lola says:

    Hello,
    My daughter (15 years old) recently was diagnosed with multiple holes in retina. She is also has astigmatism and myopia. Does stem cells treatment help to her?

    Thanks a lot.

    • Eye on the Cure says:

      Hi Lola, thank you for your message. Unfortunately, we can only speak about research for retinal degenerative diseases that affect the retina in the eye. Your doctors and other eye specialists will be best suited to answer your questions about what treatments are available for your daughter’s condition.

      You may want to visit the website of the National Eye Institute (http://www.nei.nih.gov/) or reach out to someone at the institute – they may be able to point you in the correct direction on other resources or organizations who specialize in her condition.

      We wish you all the best as you try to find out more.

  15. Rosalie Irwin says:

    I live in New Zealand and my son is 23 and has Stargardts disease and we hope and pray that the research will be able to help all who have this problem as it is devasting. Regards Rosalie Irwin

  16. Susan Cox says:

    I am 55 years old and a little over a year ago I was diagnosed with small cataracts in each eye. This clearly upset me as my vision is so important to me. Is there a chance that stem cell therapies will be able to take the place of cataract surgery in the near future? Stem cell research seems to be going at a epidemic pace. It would be wonderful if we can restore our vision through stem cell therapy. Good luck with your research.

  17. Karen Lank says:

    My niece is 21 years old. She has been legally blind since birth. She has had a diagnosis of Retinitis Pigmentosa and Macular Deggeneration. Her hope is to one day have sight. Thank you Karen Lank

  18. John A Hommeyer says:

    My husband is legally blind. He had his first CRVO 40 years ago. In October 2013 he woke up unable to see. His 2nd CRVO was diagnosed at that time. IAre there any plans. to do research w/ stem cells for this situatuion

    We are often in Santa Barbara visiting from Seattle and would be happy to receive any info.

    Thank you for your interest in Retinal diseases.

    Mary Hommeyer

    • EyeOnTheCure says:

      Mary, Central retinal vein occlusion (CRVO) is not a genetic retinal degeneration, so it is not a condition that falls under the scope of the Foundation. There are some treatments for CRVO – namely anti-VEGF injections (e.g., Lucentis, Avastin) as well as anti-inflammatory medications (e.g., steroids). We are not aware of any research for using stem cells to treat CRVO, but this is not our area of expertise. We suggest you discuss your husband’s condition with a clinical researcher who has experience with CRVO. He or she may know more about the research underway for the condition.

  19. Dee. Wells says:

    I had stem cells over a year ago for AMD, I don’t think it improved my eyesight but my eyesight has not deteriorated in the last 12 months. I am using the new Ocutech Sightscope glasses which helps me to continue driving. I continue to research stem cells and gene therapy but get Very frustrated because it always says it will be available in five years time this is a long time to wait why do we need to wait so long. I realize that safety issues but if the stem cells are our own then why would that be a health issue. I believe most people that are losing their eyesight would be more than happy to become guinea pigs for any of the stem cell or gene therapy treatments that are being tested at the moment. If China or Mexico are already implanting stem cells or Gene therapy for macular degeneration then I will certainly be happy to travel for this treatment

  20. Shannon Castillo says:

    My son has Velliform Macular Dystrophy aka Best Disease in his left eye. His eyesight is deteriorating and he is a 15 year who loves to play baseball. Can you please let us know when we can sign up for this clinical trial? I know he and I would love to have a chance at saving his eyesight.

    Thank you,
    Shannon Castillo

    • EyeOnTheCure says:

      Shannon,

      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 twww.ClinicalTrials.gov, which is maintained by the National Health Institute, you are able to search for 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.

  21. Anny says:

    hai, i’m from indonesia.
    my left eye is blind since i was 10 years old, bcause bleeding inside of my eyes cause of ITP (Idiopathic thrombocytopenic purpura) cronic. now i’m 24 years old.
    i grow up with one eye.
    my retina nerve is not working at all.
    is there any possibility or any chances or maybe any suggestions, what i am suppose to do?
    how i can get my sight?
    is there any research stem cell is working for me?
    i’m waiting for reply.

    thank you,so much hope.

  22. Marcel Hogervorst says:

    The technology in eye research over the last few years is amazing. I want to know what is new on the horizon for people who have Congenital Toxoplasmosis. I know that a bioptic implant is available but that just takes the place of the telescopic glasses which I currently wear. I have scar tissue on the retina as a result. Is there a stem cell procedure that can regrow parts of the retina?

    • EyeOnTheCure says:

      Marcel,
      Thanks for your comment. We do not fund research for congenital toxoplasmosis, so we are not familiar with how the condition affects the retina. Also, we don’t know how the condition has affected you personally. With that said, stem cell researchers are able to grow new retinal cells, namely photoreceptors and retinal pigment epithelial cells. Perhaps these cells could benefit you at some point, but only an ophthalmologist familiar with your condition can determine that. Clinical trials of stem cells for retinal diseases are just being launched — they are now being evaluated in humans. However, it is difficult for us to judge what may benefit you in the long run.

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