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The Importance of Stem Cells (a guest post from Dr. David Gamm)

Dr. David Gamm

Photograph by Andy Manis.

When I joined the University of Wisconsin (UW) in 2003, I saw stem cell research as having great potential to benefit patients with retinal degenerations. I also saw stem cells as a way to answer basic science questions about the retina and the conditions that affect it. As a scientist and a pediatric ophthalmologist, these goals were really important to me.

But to enter the field, I had to make a radical shift from my Ph.D. work and return to the drawing board. I wasn’t trained as a cell biologist – I had studied biochemistry – so I had to learn new techniques and concepts from the accomplished scientists at UW.While there clearly are challenges in this area of research, I’m very inspired by the potential of stem cells for saving and restoring vision in people with retinal degenerations. The Holy Grail for me and my colleagues is to use them to replace retinal cells that have been lost, so we can bring back some vision for those with even the most advanced disease.

We’ve made considerable strides toward that goal in a short amount of time. To think that a little more than decade ago, the entire field of stem cell biology was discovered, and now we already have human studies underway – that’s truly remarkable compared to other fields of science and medicine.

The power of stem cells is twofold: 1) They have the ability to become any cell type in the body, including retinal cells; and 2) They have the ability to self–renew, so that virtually limitless supplies of them can be made. Stem cells are said to be “pluripotent” if they possess these two qualities.

Dr. Jamie Thomson, my colleague at UW, discovered the first human pluripotent stem cells in 1998. They were obtained from blastocysts that were left over from in vitro fertilization. These are commonly referred to as embryonic stem cells.

Another big advancement in the field came in 2006 and 2007, when Jamie and scientists from Japan genetically reprogrammed, or induced, mature cells from skin to become pluripotent. This provided a new source of stem cells, called induced pluripotent stem cells (iPSC), which offer many practical advantages and don’t raise the ethical concerns sometimes associated with embryonic stem cells.

Dr. David Gamm at Microscope

Photograph by Andy Manis.

While my colleagues and I work with both embryonic stem cells and iPSC, the latter are unique in that the patient can serve as his or her own source of stem cells. This could greatly increase the chance that these cells will survive, integrate and function without sparking an immune reaction. They could, in other words, serve as a customized treatment for the patient.

Collaborating with Jamie and Dr. Sara Howden more than a year ago, we were able to take skin cells from a 27-year-old woman with a retinal disease called gyrate atrophy and reprogram them back to an embryonic-like state. We then corrected the genetic defect causing the retinal disease — something that had not been done previously— before directing them to become retinal cells. That, as you might expect, was a big breakthrough for us. Recently, we used a patient’s blood cells to develop layered retinal tissue that is closer to being suitable for transplantation back into the eye.

The challenge that lies ahead is transplanting these cells back into the patient and getting them to survive, integrate and restore vision. We’re making meaningful progress and exploring innovative ways, including the development of scaffold-like structures, to do this. It isn’t easy, and it won’t happen overnight, but I am encouraged by the advancements that we and other FFB-funded labs are achieving.

We are also using iPSC, without correcting the genetic defect, to study the underlying retinal degeneration process in a lab dish and figure out how different types of RP lead to vision loss. In turn, we can explore how well different treatments work in saving these cells and, ultimately, vision. Again, all this is happening in a dish using human cells, which are a critical tool for understanding human disease. These are important applications of iPSC not only for us, but for researchers around the world studying a variety of conditions.

I’d be remiss if I didn’t mention that the Foundation believed in the potential of stem cells at a very early stage, long before their power was demonstrated. Fortunately, it also believed in me when I was just starting out in this field. While I’m grateful for that support, mostly I’m excited about what we are doing to help patients. We are, no doubt, on the right path to saving vision.

David Gamm, M.D., Ph.D., is a Foundation-funded researcher at the University of Wisconsin-Madison, where he is also an assistant professor of ophthalmology and visual sciences and the Edwin & Dorothy Gamewell Professor at the UW Eye Research Institute.


34 Responses to 'The Importance of Stem Cells (a guest post from Dr. David Gamm)'

  1. Dear Dr.Gamm,
    I was blind for five years and I regained my sight back in one eye. I am one of the very lucky people and I my hopes are for many more to follow in my path.
    My blindness is from an optic nueritis from overlapping autoimmune diseases.I recieved major intenstive medical therapies over five years and regained my sight back in one eye after a stroke. All very rare and I am blessed beyond words to have regained my sight back in one eye.
    Thank you hardly seems enough, I hope that you get to read this and know that coming from blindness I do now know how truly important research doctors are to the future for all ages to see the light once again. Blindness know no age or color and I do belive that it is research doctors as yourself that are on the forefront for the cures for blindness.
    I want to thank you from the bottom of my heart for all of your outstanding and extraordinary research and for your dedication to strive for a cure for blindness.
    Your research will undoubtedly change the lives of so many that have hope for the cure for blindness.
    I wholeheartldly support the Foundation Fighting Blindness in my small way as a volunteer chapter leader in California and wish you all the very best in your research that is so very important.
    Randyce Wechter

    • EyeOnTheCure says:

      Randyce, thank you so much for your support and for sharing your story with us. It’s wonderful to hear such great feedback from a supporter and I look forward to updating you more on our progress in the future.

  2. RobertAZ says:

    I honestly believe that within 50 years Genetic Research will not only lead to treatments and cures to most disease, but will also have the potential to prolong the quality of life and even dramatically improve one’s abilities in ways that you would think of as “Super”. My hope for normal eyesight is in Science but I am concerned that some Religious groups could slow or even stop progress with genetic therapy JMO.

  3. Congratulations on your important work. When you get close to human trials, I would be most interested in being a subject. I have dry macular degeneration so have only peripheral vision. This is so exciting and know that so many will be cheering you on…

    • EyeOnTheCure says:

      David, we’ll be following the progress that Dr. Gamm and his team make and will certainly keep you and our other readers updated on their work. Stay tuned!

    • Debbee Davis says:

      I couldn’t agree more!!!!. I have Stargardts’ Macular Degeneration since I was 16… Thanks to Technology I recently received my BA 2011.. This news of Stem cells trial that began ACT. the same year I graduated was the best gift I could have heard to be happening in my lifetime. I lived with this for 38 years and truly believed one day a cure would be possible for me and so many others who suffer from so many retinal diseases.. My gratitude goes out to those who work so hard doing this research. From the day I learned about Gene Therapy, my last 2 years of college then the year I graduate Stem Cells? Hope and Faith resides front and center. Added myself to any clinical trials coming up to help the future of what I heard for so many years “No Cure” .. and would do anything to help change this!!!! and never to have any child or adult hear this 2 words ever again…. I realize the challenges ahead but feel the gifts given to those who dedicate their lives to cures is the gift we receive from their hard work and the rewards are grand for all!. THANK YOU FROM the BOTTOM of MY HEART for all your efforts.

      • EyeOnTheCure says:

        Thank you so much Debbee! None of the research we do would be possible without people like you. Stay tuned as we learn more about gene therapy. We will certainly be updating our readers on this area of research.

  4. Cathy scarbeau says:

    Dear Dr. Gamm,
    There are no words to describe how grateful we are to you and other researchers. Your life mission to cure blindness is truly appreciated

  5. Cynthia says:


    I am waiting for a cure for RP. While I realize I am lucky to still see – I have a four month old and I REALLY want to see what she looks like at 40! Please keep working and working faster.

    Thanks for all you do

  6. Natalie Watkins says:

    Dr. Gamm,

    Thank you so much for the post! I look forward to following your work and seeing the miracle of cell integration and improved visual function! It CAN happen. There is so much transplantation work that has come before you; ample opportunities to learn from others’ RPE transplantation challenges.

    Keep striving and believing. I hope to see my children’s faces ten years from now. People like you could make that happen.


    Natalie Watkins

  7. Ione Thacker says:

    Dear Sir, My husband been blind for 6years, due to surgery, he lost all of his blood and it was like a stroke hit the optic nerve. Can you help? We were told that stem cell was the answer. Thank you Mrs Thacker

    • EyeOnTheCure says:

      Mrs. Thacker,

      Thanks for posting. We are focused on the research behind retinal diseases and cannot speak to treating medical conditions on an individual basis. We would recommend discussing possible treatment options with your eye doctor or a vision specialist. We wish you and your family all the best.

  8. Muhammad says:

    Dear Dr. Gamm.

    Weldon for the work you have done.
    I congratulate you on your success and hope that treatment will come soon.



  9. Faye Aul says:

    I am so excited to hear about all of the discoveries with stem cell research. My son, who is 32 years old has RP. I am greatly encouraged by this news. How soon will there be clinical trials? Will there be some trials, in close proximity to Tulsa, OK?
    Thanks again for all you are doing.

    • EyeOnTheCure says:

      Faye, right now, the Retina Foundation of the Southwest in Dallas is the clinical trial center closest to Tulsa for people with inherited retinal diseases. They are running a study of valproic acid for people with autosomal dominant RP, and they are recruiting participants. They are also conducting a clinical trial of DHA for males with XLRP, however they are no longer recruiting, but hope to report results by the end of 2012.

  10. Robert Clark says:

    I am so thrilled to learn of your breakthroughs in stem cell research. I suffer from macular degeneration in both eyes. When you begin testing on humans, I would love to volunteer.

    Thank you for the great work you are doing!!

  11. Marlow Lim says:

    Dear Dr David Gamm,

    Thank you for bring hope to the people of the world who can’t see or have eye diseases.
    Your reasearch truely give hope to many.
    I do my small part surportting FFB.

    Wish you every success in your reasearch foe eye diseases.

    Best Regards
    Marlow Lim (Singapore)

  12. tv cover says:

    RSS Feed? I’d like to add to my blog reader.

  13. Michael Clavet says:

    Dr. Gamm,

    Thank you for all your work and research to help those of us with vision impairment. I was diagnosed with Gyrate Atrophy over a year ago and until reading your post tonight I had little hope that anything could be done about it. I am a strong believer in stem cell research for finding cures for many diseases. So far I have been told that there are no studies on my condition. I would appreciate any information you could share on any treatments or studies for Gyrate Atrophy.

    Best Regards,

    Michael Clavet

  14. Carol Correggia says:

    My 16yr old granddaughter was born deaf, had cochlear implant at 18 months. We only learned in Feb that she has Usher’s Disease and yesterday was declared legally blind. We pray to find a clinical trial using stem cell
    therapy that she can participate in. How soon will you be starting one?

    • EyeOnTheCure says:

      Carol, thanks for your comment.

      A company called ReNeuron is currently planning a clinical trial for 2014 of its stem cell treatment for people with retinitis pigmentosa. Because your granddaughter is relatively young and has Usher syndrome, we would suspect that she would not be eligible for the study. Though the retinal manifestation of Usher syndrome is retinitis pigmentosa, the trial investigators may not enroll Usher patients. However, if the therapy gets regulatory approval after the clinical trial is completed, it might be a treatment that can benefit her.

      We hope this information is helpful and wish you and your family all the best.

  15. Jack stark says:

    I have wet macular degeneration. I am very impressed with your studies and was interested in this treatment. Can you give me more information.

  16. Johnc373 says:

    What a video it is! In fact amazing and fastidious quality, please upload more video clips having such good quality. Thanks. bfbcedfbcfab

  17. Robin Taylor says:

    As with the previous posters, I too would like to be updated as to when and where the soonest and nearest human trials will be practiced. My wife has RP and will be totally blind in the not so distant future. Thank you for all your team has been doing

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