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Stem Cell Therapy Clinical Trial Begins With Initial Participant

Dr. Rand Spencer, left, and Dr. David Birch, surgeon and lead investigator, respectively, in the StemCells, Inc., clinical trial.I was very excited to learn that another clinical trial using stem cells for the potential treatment of a retinal disease — in this case, dry age-related macular degeneration (AMD) — is now underway. StemCells, Inc. (got to love the company’s name) announced last week that its first patient was treated at the Retina Foundation of the Southwest in Dallas.

While the 16-person Phase I/II study will evaluate the transplanted neural cells’ ability to preserve vision, the primary goal for now is to demonstrate safety. If the Phase I/II trial is successful, later stage studies will take a closer look at efficacy.

When I hear about groundbreaking studies like these, I always wonder who the courageous patient was who agreed to be the first to receive the treatment.  I don’t know if I’d personally have the chutzpah to be the first. The people who volunteer to be in these early-stage human studies for cutting-edge treatments are the true pioneers and heroes.

In addition to receiving a  potential treatment with very little (or no) track record in people, they have to make numerous clinical visits with extensive retinal exams and vision tests. Not only are they taking a risk, but clinical trial participants make a big commitment of time and energy.

I’d be remiss if I didn’t acknowledge the indispensible role of the clinicians and researchers who run the trials and perform the related procedures. They, too, are true pioneers. In the case of StemCells, Inc., Dr. David Birch is the principal investigator, with Dr. Rand Spencer performing the surgeries.

A researcher funded by the Foundation Fighting Blindness, Dr. Birch is no stranger to clinical trials. He currently serves as the principal investigator on human studies of valproic acid for the treatment of autosomal dominant retinitis pigmentosa (RP) and docosahexaenoic acid (DHA) for X-linked RP.

In recent years, he also led clinical trials of Neurotech’s encapsulated cell technology and Second Sight’s “bionic” retina. He clearly has a penchant and talent for living on the cutting edge of retinal research.

While the first results from the StemCells, Inc. study probably won’t be reported for a while, I look forward to reporting on them when they are announced. Stay tuned.

Pictured above: Dr. Rand Spencer, left, and Dr. David Birch, surgeon and lead investigator, respectively, in the StemCells, Inc., clinical trial.


20 Responses to 'Stem Cell Therapy Clinical Trial Begins With Initial Participant'

  1. Dave says:

    Very interested to read this article and would thank you for taking the time to post it. However, I am slightly confused as to what the goal of this trial. Is it similar to what ACT are doing in that the stem cells are to repopulate the retinal pigmented epithelial layer in order to replace those support cells or is there something else it is attempting to doing (i.e. replace photoreceptors that have been damaged). I know that initially the only result they are hoping for is safety and efficacy comes later, but was curious to know more.
    Hope someone can clarify.
    Regards,
    Dave.

    • EyeOnTheCure says:

      Hi Dave, Excellent questions. ACT’s treatments consists of stem cells that are differentiated into retinal pigment epithelial (RPE) cells before they are transplanted. RPE play an important role in supporting photoreceptors – waste disposal, nutrition, and delivery of protective proteins (survival factors). It is still unclear how much the newly introduced RPE cells will mimic native RPE cells. The more the better. StemCell, Inc.’s treatment consists of adult neural stem cells, which will only deliver a mix of survival factors – they don’t function like RPE or other retinal cells. The clinical trials will tell us more about which stem cell treatments are effective. Stay tuned.

      • Dave says:

        Thanks very much for your response, that’s all much clearer now.
        Have a feeling the world is right on the edge of something unique and just plain great – to be able help a lot of people who are suffering from vision loss to rediscover the images of the world again.
        Kind Regards,
        Dave.

  2. Shirley pruitt says:

    Hello I am interested in the clinical trials for Retinitis Pigmentosa my mother has this terrible disease that has robbed of a normal life we have just lost our Father and he was her eye and to she her greive is so hurtful to be in darkness with someone you love gives you a comfort but to be in darkness alone is horrifying please tell me how can I get her in this clinical trail

    • EyeOnTheCure says:

      Hi Shirley,

      I am sorry to hear about the loss of your father and your mother’s challenges with RP. That creates a difficult situation. Please keep in mind that clinical trials are experiments and people entering them should be doing so because they want to help advance research – not because they expect their vision to be saved. With that said, the clinical trial mentioned (StemCells, Inc.) is for people with dry age-related macular degeneration. A stem cell clinical trial for people with RP (http://goo.gl/FFb53) is planned for 2013 or 2014. If you would like more information for potentially helping your mom, call us at 1-800-683-5555 and we can make some suggestions based on your mom’s individual situation.

  3. DrJAndrews says:

    As an MD, I love research, and moreso, progress. With respect, I haven’t seen any of Dr Birch’s clinical overseeings amount to anything I’d call successful. RP still causes blindness.. The bionic eye, which I
    I’ve investigated, gives extrememly poor vision, (was up to 32 or 64 pixels when I last updated), if even worth having at this point, with the risk of infection, etc., during the implant procedure..
    Don’t get me wrong, I hope things work out. With this StemCells Inc, trial, what type of neural cells are they? Are they from an embryonic stem cell source, like ACT (Advanced Cell Technology), who’ve successfully used Embryonic Stem cells, or, are they simply transplanted neurons? Let us know Dr Rose..

    • EyeOnTheCure says:

      Dr. Andrews,

      Thank you for your note and questions. I think it is important to keep in mind that clinical trials are experiments and most treatments don’t make it through to FDA authorization. Even if the treatment doesn’t make it through, however, we learn a lot from these human studies. Five years ago, there were virtually no clinical trials underway for inherited retinal diseases. Today, there are several which I list in a recent blog post (http://goo.gl/95dMl). Some will succeed – some won’t. Most are still at an early stage. But there has never been a more promising time for people with retinal degenerations.

      An FDA panel has in fact recommended the Argus II retinal prosthesis for approval. Yes, it only provides rudimentary vision now, but for people who are completely blind, that is quite meaningful. Several companies are working on devices that will hopefully provide better vision. The StemCells Inc. trial is for neural stem cells that deliver a mix of survival factors that have been shown to slow degeneration. First, the researchers will be evaluating safety, then efficacy.

      Please keep in mind that Dr. Birch is one of the world’s leading retinal researchers and we are tremendously fortunate to have him supporting our mission and leading human studies. I encourage you to come to our VISIONS conference (http://goo.gl/IeKDI) next summer to meet him and other researchers in person. They are amazingly dedicated and knowledgeable scientists, who dedicate their lives to fighting blindness.

  4. As someone who has just been diagnosed with dry AMD (at 50 years old) it gives me some hope to see any kind of clinical trials being undertaken. I certainly have no understanding of the science, eventually perhaps I will grasp some of the finer points, but I can only take some comfort in the fact that there is promise.

    I am grateful for this website and will keep tuned.

  5. LATRICIA H says:

    I’d like to participate in a study

  6. Alina Ishikawa says:

    I just want to thank you for the interest all of you have about the RP, I have this disease and It`s really terrible, because I am a mom and I live with the fear of my children have the rp too.
    anyway I hope something come out soon.

  7. Ruth Mohwinkel says:

    Our 42 year old son was diagnosed with Central Serous Chorioretinopathy.
    We attended the FFB in Mpls this summer and found no one who knew of CSR and yet he has seen retina specialists in WI, Mpls, MN and Mayo in Rochester, MN., all with same diagnosis. We are searching for information from others familiar with CSR. Prayfully any trials related to the retina will also be helpful to CSR patients.

  8. Bobby Taylor says:

    I would like to sign up for A Mac Tel study.Where can I get info.

  9. MG says:

    Hello,
    My partner got blind 2years ago because of severe glaucoma. She also have diabetes for like almost 20 years.
    She is 29 at the moment. Thing is that her eyes are in flames and they did laser surgery to lower her eye pressure. Surgery managed to lower the pressure but now she gets this pain and she keeps vomiting because of it. Doctors said that they never seen anything like that before. They also said if the pain wont go away in next 3-5 weeks then they will have to take her eyes out..

    So I was wondering if they will take her eyes out will she still be able to have artificial bionic eyes in the future or not any more?
    She also would like to go for trials and etc.. She understands the risk and she said there is nothing she would lose.
    So we were wondering if there is a place that she could sign in and be one of the test subjects.

    We live in UK.

    • Eye on the Cure says:

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

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