Stem Cells for Stargardt's Macular Dystrophy and Macular Degeneration

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Stem Cells for Stargardt's Macular Dystrophy and Macular Degeneration

RPE Transplantation for Stargardts Disease

 

Advanced Cell Technology recently announced the beginning of their landmark trial where stem cells are transplanted into patients with Stargardt’s Macular Dystrophy (aka Stargardt’s Disease) and dry macular degeneration.

Clinical Trials for Stargardt’s Begin

The company announced that their phase I/II clinical trials started in mid-July with one patient each of either Stargardt’s or macular degeneration.  Each patient received a relatively small dose of cells and subsequent patients will receive larger amounts of cells.  The first patients received about 50,000 cells.

The goal of the study, at this phase, is to test the safety and tolerability of the stem cell treatment over a 12 month period, that is, phase I/II will answer the question as to “how safe is the procedure?” and will also asses if this is a viable way to transplant stem cells.

Retinal RPE Cells are Replaced

The cells to be replaced are called RPE (retinal pigment epithelial) cells and are located underneath the top layer of the retina.  If you liken the retina to be an open faced sandwich with a slice of ham with cheese on top, the RPE cells are the slice of ham.  The “rods and cones” (otherwise known as photoreceptors) are located on the underside of the cheese.  The RPE cells nourish the photorecptors.

In cases of Stargardt’s disease and dry macular degeneration, the faulty RPE cells can no longer nurture, or feed, the photoreceptors, hence, the loss of vision.  It is hoped that by replacing the sick retinal pigment epithielial cells with stem cells, the vision can return.

What Does This Mean? This is really promising and exciting news.  On the horizon is the potential for a true “fix” for two of the most common forms of “blindness” in the world.  Actually, most of these patients are legally blind as you remember that the macula gives us central vision.  Thus, RPE transplantation should improve the central vision.

In my view, there are two large variables or areas of concern.  First, the actual technique of implanting the cells must be refined and perfected and at the same time, we need to see how long the stem cells can live or remain healthy.

While this is very promising, this may still be years away from becoming a viable treatment option.

Here is further information about patient inclusion into the study for either Stargardt’s Macular Dystrophy and dry ARMD.

 

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30 Comments

  1. Lawrence Barrett says:

    T^hat’s exciting news on ste, cell research! Thanks!!

  2. richard sievers says:

    Doctor—-thank you for writing about the Advanced Cell human trials using RPE embroyic stem cells-(occurring at UCLA)….the word needs to be gotten out that these trials are going on. I have been following this for about 3 years (my 36 year old daughter has Stargardt’s) and am amazing at how little notice these FDA approved trials have gotten. This process, if it works, could be one of the most startling and helpful medicial advances in many many years—-regenerative medicine holds out the potential for cures rather than treatents. It is exciting to watch—-(and early investors in ACTC could make a ton of money….think microsoft in the beginning days) Hopefully you will continue to keep your readers informed. You can direct readers to teh ACTC web site for more information. The key appears to be whether the cells will attach in humans as they did in the animal tests. thanks again. Richard .

  3. John D. says:

    This is good news to hear, to date the only news I have heard to combat blindness is the development of a robotic eye, perhaps still a viable option for some in the future.

    Though we are years away from an effective treatment hopefully this will start a domino effect in research. Not enough money and Brain power for research.

    Does it ever seem like we are in the twilight of medicine?

    So far advanced that we can cure people that would have died 100 years ago. Yet we are far from cures like cancer, blindness, AIDS, and the number of rare diseases.

  4. John D. says:

    Richard S. I hope the best for you and your daughter. Eye problems suck.

  5. Michael HAwkins says:

    I have stargardt’s disease how do i get into the steam cell trial ?

  6. anne siddins says:

    My husband has Stargardts Macular Dystrophy. Aged 61 years old, who is in good health, both physical and mental, fitting the criteria as listed. He has been seen by Emeritus Professor Dr Frank Billson,Clinical Ophthalmology & Eye Health,from the Sydney University, Email: proffbillson@gmail.comfbillson@bigpond.net.auEmeritus Professor Dr Frank Billson has Glenn`s visual reports. Glenn is presently retired from years of working as a Hospital Orderly. We live in NSW, Australia. Will you be recruiting any subjects from overseas, if so Glenn possibly would be interested. There is no known history of SMD in his family . We have two daughters with good vision aged 28 and 25 years, and both girls have been screened for SMD, when they were young. Glenn was diagnosed at the age of 7 years, and has never had any treatment for his condition. And the limited amount of sight he has is still deterioating. If by chance we can help with any questionaire`s or lifestyle scores, Glenn will be happy to assist. With thanks

  7. gary lough says:

    i am currently a patient of dr. nolan from farmington conn. i have stargardts. i am 53. i acquired it about 6-8 years ago.

    i had an appt w/dr. nolan 10/17/11. he said i am a candidate for stem cell research. as i am a good canidate for it. i am inquiring about any trials now on the east coast, so i can get a slot in it.

    i can send you my papers, etc. from dr. nolan if needed.

    please, please, advise me of any upcoming research trails that i may participate in. i am only about an hour from your site, and i can come and go to see you as often as needed.

    please reply…………

    desperate,
    gary lough

    pestdoctors@aol.com
    gary lough
    200 bridge ave – rear
    bayhead, nj 08742
    732 267-7908

  8. Leona Saunders says:

    Hello Randy I was diagnosed with SMD 6 years ago when I was 60 years of age.About 3 yearsi started getting Avastin inj.in my right eye because of swelling under the retina.A few hours after the 4th inj.I was in a lot of pain – something had gone wrong and I ended up with 20/400 vison.I have been getting inj. of Lucentis in my other or left eye for the same reason for the last 14 months. the inj. have been stopped for two month as the swelling is worse.My OphthalmolIigist does not know why this is happing and will be checked again in Jan. 2012.I have heard of patients getting extra inj.to reduce swelling . Do you have any thoughts on these statements as it would be appreciated. yours Leona Saunders

    • Dear Leona,

      1. You have Stargardt’s, correct?
      2. Avastin given for macular edema?
      3. After the 4th injection and “the pain,” how long did it take to resolve.
      4. The recurrent swelling….not my experience that there is much swelling with Stargardt’s
      5. Second opinion sound warranted.

      Randy

  9. Leona Saunders says:

    Hi Randy I am going to aguire more info. for you .

    thanks ps my name is ken saunders husband and am typing for leona

  10. Leona Saunders says:

    Hello Dr WONG
    Dear Dr Lypka [optometrist]
    Leona will be contacting your office for a update refraction .As you know she will have significant maculopathy related to stargardt`s fungus flavim aculatus and has actually developed exudative disease in both eyes. She was treated with anti-vegf injections in the right eye initially with stabilization and no active exudation but she has had very limited central vision for quite some time. Unfortunately over the past year or two there has been development of exudation in the lrft eye for which she has also under gone Lucentis injections.More recently it seems as though she was not getting much added benefit from monthly Lucentis injections and so we have decided to watch consefvatively for a short period.She did have Lucentis injections in november and i am having her come back for review in two months time to see how things are holding.
    She has also undergone bilateral cataract surgery for significant cortical cataracts, the left september 14 2011 and the right november 9 2011 . She has healed well with the cataract surgery but vison is at a count fingers right and 20/50 left uncorrected .She will be stable for a updated refraction by mid to late december and will contact your office for this.
    Sincerely ,
    Dr. Kevin M Colleaux,B.Sc,MD, FRCSC*

    LEONA WRITES

    Please tell me why light [ any light] is so difficult to deal with . Is it simply because my pupils don`t close fast enough ? THANK YOU Leona Saunders

    • Dear Leona,

      Sorry I missed this…

      It’s hard to know why you have such difficult with light. Possibilities include the pupils, recent surgery (i.e. still some inflammation from healing), significantly more light now entering the eye and shining on a “less than optimal functioning retina.”

      Is one eye more affected than the other?

      Randy

  11. Leona Saunders says:

    Hello Dr. Wong

    The answer to your question regarding light bothering my eyes is that my better eye is affected more.
    I was at my specialist today and he said there was still swelling and is not sure what to do now.There are cyst forming on the left eye now,same as the right eye has had for some time .I am wondering what your thoughts are on this and what Dr. Colleaux said on the previous paragraph from me dated Dec 13th.

    Your Leona Saunders

  12. Levina says:

    I am stargardt patient too..is really a good news for me because it mean a hope. I was seeing a specialist two months ago and he told me no one investigate on this untreatable disease but this article shows me an evidence that what the doctor said is totally WRONG..Really thank you…

  13. Levina says:

    really happy to hear that. Do you have any suggestions on how to take care of my eyes in order to avoid decrease on my vision..

  14. ilka asparuxova says:

    Hi Dr. Wong
    I am from Bulgaria in Europe.
    I have two sons.
    found Stargard disease of the eyes.
    at large is highly developed disease and 29 years old and one 24.
    I want to ask you what can be done because the disease is incurable,
    In our country do not even know much about the disease is rare for our region. I learned about the transplantation of stem cells.
    We appeal to you for advice and direction.
    I would be very grateful if you answer me.
    I wish you health!
    With greeting Ilka!

  15. Alexis Eddington says:

    Dr. Wong,

    I am 21 years old and have had Stargardt disease since I was 6 years old. I am seeking more information on stem cell therapy for my disease. What are your suggestions?

    Thank you,
    Lexie

    • Dear Lexie,

      My suggestion is to pay close attention to the early stem cell research and results of the early trials by Advanced Cell Technology, Inc. Wait until there is some good hard data the proves this is a viable therapy.

      There is too much emotion right now in stem cell research or otherwise. It’s a hot topic.

      My guess is that this will take several more years before we have concrete answers. You have age on your side!!!

      Randy

  16. Christina says:

    Dr. Randall,

    My husband to be is a health 38 year old with Stargardts and Glaucoma. He was just diagnosed 3 years ago. He was told that he would be legally blind by the age of 50. It seems to me that there is now hope that he may not loose his vision.
    Do you know how we can get into a clinical trial? We live in Florida but we are willing to travel.

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