Stem Cells Get FDA Nod for Stargardt's Disease

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Stem Cells Get FDA Nod for Stargardt's Disease

Advanced Cell Technology, Inc. gets FDA approval to use stem cells as a possible treatment for Stargardt's disease, a inherited juvenile type of macular degeneration.

Advanced Cell Technology, Inc., received FDA approval for starting Phase I/II clinical trial to use stem cells for the treatment of Stargardt’s disease.  Stargardt’s disease is an inherited type of macular degeneration affecting individuals at age 10-20.  There is no cure.

Similar to other macular diseases, the photoreceptors, and the layer of cells just beneath, call the retinal pigment epithelium (RPE), become destroyed due to a genetic defect.  Due to the macular involvement, central vision is lost.

Stargardt’s disease is the a common form of “macular degeneration” that afflicts the young.  It is estimated to affect about 30,000 people worldwide.  It shares in common with ARMD (age related macular degeneration) the loss of photoreceptors secondary to RPE damage and degeneration.

Advanced Cell Technology (ACT) claims the ability to generate healthy RPE cells from human embryonic stem cells.  The idea is to replace the genetically diseased RPE cells with healthy replacements.  In theory, the healthy RPE cells should prevent loss of the photoreceptors, thereby preserving vision.

The phase I/II study will involve 12 patients enrolled into several centers across the United States.  The initial experiments will determine if the RPE cells are indeed safe and if they can be tolerated by the human recipients (i.e., does the body reject the new RPE cells?).

What Does This Mean?  Stargardt’s disease has no cure, and therefore, are great subjects to consider for this possible treatment.  In a way, there is nothing to lose.  Stargardt’s patients also have or had vision, that is, they have experience with vision.  We know that these patients have fully developed visual pathways.

While this group is very small in comparison to patients with macular degeneration, the significance this holds for a potential, effective treatment can only be left to our imagination.  Even a small success in this trial is exciting.

The news of this trial is exciting, but remember further testing (clinical trials III and IV) need to be completed.  Also, the techniques for introducing the cells safely underneath the retina need to be accomplished, too.

More later…I hope.

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Comments
  • elizabeth machado December 9, 2010 at 1:13 pm

    Randy,Is there any trial experiment with estem cells for diabetic retinopaty and DME ?
    Elizabeth
    HAPPY HOLLIDAYS

  • Irv Arons December 9, 2010 at 2:01 pm

    Elizabeth, the short answer is yes, the longer answer is please take a look at my Primer on the Use of Stem Cells in Ophthalmology (link: http://tinyurl.com/ophthstemcells)

    Irv Arons

  • Christine Yeung December 9, 2010 at 5:25 pm

    Hi Dr. Wong, is there any risk with use of embryonic stem cells? Would use of adult stem cells prove to be safer? Would adult stem cells provide the same potential benefit?
    – Christine

    • Randall V. Wong, M.D. December 11, 2010 at 5:39 pm

      Dear Christine,

      Not sure what you mean by risk? To my knowledge, while there are many differences between the two types, embryonic stem cells replicate easier, yet adult cells specialize further.

      The hope of stem cell research is to provide an endless supply of cells that may develop into specific types of cells and function.

      Randy

  • Sharon Bassham December 15, 2010 at 11:05 pm

    Dear Dr. Wong,
    Our 12 year old son was diagnosed with Stargardt’s Disease in February 2010. We are anxiously awaiting the outcome of the clinical trials. Eventhough the diagnosis of Stargardt’s has been extremely difficult for us, we feel fortunate that researchers like yourself are working diligently to find a treatment for this disease. Do you have any idea when the clincal trials III and IV will be conducted? Also, any idea when news will be available on trials I and II? Our son’s vision is 20/120 currently. We feel like we are racing against the clock to find treatment before his vision loss increases. Thank you and May God Bless you and the work you are doing ~ Sharon Bassham

    • Randall V. Wong, M.D. December 15, 2010 at 11:54 pm

      Dear Sharon,

      It must be terrifying for you to know all about this, but have little ability to help.

      The process is most likely going to take several years. I don’t know the specifics of the trials, but we’ll get some news periodically. My best guess is that the initial studies may take a year or two. Patients need to be recruited, the operation performed, then study the results for a moderate period of time.

      Rest assured that this technology is promising for you son during his lifetime, but not in the immediate future.

      Also, keep in mind that your son is not experiencing the amount of loss that you are imagining for him! He has never had vision as good, nor as long, as you, thus his frame of reference is much different.

      I wish I could have more concrete news for you.

      Stay in touch.

      Randy

  • Sharon Bassham December 16, 2010 at 9:39 pm

    Dr. Wong,
    We feel blessed that there is a good possibility that a treatment will be perfected in the near future.
    I will be checking this site frequently for new updates! Thank you and I will stay in touch!
    Warm regards,
    Sharon Bassham

  • mary mayfield December 20, 2010 at 10:17 pm

    My son was diagnosed at 18 on his birthay he went through several specialists through the years who neglected him and said it was psychological and would pass, Please understand he went through his entire school education as a normal child but couldnt see at distance detail at all he must stand in front of the computer very closely to read cannot read the road signs at all he completed his emt school and graduated and wants to become a doctor He says he sees and no blindspot or phantom vision or come an go effect I read about on the internet it is scary he says his vision he learned to see like this and it hasnt changed in 5 or 6 years it is the same he had it from birth we believe because he alway stood close to the tv from 2 yrs old and detected in kindergarten.. Any information on the stages and if it is settled here and remain this way or can it get better …………… The doctor who diagnosed him in May of 2010 said it is here and will not get better or worse it is his final stage and not oto worry about decrease and internet forums and information and what about the gene therapy is there a cure forhim

    • Randall V. Wong, M.D. December 21, 2010 at 9:43 am

      Dear Mary,

      Your son sounds amazing…whether or not he has vision problems! I wish him all the best.

      I am a bit confused as to how Stargardt’s was actually diagnosed. Regardless if he were “neglected,” the outcome is the same, but it is hard to realize there was no hint of it over the years.

      For now, stem cell therapy yields the best hope, but it is years away and I wonder how bad your son really is doing?

      Please let me know and we can discuss further.

      Randy

  • Herb Smith Germantown,MD December 26, 2010 at 11:30 am

    I have had 3 surgeries to my left eye 2004,05 and 06 to close a macular hole.Two face down gas bubble injections and one cateract removale and lens replacement.My vision has gone from 20/80 to 20/300 since then .I need vision of 20/60 or better in both eyes to be a CDL tractor trailer driver.In 2009 the doctor did a retina scan and showed me on a picture that the hole is closed but ther is thinning of the macular and there may be possible nerve damage.He said there is nothing he can do just i must protect my right eye which is 20/20.i asked him about stem cells and he said that may be a posible cure but that treatment is not ready yet.I am following the clinical trails and praying for succes.I found yor site and would like your input on my condition.Thank You and may God keep blessing you doc.

    • Randall V. Wong, M.D. December 29, 2010 at 1:00 pm

      Dear Herb,

      I agree that stem cells might be a viable treatment…when it is ready for routine use. I think that day is many years away.

      I am happy that you remain functional despite the repeated surgeries to the right eye.

      Happy New Year!

      Randy

  • Sharon Bassham December 27, 2010 at 8:34 pm

    Dr. Wong,

    Are there any vitamins or supplements that have been proven will help nourish the macula and help maintain the stability of cones and rods? Is Vitamin A contraindicated in Stargardts?

    Thank you,
    Sharon Bassham

    • Randall V. Wong, M.D. December 29, 2010 at 1:09 pm

      Dear Sharon,

      There are NOT any supplements PROVEN to nourish the macula. I am not aware that vitamin A is contraindicated in Stargardt’s.

      Randy

  • Randall V. Wong, M.D. December 29, 2010 at 11:19 am

    Dear Sharon,

    Stay in touch and keep your fingers crossed!

    Happy New Year.

    Randy

  • Michelle Chadwick January 29, 2011 at 4:19 pm

    Dr. Wong,

    My daughter Emily was diagnosed back in 1995 with Stargardt’s Disease. She was in 2nd grade; her vision went from 20/20 to 20/200 in one year. She is now a graduate of BU and is a certified Athletic Trainer. She is an amazing young woman and has accomplished so much in her life. She has not allowed herself to be held back and told that she could not do something.
    My question to you is how does she become part of the trail? Her case has been documented since 1995 when we first learned of her problem. She has been seen by Dr. Eliot L. Berson, MD at Mass. Eye & Ear in Boston every three years.
    Would love to get her into these trails.
    Thanks for any help you can offer.
    Sincerely,
    Michelle Chadwick

    • Randall V. Wong, M.D. January 30, 2011 at 11:13 am

      Dear Ms. Chadwick,

      I would keep an eye open for news about Advanced Cell Technology. They are the company that received the recent FDA approval for the stem cell research. The chief medical officer is Dr. Robert Lanza. I’d focus my efforts to see if the initial trial is open or closed. The FDA approval mentioned 12 patients. There will be specific criteria for becoming a patient.

      Having said all this, I’d be patient as your daughter seems to be doing so well. The initial trials will simply see if this is an viable technique to help previously sighted people. There is no guarantee as to the safety, nor efficacy, of this new research. We’ll know if a few years if this is a viable area of research.

      You are a great mom.

      Randy

  • Lyn Voss March 23, 2011 at 2:44 am

    Dr. Wong,

    Our son is now 15yrs old he was diagnosed with Stargardt’s in June of 2009, when he was 13yrs old. It was really hard for all of us. I remember the day that the vision specialist told me that he would never be able to drive. That is one of the most important thing to a teenage boy. He hadn’t heard this yet. I sat and cried for hours, blaming my self. Since he had to have gotten one gene from me and one from his father. Neither one of us know of anyone in our families. The craziest thing that we have found, is that Stargardt’s effects 1 out of 20,000 children. Well the school district that we are in has 12,000 students and there are 3 children in the school district that have Stargardt’s. We have seen doctors at the University of Utah Moran Eye Institute. They drew blood and did genetic testing and are using his blood for research. I think that we really need to get this disease out in the public more so then, maybe we can get more help trying to find a treatment or better yet a cure. Sometimes my son gets up set because it isn’t a very well know disease and think that people care more about finding ways to cure or treat other diseases. Do you have any suggestions on how we can get this disease recognized? We live in the small state of Wyoming and there isn’t very much here. Anything would help!!!

    Thank you,
    Lyn

    • Randall V. Wong, M.D. March 23, 2011 at 7:48 pm

      Dear Lyn,

      Your story, unfortunately, is not unique. I don’t know why early teens seems to be a popular time for developing visual problems and then eventually diagnosis.

      I am on the “practical” side of medicine, that is, I implement what the researchers and public officials deem appropriate.

      I can tell you that Stargardt’s has become more popular due to the potential benefits of stem cell research. While I don’t know when this will become a reality for anyone let alone Stargardt’s, the good news is that your son is young and has every hope of getting some help in the future vs. an older person who is also blind from retinal disease.

      I will keep you in mind. I would also recommend, if you haven’t already, subscribing to the blog…that way you won’t miss any news.

      Best of luck. Parenting should be easier.

      Randy

  • Carl Kephart April 14, 2011 at 10:25 pm

    Thanks Dr Wong for keep us up to date on the stem cell trials. This is a great hope builder for those of us who are personally affected by Stargardt’s.

    I realize that this article is about stem cells as a treatment which is not yet available to the public in the U.S. But could you mention your experience with other options that patients can do today while they await research results? Options such as microcurrent therapy, omega 3 oils, the many other supplements readily available, and tozal which was recently switched over to prescription multivitamin like prenatals.

    • Randall V. Wong, M.D. April 18, 2011 at 10:30 pm

      Dear Carl,

      This is a tough answer. You are asking if I have an opinion about unproven treatments. I don’t prescribe any of them and, thus, have no experience with them. There are many “treatments” out there, but so far none has really been proven to be amazing. Early data make Omega 3 research quite compelling, but supplement studies are really tough to interpret because they are so nonstandard.

      This was a long way to say I don’t use them, but can’t discount them either.

      Randy

  • Abby Chandler April 19, 2011 at 12:17 pm

    This blog is wonderful! I am very impressed with your quickness to answer and the answers you are giving! Thank you for taking the time to do this for families like mine.

    My story is a bit different from the others as it is my mother who has stargardt’s. She was diagnosed in 2003 and is no longer able to drive at all. I am currently pregnant with twins and I am hoping to help her with her grandchildrens’ stem cells. I am having trouble finding any solid information. I was told that she has a 25% chance of matching my babies’ stem cells and that a test can be done with a mouth swap to determine compatibility.

    Who do I get to do the test? How long would it take to get results?
    Is storing the stem cells worth the thousands of dollars to save them (if she is a match) for future use?

    If I knew that she could see again price would not be a question, it all just seems so up in the air. Your opinion would be very helpful.

    Again thank you for your time,
    Abby

    • Randall V. Wong, M.D. April 20, 2011 at 9:59 am

      Dear Abby,

      I would first seek the aid of a hematologist/oncologist for matching, but I don’t think it’s worth the time, right now. Although there are a few studies ongoing, the technology is far from becoming useful.

      You are very kind.

      Randy

  • Eric September 20, 2011 at 5:34 pm

    Dear Dr. Wong,

    Where are the stem cells obtained? My wife suffers from Stargardt’s and if we have a baby, is there anything we should do to preserve any specific cells from the embryo? I heard people are freezing umbilical cords. Thank you.

    • Randall V. Wong, M.D. September 21, 2011 at 11:54 am

      Dear Eric,

      And don’t forget about bone marrow!

      Remember, the people undergoing the Stargardt’s trial didn’t have a frozen umbilical cord as a source, that is, I’m not sure you need to worry about that!

      r

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