Stem Cells Reported to Improve Vision

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January 19, 2012
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February 8, 2012
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Stem Cells Reported to Improve Vision

UCLA researchers announce improvement in 2 patients receiving stem cell transplants for retinal eye diseases; macular degeneration and Stargardt's Disease.

Two patients receiving embryonic stem cells for their blinding retinal diseases reported improvement in their vision. The results were released just last week online in the medical journal Lancet.

Each patient received retinal epithelial pigment cells (aka RPE cells) grown from human embryonic stem cells. RPE cells are the faulty cells in macular diseases such as ARMD and Stargardt’s Disease. In healthy patients, these cells are normal and function appropriately.

Dysfunctional, sick or degenerated retinal epithelial cells lead to progressive loss of vision. As the healthy RPE cells begin to dwindle, so does the vision. The RPE cells are a key component to translating light to vision.

Researchers have been hopeful to replace sick RPE cells with the healthy replacements derived from the stem cells.

Hence, the excitement. Two individuals, one with dry ARMD and the other with Stargardt’s underwent RPE cell transplantation and the reports at 4 months after the operation are encouraging. Both noted improvement and, according to the report, no complications occurred either to the eye or to the patient.

Both patients underwent surgery at UCLA. The research was funded by Advanced Cell Technology, the same company conducting stem cell research on patients with Stargardt’s Disease.

What Does This Mean?

At most, this is encouraging news.  This does NOT mean that stem cell transplantation works.

There are lots of flaws; only 2 patients involved, inability to objectively measure improvement in vision, funding company (Advanced Cell Technology) is involved and adds bias to the study results, etc.

On the other hand, I am very excited that the research seems to be moving forward. Yes, the results are tainted, but it does demonstrate several “successes” in solving some of the technical challenges of stem cell research;

  • This is pioneering work. Regardless of visual outcomes, researchers have designed techniques to deliver the RPE cells to the correct anatomic location. That is, retinal surgeons have figured out how to transplant the cells effectively.
  • The cells may indeed remain “alive” when transplanted. How long? We don’t know, but it seems to be at least 4 months!
  • This is great news in the face of recent failures of stem cells used in spinal cord injury.
  • There were apparently no complications.

This certainly is enlightening and exciting.  With time, we will need more extensive research and objective data.  This is no different than a new “drug” fighting for FDA approval.

There is still many questions to answer the ultimate questions;  Is the technique safe?  Is the technique effective?

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Comments
  • Sid January 31, 2012 at 3:27 pm

    Hi Doc,
    Interesting development indeed.
    I have this doubt about stem cell therapy …Stem cells can potentially form tumors when transplanted into patient’s body…isn’t this a very serious safety issue for the therapy…especially when used for treatment of non-life threatening conditions (like retinal degeneration)? I mean there is a possibility that the patient will get cancer or am I missing something?

    • Randall V. Wong, M.D. February 1, 2012 at 11:54 am

      Sid,

      In theory, a tumor could result from unrestricted growth of stem cells. I don’t know if this is just a perceived risk or not.

      Randy

  • kitty January 31, 2012 at 6:34 pm

    Delighted that you have addressed this subject so quickly.

  • Malvas75 January 31, 2012 at 7:28 pm

    Come on 🙂
    Read here: (The CEO of the company)
    http://www.thechairmansblog.com/gary-rabin/

    Advanced Cell Technology is the future of the regenerative medicine!
    Read for believe!! 🙂

    • Randall V. Wong, M.D. February 1, 2012 at 11:55 am

      Dear Malvas75,

      Thanks so much for your comment and contribution. Great link.

      r

  • Bill Stockmun January 31, 2012 at 7:48 pm

    Thank you for writing about this exciting news. I have been following this trial for quite a while. The leading investigative scientist, Dr. Schwartz made an interesting presentation and a med conf in Mesa AZ this past fall where in he stated that he had no financial connection to Advanced Cell technology and went on to explain in detail his work. Here is a link to that video if you wish http://thesciencenetwork.org/programs/stem-cell-meeting-on-the-mesa-2011/panel-4-steven-schwartz.
    In addition the Lancet published paper does some good work in illuminating the study so far. The announcements in the past week that the prestigious Wills Eye Hospital in PA and the Aberdeen Royal Infirmary in Scotland have joined UCLA’s Jules Stein Clinic and Moorfields Eye Hosptial in London in enrolling patients, speaks well to the quality of the surgeons and facilities that will be tracking the 36 patients in this FDA phase 1/2 trial.
    Having had the great personal pleasure of meeting Sue Freedman last week, the first AMD patient injected at UCLA and watching her navigate a room full of people was an eye opener. I would have to say, stay tuned, there will be lots more information coming for both AMD and SMD as the year progresses.

    • Randall V. Wong, M.D. February 1, 2012 at 12:47 pm

      Dear Bill,

      Thank you very much for your knowledge and insight! Thanks also for sharing the link.

      Randy

  • Pol January 31, 2012 at 10:13 pm

    Sid,
    Animal studies that preceded this clinical trial on humans, indicated NO teratomas. At six months, none in humans either. Dr. Lanza has stated” However, the nature of the retina may limit how much these trials can teach researchers about stem cell medicine in general. “The eye is considered an immune-privileged site, which means the chances of the cells being rejected are considerably reduced,” Lanza said. Additionally, thanks to the nature of the RPE, the chances that the cells—either iPSCs or hESCs—will grow out of control or cause tumors may be considerably less in the retina than other parts of the body, said Takahashi, who has yet to see any signs of cancer in her mouse experiments. –Hong Kong Stem Cell Centre (12/1/11)

  • ACTC February 1, 2012 at 2:27 pm

    Dr Wong

    When you used the word “TAINTED”, meaning the preliminary result was souped up and reported to the Lancet. Do you think the Lancet is that stupid and ignorant? To publish that kind of “Tainted” report.
    I think you need to do more on your research to be familliar with the technique that ACT uses in this clinical trial.
    Good luck

    • Randall V. Wong, M.D. February 2, 2012 at 8:39 am

      Dear ACTC,

      I used the word to emphasize that in no way is this to be considered a “study,” as this is far from an objective assessment. Perhaps I should have chosen a different word, such as “bias.” I am not critical of the methodology, research or science of ACT.

      As I stated, I am very excited about the “news.” In a time where stem cell industry needs a boost, they got it! To me this is great news.

      Randy

  • GumbyJ February 1, 2012 at 9:53 pm

    There is some confusion about this therapy and stem cell therapies in general. The cells injected are no longer stem cells when injected. Before injection stem cells must be differentiated into another cell type, otherwise you will indeed grow tumors or the wrong “thing”. As noted in the article, in this case they are RPE cells at the time of injection. Also, ACT has a process where the cells used for injection undergo an extensive screening process in which a single undifferentiated cell can be detected in a million.

    I am confused by the comment “Yes, the results are tainted”. I understand the Lancet to be a highly regarded journal. I am not in the medical field but after some researching most sites agree with Wikipedia’s definition of “The Lancet is a weekly peer-reviewed general medical journal. It is one of the world’s best known, oldest, and most respected general medical journals.” Are the Lancet and the Dr’s listed in the article, some of which are not affiliated with ACT, including Dr. Schwartz somehow in cahoots and the data can’t be trusted?

    • Randall V. Wong, M.D. February 2, 2012 at 8:45 am

      Dear GumbyJ,

      Not the first to note my choice of words, re: tainted. I was simply highlighting that this, in no way, should be considered a study. Because of all the bias, there is no way one can make any conclusions whatsoever….but it makes for a very compelling story. That’s as much as meant by choosing the word.

      With regard to publication in the Lancet. I don’t know any of the Drs., including Schwartz. I have reason to question their methods or science. I did not intend to question their research. As far as I know, they are to be trusted.

      Again, publishing “results” (and I use that word with hesitation) in this manner piques curiosity. No scientific conclusions can be made due to the “bias” (study number way too small, subjective claims of vision in both eyes, yet only one was treated, ACT performs research and conducted the study, etc.).

      Last, everyone should be applauded for their position as pioneers. It is truly an exciting time not only for patients with macular disease but for those of us that may actually see this as a viable treatment….and before we retire!

      Thanks for input about the cell differentiation and screening.

      Randy

  • pacman February 2, 2012 at 3:16 am

    Dr Randall, good site and glad you posted on the hESC trials at Jules Stein Eye Institute. The Lancet article outlined the 4 month outcomes of the trial showing attachment and safety of using these terminated RPE cells. These cells are terminated, meaning that they cannot revert back to stem cells, thus unable to form teratoma tumours. The camera used in the trials is able to determine 1 in 2 million cells, the FDA are satisfied no teratoma have formed and have approved the resumption of the trial.
    We are all very excited that those family and friends with Stargardts and AMD, now have the hope of being able to halt the progression of the disease and to gain improved vision. It will be wonderful eye surgeons like yourself who will be undertaking these operations in the future. I hope that you will be in the forefront of the next generation of eye treatment this trial heralds and will be able to post of your successes encouraging others. Thanks again for your web site.

    • Randall V. Wong, M.D. February 2, 2012 at 8:47 am

      Dear Pacman,

      Thank you so much for sharing your kind thoughts (I needed a few!).

      Thank you also for explaining the “safe” selection of the cells.

      Randy

  • ACTC February 2, 2012 at 10:02 am

    Thanks for posting and replying to my comment.
    You are right about only two persons have been treated so far and they have regained some vision improvement to get any definitive conclusive results. A larger pool of patients will be required.
    You are also right “that in no way is this to be considered a study” because the study had been done in crossed species experiments.
    This is the clinical trials that were designed and approved by the US FDA. You can check clinicaltrials.gov and search for MA09. In addition, the UK had just treated 1 male SMD patient funded by ACT. JSI also had just treated one more SMD patient last week. In Scotland, Aberdeen Royal Infirmary was announced as additional site for Phase 1/2 clinical trial using ACT’s hESC-Derived RPE Cells for Macular Degeneration. In the US, we have JSI and Will Eye Institute as the first 2 trial sites. More to come, I guess….
    As the clinical trials progressing, more and more patients with improved their central vision will definitely prove to you that the “STUDY” is not “FLAW” and the results are not “BIAS or TAINTED”.

  • Joseph Exline February 3, 2012 at 1:43 am

    I am very interested in your willingness to comment inaccurately on an ongoing clinical trial led by a team that has not only acquired the approval of the FDA,but has some of the finest facilities in the world participating in their clinical trials.There is plenty you could have researched in just a few hours that would have allowed you to be true to those that read this spot of yours.
    Biased ,Tainted,Really?The Lancet, Jules Stein Eye Institute, Dr.Schwartz, the DSMB, the FDA, they are all willingly compromising their credibility for what was it?
    The good news is you have a bit of time to research thoroughly before you have to report on the next study,I mean clinical trial results pertaining to the injection of embryonic stem cells,I mean retinal epithelial pigmented cells.
    Also,this is far from being similar to a new drug fighting for approval.This is the first ever ongoing clinical trial utilizing human embryonic stem cells . ACTC and the FDA are paving a whole new pathway for future stemcell related trials and ventures.Undoubtedly ACTC, because they are blazing the trail so to speak,will progress at a much slower pace in these trials than will those that follow. They have to be flawless the first time.The FDA has to be flawless the first time.They have to be deliberate,cautious,precise,unhurried,and flawless.They are making history.

    “In theory, a tumor could result from unrestricted growth of stem cells. I don’t know if this is just a perceived risk or not.”
    Someone has already addressed this statement with you,I just wanted to show you what caused me to fall out of my chair.

    • Randall V. Wong, M.D. February 7, 2012 at 9:18 am

      Dear Joseph,

      Thank you for taking the time to share your opinion.

      Perhaps I wasn’t clear enough in that I applaud this work. It is to be respected.

      At the same time, as with everything, it must face up to a process and that process is just beginning.

      Thanks again for contributing.

      Randy

  • Catch February 9, 2012 at 2:43 am

    Thank you Dr Wong for writing about this FDA approved human trial with RPE cells differentiated from totipotential embryonic stem cells. The comment was made that the group had found a way/procedure to deliver the cells to the correct site. Are there other surgical procedures that go to that specific anatomical site? I wonder if the surgical insult to the site where the RPE cells were not functioning and the delivery of the healthy cells could have initiated healing that also resorbed the new cells? The new cells were not found to have attached but the legally blind woman did have improved vision. The cells might not be the permanent solution but the cells probably were a significant part of the healing/improved vision that resulted. It seems that would be good news for patients who presently have no treatment choices.

    • Randall V. Wong, M.D. February 14, 2012 at 12:11 pm

      Dear Catch,

      The RPE cells normally form a layer of cells underneath the retina. Not sure I understand your question about other surgical procedures. I think simply placing/injecting them underneath the retina is pretty benign and does not seem to initiate a process whereby the new cells are absorbed.

      Nice observations.
      r

  • mike February 13, 2012 at 10:29 am

    I believe ACT spiked their pure differentiate RPE cells with 10% undifferentiated stem cells and did not have any adverse effects in PREclinical studies… (check their slides from meetings/presentations)
    but their assay can find 1 out of 1million cells…

    • Randall V. Wong, M.D. February 14, 2012 at 12:30 pm

      Dear Mike,

      Thank you for highlighting this. I understand the same thing.

      Randy

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