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Embryonic stem cells to cure eye disease?

6701730f1.jpgHuman embryonic stem cells could be one step closer to the clinic. Santa Monica, California-based Advanced Cell Technology (ACT) announced today that it has applied to US regulators to launch a new clinical trial aimed at reversing vision loss with retinal cells recreated from embryonic stem (ES) cells.

The company plans to test the stem cell-derived retinal cells in 12 patients suffering from Stargardt’s disease, a form of inherited juvenile macular degeneration that affects around one in 10,000 children.

ACT researchers previously showed that ES cells could give rise to retinal pigment epithelium cells, the photoreceptors that go awry in the disease. They then demonstrated that the cells could restore vision in a rat model of retinal disease. And in September, the researchers reported that the cells were long-lasting and safe in a mouse model of Stargardt’s.

“Our research clearly shows that stem cell-derived retinal cells can rescue visual function in animals that otherwise would have gone blind,” said Robert Lanza, ACT’s chief scientific officer, in a statement. “We are hopeful that the cells will be similarly efficacious in patients.”

ACT’s investigational new drug (IND) application is only the second filing with the US Food and Drug Administration for a therapy involving human ES cells. The first company out of the gate, Menlo Park, California-based Geron Corp., had its stem-cell derived therapy to treat spinal cord injury patients approved last January. But the FDA put a hold on the trial before a single patient had been injected with the cells, citing safety concerns. Geron now says it plans to restart the trial in the second half of next year.

For more on why stem cell-derived transplants could work to delay or prevent blindness, see the June 2009 news feature from the sadly now-defunct Nature Reports Stem Cells.

Image: The left eye of a Stargardt’s patient from Özdek et al., Eye 19, 1222–1225 (2005).


  1. Report this comment

    Marcie said:

    About 9 years ago my husband was diagnosed with Stargardt’s. At the time he was told that the disease is rare enough that there would likely never be treatment, would never be research dollars put toward it, and the best he could hope for was a treatment being found accidentally while researching a more common retinal disorder. Knowing the advances being made over the last several years we have not given up hope, regardless of that very grim assessment of the chances for treatment given by his retinal specialist.

    This study makes us even more hopeful than we’ve been through the years that someday soon he’ll regain some of his vision. Let us hope that the trials go well, and this opens the door for more treatments in the future.

  2. Report this comment

    ali said:

    i am a patiten of stargared’s I am realy happy when i heard that now there is hope to treat the disese i am v happy may be i will be normal person again .please contact me if any more advance information available .

  3. Report this comment

    Carl said:

    The very best of luck to the researchers.

    I have had Stragardt’s for a number of years now and have undergone surgery in Russia but to no avail.

    I am 45 years olfd and occasionally still get frustrated with the limititations of having poor eyesight (having had excellent vision up to 20 yeras old).

    This project must go ahead if progress is to be made and for many people to be relieved of their suffering.

  4. Report this comment

    Amod said:

    I am 32 and have had this since I was 7yrs old. I dont remember ever that I was able to see clearly. Even my dreams are fuzzy and my eyes feel weak with each passing day. I have met so many doctors around the world, some motivating me and some driving me to mad frustration & I had given up hope of ever getting better. Guess it is bad karma!!….but I must admit that this is the best news i have heard in the last 20 years. I hope that this is true and really will be praying it works…Atleast if it gives me the chance to see my child grow, i would be the happiest man on the planet. God willing this will be good

  5. Report this comment

    sonya said:

    Do you know if there are any stem cell trials or research for ocular histoplasmosis/macular degeneration?

    either way could you please contact me about what you know?

  6. Report this comment

    Mutuelle said:

    We cross our finger and hope that the cells will be similarly efficacious in patients as it was successful in the test . So that the research could pay off the time and hours spend.

  7. Report this comment

    Jinal parikh said:

    My husband is 30 yr old male and was diagnosed of Stargardts disease 3 years back in both the eyes. i really wish and pray to god that the research comes successful and help to cure my husbands eyes ..thanks to all researchers!!!!!pls make it fast .awaiting your reply

  8. Report this comment

    william blount said:

    It was good reading these developments as well as the positive comments and personal accounts. My son is in his late teens and is progressively loosing his vision. this has place an incredible strain on our family, as recently he was diagnosed with cone dystrophy and while we search for solutions we also search for possible means of his being enrolled as a candidate for clinical studies and treatment. If anyone out there knows or can guide me in obtaining this information, however small, I would be greatly appreciative of this help.

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    dilip sequeira said:

    Thrilled to read that there is some hope in stem cell theraphy. Now i hope & pray that the cells will be similarly efficacious in patients. My fifteen year old son was diagnosed of Stargardts disease last year and it hurts us to see him lose his vision.

  10. Report this comment

    bassim mejbel said:

    I am a 45 y old male ,I was diagnosed with Stargardts disease in both eyes ,I am praying and waiting for any treatment ..

  11. Report this comment

    Rachana said:

    I am having retinal detachment in left eye alone for the past 18 years. Now i am 22 and have cataract,glucomo and astigmatism.Ifs there any treatment for my eye andIf u are conducting any expiriments do let me know please.

  12. Report this comment

    Jessica said:

    After 6 months of offering stem cell therapy in combination with the venous angioplasty liberation procedure, patients of CCSVI Clinic have reported excellent health outcomes. Ms. Kasma Gianopoulos of Athens Greece, who was diagnosed with the Relapsing/Remitting form of MS in 1997 called the combination of treatments a “cure”. “I feel I am completely cured” says Ms. Gianopoulos, “my symptoms have disappeared and I have a recovery of many functions, notably my balance and my muscle strength is all coming (back). Even after six months, I feel like there are good changes happening almost every day. Before, my biggest fear was that the changes wouldn’t (hold). I don’t even worry about having a relapse anymore. I’m looking forward to a normal life with my family. I think I would call that a miracle.”

    Other recent MS patients who have had Autologous Stem Cell Transplantation (ASCT), or stem cell therapy have posted videos and comments on YouTube.

    Dr. Avneesh Gupte, the Neurosurgeon at Noble Hospital performing the procedure has been encouraged by results in Cerebral Palsy patients as well. “We are fortunate to be able to offer the treatment because not every hospital is able to perform these types of transplants. You must have the specialized medical equipment and specially trained doctors and nurses”. With regard to MS patients, “We are cautious, but nevertheless excited by what patients are telling us. Suffice to say that the few patients who have had the therapy through us are noticing recovery of neuro deficits beyond what the venous angioplasty only should account for”.

    Dr. Unmesh of Noble continues: “These are early days and certainly all evidence that the combination of liberation and stem cell therapies working together at this point is anecdotal. However I am not aware of other medical facilities in the world that offer the synthesis of both to MS patients on an approved basis and it is indeed a rare opportunity for MS patients to take advantage of a treatment that is quite possibly unique in the world”.

    Autologous stem cell transplantation is a procedure by which blood-forming stem cells are removed, and later injected back into the patient. All stem cells are taken from the patient themselves and cultured for later injection. In the case of a bone marrow transplant, the HSC are typically removed from the Pelvis through a large needle that can reach into the bone. The technique is referred to as a bone marrow harvest and is performed under a general anesthesia. The incidence of patients experiencing rejection is rare due to the donor and recipient being the same individual.This remains the only approved method of the SCT therapy.

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