Embryonic stem cells to cure eye disease? - November 19, 2009
Human 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).
Comments
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.
Posted by: Marcie | November 23, 2009 02:32 PM
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 .
Posted by: ali | December 4, 2009 03:21 PM
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.
Posted by: Carl | December 11, 2009 03:56 PM
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
Posted by: Amod | January 8, 2010 09:42 PM