Stem cell trial for eye disease receives FDA approval

The second clinical trial involving stem cells from human embryonic cells has received the go-ahead. The news came from Advanced Cell Technology Inc. this Monday. The company announced that the U.S. Food and Drug Administration has cleared the way for its Phase I/II trial of retinal cells for patients with Stargardt’s macular dystrophy, a childhood version of macular degeneration.

Up to 12 patients will be enrolled at several sites across the country, including the Casey Eye Institute in Portland, Ore., the University of Massachusetts Memorial Medical Center in Worcester, and UMDNJ – New Jersey Medical School in Newark.

Stargardt’s macular dystrophy is a childhood disease that affects about one in 10,000. Initially patients begin to lose their central vision between the ages of 6 and 20. As SMD progresses, things may look blurry and distorted and patients may have trouble adjusting to low light. About half of victims are legally blind by age 50. There is no cure.

This new stem cell technology would treat these patients by giving them new retinal pigment epithelium cells to replace the ones that are lost to the disease. The therapy has been successful in lab animals. The cells were grown from a line of human embryonic stem cell that was made by extracting a single blastomere cell from an eight-cell embryo. The technique is commonly used in pre-implantation genetic diagnosis and does not require the destruction of an embryo. Last month, a patient in Atlanta became the first patient to be treated in an FDA-approved clinical trial involving a therapy derived from human embryonic stem cells.

Robert Lanza, ACT’s chief scientific officer, said that these RPE cells have undergone strict tests to meet safety standards. The protocol involves injecting 50,000 to 100,000 RPE cells into the vitreous cavity of the eye. If successful, the treatment could be extended to address other degenerative eye diseases as well, including retinitis pigmentosa and macular degeneration. Lanza said, “We’re starting out with a safety study in those with advanced disease…But hopefully if we get in earlier in the progression of the disease we might see greater impact on visual improvement. What this approval shows is that the readiness of the FDA to work with researchers to move exciting new stem cell based therapies out of the lab and into the clinic.”

Dr. Ananya Mandal

Written by

Dr. Ananya Mandal

Dr. Ananya Mandal is a doctor by profession, lecturer by vocation and a medical writer by passion. She specialized in Clinical Pharmacology after her bachelor's (MBBS). For her, health communication is not just writing complicated reviews for professionals but making medical knowledge understandable and available to the general public as well.


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  1. sonny sonny United States says:

    Great news, hopefully they will be able to help my wife soon as she is going down hill fast. please pray for her,( Kelly)

    god bless

  2. Ryan Taylor Ryan Taylor United States says:

    I honestly don't know what to say other thank you for a glimmer of hope.  I have a beautiful, determined and wonderful 17 year old daughter that has been progressively losing her eyesight to stargardt's since she was 7.  She is now legally blind and what eyesight she has left is measured in a matter of years.  Speaking for my wife as well I know nothing would mean more to us than for our daughter to be able to see again.  Despite this disease she is still fighting for a full life and looking forward to attending college next year in order to become a pharmacist.  Hopefully this research will one day prove effective in giving back these children and our daughter the gift of sight.

The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
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