FDA clears second clinical trial involving human embryonic stem cells


Another therapy derived from human embryonic stem cells is headed for clinical trials.

Advanced Cell Technology Inc. said Monday that the 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.

Here’s a description of Stargardt’s macular dystrophy from a previous Booster Shots post: “The disease is a childhood version of macular degeneration and affects about one in 10,000 kids. Patients typically 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.”

Advanced Cell Technology hopes to treat these patients by giving them new retinal pigment epithelium cells to replace the ones that are lost to the disease. The therapy has restored vision in rats and mice, according to published studies. The Phase I/II trial is designed to test the safety and tolerability of the RPE cells in humans.


The RPE cells were grown from an unusual line human embryonic stem cells 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. That clinical trial is testing cells made by Geron Corp. to treat people with spinal cord injuries.

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