ACT wins FDA’s approval to test stem cell therapy

Posted AtBoston.com

Advanced Cell Technology, a stem cell company with a research laboratory in Marlborough, has received federal approval to test a human embryonic stem cell treatment on patients with a rare disease that causes blindness.

ACT has withstood financial struggles over the past few years, and its stock price closed on Friday at under 5 cents a share. But the small clinical trial that it has gained approval for - to treat 12 patients with Stargardt's macular dystrophy - will make it the second company in the United States to test a human embryonic stem cell treatment.

"It's been a long road. . . . We've had our telephones turned off a few times," said Dr. Robert Lanza, chief scientific officer for ACT. "We've hung in there."

ACT filed an investigational new drug application with the Food and Drug Administration a year ago to use retinal cells derived from human embryonic stem cells to treat patients with Stargardt's, a rare genetic disease that affects about 30,000 people in the United States. The disease causes progressive vision loss, starting in patients between 10 and 20 years old, and results in blindness.

The study will be conducted at a number of sites, including the University of Massachusetts Memorial Medical Center in Worcester, where it will be overseen by Dr. Shalesh Kaushal, chairman of the department of ophthalmology at UMass Medical School.

Kaushal said the current standard of care in the disease is simply to follow patients, since there are not drugs or treatments to reverse the vision loss.

"It's a largely unmet medical need, in terms of therapeutics," Kaushal said, adding that he will be careful to caution patients the initial trial is preliminary and primarily a test of the safety of the treatment.

In the disease, Kaushal said, retinal cells that are the "photographic film of the eye" degenerate, and photoreceptors that sense light also die.

In the trial, retinal cells will be injected into patients' eyes in varying doses, and patients will be closely followed to gauge the safety and efficacy of the treatment. The disease has certain characteristics in common with a form of age-related macular degeneration, a disease that also causes vision loss.

Lanza said the company will soon apply for approval to test the therapy in patients with age-related macular degeneration.

Nov 23, 2010