Nature Medicine | Spoonful of Medicine

Results from two patients signals possible efficacy of stem cell-based eye therapy

Geron may have been the first company to launch a US trial with regulatory clearance involving a human embryonic stem cell (hESC)-derived product. But Advanced Cell Technology (ACT) made history itself today, becoming the first biotech to report results about such a product in a major peer-reviewed journal.

In July of last year, ACT started treating subjects with retinal pigment epithelial cells derived from hESCs in each of its two ongoing US trials—one for a rare form of blindness that typically begins in childhood, the other for a common cause of blindness in the elderly. Now, a team led by Robert Lanza, chief scientific officer of the Marlborough, Massachusetts-based company, offers a four-month status report on the first participant in each trial. Writing in The Lancet, the researchers describe how the transplanted retinal cells appear safe and both study subjects experienced some improvement in vision.

“The results are impressive,” Anthony Atala, director of the Wake Forest Institute for Regenerative Medicine in Winston-Salem, North Carolina, wrote in an accompanying commentary. The authors “have realized the potential to use hESCs therapeutically in human beings.”

In a conveniently timed press release, ACT also announced today that the first participant in the company’s European trial was treated on Friday at the Moorfields Eye Hospital in London. With all this forward momentum, the company, which was profiled in a January 2012 news feature in Nature, hopes it can avoid the fate of Geron. In November, the Menlo Park, California-based biotech announced that it was abandoning its hESC work to focus instead on cancer drugs. Earlier this month, the company also said that it had hired a brokerage firm to help find a buyer for its stem cell portfolio.

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