FDA To Allow First Clinical Trial Of Human Stem Cell Therapy 

The Food and Drug Administration has cleared the way for the world’s first study of human embryonic stem cell therapy, Geron Corp said on Friday.

The California biotechnology company plans to start a clinical trial to try to use the stem cells to regrow nerve tissue in patients with acute spinal cord injury.

“This marks the beginning of what is potentially a new chapter in medical therapeutics — one that reaches beyond pills to a new level of healing: the restoration of organ and tissue function achieved by the injection of healthy replacement cells,” Geron Chief Executive Thomas Okarma said in a statement.

Shares of Geron rose nearly 30 percent to $6.75 in premarket electronic trading on Nasdaq.

The FDA rejected his company’s first request to conduct the trial of GRNOPC1, Oligodendroglial Progenitor Cells. It put the trial on hold in May.

“If GRNOPC1 eventually does become the first human Embryonic Stem Cell drug to receive an approved Biologic License Application it could be as important to drug therapy as the discovery of Salvarsan or penicillin,” drug analysts Stephen Brozak and Daniel Mallin of WBB Securities LLC said in a statement.

Salvarssan was the first modern drug, used to treat syphilis early in the last century.

Joel Sendek of Lazard Capital Markets was cautious, saying he was recommending a hold in Geron shares. “Cellular-based therapies carry substantially more regulatory risk than do traditional small-molecule drugs or protein therapeutics,” he said.


Former President George W. Bush had been at odds with Congress, researchers and advocates for years over the issue and by executive order restricted federal funding of work involving human embryonic stem cells.

President Barack Obama, who succeeded Bush on Tuesday, had been widely expected to rescind that directive. Although the FDA says it does not make decisions based on politics, the company made the decision public just days after Obama was sworn into office.

Stem cells are the body’s master cells, giving rise to all the tissues, organs and blood. Embryonic stem cells are considered the most powerful kinds of stem cells, as they have the potential to give rise to any type of tissue.

But they are difficult to make, requiring the use of an embryo or cloning technology. Geron and some other companies have been pursuing the goal without the use of federal funds.

Advocates say stem cell-related research could lead to a whole new field of regenerative medicine, in which patients could get transplants and treatments for Parkinson’s, juvenile diabetes, cancer, injuries and a range of other ills.

“If safe and effective, the therapy would provide a viable treatment option for thousands of patients who suffer severe spinal cord injuries each year,” Richard Fessler, professor of neurological surgery at the Feinberg School of Medicine at Northwestern University said in a statement.

“There has been so much speculative and unsubstantiated information about the use of stem cells in spinal cord injury; it is important to study these cells in a rigorously designed clinical trial that is monitored by the FDA,” said Peter Wilderotter, president and CEO of the Christopher & Dana Reeve Foundation. 

Actor Christopher Reeve died in 2004 after having broken his spine years earlier in a horseback riding accident and was a vocal advocate of embryonic stem cell research.

Via Reuters