FDA OKs First Human Trials of Embryonic Stem Cells

The Federal Drug Administration has approved the first human trials of embryonic stem cells — a sign of a new, liberal attitude toward stem cell research, which was hamstrung by the Bush administration.

Starting this summer, the biotech firm Geron will treat a small group of spinal-cord injury patients using neurons derived from stem cells, marking the first time embryonic stem cells will be tested in humans.

The trial is designed to test the safety of the treatment, not how well it works. Nonetheless, it's a huge first step for the field.

"It signals to me that we have the primary regulatory authorities on board for embryonic stem cells," said Alan Trounson, president of the California Institute of Regenerative Medicine, a $3 billion state initiative to support stem cell research. "That really is a tremendous piece of news."

Under the Bush administration, stem cell research was slowed by an executive order, signed in August 2001, that (severely) restricted the types of stem cells and stem cell research that could be conducted. President Barack Obama is widely expected to lift Bush’s executive order, perhaps as soon as next week.Working in a handful of medical centers around the country, Geron will treat eight to 10 recent paraplegics, who can use their arms but not their legs. The patients will receive an injection of neurons to the site of the damage, followed by a short treatment of anti-rejection drugs.

Previous animal studies suggest the new neurons will repair damaged neurons and secrete substances to help nerves function and grow.

Amy Rick, president of Coalition for the Advancement of Medical Research, a group of dozens of research institutions that support stem cell research, said the Geron trial is a milestone.

"It's hugely significant in the sense that it's the first approval of a human embryonic stem cell trial," she said. "In this week of hope and change, it feels even better."

While Geron scientists waited months for FDA approval of the stem cell treatment, they are reluctant to link the go-ahead directly to the inauguration of Obama.

A Geron spokeswoman said that the company had no evidence of political influence aiding their application.

“It’s just coincidental timing,” the spokeswoman said.

Karen Riley, an FDA spokeswoman, echoed that the timing was coincidental. "We make science-based decisions and politics is not a factor," she said.

But the new president surely didn't hurt matters. The chairman of Trounson's organization told the New York Times, "I think this approval is directly tied to the change in administration."

The approval is expected to the first of several trials involving embryonic stem cells. A recent CAMR report found that nine companies, including Geron, were in the process of developing human embryonic stem cell treatments.

Embryonic stem cells are like blank slates that can be transformed into different types of tissue. They've been hailed as the next big thing in medicine ever since University of Wisconsin scientist James Thomson showed their ability to regenerate in 1998. Since then, stem cells have been like a high school star turned NBA draft pick — talented and expensive but undisciplined and perhaps not quite ready for the glare of the big game. Like many biotechnology techniques, the lag between scientific discovery and clinical treatment can be decades.

Still, the Regenerative Medicine Institute’s Trounson, who was a stem cell scientist in Australia before heading the California institute, said that experimental treatments are outpacing his expectations.

"We're running an agency funding this work and I'm astounded at what's happening in this space,” he said.

Trounson said there’s evidence in animal trials that stem cells are effective in treating ailments as varied as diabetes, Alzheimers, multiple scleorsis and macular degeneration.

“It’s just fantastic,” Trounson said. "And I would expect some of these to enter clinical trials sooner, rather than later."

His agency expects to fund up to a dozen scientists who think they can submit their stem cell work to the FDA for clinical trial approval within four years.

From there, those so-called investigational new drugs will have to follow the path that Geron's treatment did. The company submitted its application early in 2008. It was then put on hold in May 2008 and kicked back to the company for further review. Seven months later, the company resubmitted the application and received approval Wednesday, the day after the inauguration.

That said, Obama's political influence is likely to invigorate a field that — despite impressive state-level and private efforts — has been ham-strung by Federal regulation and the specter of increased government regulation.

"With President Obama there, there will be a big change not only in government administration and the public sector, but I think it will encourage the pharmaceutical companies to be involved as well," Trounson said.