Approval of Clinical Trial Opens Door to Next Steps in Regenerative Medicine

FDA’s approval of a clinical trial for stem cell therapy last month is a milestone for regenerative medicine, potentially heralding a new era of treatment and a new generation for research.

With any measurable success in treating humans, coupled with a lifting of restrictions on federal funding and support of human embryonic stem cell research, the future looks bright for at least one segment of health care.

President Obama is expected to reverse restrictions former President George W. Bush imposed on federal funding for stem cells in 2001.  The new president has not made it clear whether he intends to issue his own executive order rescinding Bush’s — a move that could come early this month — or whether he plans to wait for Congress to pass a bill that would create new, unrestricted funding for stem cell research.

Several organizations and companies are well positioned for the new stem cell era, and two of the most notable are in California: the taxpayer-funded California Institute for Regenerative Medicine and Geron, a biotech firm that won FDA approval for the first trials in humans.

‘This Opens The Door’

CIRM, created and funded by the passage of Proposition 71 in 2004, has invested $625 million in stem cell research. So far, most of the money has been used to lay foundations — scientific, brick-and-mortar laboratories and stem-cell specific IT applications — for what was to come.

While much of the foundation work still needs to be done, the next era is starting, according to Patricia Olson, director of scientific activities for the institute.

“The FDA’s approval for initiation of a clinical trial in patients using a cell therapy derived from embryonic stem cells is a landmark for regenerative medicine,” Olson said, adding,  “This opens the door for clinical testing of additional stem-cell derived cell therapies which may lead to restoration of function for patients.”

Early this month, the institute will announce a new Disease Team Research Award program aimed at bringing additional embryonic stem cell-based therapies to clinical trials. Disease Team grants will fund interdisciplinary teams to share expertise and bring products to clinical trials within 48 months, Olson said.

CIRM’s existence has sparked partnerships with universities and other research organizations and attracted other stem cell companies.

Geron Trial Begins This Summer

Geron will implant embryonic stem cells in eight to 10 paraplegic patients who can use their arms but cannot walk. Stem cell injections will be given within two weeks of the injury.

Researchers hope embryonic stem cells — the most versatile of human cells believed to be capable of growing into any type of cell — will help patients make modest gains after their injuries.

The trials will begin this summer and could involve as many as seven medical centers.

Scientists hope to use the transformational qualities of stem cells to treat a variety of diseases and injuries affecting millions of people worldwide. Because stem cells can turn into many other cell types with the right prompting, doctors might be able to replace tissues and organs damaged by disease or injury to restore healthy function. Supporters of stem cell research say it could lead to treatments for Parkinson’s disease, diabetes, Alzheimer’s disease, multiple sclerosis, stroke, heart attacks, spinal injuries, bone and bone marrow diseases and other ailments.

“This marks the dawn of a new era in medical therapeutics,” Thomas Okarma, Geron’s president and CEO, said during a teleconference announcing the trials last week. “This approach is one that reaches beyond pills and scalpels to achieve a new level of healing.”

Regulatory Risk Reduced

New technologies and applications are more likely to be brought forward in stem cell research now as a result of Geron’s trials, Olson said.  

“The regulatory risk has been reduced in the sense that, under appropriate risk-benefit circumstances and based on adequate data, FDA is willing to approve clinical studies with embryonic stem cell-derived cell therapies,” Olson said.

By establishing CIRM, California already has shown how public funding can generate more investment. Could Geron’s clinical trial have a similar impact in the private sector?

“Yes,” Olson said, “but realistically, this is a first step along the stem cell-derived-regenerative medicine path to therapies and cures. But to those who invest in this space, it is a landmark step,” Olson added.

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