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Geron Gets FDA Nod for First Embryonic Stem Cell Trial in the U.S.

by Lynn Shapiro, Writer | January 28, 2009
Can embryonic stem cells
regrow nerve tissue
in patients with
crushed but not
severed spinal cords?
The FDA has permitted Geron Corporation to find out whether embryonic stem cells can regrow nerve tissue in patients with crushed but not severed spinal cords, in effect announcing that the Obama Administration has lifted the ban on federally-funded embryonic stem cell research put in place by former President George W. Bush on August 9, 2001.

"For us, it marks the dawn of a new era in medical therapeutics. This approach is one that reaches beyond pills and scalpels to achieve a new level of healing," Geron's chief executive, Dr. Thomas Okarma, said in a briefing.

As the biotech community celebrated the news, shares of Geron jumped more than 53 percent to $8 on Monday, January 26. The FDA said that it had already been conferring with Geron on important scientific issues, as President Obama had been expected to allow federally-funded stem cell research.

Geron plans to initiate a Phase I multi-center trial that is designed to establish the safety of its stem-cell product, called GRNOPC1. For starters, Geron will inject eight to 10 recently injured patients with GRNOPC1.

"The ultimate goal for the use of GRNOPC1 is to achieve restoration of spinal cord function by the injection of [embryonic] oligodendrocyte cells directly into the lesion site of the patient's injured spinal cord," Dr. Okarma said. GRNOPC1 has demonstrated remyelinating and nerve growth stimulating properties leading to restoration of function in animal models of acute spinal cord injury, he pointed out. (Journal of Neuroscience, Vol. 25, 2005).

Heralding the research, Richard Fessler, M.D., Ph.D., professor of neurological surgery at the Feinberg School of Medicine at Northwestern University, said, "The neurosurgical community is very excited by this new approach to treating devastating spinal cord injury. Demyelination is central to the pathology of the injury, and its reversal by means of injecting [embryonic] cells would be revolutionary for the field. If safe and effective, the therapy would provide a viable treatment option for thousands of patients who suffer severe spinal cord injuries each year."

Patients eligible for the Phase I trial must have evidence of functionally complete spinal cord injury with a neurological level of T3 to T10 spinal segments and agree to have GRNOPC1 injected into the lesion sites between seven and 14 days after injury, the company said. Geron has selected up to seven U.S. medical centers as candidates to participate in this study. The sites will be identified as they come online and are ready to enroll subjects into the study.

Although the primary endpoint of a Phase I trial is safety, this Phase I protocol includes secondary endpoints to assess efficacy, such as improved neuromuscular control or sensation in the trunk or lower extremities. Once safety in this patient population has been established and the FDA reviews clinical data in conjunction with additional data from ongoing animal studies, Geron plans to seek FDA approval to increase the dose of GRNOPC1 and expand the trial to include as many severe spinal cord-injured patients as possible.

The cells may also be useful for people with other diseases such as multiple sclerosis, in which nerve cells have been stripped of insulating myelin sheaths, or possibly for people who have had strokes, Dr. Okarma said. What is more, he said, the cells are easy to clone in vats so that treatment will not be costly.