Geron Gets FDA Approval To Resume Stem-Cell Study

NEW YORK (Dow Jones)–Geron Corp. (GERN) can move forward with its early-stage trial of an embryonic stem cell treatment after the Food and Drug Administration lifted a clinical hold after almost a year.

Shares of the company rose 11% to $5.31 on the news.

The Menlo Park, Calif., company initially launched the study of GRNOPC1, which could potentially fix spinal cord injuries, in early 2009 but it was halted seven months later after safety concerns were raised by an animal study. No patients were enrolled in the study.

Late Thursday, Geron reported a second-quarter loss of $17 million on revenue of $1 million, reflecting collaboration agreements, and royalty and license fees. Aside from stem cell derived drugs, the company is also developing several cancer treatments.

The resumption of the GRNOPC1 study, which the company called the world’s first clinical trial of embryonic stem cell-based therapy in humans, marks the beginning of a multiyear journey for the company to potentially market the therapy. The initial testing in humans is primarily designed to test the safety of the drug and its effectiveness must still be proved.

The drug is part of a hopeful field of regenerative medicine. Embryonic stem cells are building-block cells that differentiate into more specific cells, and their use is hoped to regenerate cells that can’t be replaced because of injury or disease.

Geron’s drug contains living cells, called oligodendrocyte progenitor cells, that help restore nerve fibers and myelin, which is a protective sheath that protects nerves in the central nervous system. Repairing the nerves could lead to restoration of sensory function or use of extremities.

The study will evaluate safety in eight to 10 patients with recent severe spinal cord injuries. The company agreed with the FDA to leave 30-day intervals between the first patients, for safety reasons.

The halt of the trial came after an animal study showed an increased frequency of small cysts within the injury site in the spinal cord than previously seen.

In response to those results, Geron developed new testing methods that essentially ensure the purity of the drug before it is used. GRNOPC1 is actually a mix of different cells and the tests ensure that the drug doesn’t contain cells that shouldn’t be there.

As a secondary measure, the data will measure effectiveness of the drug, which has only been seen in animal testing. The company is currently testing the therapy in animals to determine its potential in nervous-system diseases such as Alzheimer’s Disease, multiple sclerosis, and Canavan Disease, a fatal neurological disorder.

Research in embryonic stem cells has been controversial because it may involve destroying human embryos to obtain the cells.

Congress banned the use of federal funds to create or destroy human embryos solely for research purposes in 1996. In 2001, President Bush loosened the restriction slightly by allowing federal funds to be used for research on a few then-existing stem-cell lines. Those limits were still viewed as restrictive to research by many in the field and they were lifted in March 2009 by President Obama.

-By Thomas Gryta, Dow Jones Newswires

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