FDA Approves World's First Embryonic Stem Cell Therapy in Humans. Will They Walk Again? |
Geron Corp will regrow nerve tissue in spinal cord injuries |
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Editors published 1/23/2009 9:25:00 AM
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The FDA announced today that Geron Corp, a California biotechnology company, will perform the world's first embryonic stem cell (hESC) therapy on humans.
Geron will inject hESC cells directly into the damaged sites of paraplegic patients with spinal cord injuries in an effort to regrow nerve tissue.
What are these cells?
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.
What are the political implications?
The embryonic stem cells used in this study were derived as a result of Bush administration policies.
According to Geron's website, these cells fall within the Bush policy guidelines for Federal funding.
However, Geron does not use federal funding for its research.
The cells are derived from the H1 human embryonic stem cell line, which was created before August 9, 2001. Studies using this line qualify for U.S. federal research funding, although no federal funding was received for the development of the product or to support the clinical trial.
Who are the patients?
Geron will try to use the stem cells to regrow nerve tissue in patients with crushed, but not severed, spinal cords. Technically, these are "complete" American Spinal Injury Association (ASIA) grade A subacute thoracic spinal cord injuries (with no motor and sensory function).
Paraplegics can use their arms but can't walk.
According to Reuters health editor Maggie Fox:
Geron will recruit eight to 10 recently injured patients [within the last 14 days] and inject them with small numbers of human embryonic stem cells manipulated to become the oligodendrocyte cells that insulate nerves, and that produce compounds to stimulate the growth of nerve cells.
While the patients will get low doses of immune-suppressing [anti-rejection] drugs for the first two months, [they are] confident the cells will escape immune system recognition and patients will not have to endure the treatments that organ and tissue transplant recipients usually do. Treatment on the first patient should begin this summer.
They are not expecting the patients to get up and walk. This particular study is primarily testing to determine the therapy's safety and to look for signs of patient improvement like return of sensation or movement in the legs.
What are the Possibilities?
Geron CEO Dr. Thomas Okarma said the treatment should eventually become cheap and easy to mass produce because the cells can be grown in vats. He believes the cells may be useful for other diseases such as multiple sclerosis, in which nerve cells are stripped of their insulating sheaths, and perhaps strokes.
Financial analysts celebrated. Stephen Brozak and Daniel Mallin of WBB Securities LLC said it could "as important to drug therapy as the discovery of ... penicillin."
Financial Implications
Geron shares rose more than 50% on the news. The company is debt free. They have spent more than $100 million on embryonic stem cell research since 1992.
Update 2/6/2009 per Illinois State University
"Politics played no role in the FDA's decision," Karen Riley, FDA press officer said. "These things are a process."
"The timing was governed by the waiting for the company to come back to us with an answer to one of our previous questions. It's about science, not politics."
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