Stem cell trial re-starts
pharmafile | October 12, 2010 | News story | Research and Development | Geron, Stem cells, spinal injury
Geron Corporation has enrolled the first patient in its restarted clinical trial of human embryonic stem cells (hESC).
The world’s first phase I clinical trial of a stem cell therapy was approved in January last year, but was stopped nine months later.
The FDA put it on ‘clinical hold’ after a pre-clinical trial showed a higher frequency of test animals developed cysts at the injury site than had been noted in earlier studies.
But the US regulator gave California-based Geron the green light in August to begin treating patients with spinal cord injury using GRNOPC1, which contains hESC-derived oligodendrocyte progenitor cells.
These have been shown to help nerve growth, which in turn has led to restoration of function, in rats with acute spinal cord injury.
Stem cells have the potential to become different cells in the body, and their use – while controversial – is therefore seen as having potential to find treatments for a range of conditions.
“Initiating the GRNOPC1 clinical trial is a milestone for the field of human embryonic stem cell-based therapies,” said Geron chief executive Thomas Okarma.
Geron’s trial will focus on safety, assessing whether there are risks to patients, the first of who will be treated at the Shepherd Center in Atlanta. Six other US sites, including Northwestern Medicine in Chicago, may also be used.
Study participants will receive GRNOPC1 within 14 days of their injury. Previously, histological examination of the injured spinal cords treated with GRNOPC1 showed improved axon survival and extensive remyelination surrounding the rat axons.
“Our medical staff will evaluate the patients’ progress as part of this study,” said Donald Peck Leslie, medical director at the Shepherd Center. “We look forward to participating in clinical trials that may help people with spinal cord injury.”
Geron started working with hESCs in 1999 and some observers predicted it would be “decades before a cell therapy would be approved for human clinical trials”, added Okarma.
The company is also exploring GRNOPC1’s application for use against neurological diseases including multiple sclerosis, stroke and Alzheimer’s disease.
The pharma sector will be watching closely, calculating whether their interest in this unproven science would be best served through patnering with academics and biotechs or investing in their own research.
Along with human embryonic stem cells, adult stem cells (largely sourced from bone marrow) and induced pluripotent stem cells provide the research options.
In 2008 Pfizer set up Pfizer Regenerative Medicine with scientists in Cambridge, UK, looking at neural and sensory disorders while those in Cambridge, US, concentrate on cardiac and endocrine disorders.
Meanwhile, in April a UK trial of ReNeuron’s ReN001 began with12 patients who have suffered an ischaemic stroke, implanting neural stem cells directly into the brain.
Adam Hill
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