A potential treatment for spinal cord injury that made headlines in 2010 as the first human embryonic stem cell therapy to be tested in humans is pushing ahead after a stalled clinical trial and a change of ownership. Asterias Biotherapeutics announced the results of an initial safety study of the therapy earlier this month, and yesterday the company won $14.3 million from the California Institute for Regenerative Medicine (CIRM) for the next round of trials.
CIRM had given $25 million to Menlo Park, California–based Geron, which developed the cells, to test the treatment, which uses neuronal cells derived from embryonic stem cells intended to restore function to spinal cord injury patients. But Geron halted the trial a year later to focus on anticancer therapies, fueling concerns that the treatment might not be as promising as hoped.
Asterias took over the project in 2013, and last week the company’s president of R&D, Jane Lebkowski, presented results from the first trial at the annual meeting of the American Society of Gene & Cell Therapy in Washington, D.C. The five patients with spinal cord injury who received a low dose of the treatment saw no adverse effects, she reported, and there was some evidence that further degeneration had been prevented at the injury site for four of them.
The next study will focus on the upper part of the spine rather than the lower part. (Initial clinical trials for spinal cord injury often target the lower spine, as there is less risk of adverse consequences causing serious damage.) The company also plans a phase II trial to study the effect of higher dosages.
The therapy was one of two to gain approval yesterday as part of CIRM’s Strategic Partnership program. It provides funding to help propel a therapy through clinical trials on the condition that the recipient matches the investment.