When Geron decided, in 2010, to halt the first ever clinical trial of stem cells for spinal cord injury it was a disappointment to many people, particularly for those with spinal cord injuries (SCI) who were hoping it might help them. But now that therapy is back, and the company behind it this time, Asterias Biotherapeutics, has just treated its first patient.
The patient was dosed at the Shepherd Center in Atlanta, Georgia. This trial participant is the first of 13 patients who will be given escalating doses of up to 20 million AST-OPC1 cells to see if they can benefit patients. Up to 8 centers will be involved in the trial, including some in California.
The AST-OPC1’s are a kind of stem cell called an oligodendrocyte progenitor, which can turn into cells that protect nerves and help insulate their signals, much like the insulation of a bare electric wire. In the trial, these progenitor cells are injected into the injured spine. Earlier animal experiments showed the AST-OPC1 cells helped regenerate nerve tissue at the site of injury.
In a news release Dr. Donald Peck Leslie, the medical director of the Shepherd Center, said the people being treated have essentially lost all sensation and movement below their injury site with severe paralysis of the upper and lower limbs:
“If AST-OPC1 could deliver even modest improvements in motor or sensory function, it would result in significant improvements in quality of life for people with SCI.”
The primary goal of the trial – which CIRM is helping fund – is to make sure this approach is safe; so the first three patients treated will get just 2 million cells. If that first group show no bad side effects then the next five patients will get ten million cells, and the next five after that will get 20 million cells.
For people with spinal cord injuries this is clearly a hopeful development. Last year alone 12,000 people in the U.S. suffered a severe spinal cord injury leaving them completely or partially paralyzed. There is no effective treatment.
For the Asterias team this is also an exciting time. Several of them were with Geron when that first trial was cancelled because the company changed its business strategy. Since then the Asterias team has worked long and hard to get the approach revived. This latest news is a recognition that all that effort is paying off.
Of course this is just the start, and there is a long way to go before we know if this approach, these cells, will provide any lasting benefits to people with spinal cord injuries. But the results from the five people treated with these cells at Geron are encouraging. Follow-up studies on those patients have shown no serious side effects due to the therapy and in four of the five patients, MRI scans have shown that the actual injury site had shrunk.
Pedro Lichtinger, President and CEO of Asterias, was full of praise for those agreeing to get the treatment:
“Individuals with SCI have severe disabilities that can significantly shorten projected lifespan, impact quality of life and result in lifetime costs of care of $3 million to $4 million. We are grateful for the interest of patients with SCI to participate in this program.”
That praise for the people who are getting the therapy is well deserved. They have just experienced a life-changing, traumatic experience and have volunteered to be part of a clinical trial where they may not get any benefit because, at least for this first phase, the number of cells being used is comparatively small.
The people volunteering know that this trial is all about safety. But they also know that without these first steps, without their involvement, there can be no progress. They are, in every sense of the word, pioneers in this research. For that they deserve our recognition, and our gratitude.