What can we expect from the first U.S. human clinical trials of stem cell therapy for spinal cord injury (SCI)?
In an exciting development, StemCells, Inc., has announced that it has received FDA authorization to begin clinical trials of its Investigational New Drug (IND) application of purified stem cell therapy to treat chronic spinal cord injury. This means that the breakthrough therapy for stem cell injuries can now begin testing on human patients in the United States.
The company, which focuses on the research and development of therapies and tools for use in stem-cell-based research, began trials about a year ago in Switzerland and Canada, and sees the potential breakthrough therapy currently being tested on a total of seven patients in those countries. The company calls the therapy HuCNS-SC, which stands for “human neural stem cells.”
Research for the therapy has been ongoing since 2002, when pre-clinical studies conducted with researchers at the University of California, Irvine showed that human neural stem cells engraft long-term, migrate to the area of injury, and transform themselves into specialized cells that create the protein myelin, a vital substance in treating spinal cord injury. The research eventually showed that transplantation of human neural stem cells can restore lost motor function. This restoration of function occurs even when treatment is administered after the initial, acute phase of injury.
The FDA approval for human clinical trials is a highly anticipated next stage.
“This IND is a significant step forward for our spinal cord injury program,” said Stephen Huhn, MD, FACS, FFAP, the company’s VP of CNS Clinical Research. “With regulatory authorization from Switzerland, Canada, and now the United States, we have the first international trial of a stem cell therapy for spinal cord injury.” He goes on to say, “We now look forward to enrolling patients at U.S. and Canadian sites and easing the travel burden for North American patients.”
Roman Reed, president of the Roman Reed Foundation, says, “This is great news for the spinal cord community. StemCells…have reported truly exciting preliminary results, with gains in sensory function persisting for 12 months after transplantation.”
The trial is designed to measure not only the effectiveness of the treatment for SCI, but the safety issues inherent in stem cell treatment as well. The current plan is to enroll a total of twelve patients (including the seven already taking part) with chest-level injuries classified as complete or incomplete, according to the American Spinal Injury Association Impairment Scale.
Previously, the study has been funded by a five year, $1.4 million grant from the National Institutes of Health (NIH) and a one-year $342,000 grant from the National Institute of Neurological Disease and Stroke (NINDS). The company intends to raise more funds for the study by selling shares of stock and warrants, which it expects to amount to $16.2 million, with shares going for an initial cost of $1.45 each. The effort is somewhat risky for a company as small as StemCells, Inc., with no approved products, but the company is confident that the research bears out the effort.
It is important to remember that as dramatic as the breakthrough is, stem cell therapy is still very much an experimental field, and it could be years before this therapy is available in any commercial sense. Still, it represents a very hopeful new era in the treatment of SCI. The video features a woman who participated in the initial trial speaking about her experience!