U.K. doctors injected fetal stem cells into the brain of a male stroke patient this week, opening the first-ever stem-cell trial for treating stroke in humans.
The study, known as Pilot Investigation of Stem Cells in Stroke (PISCES), marked the world's first fully regulated clinical trial of a neural stem-cell therapy for stroke patients. The treatment occurred last weekend at Glasgow's Southern General Hospital and the patient was discharged Monday, according to professor Keith Muir, principal investigator for the trial.
A stroke, sometimes called a "brain attack," is a interruption in the brain's blood supply sometimes causing cells in certain parts of the brain to die.
"We have no treatments that can repair the damage caused by stroke at present. A lot of stroke survivors have permanent disability despite acute treatment and physical therapies to maximi[z]e recovery," Muir said. "Stem cells promise possible regeneration of brain tissue, although exactly how they work is not clear."
After drilling a hole into the skull of the patient, a thin needle was used to inject roughly 2 million neural stem cells directly into the damaged area of the brain. Although there is the potential for the patient's immune system to react negatively to the foreign stem cells, Muir said, it is unlikely. The patient and 11 others expected to receive the treatment will be watched closely for the next few years.
"This is a safety study so we are interested in following up for any safety problems," he said. "This means a series of clinical checks, scans and blood tests over the next two years to begin with."
The trial is being carried out by the University of Glasgow and ReNeuron, a company that builds stem-cell technologies, is the first stem cell-based clinical trial to receive regulatory approval in the U.K. The trial uses neural stem cells derived from a 12-week-old aborted fetus.
"The initiation of the PISCES clinical trial is a major and hard-won milestone for ReNeuron and a significant milestone in the development of therapies to address the severely disabling effects of ischaemic stroke," Michael Hunt, chief executive officer of ReNeuron, said in a news release.
Muir said PIECES has been in the works for ReNeuron for two years now, although the supporting research has been developing in the past decade. It was a little under a decade ago that researchers at Albert Einstein College of Medicine in New York successfully repaired stroke damage in rats by injecting their brains with their own stem cells.
"Animal studies do suggest benefit, but that is a long way from proving clinical benefit in humans," said Dr. Larry Goldstein, director of the Duke Stroke Center in Durham, N.C.
"Even if efficacious, the effects might be different in strokes affecting different brain regions and strokes of different severities. Well designed studies are needed, hopefully this will provide a start."
The first embryonic stem-cell trial in humans in the United States was approved in 2009 for spinal cord injury and since then hundreds of Phase 3 clinical trials utilizing stem cells are underway, several of which will test stem cell treatments for stroke.
Stroke is the third leading cause of death in the United States and accounts for nearly 136,000 deaths each year, according to the Centers for Disease Control and Prevention.