A new stem cell production facility at Children’s Hospital of Orange County (CHOC) will allow researchers to produce patient-specific cells for future immune-matching therapies that could positively impact fatal neurological diseases in children.
The state-of-the-art softwall clean room is one of less than a dozen of its kind in the nation, and the only one focused on immune matching rather than immune suppression.
Built at the fraction of the cost of a larger, more complex laboratory, the facility will allow CHOC researchers to study a stem cell-based therapy for the treatment of mucopolysaccharidosis (MPS-1), a rare and progressive neurodegenerative disease that typically claims patients before age 10.
“Based on the results of animal trials we’ve conducted so far, we have a high degree of confidence that stem cell-based therapy will work to treat MPS-1,” said Philip Schwartz, Ph.D., senior scientist at the CHOC Children’s Research Institute and managing director of the National Human Neural Stem Cell Resource.
“If our research is successful, the approach could be used to treat a number of other immune-based diseases that damage the nervous system, like multiple sclerosis,” Dr. Schwartz said.
The approach involves using umbilical cord blood to replace a patient’s immune system, then implanting neural cells derived from the same blood into the brain to repair and prevent brain damage.
While implanting cells directly into the brain isn’t new, current treatment protocols require that patients take immunosuppressant drugs to reduce the risk of rejection, which leaves them vulnerable to a host of infections.
Standard procedures for replacing the immune system, like bone marrow transplants, aren’t effective for patients with brain disorders caused by their underlying disease because the transplanted cells don’t cross the blood-brain barrier and therefore don’t slow the progression of brain disease.
Dr. Schwartz estimated that about five years of work would be required to establish a program before approaching the U.S. Food and Drug Administration for approval to begin Phase I clinical trials. The current research project is supported by a $4.27 million grant from the California Institute for Regenerative Medicine.