ABOVE: A neurosurgeon injects neural stem cells into the frontal lobe of a child with Pelizaeus-Merzbacher disease who participated in a Phase 1 clinical trial of the transplantation.
NALIN GUPTA
Pelizaeus-Merzbacher disease is a genetic malady that leaves neurons without their myelin coating. This deficit has devastating consequences for the boys—it’s X-linked—who have it. “These children have severe developmental delay, so they have inability to walk, inability to talk and perform self-care,” says Nalin Gupta, a professor of neurological surgery and pediatrics at the University of California, San Francisco (UCSF). “Their neurologic function typically does not improve, and usually they actually die during childhood.”
About a decade ago, the biotech firm StemCells Inc. was looking for a neurosurgeon to try out an intervention that might finally offer some help for these children. Because Gupta had experience conducting surgical clinical trials in kids with disabilities, the company approached him to see if ...
