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.
…
In 2012, [neurosurgeon Nalin] Gupta and colleagues reported that four boys with PMD who had received pluripotent neural stem cells in a Phase 1 clinical trial tolerated the procedure, and imaging techniques that indirectly detect myelin indicated they may have had myelination in their brains one year following the transplant. This August, the researchers reported the results of a long-term follow-up study of those patients—all four are still alive at ages 10, 11, 12, and 13. Patients like these who have symptoms of the disease starting at birth typically die in their teens.
…
Gupta’s study is the latest report in a series of clinical trials on neural stem cell transplantation, in which pluripotent neural cells taken, in most cases, from the brains of aborted fetuses are expanded in the lab and then injected into the brains or spinal cords of patients with incurable neurological disorders. These include stroke, multiple sclerosis, ALS, spinal injury, and Parkinson’s disease. But for all the effort that has gone in to testing these cells, none have been able to work themselves out of trials and into clinical practice.
Read full, original post: Neural Stem Cell Transplantation Crawls Toward the Clinic
