Human neural stem cells developed by Stem Cells, Inc. were injected directly into the brains of four young children with an early-onset, fatal form of a condition known as Pelizaeus-Merzbacher disease (PMD). The neural stem cells successfully engrafted into the brains of patients and appear to have produced myelin.
Patients with early-onset PMD cannot walk or talk, often have trouble breathing and undergo progressive neurological deterioration leading to death as a result of an inherited genetic defect that prevents brain cells called oligodendrocytes from making myelin.
The investigators found evidence that the stem cells had successfully engrafted, receiving blood and nutrients from the surrounding tissue and integrating into the brain, a process that David H. Rowitch, MD, PhD, professor of pediatrics and neurological surgery at UCSF, chief of neonatology at UCSF Benioff Children’s Hospital and a Howard Hughes Medical Institute Investigator, likened to “a plant taking root.”
Condensed and adapted from the UCSF announcement.