Four boys with a rare and often fatal brain disease were implanted with stem cells that began fixing damage that impeded their ability to walk, talk and eat, a trial found. The findings, published today in the journal Science Translational Medicine, are from the first stage of human tests funded by StemCells Inc. (STEM), a Newark, California-based company.
The children have a genetic disorder called Pelizaeus- Merzbacher, in which the brain can’t make myelin, the fatty insulation for nerve cells that helps conduct brain signals. The children all had evidence of myelin growth a year later. The increased abilities shown by three of the boys in the University of California San Francisco study may bode well for other diseases caused by a lack of myelin insulation, including multiple sclerosis and cerebral palsy, the authors wrote.
“Those were severely impaired children,” said Stephen Back, a professor of pediatrics and neurology at Portland’s Oregon Health & Science University, in a telephone interview. “The fact that they showed any neurological improvement is very encouraging.”
Back did work in mice that preceded today’s work in humans, which he wasn’t directly involved in. His study, published simultaneously, showed that the animals with no myelin at all grew some after being implanted with human stem cells.
Pelizaeus-Merzbacher disease causes the degeneration of the nervous system, and there is no cure or standard treatment. People with the illness experience a loss of coordination, thinking and motor abilities. It’s one of several disorders linked to genes that control myelin production.
The incidence of the disease is 1 in 200,000 to 500,000 people, according to today’s study of the boys.