Recent news reports highlight two interesting stem cell trials involving children, one completed and one just getting underway.
Researchers at the Sutter Neuroscience Institute in Sacramento, CA, in conjunction with CBR (Cord Blood Registry) – which bills itself as the world’s largest stem cell bank – announced on Friday that the US Food and Drug Administration (FDA) has granted approval to proceed with a single-site trial of cord blood stem cells in 30 children with a diagnosis of autism. According to the press release, this is “the first FDA-regulated clinical trial to assess the use of a child’s own cord blood stem cells” for this indication. Children who are enrolled in the 13-month study will receive two infusions of their own cord blood stem cells or placebo at 6-month intervals. The study will use a case-crossover design, in which children initially placed in the placebo group will cross over to the cord blood stem cell group at the 6-month mark, and vice versa. This allows for a randomized, double-blind study with each child serving as his or her own control. Endpoints include changes in language, learning and behavior.
Also last week, researchers at the University of California, San Francisco Children’s Hospital published a small study of neural stem cell transplantation in children with a rare, usually fatal condition called Pelizaeus-Merzbacher disease (PMD) that affects the central nervous system. Human central nervous system cells were transplanted into the brains of 4 young children with PMD, after which they were followed for one year. Safety was the primary endpoint in this study, which did not include any controls. Three of the four children exhibited modest improvements in their neurologic function and no serious safety issues were reported. Although not definitive, the results suggest that research on this potential therapy should continue. There is currently no cure for PMD. The children enrolled in this study have the most severe form of the disease, which is referred to as connatal (i.e., congenital) PMD.