Stem cells show preliminary success in rare childhood brain disease

Yesterday, Newark-based StemCells Inc. announced a significant milestone in regenerative medicine. They injected their purified neural stem cells into the brains of four children with a disease that results in insufficient amounts of an insulating layer called myelin around their nerves. The injected cells were able to form new myelin, providing hope that the cells might also be effective in other demyelination diseases such as multiple sclerosis.

Alan Trouson, CIRM President, had this to say about the results:

"Even though the numbers are small and any conclusions are preliminary, the team hit four separate milestones the field has been striving for—they got new myelin to grow, it appears to be durable, they withdrew the immune suppressants and did not see rejection of the new tissue, and they saw very early signs of functional improvement. This is very exciting data for all patients with demyelinating diseases such as MS because it suggests that with time, and a bit more patience, we should be able to work out how to replace this vital protecting layer for neurons."

StemCells Inc. presented the results at the 2012 European Leukodystrophy Association Families/Scientists Meeting in Paris. After the presentation they released a statement describing the disease under investigation, called Pelizaeus-Merzbacher disease (PMD).

Patients with PMD have a defective gene, which leads to insufficient myelin in the brain. The disease occurs only in males, and those with the most severe form of the disease, connatal PMD, are significantly disabled from birth and usually die, within the first decade of life. The study was the first to test transplantation of neural stem cells as a potential treatment for a myelination disorder. Myelin is the substance that surrounds and insulates nerve cells' communications fibers (also known as axons). Without sufficient myelination, these fibers are unable to properly transmit nerve impulses, leading to a progressive loss of neurological function, and death.

The San Francisco Business Times wrote about other clinical trials by StemCells Inc. involving these same purified neural stem cells:

StemCells, which purifies neural stem cells extracted from fetal brain tissue, has transplanted those cells in patients with spinal cord injuries and a rare childhood neurodegenerative disorder, called Batten disease, though it abruptly ended that trial last year. It also received approval from the Food and Drug Administration in February to use the stem cells in a Phase I/II trial of patients with an eye disease, dry age-related macular degeneration.

Scientists from StemCells Inc spoke to the CIRM governing board about their clinical trial for Batten Disease in 2008. The video of the talks are available on the CIRM website. CIRM has also funded an award providing funds for StemCells Inc to plan a full Disease Team Award that would use these same cells to develop a therapy for Alzheimer’s disease. Here is a summary of that award. All disease team research award applications will be reviewed by CIRM's Grants Working Group, which provides funding recommendations to our governing board. The governing board will make the final decisions on which applications to fund at a meeting this summer.

A.A.