Stem-Cell Test Is Set for Children
With an Incurable Brain Disease
By DAVID P. HAMILTON
March 10, 2006; Page B4
Scientists in Oregon are preparing to begin a clinical trial in which they will test transplants of human neural stem cells as a treatment for young children with an incurable brain disease.
On Wednesday, a review committee at the Oregon Health & Science University in Portland cleared the proposed trial, which will involve the transplantation of "purified" neural stem cells developed by StemCells Inc., a biotechnology company in Palo Alto, Calif.
The trial won't involve embryonic stem cells, which can transform into any form of tissue, but rather neural "precursor" cells that can transform themselves into various types of brain and nerve tissue. The trial will enroll six children from the ages of 18 months to 12 years who suffer from Batten disease, a genetic brain disorder that leads to progressive loss of motor control, seizures and, ultimately, death. Batten disease results from the absence of an important enzyme in brain tissue.
The children will receive several injections of the neural stem cells directly into their brains, after which researchers will closely monitor their physical and mental well-being. While the trial is designed primarily to study the safety of the StemCells cell therapy, researchers also hope it might turn up evidence that the transplanted cells can produce enough of the missing enzyme to slow progression of the disease.
StemCells derives its cells from fetal brain tissue, which the company obtains via a third party from aborted, miscarried or stillborn fetuses. Its cell therapy so far has never been tested in humans.
In general, scientists have tried several times over the past two decades to treat neurological problems such as Parkinson's disease with transplants of fetal cells, but many prominent attempts have ended in failure.
StemCells, by contrast, purifies and grows just the fetal stem cells, which it believes can "differentiate" into healthy brain cells once injected. Robert Steiner, an OHSU pediatric geneticist who is leading the trial, said the research team hopes to enroll its first patient within the next month or so. The Batten-disease trial has been delayed several times over concerns about the safety and ethics of the trial.
Write to David P. Hamilton at david.hamilton@wsj.com |
