logo
Share SHARE
FONT-SIZE Plus   Neg

StemCells' Phase I Trial In PMD Shows Evidence Of Progressive Myelination

StemCells, Inc. (STEM) revealed preliminary evidence of progressive and durable donor-cell derived myelination in all four patients who underwent transplantation with the company's proprietary HuCNS-SC cells in its clinical trial for Pelizaeus-Merzbacher disease, or PMD, a rare hypo-myelination disorder in children.

In addition, clinical assessment disclosed small but measureable gains in motor and/or cognitive function in three of the four patients; the fourth patient remained clinically stable.

The company noted that the open-label Phase I trial enrolled four patients with the connatal form of PMD, between the ages of 14 months and 5 years, and was designed to assess safety and preliminary efficacy of the intervention. The study used magnetic resonance, or MR, imaging, commonly employed in other neurological diseases, to explore signs of myelination related to the transplanted neural stem cells.

At the one-year interval, MR imaging showed changes compatible with increased myelination in the region of the transplantation.

Further, the company said that a separate four-year observational study will continue to monitor and report the future progress for all four patients.

"The finding of myelin formation in this first exploratory study is indeed very encouraging," said Stephen Huhn, Vice President and Head of the CNS Program at StemCells, Inc. "We believe that the results of this trial provide proof-of-concept and a compelling rationale for the Company to begin planning for a controlled Phase II study in PMD. These results may also have implications for other leukodystrophies, as well as more common myelin disorders including transverse myelitis, multiple sclerosis and periventricular white matter injury seen in Cerebral Palsy..."

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

Business News

Quick Facts

Editors Pick
Embark Veterinary Inc., a startup that makes dog DNA-testing kits for pet owners, vets and breeders, said it has raised $4.5 million in funding from investors and relocated its headquarters to Boston. The company intends to use the new funding to expand its canine genetic testing suite for pet owners, breeders, and veterinarians. Intel Corp. has completely shut down its wearables division about two weeks ago, CNBC reported, citing a person familiar with the matter. The company reportedly intends to focus on augmented reality. According to the CNBC report, Intel laid off about 80 percent of the team that made the Basis fitness watch in November 2016 and has now eliminated the division entirely. The Federal Trade Commission is investigating allegations that Amazon.com Inc. misled consumers about its pricing discounts, Reuters reported, citing a source close to the probe. The FTC probe is reportedly part of the agency's review of Amazon's proposed $13.4 billion deal to buy grocery chain Whole Foods Market Inc.
comments powered by Disqus
Follow RTT