Sunday, April 30, 2017

Asterias and Capricor: Good News for California's Stem Cell Research Effort

Pat Furlong, CEO of Parent Project Muscular Dystrophy, spoke earlier 
this year about losing her two sons to Duchenne muscular dystrophy

California's $3 billion stem cell agency last week reported encouraging results from two of its clinical trials, news that came as the prestigious journal Nature was declaring that the state effort was in its "last stage."

The trials are being conducted by Capricor Therapeutics, Inc., of Beverly Hills, Ca., and Asterias Biotherapeutics, Inc., of Fremont, Ca. Both are publicly traded firms.

The news was reported by The Stem Cellar, the blog of the California Institute for Regenerative Medicine (CIRM), as the Oakland-based agency is formally known.

CIRM declared,
"Asterias Biotherapeutics continues to keep a steady pace and to hit the proper milestones in its race to develop a stem cell-based treatment for acute spinal cord injury."
The agency has pumped $20.7 million into the company's program, including $6.4 million when the research was being performed by Geron Corp. CIRM said that the latest data show that the treatment is safe and should continue with 10 million and 20 million cell doses with new trial participants. CIRM  blog author Todd Dubnicoff said,
"About a month ago, Asterias reported that six of the six participants who had received a 10 million cell dose – which is transplanted directly into the spinal cord at the site of injury – have shown improvement in arm, hand and finger function nine months after the treatment. These outcomes are better than what would be expected by spontaneous recovery often observed in patients without stem cell treatment. So, we’re hopeful for further good news later this year when Asterias expects to provide more safety and efficacy data on participants given the 10 million cell dose as well as the 20 million cell dose."
CIRM's Karen Ring reported on the good news from Capricor, which has received $23.2 million from CIRM, not all of it for Duchenne muscular dystrophy, however. The disease follows a devastating course and significantly reduces life expectancy. Ring wrote,
"The company reported positive results from their Phase I/II HOPE trial that’s testing the safety of their cardiosphere stem cell-based therapy called CAP-1002. The trial had 25 patients, 13 of which received the cells and 12 who received normal treatment. No serious adverse effects were observed suggesting that the treatment is 'generally safe' thus far."
Linda Marban, president of the company said that it plans to seek permission from the FDA to move into one of the agency's programs to expedite making the treatment available.

The announcements on the trial results came during a week when Nature, which reports only intermittently on CIRM, did something of an overview of the California research program. The Nature headlines said,
"California’s $3-billion bet on stem cells faces final test
"Major investment in regenerative medicine enters its last stage — and the money might run out before treatments are ready."
Capricor's stock closed at $3.12 Friday with a 52-week price range of $2.12 to $5.40. Asterias closed at $3.70 with a 52-week range of $2.30 to $5.80.

Below is an Asterias video on its research.

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