Alpha Clinic Kickoff: Launch of Capricor Trial for Duchenne in $50 Million California Stem Cell Network

UC Davis this week inaugurated its Alpha Clinic program, part of a $50 million, statewide network created by California's 13-year-old stem cell research agency.

The school kicked off its program with the start of a "HOPE-2" clinical trial for Duchenne muscular dystrophy – a fatal genetic disorder mainly affecting boys and young men. About 200,000 persons worldwide suffer with the affliction. There is no cure. Treatment options are limited.

Craig McDonald
UC Davis photo

The lead trial investigator, Craig McDonald of UC Davis, said in a news release,

"Collaborating with the Alpha Clinic team enables us to capitalize on their research infrastructure and expertise for clinical trials focused on cell-based therapies.

"It complements the skills of UC Davis’ Neuromuscular Research Unit, which is a national leader in conducting Duchenne trials. We believe this unique partnership could be a model for translating stem cell discoveries into meaningful treatments for patients with muscular dystrophy and other serious progressive neurologic diseases.”

The trial will test the safety and efficacy of a therapy developed by Capricor, Inc., of Beverly Hills -- CAP-1002. The state's stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM), in 2016 awarded the firm $3.4 million, part of a total of $17.8 million that the firm has received from the state. CIRM has also awarded another $7 million to Cedars-Sinai for early development work that led to the creation of Capricor.  

(Here is a link to the summary of the review of the Capricor application, CLIN2-08334, submitted by Deborah Ascheim, chief medical officer at Capricor.)

CIRM governing board member Jeff Sheehy said in 2016,

"This is pretty much a pure CIRM product. They came into our first disease team to develop the product. We've supported two of the three clinical trials. So if this turns out to be a major success, this will be a real feather for CIRM. We've been with them all the way. So I'm optimistic."

Linda Marban, president of the firm, said in a news release that the research is "one of the very few clinical initiatives to focus on helping boys and young men whose ability to walk has been seriously impaired by the loss of muscle function that occurs as Duchenne muscular dystrophy progresses."

Capricor is a publicly traded firm, whose stock closed at $1.32 today. The 52-week high on the stock was $4.25 and its  52-week low 63 cents.

The Alpha Clinics are one of the signature programs of the stem cell agency. In addition to Davis, UC San Francisco, the City of Hope, UCLA, UC Irvine and UC San Diego are part of the network. UC Davis last year received $8 million from CIRM to help create its program.