Bones, Stem Cells and Bridging the Gap

California's $3 billion stem cell research effort chalked up a small score yesterday with the announcement that a $5.2 million investment is making progress towards development of a therapy to regenerate broken bones. 

Writing on the the state stem cell agency's blog, Karen Ring, social media manager for the agency, said,

"Scientists from Cedars-Sinai Medical Center have developed a new stem cell-based technology in animals that mends broken bones that can’t regenerate on their own. Their research was published today in the journal Science Translational Medicine and was funded in part by a CIRM Early Translational Award."

The award went to Dan Gazit and Hyun Bae at Cedars. Their year one and two progress reports involving the adult stem cell therapy can be found at the link in the above paragraph. 

Ring's  blog item also carried a nifty graphic on the work and a link to a video on the research. 

Ring wrote,

 "Over two million bone grafts are conducted every year to treat bone fractures caused by accidents, trauma, cancer and disease. In cases where the fractures are small, bone can repair itself and heal the injury. In other cases, the fractures are too wide and grafts are required to replace the missing bone.

"It sounds simple, but the bone grafting procedure is far from it and can cause serious problems including graft failure and infection. People that opt to use their own bone (usually from their pelvis) to repair a bone injury can experience intense pain, prolonged recovery time and are at risk for nerve injury and bone instability."

$20 Million, California-backed Stem Cell Trial Discloses Disappointing News

Capricor Therapeutics, Inc., today announced some bad news about its state-of-California financed clinical trial for a cardiac, stem cell therapy and said that it planned to lay off an unspecified number of employees.

Capricor's stock price plummeted 62 percent following what the company called the "unexpected" news, dropping from $1.89 to $1.16. California's stem cell agency has backed the trial with $20 million, plus an additional $7 million for earlier, related research.

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), said on its blog,

"Obviously this is disappointing news for everyone involved, but we know that not all clinical trials are going to be successful. CIRM supported this research because it clearly addressed an unmet medical need and because an earlier Phase 1 study had showed promise in helping prevent decline in heart function after a heart attack."

In response to a question, Kevin McCormack, senior director of communications, said the agency is talking to Capricor about the next steps. The agency regularly halts funding of awards when recipients do not meet milestones. McCormack did not respond to a question about how much money the firm had already received from CIRM.

Capricor, which is headquartered in Beverly Hills, said in a press release that an interim analysis on the phase two trial has "has demonstrated a low probability (futility) of achieving a statistically-significant difference in the 12-month primary efficacy endpoint." The company said there was "no notable difference" between treatment groups.

The company said it would cut its workforce to focus more sharply on its treatment of Duchenne muscular dystrophy, which the stem cell agency is also backing with $3.4 million. The Duchenne treatment had better news the last month, clearing its phase one trial with no adverse effects.

Timothy Henry and Rajenda Makkar of the Cedars-Sinai Heart Institute in Los Angeles are the principal investigators for the clinical trial.

The highest price for Capricor stock over the last 12 months was $5.40 and the lowest was $1.13.

Here are links to additional news stories today on Capricor: BiopharmaDive, MarketWatch, Genetic Engineering News, 

Cedars-Sinai Researcher Snags $5 Million for Stem Cell Eye Research

Another winner in last week's California stem cell awards was scientist Shaomei Wang of Cedars-Sinai, who will receive nearly $5 million for her work to find a treatment for retinitis pigmentosa. 

Shaomei Wang, Cedars photo

The board last Thursday ratified the decision of 15 reviewers who unanimously approved the Wang application. It was one of two awards approved in the first round of CIRM 2.0, the new effort by the agency to speed cash to researchers. It was the first agency award to Wang.

(The other award last week was for nearly $18 million to NeoStem, Inc., of  New York.)

CIRM, as the agency is known, said in a press release that the funds will go for "the late-stage research needed to apply to the Food and Drug Administration for approval for a clinical trial in people. The therapy will involve injecting neural progenitor cells under the retina at the back of the eye. The hope is that this will slow or even halt the progress of the disease."

The press release oddly, however, did not identify Wang as the recipient, only mentioning that the award went to Cedars. In the past, the agency has identified researchers by name in award press releases and has provided information that has included links to summary information about the research.  

Cedars has been awarded $43 million by the agency, not including the latest $5 million. It has had a representative on the CIRM governing board since its inception as do nearly all of the recipients of funds from the $3 billion state program.

Here is a link to a summary of the review of Wang's proposal. Here is Cedars' press release on the award.