Monday, December 23, 2013

Golden State Milestone: Stem Cell Agency Announces its First Phase Two Clinical Trial

The California stem cell agency today scored a first with the announcement that one of its projects – a heart disease therapy -- has now advanced to a phase two clinical trial after successfully completing a phase one safety trial.

In a press release, Jonathan Thomas, chairman of the $3 billion research effort, hailed the Allstar trial by Capricor Therapeutics, Inc., of Beverly Hills. He declared,
"This is a highly significant announcement for us at CIRM as it's the first time we have funded a therapy into a Phase 2 clinical trial.
"Heart disease claims around 600,000 American lives every year, so clearly there is a huge need for new approaches and more effective therapies. We are hopeful this is the first of many treatments to turn the tide against this disease, and that this will be the first of many projects we are funding to get to a Phase 2 trial."
The adult stem cell therapy “uses unrelated donor-derived stem cells, called cardiosphere-derived cells, that are then infused into a patient’s artery with the aim of reducing scarring caused by heart attacks,” CIRM said.

Capricor Therapeutics, which has its roots in Cedars-Sinai in Los Angeles, describes itself as as “a diversified heart failure biotechnology company.” It came into being last summer as the result of the merger of Capricor and Niles Therapeutics. The company has benefited from about $27 million in support from the stem cell agency, including $6.9 million for early work by one of its founders Eduardo Marbán at Cedars-Sinai.

The company's stock price closed at $2.48 today, down 17 cents. Its 52-week range is from $2.48 to $2.65.

The firm's CEO, Linda Marbán, said the move into phase two was a “giant leap” for the firm and the heart therapy field.

The executive chairman of the firm is Frank Litvack, a heart surgeon who was the only other candidate for chairmanship of the stem cell agency when Thomas won the job in 2011.

The announcement today is a milestone for the stem cell agency, which is aggressively seeking results that will help to generate financial support after 2017, when its current state funding runs out.

The CIRM news release said,
“The next phase will involve an estimated 300 patients who have had heart attacks, and they will be evaluated in a double-blind, randomized, placebo-controlled trial.  This will be further broken down into two groups: one will include patients 30-90 days post attack, the second will be 91 days to one year after the incident.”
The phase two trial is to determine the therapy's effectiveness and further study its safety. If successful, it can move into a phase three trial to confirm its effectiveness, monitor side effects, compare it to common treatments and collect information that will allow the drug or treatment to be used safely.

Here is a CIRM video in which Eduardo Marbán discusses some of the issues involved in the therapy. Here is a link to a 2011 presentation to the CIRM governing board about the early research – a presentation that generated some excitement on the board.
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