Assistant Managing Editor

Finding ways to combat the cardiac degeneration that follows a heart attack has been one of the challenges of the regenerative medicine space, but Capricor Inc. has yielded impressive preclinical data so far using its cardiosphere-derived stem cells (CDCs) and hopes to mirror those results in clinical testing, expected to start early next year.

A 2006 spinout of Johns Hopkins University School of Medicine, Capricor was founded by Eduardo Marbán, then the chief of cardiology at Johns Hopkins, who along with Italian collaborators discovered stem cells in the heart. Marbán then "developed a method to grow the stem cells," creating CDCs, said Oliver Foellmer, Capricor's president and CEO.

Capricor licensed rights to that technology from Johns Hopkins and the University of Rome and began making headway on the lead product candidate, CPI-003, an autologous adult stem cell product initially aimed at treating myocardial infarction and chronic heart failure, Foellmer said.

The process is fairly simple. Heart stem cells from a recent heart attack patient are obtained via catheter in "an extremely small routine biopsy procedure," Foellmer told BioWorld Today. Then those cells are "put into a proprietary culture and expanded to dose levels."

The CDCs are put back into the patient about six to eight weeks later and, because they are the patient's own cells, there's no risk of rejection.

Preclinical data recently presented at the American Heart Association in New Orleans showed that when the company's CDCs were transplanted into a mouse model of myocardial infarction, they were able to protect the heart significantly better against progressive degeneration following a heart attack compared to highly purified cardiac stem cells or heart-derived mesenchymal-like progenitor cells.

In chronic heart failure, Capricor is hoping its CDCs can "shift the disease paradigm," Foellmer said. Chronic heart failure patients, who usually have had a myocardial infarction in the past, typically face a decline in heart function and possibly ischemic trouble until it leads to death. By delivering CDCs to the damaged heart muscle, Capricor's technology actually might be able to benefit patients by maintaining, or even improving, heart function.

The company is in the process of setting up a Phase I safety trial in heart attack patients. "We're hoping to submit an investigational new drug application by the end of this year," Foellmer said, "and to start the trial in the first quarter of 2009."

He added that based on discussions with the FDA, the company might be able to move from Phase I - which is expected to test two different dose levels and, hopefully, provide "a good indication of efficacy" - straight into pivotal studies.

Capricor likely will look to partner the program down the road, and probably will continue working with academic collaborators. The firm raised $3 million in late 2006 and is in the process of raising additional money, which Foellmer conceded is tough to do in the current market, especially for a small firm.

"It's also very difficult to get funding in stem cells," he added. "Just like the antibodies of the past, they need to prove themselves.

"So it's been a difficult road, but we're confident we can raise money," he said, adding that the company is working with some great existing investors.

Originally based in Baltimore, Capricor moved to Los Angeles in mid-2007 to accommodate founder Marbán, who accepted the position of director of the Heart Institute at Cedars Sinai Medical Center.