Autologous bone marrow cells are grown in vitro prior to patient administration.
The company released results in November 2010 from the C-Cure study done in Belgium, Serbia, and Switzerland that enrolled 45-patients with severe ischemic heart failure. Six months after treatment, the 21 patients who received injections of the stem cells had an average 18% relative increase in their left ventricular ejection fraction compared with baseline, versus a 4% relative increase over baseline among 24 control patients. Speaking in March at the American College of Cardiology meeting, Dr. André Terzic, who led the study, provided a few more details from the results. Average ejection fraction in the actively-treated patients rose from 28% at baseline to 33%. The treatment was especially effective for the patients who entered the study with an ejection fraction below 28%, said Dr. Terzic, a professor of medicine and cardiology, and director of the Center for Regenerative Medicine at the Mayo Clinic. The 6-month results also showed other indications of benefit in the patients treated with the cardiopoietic stem cells, including significantly increased left ventricular end systolic volume, and reduced left ventricular mass index.
Cardio 3 Biosciences is now planning to start a "late phase II" or phase III trial with this material, said Dr. Simari, who is not directly involved in these studies.
What Comes Next?
Despite 11 years of work, "it’s still very early in the field. We’re still trying to figure out the cells and out targets," said Dr. Simari. "This is the haze of early days, of learning from groups of 10, 20, or 80 patients. Only in retrospect, and as we get to larger trials will the truth emerge. Right now, there is a lot of work needed and few headlines, and it will probably stay that way for awhile. This is not a game for people with short attention spans.
"The business model for autologous cells remains uncertain," he added. "For allogenic products that are generated from young, healthy donors and made into thousands of doses, the business model is different and the costs might be more reasonable. Hopefully, as our knowledge grows, we will develop treatments that end up costing less."
"I have no doubt that cell treatment is one of the great avenues of future medicine; it’s just a matter of figuring it out," said Dr. Perin. "Having phase III trials now starting is a step forward. Things are starting to come into focus. Unless something totally unforeseen happens, we will eventually have an approved product," he predicted.
Dr. Perin said that he has been a consultant to Amorcyte, Teva, Cytori, and Aldagen. Dr. Henry said that he has served on steering committees for and received research grants from Aastrom and Mesoblast. Dr. Marbán said that he is a founder of and holds equity in Capricor. Dr. Simari said that he had no disclosures. Dr. Zeiher said he is a cofounder of and scientific advisor to t2cure, a company set up to run the BAMI trial, sponsored by the European Union Dr. Losordo is an employee of Baxter International. Dr. Terzic said that he has received research grants from Cardio 3 BioSciences.