Heart disease is the leading cause of death in the United States with over 600,000 deaths occurring per year. Patients with heart disease or heart failure are given treatments that attempt to prevent their condition from getting worse or improve some of their symptoms. However, no treatment exists that can completely restore their heart function except for having a heart transplant – a risky procedure that has significant obstacles associated with it including transplant rejection and limited donor availability.
Regenerative medicine research for heart disease is an up-and-coming field. Scientist and companies are testing stem cell-based therapies to treat patients with heart disease in hopes of improving or restoring heart function.
CIRM is funding a company called Capricor Therapeutics located in Los Angeles, California, that’s testing a stem cell-based therapy in a Phase II clinical trial for cardiac dysfunction called ALLSTAR (ALLogeneic Heart STem Cells to Achieve Myocardial Regeneration). The treatment is called CAP-1002, which is an infusion of allogeneic cardiosphere-derived cells (CDCs). Capricor has shown that CDCs can regenerate tissue in the injured human heart in a previous Phase I clinical trial called CADUCEUS, which treated patients one to three months after they had a heart attack.
This week, Capricor reported that it has passed another milestone in the ALLSTAR trial and finished patient enrollment. Compared to the CADUCEUS trial, the patient population in ALLSTAR was expanded to include individuals that had a heart attack in the past 12 months. The purpose of this expanded patient population is to determine whether CAP-1002 is beneficial to patients with older heart injuries. A total of 142 patients were enrolled in the trial and 134 of those patients received either a single injection of CAP-1002 or a placebo treatment into their coronary artery associated with the heart injury.
In a news release, Capricor President and CEO Linda Marban explained the logic behind the CADUCEUS and ALLSTAR trials for cardiac dysfunction:
“As we and others have shown, CAP-1002 possesses the ability to promote therapeutic regeneration in the injured heart, a powerful concept for the treatment of heart disease. In the CADUCEUS clinical trial, CDCs decreased scar size and increased viable tissue in the hearts of patients who had suffered a large heart attack. In ALLSTAR, not only are we studying a population similar to the one that delivered such astounding results in CADUCEUS (30 – 90 days post-MI), but we have also included patients that were 91 – 365 days post-MI to see if we could extend the indication window. We have also moved to an allogeneic platform from autologous cells.”
ALLSTAR patients will be monitored carefully over the next year to make sure the CAP-1002 treatment is safe. After a year, Capricor will assess the potential regenerative capacity of CAP-1002 by measuring the size of the heart injury and looking for a reduction in scar tissue using magnetic resonance imaging (MRI).
“With the last patient in ALLSTAR having been dosed on September 30th, we expect to report top-line 12-month primary efficacy outcome results in the fourth quarter of 2017,” said Marban. “We are very much looking forward to seeing the results of the ALLSTAR trial because they may show, for the first time in a Phase II clinical trial, that cells can reduce scar and potentially improve outcomes.”
CIRM is also funding another clinical trial by Capricor that’s evaluating CAP-1002 in young boys with cardiomyopathy – diseases that affect heart muscle – resulting from Duchenne muscular dystrophy. The Phase I/II trial called HOPE recently completed its patient enrollment and you can read more about it here on the Stem Cellar.