ALLSTAR: Mixed results for cardiosphere-derived cells in MI, reduced LVEF
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In patients with MI, reduced left ventricular ejection fraction and large LV scar size, cardiosphere-derived cells did not reduce scar size but lowered LV volume and levels of a key biomarker, according to results of the ALLSTAR trial.
The trial results, recently published in the European Heart Journal, were initially presented at the 2017 American Heart Association Scientific Sessions.
Raj R. Makkar, MD, vice president of cardiovascular innovation and intervention and the Stephen R. Corday, MD, chair in interventional cardiology at Cedars-Sinai, and colleagues randomly assigned on a 2:1 basis 142 patients (mean age, 55 years; 85% men), of whom 134 were treated, to receive an intracoronary infusion of cardiosphere-derived cells (CAP-1002, Capricor Therapeutics) or placebo.
All patients had MI 4 weeks to 12 months prior to randomization, LVEF 45% or less and scar size 15% of LV mass or more on MRI (mean LVEF, 40%; mean LV scar size, 22%).
The trial was stopped early for futility after a 6-month interim analysis of the primary efficacy outcome of relative percentage change of infarct size after infusion as assessed by cardiac MRI, the researchers wrote.
"We think we may have chosen the wrong endpoint," Eduardo Marbán, MD, PhD, executive director of the Smidt Heart Institute and the Mark S. Siegel Family Foundation Distinguished Professor at Cedars-Sinai, said in a press release. "This happens in science because you have to design the trial a year or more before you begin, and sometimes you bet on the wrong horse ... but that doesn’t necessarily mean the therapy is ineffective.”
At 6 months, there was no difference in the primary efficacy outcome between the cell therapy and placebo groups (P = .51). There were no instances in either group of the primary safety outcome of acute myocarditis attributable to allogeneic cardiosphere-derived cells, ventricular tachycardia- or ventricular fibrillation-related death, sudden unexpected death, or a major adverse cardiac event — defined as death, hospitalization for HF, nonfatal MI or need for LV assist device or heart transplant — at 1 month.
However, compared with the placebo group, the cell therapy group had reductions in LV end-systolic volume, LV end-diastolic volume and N-terminal pro B-type natriuretic peptide (P = .02 for all) at 6 months, according to the researchers.
“To me, these data are very reassuring that there really is therapeutic benefit,” Marbán said in the release. “There is a growing body of evidence that this cell treatment does work.”
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