Treatment with the cell-based therapy CAP-1002 may reduce scar size in hearts of patients with Duchenne muscular dystrophy (DMD), according to early clinical data from the HOPE-Duchenne trial presented Nov. 15 at AHA 2017 in Anaheim, CA.

Ronald G. Victor, MD, FACC, et al., looked at 25 male patients with DMD and a mean age of 17.8 years who were randomized to receive either standard medical care plus cell-based therapy CAP-1002 (75 million allogeneic cardiosphere-derived cells) or standard medical care alone.

Results showed that at six months, there was approximately a 5 percent reduction in scar size in the hearts of those who received CAP-1002 as measured by MRI vs. the standard medical care group. The researchers explain that "although this did not achieve statistical significance (p=0.09), this is a notable observation since scar progressively increases over time in DMD."

The researchers conclude that "exploratory efficacy analyses signal a potential benefit for CAP-1002 for patients with advanced DMD." They explain that skeletal and cardiac muscle function in this population will be the primary endpoint in the planned phase 2, double-blind, randomized HOPE-2 Trial expected to begin in early 2018.

"This small study showed no statistical benefit of the cell therapy. Therefore we have no confidence that this highly expensive treatment actually works. We need much larger studies in order to tease out whether there is benefit and if so, in which patients," commented Kim A. Eagle, MD, MACC, editor-in-chief of ACC.org.

Keywords: AHA17, AHA Annual Scientific Sessions, Cardiomyopathies, Albumins, Polyesters

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