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 November 15, at American Heart Association (AHA) Scientific Sessions 2017.

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 five 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 explained 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.