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Cardiosphere-derived cells may reverse Duchenne heart scarring

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Elevated troponin a concern

This is a tough disease, and therapies are desperately needed.

The HOPE-Duchenne data look pretty good, and everything seems to be moving in the same direction, but 13 people treated for even 12 months doesn’t really tell us much about safety.

Catherine Hackett/Frontline Medical News

Dr. Robert M. Califf

There is a signal for atrial fibrillation, and every one of these boys had a troponin elevation. That is a signal of some harm, although not clinical harm.

The pipeline for this disease is filling up, but a lot of therapies are aimed at younger patients with the hopes of keeping them functional for as long as possible. I’m glad to see a treatment aimed at more advanced disease.

In general, when the choice is made to enter a clinical trial, it may mean you can’t enter another clinical trial, and these people are in a desperate situation. We need to sort out what should be tested, not just what can be tested.

Robert M. Califf, MD, is professor of cardiology at Duke University, Durham, N.C. He was the study discussant, and wasn’t involved in the work.



On the safety side, nothing unusual happened in the control arm, except a femur fracture.

One boy in the treatment arm went into ventricular fibrillation during the diagnostic angiogram. Five had periprocedural atrial fibrillation. All 13 had periprocedural troponin elevations, versus two boys in the control group over the entire course of the study. Dr. Victor didn’t report any deaths, but safety concerns are probably why Capricor is shifting to intravenous infusion for the next trial, HOPE-2.

Dr. Victor noted that troponin leaks “wax and wane” over time in Duchenne, so the transient increases in the treatment arm “were superimposed on a baseline of abnormal cardiac enzymes.”

The Food and Drug Administration has green-lighted the company’s HOPE-2 trial to start enrollment in early 2018, with a larger number of patients and intravenous CDC delivery at 3 month intervals.

Several investigators were Capricor employees. Others were consultants or reported ownership interests. Dr. Victor was the principal investigator at Cedars-Sinai, and was on the trial’s steering committee. The University of Florida, Gainesville, and Cincinnati Children’s Hospital Medical Center, Ohio, were the other two study sites.

SOURCE: Ronald Victor, MD; 2017 AHA Scientific Sessions abstract number S1177

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