American Heart Association
American Heart Association
November 17, 2017
3 min read

HOPE-Duchenne: Cell therapy shows promise for treating Duchenne cardiomyopathy

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Ronald G. Victor

ANAHEIM, Calif. — Cardiosphere-derived cells to treat patients with cardiomyopathy caused by Duchenne muscular dystrophy was safe and well-tolerated, according to data presented at American Heart Association Scientific Sessions.

Ronald G. Victor, MD, director of the Hypertension Center, associate director of the Heart Institute for Translational Research, professor of medicine and Burns and Allen Chair in Cardiology Research at Cedars-Sinai, and colleagues analyzed data from 25 patients with Duchenne muscular dystrophy aged 12 to 22 years on a stable steroid regimen, left ventricular scar in greater than four segments and an LV ejection fraction greater than 35%.

Patients were assigned to usual care and cardiosphere-derived cell therapy (CAP-1002, Capricor Therapeutics; n = 13; mean age, 17 years) or usual care alone (n = 12; mean age, 19 years). Those assigned cell treatment received 75 million cells through one-time, multivessel intracoronary delivery. Follow-up was conducted at 6 and 12 months. Endpoint of interest included safety, in addition to exploratory efficacy such as cardiac MRI and upper limb performance.

The two groups were well-balanced regarding baseline characteristics, according to the presentation.

“Because the average age was the late teens, which is much older than other Duchenne trials, the majority of patients were non-ambulatory, even more so in the active treatment group,” Victor said.

Serious adverse effects included femur fracture in the usual care group and fever, confusion, urinary tract infection and ventricular fibrillation (one patient during diagnostic angiography before cell infusion) in those assigned cardiosphere-derived cells. Adverse events were consistent with an intracoronary infusion procedure, with five patients having transient atrial fibrillation.

“An important observation is these patients have both continual troponin and [creatinine kinase] leaks that wax and wane over time, so these transient periprocedural increases were superimposed in a baseline of abnormal cardiac enzymes to start with,” Victor said.

A reduced cardiac scar size was seen in patients assigned cardiosphere-derived cells at 6 and 12 months. At 12 months, the scar increased in size in the usual care group by 5% and decreased in the active treatment group by 7%, yielding a statistically significant treatment effect in the change scores (P = .03).

“Decreased scar is really a big deal because it’s counter to the natural history of Duchenne, where the scar always progresses,” Victor said.

Regional systolic wall thickening improved in patients assigned cardiosphere-derived cells, particularly in the inferior wall, the specific segment that is disproportionally affected by Duchenne (P = .04 at 6 months; P = .09 at 12 months). There was a similar trend in the anterior wall and a lesser trend in both the lateral walls, with the septum being least involved with little room for improvement.

Robert M. Califf, MD
Robert M. Califf

At 12 months, upper limb performance either remained unchanged or decreased in the usual care group, but about half of patients in the active treatment group improved. Post hoc responder analysis found that, at 12 months, the performance of the upper limb was either sustained or improved in 89% of patients treated with cardiosphere-derived cells compared with no patients treated with the usual care (P = .007).

“For patients who’ve lost ambulation, this is absolutely key to quality of life, so the ability to use a joystick to drive a scooter, the ability to feed oneself and to use a computer and a cellphone,” Victor said. “Based on these promising early data, further research is warranted and already being planned.”

“The pipeline of therapies for this disease is filling up, but to my knowledge, not in this population,” Robert M. Califf, MD, MACC, professor of medicine and Donald F. Fortin, MD, Professor of Cardiology at Duke University School of Medicine, member of the Duke Clinical Research Institute and former FDA commissioner, said during the presentation. “Most of the therapies are aimed at younger patients with the hopes of keeping people as functional as possible for as long as possible. I’m glad to see a treatment aimed at more advanced disease.”

Eduardo Marbán

“The need is great because there is no current treatment to address heart failure in these patients,” Eduardo Marbán, MD, PhD, director of the Cedars-Sinai Heart Institute and developer of cardiosphere-derived cell technology, said in a press release. “Generally, the primary cause of death in these patients is heart failure. If we can slow or reverse heart failure in Duchenne patients, it will be a step forward.” – by Darlene Dobkowski


Victor RG, et al. LBS.07. Innovative therapies and novel applications. Presented at: American Heart Association Scientific Sessions; Nov. 11-15, 2017; Anaheim, Calif.

Disclosures: The study was sponsored by Capricor. Victor reports he is a site principal investigator for Capricor, Catabasis Pharmaceuticals and Eli Lilly; serves on a steering committee for Capricor and Eli Lilly; is a research grant principal investigator for Coalition Duchenne; and is a global principal investigator for Eli Lilly. Marbán reports financial interests in Capricor. Califf reports no relevant financial disclosures.