Interim results from the Phase 1/2 trial clinical trial investigating CAP-1002 as a treatment for heart disease associated with Duchenne muscular dystrophy (DMD) are being presented today at the 2017 Parent Project Muscular Dystrophy (PPMD) Annual Connect Conference taking place in Chicago.
Linda Marbán, president and CEO of Capricor Therapeutics, is talking about the company’s plans to advance the development of CAP-1002 after a recent meeting with the Food and Drug Administration (FDA).
Those interested in the conference, which concludes July 2, may watch live by clicking here; the streaming is provided by PPMD.
CAP-1002 is a cardiac stem cell therapy under investigation as a treatment for heart disease. This treatment involves using cells from a healthy donor’s heart tissue, which are grown in a laboratory. When ready, these cells are directly introduced into the patient’s heart via infusion into a coronary artery, using regular cardiac catheterization methods.
The trial, called HOPE (NCT02485938), has enrolled 25 patients (ages 12 or older) with DMD-associated heart disease. Researchers are comparing the safety and efficacy of CAP-1002 to usual care. CAP-1002 is administered to the three main coronary arteries to enable broad delivery across the myocardium (the muscular tissue of the heart). After the treatment, the patients will be followed and their hearts examined using magnetic resonance imaging (MRI) scans over the course of one year.
Results released after the first six months of treatment demonstrated that CAP-1002 induces significant improvements in the heart and arm functions, suggesting that this treatment may be a reliable option to improve muscle activity in different parts of the body.
“In HOPE, we saw potential effects in both the heart and skeletal muscle that appear quite compelling in an exploratory trial,” John Jefferies, MD and trial’s principal researcher, said in the April news release. “These results clearly support the conduct of a confirmatory clinical trial in DMD to further evaluate the potential of CAP-1002. We look forward to an effective medication becoming available for people with this progressive and fatal disease, one that is poorly met by current options.”
DMD is characterized by the loss of muscular function, including in the heart and lungs, due to the presence of mutations in the gene encoding the dystrophin protein. In fact, mortality is frequently associated with cardiopulmonary failure due to heart failure. Electrocardiography can detect cardiac impairment in more than half of patients with DMD ages 6 to 10, but early symptoms of heart failure can go undetected.