Cell-based Therapy Shows Promise in Treating Duchenne Muscular … – Bioscience Technology

Clinical-stage biotech company Capricor Therapeutics Inc., has announced positive six-month results from a randomized 12-month Phase 1/2 trial in patients with Duchenne muscular dystrophy (DMD), which was designed to analyze safety and exploratory efficacy.

DMD is a rare, genetic disorder that often occurs in boys. It involves progressive muscular weakness and patients often experience frequent falls, have trouble getting up, or performing daily tasks such as eating, as well as learning disabilities, and treatment options are limited.

Cardiac disease is the most common cause of death among those with DMD, who often dont survive past their twenties, and early results from the trial showed statistically significant improvements in measures of cardiac and upper limb function in patients treated with the cell-based therapy called CAP-1002.

CAP-1002 is comprised of allogeneic cardiosphere-derived cells, or CDCs, which are a type of progenitor cell.

The HOPE trial, involved 25 patients, aged 12 years an older, with DMD who had cardiomyopathy, or heart disease secondary to DMD. Thirteen patients were randomized to receive a single dose of CAP-1002, while 12 received usual care. The cell therapy was infused into the three main coronary arties, with a total dose of 75 million cells.

MRI assessments showed that those receiving the cell-therapy showed significant improvements in systolic thickening of the inferior wall of the heart.

The observed signal in global cardiac scar reduction and the increase in the thickening of the left ventricle during contraction are very encouraging, Joao A. C. Lima, M.D., professor of medicine and Director of the magnetic Resonance Imaging Core Lab at Johns Hopkins University School of Medicine, said in a prepared statement. The population treated in HOPE had very advanced cardiac involvement, and to see such positive results following just a single-dose of CAP-1002 is remarkable.

There were also statistically significant improvements in upper limb function, which was assessed using a test designed specifically for use in a DMD setting, called the Performance of the Upper Limb test (PUL). The test simulates common daily activities that patients with DMD often have trouble performing, such as tearing paper or removing a container lid.

The treatment was generally safe and well-tolerated no patients experienced a major adverse cardiac event.

In HOPE, we saw potential effects in both the heart and skeletal muscle that appear quite compelling in an exploratory trial, principal investigator of the trial, John L. Jefferies, M.D., professor of pediatric cardiology and adult cardiovascular diseases at the University of Cincinnati said in a prepared statement. 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.

Capricor plans to request Breakthrough Therapy or Regenerative Medicine Advanced Technology designations for the therapy from the Food and Drug Administration.

The company said it anticipates releasing top-line 12-month data results from the trial during the fourth quarter of 2017.

More here:
Cell-based Therapy Shows Promise in Treating Duchenne Muscular ... - Bioscience Technology