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Duchenne MD Patients Improve After Stem Cell Therapy.

ANAHEIM, Calif., November. 15, 2017 -- Boys and young men in advanced stages of Duchenne muscular dystrophy experienced significant and sustained improvements in cardiac structure and function, as well as skeletal muscle function, following treatment with Capricor Therapeutics' (CAPR) lead investigational therapy (CAP-1002).

These findings were reported at a late-breaking science session of the American Heart Association Scientific Sessions 2017.

Those treated with CAP-1002 had improvement in cardiac muscle function and reduction in cardiac scarring that were statistically-significant in comparison to the control group, according to a pre-specified analysis.

In addition, in a post-hoc analysis, 89 percent of the CAP-1002 treated patients who were more severely impaired demonstrated sustained or improved skeletal muscle function at 12 months, as compared to none of the participants in the control group.

The positive data are from 12-month results of the randomized, open-label, early stage Phase 1/2 HOPE clinical trial, designed to evaluate safety and explore efficacy.

It enrolled 25 boys and young men in advanced stages of Duchenne muscular dystrophy and was conducted at three U.S. centers. All participants had significant cardiac scarring and approximately two-thirds were wheelchair-dependent at the time they began the trial.

All patients received standard-of-care for Duchenne muscular dystrophy, including oral steroids, and 13 also received one dose of intracoronary CAP-1002 upon randomization.

CAP-1002 consists of allogeneic cardiosphere-derived cells which have been reported to improve muscle function and increase new muscle cell generation in preclinical models of Duchenne muscular dystrophy.

To assess skeletal muscle function, investigators used the Performance of the Upper Limb test (PUL). The PUL tests manual tasks that relate to activities of daily living that are very important for quality of life. PUL has been validated for the assessment of upper limb motor function in individuals with Duchenne muscular dystrophy.

The functional tasks of the PUL are subdivided into three subscales reflecting disease progression from proximal to distal (from the middle of the body outward): (1) High-level: shoulder dimension; (2) Mid-level: elbow dimension; and (3) Distal-level: wrist and finger dimension.

As shoulder function had already been lost in most of the HOPE participants, investigators used the combined Mid-Distal PUL subscales to assess changes in skeletal muscle function and found significant improvement in those treated with CAP-1002 (defined post-hoc).

To assess cardiac structure and function, investigators used magnetic resonance imaging (MRI).

Investigators also found that scarring of the heart muscle among those treated with CAP-1002 decreased relative to the control group. Progressive cardiac scarring eventually impairs the heart's pumping ability and is currently the leading cause of death in Duchenne muscular dystrophy.

CAP-1002 was generally safe and well-tolerated in the HOPE trial. There was no significant difference in the incidence of treatment-emergent adverse events in either group. There were no early study discontinuations due to adverse events.

Duchenne muscular dystrophy causes muscle degeneration and leads to death, generally before the age of 30, most commonly from heart failure.

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Publication:Stem Cell Business News
Date:Nov 27, 2017
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