LOS ANGELES, July 27, 2017 — Capricor Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology company developing biological therapies for Duchenne muscular dystrophy (DMD) and other rare diseases, today announced that it has received official minutes of the meeting held recently between the U.S. Food and Drug Administration (FDA) and Capricor to discuss the development of intravenous CAP-1002 (allogeneic cardiosphere-derived cells) for the treatment of Duchenne muscular dystrophy (DMD).
How are Stem Cells Making an Impact on Treatment for Duchenne Muscular Dystrophy?
Hear Dr. Eduardo Marbán, Director of the Cedars-Sinai Heart Institute, speak about how breakthroughs in the application of stem cells for diseases related to the heart have led to new possibilities in treating Duchenne muscular dystrophy. See the video below, followed by some key points to take away from this post.
Key Points Include:
- After discovering the heart possesses its own supply of stem cells, Dr. Marbán and his team worked to isolate these stem cells and grow them. When the stem cells were reintroduced to patients who had suffered heart attacks, the cells reformed heart tissue and reduced scarring!
- With help through a grant from Coalition Duchenne, a non-profit dedicated to raising awareness of and funding for Duchenne muscular dystrophy (DMD), Dr. Marbán and his team have begun to apply their findings for treatment of DMD
- DMD is a form of muscular dystrophy caused by a lack of production of dystrophin, a protein that connects muscle fibers together. It is a rare genetic disorder linked with the X chromosome, and therefore primarily affects boys
- Trials in which stem cells were injected in the hearts of mice with a DMD-like phenotype showed that not only did their heart function improve and scar tissue shrink, but the mice showed enhanced motor function
- The success of the mice trials gives major promise for human testing. If scar tissue is shown to reduce over a longterm period following treatment, it would be a major victory for patients tackling DMD!
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