Duchenne Stem Cell Trial Halted

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2 Jan. 2019. A clinical trial testing stem cell treatments for symptoms of Duchenne muscular dystrophy is on hold after a patient reported a severe reaction. The biotechnology company Capricor Therapeutics Inc. in Los Angeles reported suspension of the study in a filing with the Securities and Exchange Commission late last month.

Capricor Therapeutics develops treatments for rare diseases including Duchenne muscular dystrophy, a rare genetic disorder resulting in progressive muscle degeneration and weakness, primarily in the shoulders, arms, hips, and thighs. The disease affects mainly boys starting at age 3 to 5, and caused by a defective gene that fails to produce the protein dystrophin for strengthening muscle fiber and protecting muscles from injury. While life expectancy can vary, people with Duchenne muscular dystrophy do not often survive past their 20s or 30s.

The company’s lead product, code-named CAP-1002, is designed to regenerate heart tissue from cardiac stem cells. The treatments take a piece of a donor’s or the individual’s own heart tissue, from which are extracted cells known as cardiospheres producing stem cells that are introduced back into the coronary arteries of the patient. Capricor says cardiosphere-derived stem cells release growth factors and cytokines, cells that stimulate specific cell responses, signaling the heart to repair itself. CAP-1002 is also intended to improve skeletal muscle function.

The mid-stage clinical trial is enrolling 84 participants, boys and young men age 10 and higher with Duchenne muscular dystrophy at 14 sites in the U.S. Participants are randomized to receive intravenous doses of 150 million cardiosphere-derived stem cells or placebo infusions every 3 months for a year. The study team is looking primarily for changes in upper limb functioning affecting participants’ quality of life, as well as changes in heart functions.

The study is also assessing safety of the treatments. According to the SEC filing, Capricor is suspending treatments in the trial “pending an ongoing safety review due to a severe allergic reaction that occurred during patient infusion of blinded investigational product.” The company adds, “The patient responded well to medical treatment and is currently asymptomatic.”

Capricor says it alerted the Food and Drug Administration of its decision. In February 2018, FDA granted CAP-1002 expedited review through the agency’s Regenerative Medicine Advanced Therapy designation for regenerative therapies addressing serious medical conditions. In November, the company said it planned to meet with FDA in the following month as part of this expedited process.

More from Science & Enterprise:

• NIH Stops Stem Cell Heart Failure Trial Over Retracted Data
• Trials to Test Stem Cell Treatments for Eye Injuries
• Patents Awarded for Stem Cell Heart Disease Technologies
• Start-Up Developing Regenerative Liver Disease Treatments
• Stem Cell Transplants Found Safe for Spinal Cord Injuries

Hat tip: Endpoints News

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