Science & Enterprise subscription

Follow us on Twitter

  • New contributed post on Science and Enterprise: https://t.co/LZVxve0cm5 Making an Excellent First Impression in Business
    about 2 hours ago
  • A research team in Europe and Israel is devising a new process for faster bio-printing of human organs with three-d… https://t.co/izmHrVwd2Q
    about 19 hours ago
  • New post on Science and Enterprise: High-Speed 3-D Organ Printing in Development https://t.co/31d99lRLcL #Science #Business
    about 19 hours ago
  • Medical and robotics labs at two universities in Pittsburgh are developing a portable, autonomous trauma care devic… https://t.co/G8HKYrLfgi
    about 23 hours ago
  • New post on Science and Enterprise: A.I., Robotics Studied for Military Trauma System https://t.co/jkPG42ohGN #Science #Business
    about 23 hours ago

Please share Science & Enterprise

RSS
Follow by Email
Facebook
Facebook
Google+
Twitter
Visit Us
LinkedIn
INSTAGRAM

Duchenne Stem Cell Trial Halted

Stop signs graphic

(kropekk_pl, Pixabay)

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:

Hat tip: Endpoints News

*     *     *

Please share Science & Enterprise ...
error

1 comment to Duchenne Stem Cell Trial Halted