Results Announced from Clinical Trial of Idebenone in Primary Progressive MS

  • Results from a 3-year clinical trial conducted by researchers at the U.S. National Institutes of Health suggest that an oral compound called idebenone, a synthetic version of a dietary supplement called coenzyme Q10, was safe in people with primary progressive MS.
  • After two years on treatment, there were no differences in disability progression or other indicators of benefit between those taking idebenone and those on inactive placebo.
  • Individuals who participated in the trial are being invited to join a one-year followup observational study in which everyone will take idebenone for a year, to further evaluate its effectiveness and safety.

“Although this trial did not have enough patients to adequately assess if idebenone canslow primary progressive MS over two years, we are encouraged that the company and investigators undertook the study. The trial design and the outcome measures used will help inform knowledge about progressive MS, and aid in the design of future studies aimed at treating progressive forms of multiple sclerosis.”

Prof. Alan Thompson, Chair of the Alliance’s Scientific Steering Committee and Dean of University College London’s Faculty of Brain Sciences.

 

Background: Idebenone is a synthetic form of a naturally occurring compound known as coenzyme Q10, a common dietary supplement. Preliminary research had suggested that idebenone might be able to limit nervous system damage to slow progressive disability in MS. Idebenone is being tested in other neurological and muscular conditions.

The Study: This 3-year, phase I/II clinical trial was conducted by Bibiana Bielekova, MD, and others at the National Institutes of Neurological Disorders and Stroke. The trial recruited 77 people with primary progressive MS; ultimately 66 completed the trial. For the first year, participants were monitored, and then for the following two years, half received oral idebenone and half received inactive placebo. A variety of testing occurred over the course of the trial, including MRI imaging, blood and spinal fluid testing, and measures of disability to monitor safety and signs of potential benefit. The team developed a disability rating scale called CombiWISE to estimate potential benefit.

The preliminary results were announced by a company, Santhera Pharmaceuticals, which markets idebenone for another condition. According to the company’s press release, the treatment was safe and well tolerated. The assessments of disability progression and other tests showed no apparent benefit of idebenone over placebo after two years of treatment.

Individuals who participated in the trial are being invited to join a one-year followup observational study in which everyone will take idebenone for a year, to further evaluate its effectiveness and safety.

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