Ten to 15 percent of people with MS are diagnosed with primary progressive MS (PPMS). PPMS is characterized by progressive accumulation of disability from the condition’s onset, without any marked improvements or relapses.
Unlike relapsing remitting MS which has a number of disease modifying therapies to mitigate the condition, there are no effective treatments for PPMS - therefore it is crucial that greater research is undertaken into every aspect of PPMS.
Idebenone, a manmade drug, is similar to a naturally occurring dietary supplement, coenzyme Q10. Some research indicates that Idebenone may be able to limit demyelination and death of brain cells and thereby slow or halt the progression of neurological dysfunction such as that occurring in MS.
Prior Idebenone research has shown it to be safe and well tolerated by participants of trials, but has provided limited evidence that the drug is effective. Santhera Pharmaceuticals are developing it under the name Catena researching its effectiveness in five conditions, including PPMS.
It has been approved for use in Friedreich's Ataxia (FA), a genetic neuromuscular disorder, in Canada though its effectiveness is still under review, and it is in varying stages of development for other neuromuscular and mitochondrial diseases.
Published data indicate that higher doses (10-50 mg/kg) of Idebenone per day are required for beneficial effects on neurological disability in comparison to the lower doses (5-10mg/kg) that are sufficient for beneficial effects on cardiac/systemic functions in FA patients.
Therefore, the objective of the upcoming clinical trial, is to assess whether Idebenone is safe and effective and hopefully gain an understanding of how it might work in people with PPMS.
For this reason high dose Idebenone or a placebo will be given to 80 enrolled participants with PPMS in order to assess the effects of Idebenone in people with PPMS.
The trial has also been designed to be adaptive in order to ensure any significant findings will be noted, in the event that either structural (imaging results) or functional (symptomatic alleviation) benefits are discovered from taking Idebenone.
We look forward to this trial progressing and the publication of its findings.