There could be a substantial advance in the treatment of multiple sclerosis (MS) on the horizon. An possibly an unprecedented advance, at that.
MS is an autoimmune disease, in which the myelin sheath (the cover of nerve cells) is attacked by the immune system, resulting in serious and progressive neurological dysfunction, especially in the brain, eyes and spinal cord. Muscle weakness, loss of balance, extreme fatigue and vision loss often lead to permanent disability. About 400,000 people in the U.S. and 2.3 million worldwide suffer from the disease.
There are two distinct types of the disease. The most common is relapsing MS, which is characterized by periods of remission and flare ups. About 85 percent of MS sufferers have this type of disease. There are now a dozen disease-modifying drugs to treat relapsing MS, but none of them treat the primary form (primary progressive multiple sclerosis, PPMS), which is more severe.
Thanks to Roche, this may change.
In Phase III trials, ocrelizumab, an antibody which is given intravenously twice per year, has been shown to treat both MS types, which has never been done before.
Dr. Sandra Horning, the Chief Medical Officer and Head of Global Product Development of the company said, "people with the primary progressive form of MS typically experience symptoms that continuously worsen after the onset of their disease, and there are no approved treatments for this debilitating condition ... ocrelizumab is the first investigational medicine to show a clinically meaningful and statistically significant effect on the progression of disease in primary progressive MS."
The goal of the 12-week trial was to determine if the drug could reduce the disability from PPMS, which it did. Furthermore, the safety profile, especially for an immune-modifying drug, was excellent, similar to that of placebo. The company stated that the most common adverse events were "mild-to-moderate infusion-related reactions." Although there were some serious adverse events associated with the use of ocrelizumab, most notably infections, the rate was similar to that of placebo.
Roche will be filing for FDA approval in early 2016.
Although Roche has not discussed the drug's price, it is likely to be high, as is the case with almost all new injectable drugs. But ocrelizumab could become a valuable weapon against a very debilitating disease.