A newly approved Drug Treatment For Multiple Sclerosis that is the first to reflect the current scientific understanding of MS is holding new hope for the hundreds of thousands Americans living with the disease.It also highlights the importance of clinician-scientists like UC San Francisco’s Stephen Hauser, MD, who are working to transform research into cures for patients.The Food and Drug Administration on Tuesday approved ocrelizumab (brand name Ocrevus) to treat both relapsing-remitting MS and primary progressive MS, the first time a therapy has been available for severe forms of the disease.This Drug Treatment For Multiple Sclerosis, the first that targets B cells in the human body, grew out of the work of Hauser, chair of neurology at UCSF, whose team persevered for decades in elucidating the science behind MS, defending findings that contradicted long-standing assumptions and translating the science into a therapy.
A Devastating Disease
In MS, the immune system attacks the protective myelin covering around nerve cells, stripping them like insulation from electrical wires. We now know, thanks in large part to Hauser and his colleagues, that immune cells known as B cells lead the attack, mistaking myelin protein for harmful agents. The attacks cause inflammation that disrupts the communication between nerve cells.The first episode of MS typically strikes in early adulthood and the disease advances over the years to devastating disability.
Depending on where the inflammation occurs – the brain, spinal cord or optic nerves – patients can develop muscle weakness, incoordination, bladder difficulties, visual impairment and cognitive problems.In relapsing-remitting MS, the most common form, people experience intermittent flares of symptoms followed by periods of complete or partial recovery.About one in 10 people with MS have the more intractable primary progressive form and experience steady worsening of symptoms without remission.There are no cures for MS, and existing treatments only partially reduce symptoms of relapsing-remitting MS. No therapies at all have been effective for primary progressive MS – until now. The approval of the ocrelizumab Drug Treatment For Multiple Sclerosis comes after studies that have shown remarkable results for both forms of the disease.
The Promise of the New Drug Treatment For Multiple Sclerosis
In December 2016, the New England Journal of Medicine published the spectacular results from a pair of major international clinical trials led by Hauser, confirming that ocrelizumab Drug Treatment For Multiple Sclerosis can block the inflammation that drives the disease. The trials, known as OPERA I and OPERA II, compared the effectiveness and safety of ocrelizumab to that of interferon beta-1a, the current standard treatment for relapsing-remitting MS. Ocrelizumab cut annual relapse rates by 47 percent, reduced disability by 43 percent and shrank inflammatory lesions in the brain by 95 percent compared to interferon.
A separate clinical trial, known as ORATORIO, found that ocrelizumab Drug Treatment For Multiple Sclerosis could modestly slow the advance of primary progressive MS, which until now has been resistant to treatment.These Phase III trials have shown that ocrelizumab, an antibody that targets the CD20 molecule on the surface of B cells, is extremely effective for relapsing-remitting MS, which is caused by B cells circulating in the blood and bound for the nervous system, where they orchestrate inflammation. These B cells – about 2 percent of the B cells in the body – are destroyed reliably and for several months with each dose of ocrelizumab Drug Treatment For Multiple Sclerosis, which is given by infusion every six months. The trials were sponsored by F. Hoffman-La Roche (Roche), the company that holds the patent on ocrelizumab.
“The availability of a highly effective and well-tolerated treatment means that people at the dawn of their MS can be treated with a therapy that will essentially completely block the inflammation in myelin that causes relapses and remission,” said Hauser, director of the UCSF Weill Institute for Neurosciences. “And we are optimistic that by doing so, the outlook over many years will be even more favorable than it is today.”Researchers believe that primary progressive MS is caused by the other 98 percent of our B cells, which live in protected areas within brain tissue. Ocrelizumab is only modestly effective at targeting these hidden B cells. But it is nonetheless the first therapy to have any effect on primary progressive MS.