After a career-long battle waged against multiple sclerosis, neurologist Stephen Hauser, MD, announced the astounding clinical trial results of a drug that stops the disease in its tracks.
Hauser is the inaugural director of the Weill Institute for Neurosciences, an ambitious effort established in June by a $185 million gift from Joan and Sanford I. Weill to accelerate the development of new therapies for diseases affecting the brain and nervous system. Hauser’s announcement, the first of many to follow from the institute, exemplifies the remarkable work made possible by physician-scientists fueled by passion philanthropy.
Hauser, the Robert A. Fishman Distinguished Professor in Neurology, led the 200-center international trial in patients with the most common form of the disease, relapsing-remitting MS. The drug, ocrelizumab, targets white blood cells called B-lymphocytes. Compared to interferon, ocrelizumab reduced clinical MS attacks, blocked the development of new myelin inflammation by over 95 percent, and delayed disability progression by 40 percent.
The results were beyond spectacular. This therapy essentially stops the inflammatory relapsing form of MS in its tracks and appears to do so safely.
“The results were beyond spectacular,” says Hauser, who spent decades collaborating across institutions and industry and bucking the conventional wisdom of a field with a singular focus on therapies that targeted the immune system’s T-cells. Hauser trained his sights on B-cell therapies instead, and the trial results validated his every instinct. Ocrelizumab is the first approved B-cell therapy for MS.
More good news followed. There’s been no therapy for the primary progressive form of the disease (PPMS), which affects 10 to 15 percent of the 400,000 Americans with MS. But a related trial showed that ocrelizumab is 24 percent more effective than a placebo in slowing PPMS’s progression and staving off brain lesions and degeneration.
“We are optimistic that with aggressive therapy that can be given safely at the beginning of the disease, the long-term outcomes, which are measured in decades, will be far superior to what they are today,” says Hauser. “These results should allow us to ask if shutting down brain inflammation at a very early stage in the disease might prevent late progressive MS altogether.”
Read the complete story in UCSF Magazine.