HOUSTON - (Dec. 21, 2016) - Positive results of an investigational medication study for primary progressive multiple sclerosis were published online in today's New England Journal of Medicine in a paper led by senior author Jerry Wolinsky, M.D., of McGovern Medical School at The University of Texas Health Science Center at Houston (UTHealth).
The study, called ORATORIO, was one of three published in the journal about the medication ocrelizumab (OCREVUS®), which was designed to target immune cells called CD20-positive B cells that contribute to myelin and axonal nerve damage. That damage can lead to disability in people with multiple sclerosis (MS).
Wolinsky, now emeritus professor of neurology at McGovern Medical School at UTHealth, was director of the Multiple Sclerosis Research Group at UTHealth at the time of the study
"Understanding what drives disability in the progressive phases of MS has been one of my focuses over the last several decades," Wolinsky said. "We are finally showing that applying highly effective anti-inflammatory therapies that substantially limit new lesion formation makes a clinical difference in primary progressive MS. This not only opens this anti-B cell therapy as a proven approach for our patients, it also should serve to redouble our efforts to find novel agents to limit tissue damage as well as attempts to facilitate repair. We have made great strides, but much work remains to be done."
Wolinsky was a co-author of a second paper published today in the journal that detailed positive results of two other studies, OPERA I and OPERA II. In those studies, ocrelizumab was compared with interferon beta-1a for patients with relapsing MS, the most common form of the disease.
MS occurs when the immune system abnormally attacks the insulation and support around nerve cells (myelin sheath) in the brain, spinal cord and optic nerves, causing inflammation and consequent damage. This damage can cause a wide range of symptoms, including muscle weakness, fatigue and difficulty seeing, and may eventually lead to disability.
Most people with MS experience their first symptom between 20 and 40 years of age, making the disease the leading cause of non-traumatic disability in younger adults.
Primary progressive multiple sclerosis (PPMS) is a debilitating form of the disease marked by steadily worsening symptoms, but typically without distinct relapses or periods of remission. Approximately 10-15 percent of people with MS are diagnosed with the primary progressive form of the disease.
There are no approved treatments for PPMS. Results of the phase 3 trial, which compared ocrelizumab with placebo, showed a reduction in the progression of clinical disability, which was sustained for 12 weeks. The secondary endpoints in all three studies were also met, including multiple measures of disability progression and brain lesion activity.
During ORATORIO, 732 patients either received intravenous infusions of OCREVUS or placebo. In contrast to the OPERA I and OPERA II studies, where the blinded treatment period was two years, the blinded treatment period of the ORATORIO study continued beyond that until all patients had received at least 120 weeks of either OCREVUS or placebo and a predefined number of confirmed disability progression events was reached overall in the study.