Research in primary progressive MS (PPMS) - National Multiple Sclerosis Society

Skip to navigation Skip to content

Research in PPMS

Share

Research is ongoing, funded by the National MS Society and others, to find solutions for people affected by primary progressive MS.
  • The experimental therapy ocrelizumab moderately slowed the progression of disability compared to placebo in 732 people with primary progressive MS – the first large-scale clinical trial to show positive results in primary progressive MS. Ocrelizumab has been granted “Breakthrough Therapy designation” by the U.S. Food and Drug Administration (FDA) for the treatment of people with primary-progressive MS. Read more
  • The Society is funding the MS Microbiome Consortium, which is comparing the gut bacteria of people with relapsing MS, people with primary progressive MS, and healthy controls. They believe that significant differences in gut bacteria exist among these groups that may drive MS progression. The findings will help the Consortium toward its goal of developing biomarkers of MS progression, as well as novel therapeutic approaches based on personalized probiotics.
  • The experimental oral therapy MN-166 (ibudilast) has been designated by the U.S. Food and Drug Administration as a “Fast Track Product” in terms of its development as a possible treatment of progressive MS, including primary progressive and secondary progressive MS. Investigators are currently conducting a phase II clinical trial of ibudilast in 250 people with progressive forms of MS, principally funded by the National Institutes of Neurological Diseases and Stroke (NINDS), with additional support by MediciNova and the National MS Society. Read more 
  • In a study of 453 people described as having radiologically isolated syndrome (specific areas of damage on MRI scans with no accompanying symptoms), about 12% eventually developed primary-progressive MS. Those who developed primary-progressive MS were more likely to be men, were significantly older, and were more likely to have MS-like lesions in the spinal cord compared to those who went on to develop clinically isolated syndrome or relapsing-remitting MS. This study provides a rare glimpse of a very early stage of disease. Finding a way to identify and track primary-progressive MS earlier may help to improve access to care for those who have it. Read more

Clinical trials

People who have received a diagnosis of PPMS are often frustrated by the relatively small number of clinical trials in PPMS compared to the large number in RRMS. MS clinicians and researchers share this frustration and are actively looking for ways to increase the number of trials of treatments for PPMS, addressing several obstacles:

  • The disease modifying medications currently used to treat relapsing forms of MS primarily target inflammation in the central nervous system (CNS). Because inflammation plays a much smaller role in PPMS than in relapsing forms of MS, these medications do not seem to be as effective in PPMS — which means that new treatment targets need to be identified.
  • In PPMS, there is a lack of easily-identifiable outcomes to measure in clinical trials. In the trials for the approved disease-modifying therapies, investigators looked at outcomes such as number of relapses and number of new lesions (also called plaques) seen on magnetic resonance imaging (MRI) to determine if people who received the treatment had lower numbers than those who received a placebo (non-active substance). The outcome measurements do not adequately quantify disease progression in the PPMS group.
  • Disease progression in PPMS can be quite slow, making the ability to identify an effect on progression difficult in a two- or three-year trial.

Researchers are working to understand why some people experience aggressive worsening of MS and others experience a milder course, and to identify other ways to measure the changes that occur in PPMS so that they can more easily test potential treatments.

Read more about research in all forms of progressive MS.

Treatment Options and Research Advances in Progressive MS

Benjamin Segal, MD, discusses treatment options and research advances in Progressive MS

Watch now

Share

Working For Me: Research In Understanding and Treating Progressive MS

Watch scientists, clinicians, and those living with the disease discuss: causes of MS progression, who is more likely to have a progressive course, and what can be done to stop progression. 

Watch now