A promising drug slowed brain shrinkage in progressive
multiple sclerosis (MS) by nearly half, according to new research
led by Cleveland Clinic. Very limited therapies are currently
available for this disabling form of the disease.
The definitive results of the phase 2 trial – published in
the New England Journal of Medicine –
showed that the drug ibudilast decreased progression of brain atrophy in
progressive MS patients by 48% versus placebo. The two-year SPRINT-MS study was
conducted at 28 sites with 255 patients.
“These findings are significant for patients with
progressive MS,” said Robert Fox, MD, the study’s principal investigator
and vice-chair for research in Cleveland Clinic’s Neurological Institute. “Our
hope is that the benefit of ibudilast in slowing brain shrinkage will also
translate to decreased progression of associated physical disabilities in a
future phase 3 trial.”
Progressive MS is associated with gradual worsening of
symptoms and increasing disability. It commonly follows relapsing-remitting MS,
for which there are more than a dozen approved treatments. However, none of
these therapies has consistently demonstrated efficacy in slowing disability
progression in patients with progressive MS, particularly those without
evidence for active inflammation.
Ibudilast, an oral drug with activity on several biologic
pathways with potential relevance to progressive MS, was approved in Japan in
1989 for use in asthma and stroke. It is also being studied in the US for
potential treatment of amyotrophic lateral sclerosis (ALS) and drug addiction.
Additionally, the SPRINT-MS study demonstrated the utility
of advanced imaging in clinical trials to measure the impact of therapies on
brain health. The potential application of imaging-based outcome measures may
extend beyond progressive MS to other neurodegenerative disorders as well.
“There is a significant need for new treatment options to
effectively delay disability progression for patients with progressive MS,”
said Dr Fox. “We are hopeful these findings will help us develop more therapies
for progressive MS and do so more rapidly and efficiently.”
Reference: Fox R,
et al. Phase 2 Trial of Ibudilast in Progressive Multiple Sclerosis. N Engl J
Med 2018; 379:846-855. Published 30 August 2018.