Results of a phase 3 clinical trial show an experimental drug called tolebrutinib can delay disability in patients with non-relapsing secondary progressive multiple sclerosis.
An experimental drug may slow the progression of a form of multiple sclerosis that increases disability relentlessly, a new study finds.
There are currently no FDA-approved treatments for non-relapsing secondary progressive multiple sclerosis or SPMS, according to Dr. Robert Fox of the Mellen Center for Multiple Sclerosis.
“This form of MS occurs later in the MS disease course, and is characterized by gradual, little by little worsening of neurologic problems. Up to half of people with MS have non-relapsing secondary progressive MS. So, treatment for this form of MS represents a major unmet need in the field of MS.”
In a phase 3 clinical trial, more than 11-hundred patients with SPMS were randomly assigned to receive either 60 milligrams of the drug called tolebrutinib (pronouncer: tohl-ehh-brutinib) or a matching placebo tablet.
Close to 23% of those taking tolebrutinib experienced progression in their MS-related disability, compared to about 31% of those taking placebo.
That’s a 31% reduction of disability with the experimental drug.
Patients in the tolebrutinib group were also more likely to
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experience improvement in disability with therapy
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achieve a sustained 20% increase in a timed 25-foot walk test after three months
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and had fewer new or growing brain and spinal cord lesions associated with MS.
But Dr. Fox says some serious side effects were a bit higher with the drug, including liver irritation.
“And about 0.5% or about one in 200 patients who received tolebrutinib had a very high elevation in liver enzymes, more than 20 times the upper limit of normal.”
If the drug is approved, patients taking it will likely need to be monitored closely during the first three months when the risk appears to be highest.
Still, Dr. Fox says these findings are very exciting. “I think a bright day has come for the have-nots with secondary progressive MS because we have finally found a treatment that may be helpful for them.”
Tolebrutinib appears to work by reducing inflammation in the brain and spinal cord.
Source: New England Journal of Medicine
Author Affiliations: Cleveland Clinic, University of Pennsylvania, University of British Columbia, Instituto de Investigación Sanitaria del Hospital Clínico San Carlos, Universidad Complutense de Madrid, Queen Mary University of London, Blizard Institute, University of Lille, Sanofi, National Institutes of Health
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