New Experimental Drug Shows Promise In Slowing Disability
A recent clinical trial has shown that tolebrutinib, an experimental drug, could help patients with advanced multiple sclerosis (MS) slow the progression of disability. The trial focused on individuals with non-relapsing secondary progressive multiple sclerosis (SPMS), a type of MS marked by gradual and relentless neurological decline. The drug was found to reduce the rate of disability progression by 31%, making it the first clinical trial to demonstrate a positive impact on this particular form of MS.
Conducted by Dr. Robert Fox from the Cleveland Clinic, the study involved over 1,100 patients aged 18 to 60 from 31 countries. Participants were randomly assigned to receive either tolebrutinib or a placebo daily. Tolebrutinib, a type of tyrosine kinase inhibitor, works by blocking enzymes responsible for cell growth. After six months, 23% of patients taking tolebrutinib experienced worsening disability, compared to 31% in the placebo group, indicating a 31% reduction in disability progression.
Beyond reducing disability, the drug also offered additional benefits, including improved mobility and a reduction in lesions in the brain and spinal cord. Patients taking tolebrutinib showed greater improvement in their condition and performed better in a timed walking test. However, the drug was associated with a risk of liver damage, as 4% of those taking tolebrutinib showed elevated liver enzymes, compared to 1.6% in the placebo group, which requires careful monitoring.
If approved by the FDA, tolebrutinib could offer a new treatment option for those with SPMS, as there are currently no FDA-approved therapies for this form of MS. Dr. Fox highlighted the drug’s potential to reduce brain and spinal cord inflammation, which could be a major breakthrough for managing MS, a leading cause of disability in young adults. However, due to the liver-related risks, it may require close monitoring during the early stages of treatment.
Funded by the pharmaceutical company Sanofi, the study has sparked optimism for new treatment possibilities for those with SPMS. While the trial results are promising, the liver risks involved underscore the need for further research and careful oversight in patients who use the drug if it becomes widely available.
Discussion about this post