According to a recent phase 2 study published in Muscle & Nerve, tocilizumab, an established rheumatoid arthritis (RA) treatment, was found to reduce inflammation in amyotrophic lateral sclerosis (ALS) patients. The drug, which was FDA-approved for RA in 2010, was also demonstrated to be “safe and well-tolerated.”
Researchers at the Barrow Neurological Institute in Arizona tested the effects of tocilizumab in 22 adults with ALS against a placebo control group. All participants were required to have high levels of inflammatory markers in their blood in order to determine that inflammation played a role in their ALS.
The study’s participants received either three intravenous injections of Actemra (the brand name for tocilizumab) once a month, or a placebo. Follow-up took place every four weeks for 16 weeks. During follow-up, patients were monitored for any adverse events, tolerability of the drug, inflammatory markers, and ALS progression. Their ALS progression was measured by the Revised ALS Functional Rating Scale (ALSFRS-R).
Actemra was found to be generally safe, although there were a few reports of side effects, including one severe incident of aspiration pneumonia. However, this occurred in the placebo group. Two patients taking Actemra declined to continue treatment due to adverse effects — one developed a bacterial infection, while the other experienced low white blood cell levels.
Actemra led to an 88% decrease in C-reactive protein (CRP), an inflammatory marker, in the treatment group. The placebo group experienced a 4% increase in CRP.
The treatment group also saw “dramatically increased” levels of IL-6, a marker associated with ALS progression. However, the researchers say this supposed increase wasn’t due to disease progression – rather, Actemra interfered with the body’s normal breakdown of IL-6 without causing an actual increase.
Future use of the drug
Despite the seemingly positive results of the study, the researchers concluded that Actemra, at its current stage, showed no clinical benefit for the treatment of ALS, although future studies could prove differently.
In a final statement, the researchers wrote: “These results warrant further study in ALS patients where IL-6R genotype and CRP levels may be useful enrichment biomarkers.”