reported by : Cherie C Binns RN MSCN
— Neither primary nor secondary endpoints met in progressive multiple sclerosis
by Judy George, Senior Staff Writer, MedPage Today October 30, 2020
High-dose pharmaceutical-grade biotin (MD1003) did not significantly improve disability or walking speed in patients with progressive multiple sclerosis (MS), the phase III SPI2 trial showed.
About 12% of patients treated with biotin compared with 9% of patients who received placebo met the study’s primary outcome of improvement in Expanded Disability Status Scale (EDSS) or the Timed 25-Foot Walk (T25-FW) test (OR 1.35, 95% CI 0.81-2.26), reported Bruce Cree, MD, of the University of California San Francisco, and co-authors.
All predefined endpoints in the study were negative, including serum neurofilament light levels.
“This study found that MD1003 did not improve multiple sclerosis disability,” Cree and colleagues wrote in Lancet Neurology. “High biotin serum concentrations can falsely alter laboratory tests that, if misinterpreted, could result in iatrogenic harm.”
“Therefore, because of this safety concern and the absence of significant efficacy, MD1003 cannot be recommended for treatment of progressive multiple sclerosis,” they added.
The outcomes dashed hopes created by the earlier MS-SPI trial, which found that the water-soluble B vitamin improved disability outcomes over 12 months in progressive MS patients.
But MS-SPI has been criticized for its methodological limitations and may have produced biased results, observed Jeremias Motte, MD, and Ralf Gold, MD, both of Ruhr-University Bochum in Germany, in an accompanying editorial.
“Specifically, the short follow-up of the placebo-controlled phase and the very small placebo effect in MS-SPI (none of the placebo-treated patients achieved the primary endpoint) favored significant differences between biotin-treated and placebo-treated patients,” they pointed out.
Biotin has been discussed as a possible way to treat progressive MS by targeting remyelination, Motte and Gold said. “Biotin, as a cofactor for four essential carboxylases, might support myelin repair by enhancing fatty acid synthesis and protect against hypoxia-driven axonal degeneration by augmenting energy production in neurons,” they wrote.