High-dose biotin, a B vitamin used at lower doses as a food supplement, does not significantly improve disability or walking speed in patients with progressive multiple sclerosis, new research shows.
For this reason, and because of the possibility of false lab reports as a result of high biotin serum concentrations, investigators of the randomized, double-blind, placebo-controlled study conclude that high dose biotin is not recommended for treatment of progressive MS.
For investigators involved in the study, the findings are a blow.
“I’m very disappointed,” lead investigator Bruce A. C. Cree, MD, PhD, professor of clinical neurology at the University of California San Francisco, told Medscape Medical News.
“I’ve spent years working on this study and was very excited about the opportunity to develop a product, a non-inflammatory drug that might improve disability in patients with MS.”
The study was published online October 23 in Lancet Neurology.
Most studies of disease modifying therapies in MS use agents designed to modulate or suppress immune function. In contrast, the biotin approach targets myelin.
Biotin is essential for breaking down proteins, carbohydrates, and fats. It’s especially important for healthy skin, hair, eyes, liver, and nervous system functioning.
At low doses, biotin, which is also known as vitamin B7 or vitamin H, is found in some foods and is used as a supplement to treat biotin deficiency, experienced by some pregnant women
MD1003 (MedDay Pharmaceuticals), the agent used in the study, is a highly concentrated oral preparation of biotin. High-dose biotin, explained Cree, potentially addresses two mechanisms — mitochondrial energy failure that seems to occur in MS, and enhancement of myelin metabolism.
Taking high doses of biotin may affect certain blood tests, such as thyroid function tests, some pregnancy blood tests, and cardiac tests.
An earlier pilot study demonstrated safety and efficacy of high-dose biotin in progressive MS, which prompted the design of a phase 2 randomized double blind placebo-controlled trial (MS-SPI).
Results of the MS-SPI study showed that compared with placebo, MD1003 improved disability outcomes over 12 months in patients with progressive MS.
“We were very excited about the results of the first clinical trial. It seemed to work and I was excited of course to see a bigger study replicate it,” said Cree.
The new study, known as MS-SPI2, was designed to extend the observations of the first study in a larger, more diverse patient population.
It included 642 adult patients with primary or secondary progressive MS without relapse in the past 2 years. It was conducted at 90 centers in 13 countries. The mean age of study subjects was 52.7 years, 54% were women, and 65% were diagnosed with secondary progressive MS.
In this patient population, the mean expanded disability status scale (EDSS) score was 5.4, and the mean timed 25-foot walk (TW25) was 11.7 seconds. About 58% of subjects required a walking aid (EDSS 6.0 or 6.5), 46% were receiving concomitant disease-modifying therapies, and 5% had at least one gadolinium-enhancing lesion on MRI.