High-dose Biotin Clinical Trial Results

Research Summary: MD1003 (high-dose biotin) for the treatment of multiple sclerosis: A randomised, double-blind, placebo-controlled study

The background

Whilst many treatments are now available for relapsing-remitting multiple sclerosis, very few have proven to be effective for progressive forms of the disease.  In fact, no currently available therapies have shown any benefits against ‘non-active’ progressive MS.

In ‘non-active’ progressive MS, the increasing levels of disability are most likely caused by accumulated damage to nerves (in the form of demyelination) and problems with mitochondria (the energy production center of the cell).

In a small pilot study, MD1003 – a formulation of biotin that is 10,000 times the recommended daily intake – was shown to be effective in people with ‘non-active’ progressive MS.   In this 23 person trial, over 90% showed some clinical improvement.  Based on these results, a larger trial of MD1003 was undertaken.

The study

The study was conducted in two phases.  The first stage was a 12 month placebo controlled trial.  The participants were randomly grouped into those receiving treatment (n=103) or placebo (n=51) and were given a 100mg tablet, three times a day.  After the completion of this phase, a second stage 12 month extension trial was performed, whereby all participants received MD1003.

All of the participants in the study were 18-75 years old with non-active progressive MS and an EDSS between 4.5-7.  Study participants were allowed to continue taking associated medications, as long as they had begun taking them at least 3 months prior to the trial starting.

The success of the trial was determined through measuring changes in EDSS and a timed 25-foot walk.  These were used as ways of assessing reduced disability.  Throughout the trial, data was also collected on fatigue, cognition and the overall well-being of the participant, as assessed by both the individual and their clinician.

The findings

The findings showed that high-dose biotin could result in an improvement in disability in a small percentage of people with progressive MS.  This was highlight from the following results:

• 12.6% of the biotin group showed an improvement in disability at the 9 month point, which was confirmed at 12 months.  This was compared to 0% of the placebo group.

• During the second 12 months, 7.1% of the group that switched from placebo to biotin showed improvements in disability.

• At 24 months, 15.4% of the original biotin group and 11.9% of the placebo to biotin group showed improvements in disability.

• EDSS progression was reduced in the biotin group at the 9 month point.  Only 4.2% of people on biotin had seen an increase in EDSS compared to 13.6% of the placebo group.

• There was no significant difference in the time taken to do the 25 foot walk.   However, the proportion of people who progressed to the point of being unable to complete this task was lower in the biotin group.

• The general well-being of participants was significantly better in the biotin group, as determined by both their own reporting and that of their clinician.

• Some adverse effects were reported throughout the study.  The levels of these were similar between both the biotin and placebo groups and no major adverse effects were associated with the biotin itself.

The outcomes

This study has shown some exciting results, as this is one of the first treatments to show the potential to improve disability in people with progressive MS.  It is important to note that these benefits were only seen in a small percentage of the people taking biotin, however, the effect was highly specific to the treatment group.

Importantly, no significant safety issues were reported across the length of the study.  However, it will be important to assess this over a longer period of time to ensure that no long-term effects are associated with the use of high-dose biotin.

The abstract for this article can be viewed here.