The treatment options for progressive MS are limited – so interest was high when the study data on the use of high-dose biotin in progressive MS was first presented at the American Neurology Congress in 2015. The result: High-dose biotin is not effective. Despite some limitations of the study (small number of cases, mixed population of secondary and primary chronic progressive MS, unusual primary endpoint), the results were surprising – while almost 13% of patients who had received 300 mg of biotin showed a confirmed EDSS improvement after 12 months, this was not the case in any patient (0%) in the placebo group. While most scientists urged caution and demanded confirmation of the results, the public reaction was enthusiastic from the start. Enterprising companies began to market biotin, pharmacies produced high-dose biotin as a compounded prescription, and many patients were swept up by the resulting “hype” – incidentally, calls for accelerated approval of high-dose biotin for progressive MS became increasingly loud.
It is therefore good that the approval authorities (as recently in the pandemic) have remained true to their principles and demanded a phase III study from the manufacturing company (the French pharmaceutical company Medday) to confirm the first “Proof of Concept” study. These study results appeared online at the end of October 2020 and in print in Lancet Neurology in December 2020 (Safety and efficacy of MD1003 (high-dose biotin) in patients with progressive multiple sclerosis (SPI2): a randomised, double-blind, placebo-controlled, phase 3 trial – PubMed (nih.gov)) – so it’s been a while, but since biotin is still occasionally asked about, I would like to address the topic one last time.
No significant advantage of high-dose biotin
It must be said about this new study that it was excellently made and very carefully conducted in over 90 academic centers in over 13 countries. In total, over 300 patients with progressive MS were included in each the biotin or placebo group. The primary endpoint was (in reference to the first “Proof of Concept Study”) a combined parameter from the proportion of patients with improvement in the EDSS disability scale and/or the proportion of patients with a relevant improvement in the 7.6 m walking distance (25 TFW) after 12 months of treatment (confirmed after 15 months) compared to baseline values. But other conventional study parameters, such as EDSS progression and the safety of the substance were examined. or the change in walking speed was measured.
With regard to the primary endpoint, 39 (12%) patients in the treatment group and 29 (9%) patients in the placebo group improved – this difference was not significant. No advantage for the treatment with high-dose biotin resulted from all other evaluations either. Treatment-related side effects occurred in 84% of patients in the biotin group and in 85% of placebo patients, serious side effects occurred in 26% of study participants each. In summary, it can be stated that biotin is a safe but ineffective substance for progressive MS – and this is also an unequivocal result considering the quality of the study: Biotin is a dead end!
Biotin as an instructive story
Consequently, the administration of high-dose biotin in MS cannot be recommended. Given that many laboratory detection systems are based on a streptavidin-biotin system, taking high-dose biotin very often leads to falsification of laboratory determinations, which can potentially be dangerous. Therefore, I would even go further and say that the intake of high-dose biotin should be completely avoided.
The story about biotin is also instructive because it again shows that the recommendation and application of a concept should only be made after effectiveness has been proven by an adequate study program – and this is still pending for many “concepts” that are currently being hyped.
Older posts on high-dose biotin on amsel.de:
