The phase 3 trial, the results of which were published on October 23 in The Lancet Neurology, found that 12 percent of people who received the high-dose form of the vitamin, called MD1003 and manufactured by MedDay Pharmaceuticals, at a dose of 100 milligrams (mg) three times per day, showed improvement on the expanded disability status scale (EDSS) and the timed 25-foot walk (T25-FW) test. EDSS and T25-FW are both commonly used assessments of disability in MS. But biotin’s performance was only slightly better than that of the trial’s placebo, according to the study authors. In all, 9 percent of the study participants with progressive MS who received placebo treatments improved their EDSS and T25-FW results. “The study did not find a beneficial effect of high-dose biotin in improving disability outcomes in persons with not-active progressive forms of MS,” says the lead author, Bruce A. C. Cree, MD, PhD, the George A. Zimmermann Endowed Professor in Multiple Sclerosis in the department of neurology at the University of California in San Francisco. “Some people with progressive MS may benefit symptomatically from high-dose biotin — for example, the study suggests that there could be improvements in walking speed — and [there may be] a beneficial effect of MD1003 after long-term treatment,” he adds. “Further study would be needed to determine if any of these possible beneficial effects could be replicated.” Biotin, or vitamin B7, is found in meat, fish, eggs, nuts, seeds, and some vegetables. But the studies on biotin for MS have used much higher amounts than are found in food — in fact, they used up to 10,000 times the amount recommended to meet nutritional needs (30 micrograms per day).
Results Don’t Support Theories for How Biotin Could Help
It was thought that the B vitamin could help slow or even reverse the loss of protective myelin around nerve fibers, which is a major cause of disability in progressive MS. It would do this by boosting several enzymes involved in producing cellular energy, allowing nerve cells to continue transmitting signals even with damaged myelin. Biotin may also increase the production of fats needed to make myelin. Earlier studies of MD1003 — which delivers a 300 mg daily dose of biotin — found that some people with progressive MS who took it saw improvements in disability. But these studies were small, and found that the vitamin only helped just over 10 percent of those taking it. The phase 3 study by Dr. Cree and his colleagues — phase 3 trials are the final step in the drug-approval process — evaluated high-dose biotin in 642 people with progressive MS, 65 percent of whom had secondary-progressive MS. In all, 326 received biotin at a dose of 100 mg three times per day and 316 were given a placebo, which has no therapeutic effect. Seven percent of the study participants who received high-dose biotin had improvement in EDSS scores at 12 months, compared with 6 percent of those treated with placebo. Similarly, 7 percent of those on high-dose biotin had improved performance on the T25-FW test at month 12, compared with 3 percent of those on placebo. Just 12 percent of study participants on MD1003 showed improvement on both measures, the researchers say.
A High Rate of Side Effects or Adverse Events
In addition, about 25 percent of people in both groups experienced at least one serious treatment-related side effect or adverse event, with nervous system disorders, most commonly MS relapse, being the most common. Administration of high-dose biotin also led to inaccurate laboratory results for tests using biotinylated antibodies — blood proteins with biotin attached to them — the researchers found. Overall, about 85 percent of participants in both groups had a treatment-related adverse event by the end of the study, the researchers say. Barry Wolf, MD, PhD, a pediatric geneticist at the Ann and Robert H. Lurie Children’s Hospital of Chicago, believes studies of high-dose biotin have given people with MS false hope and argues that side effects with the B vitamin are problematic “at such mega-doses.” Dr. Wolf specializes in biotinidase deficiency, a genetic disorder in which the body has problems processing biotin; he is not a neurologist, nor does he have expertise in MS. But biotinidase deficiency produces symptoms similar to those of MS and is treated with the B vitamin, he says. As Cree wrote in the summary of the study’s findings, “[Our] study does not support ongoing use of MD1003 and by extension other forms of high-dose biotin in persons with progressive MS. Because of the possible deleterious consequences of misinterpretation of inaccurate laboratory tests that are influenced by high serum concentrations of biotin, the risks of treatment outweigh potential benefits for most MS patients.”
