Injections of vitamin B1 improved several symptoms in patients with Friedreich’s ataxia and kept patients from deteriorating further over a two-year period, according to a recently published study.
While researchers caution that the results need to be verified, the findings open up the possibility of an entirely new way of approaching treatment in Friedreich’s ataxia.
The study, “Long-term treatment with thiamine as possible medical therapy for Friedreich ataxia“ recently appeared in the Journal of Neurology.
Without vitamin B1 in the central nervous system, numerous enzymes become unable to do their job. Recent studies also show that the vitamin, also called thiamine, may have other types of neuroprotective properties.
Studies also have indicated that vitamin B1 is low in the cerebrospinal fluid of Friedreich’s ataxia patients. A smaller study also found that treating patients with the vitamin for three months improved movement coordination, speech and swallowing while reducing fatigue.
Researchers at the Villa Immacolata Clinic and the San Martino University Hospital, both in Italy, wanted to explore the treatment for a longer period. They recruited 34 Friedreich’s ataxia patients who received intramuscular injections of 100mg of thiamine, twice a week.
Before starting the treatment, researchers examined the patients using the Scale for the Assessment and Rating of Ataxia (SARA). Some patients also were evaluated with the Fatigue Severity Scale (FSS) and another group had an echocardiogram done. The assessments were repeated after 1, 3, 6, 9, 12, and 24 months.
The time of treatment and follow-up ranged from 80 to 930 days. The treatment significantly improved movement symptoms. Improvements were seen in patients of all impairment stages, but those in stage 3 (in need of a wheelchair) improved more than patients with less disability.
The treatment also lowered the annual rate of progression compared to previously published levels.
Researchers also report that 16 out of 28 patients who had lost their deep tendon reflexes got them back after three months of treatment. Likewise, swallowing difficulties improved in 14 of 22 patients with such problems at the start of the study.
Among the 13 patients who were assessed with echocardiography, the thickness of the wall separating the heart ventricles decreased during the study. The treatment also increased the levels of frataxin mRNA, indicating increased gene activity, in half of the group, but the increase was not statistically significant.
Importantly, there were no side effects from the treatment and no patients left the study.