Friedreich’s Ataxia Treated With Vitamin B3 in Clinical Trial

Vitamin B3, a water soluble vitamin essential to processing fat and proteins in the human body, may also prove important to treating Friedreich’s Ataxia. A currently recruiting clinical trial from Imperial College London is studying the “Effect of Nicotinamide [Vitamin B3] in Friedreich’s Ataxia” to determine any subtle changes in the daily activities of patients with Friedreich’s ataxia as a result of nicotinamide treatment.

“Further studies are needed to determine the safety of high-dose nicotinamide with long-term administration and whether it can increase frataxin levels when given for longer periods,” said Dr. Richard Festenstein, of Imperial College London, in a news release. His comment was in response to the earlier published results leading to the current trial, which he is a principal investigator on, along with Drs. Vincenzo Libri and Paola Giunti.

“Finding a cure for Friedreich’s ataxia is what every researcher in the field dreams about,” said Dr. Libri. “We’re absolutely thrilled by our preliminary results and the hope it offers for the future of patients with this devastating condition and their families.” Previous results, published in “Epigenetic and Neurological Effects and Safety of High-dose Nicotinamide in Patients with Friedreich’s Ataxia: An Exploratory, Open-label, Dose-escalation Study,” in the journal The Lancet, indicated that nicotinamide is well tolerated and upregulates frataxin levels, although there are no significant improvements in clinical measures.

The study is a dose-escalation study administering 2-8 grams of oral nicotinamide, with comparisons made among groups and against a placebo group. It is important to note that these doses are much greater than the recommended daily allowance for vitamin B3 and that patients are not advised to self-medicate in response to the proposed benefits of nicotinamide treatment for Friedreich’s ataxia.

Enrollment is expected to reach 40 participants, with the primary completion date in June 2015. For an individual patient, daily administration will continue up to nine weeks, during which time patients’ levels of frataxin will be measured using an antibody dipstick assay.

Additional outcome measures include impact on clinical phenotype, clinical deficit, and biomarker expression. Researchers will also investigate correlations between functional changes and frataxin levels and determine the rate of adverse effects as a cause of treatment.

It is believed nicotinamide holds the potential to improve symptoms of patients with Friedreich’s ataxia due to its ability to remodel pathological heterochromatin in patients’ genomes. It is a histone deacetylase inhibitor and can upregulate frataxin expression, the root factor in Friedreich’s ataxia.