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Vitamin B3 treatment for ataxia shows promise in first human trial

1 May 2014

An essential vitamin commonly known for processing fat and proteins in the body may hold the key to slowing the progression of Friedreich’s ataxia, according to findings published today in The Lancet.

Dr Vincenzo Libri

In the first clinical trial of its kind, Dr Vincenzo Libri (Head of the Leonard Wolfson Experimental Neurology Centre at UCL and former Head of Clinical Studies at the NIHR/Welcome Trust Imperial College Clinical Research Facility), Professor Richard Festenstein (Professor of Neurology at Imperial College London), Dr Paola Giunti (Head of Ataxia Centre, UCL Institute of Neurology) and their research teams, tested the ability of nicotinamide (a form of vitamin B3) to increase levels of frataxin protein that is abnormally low in Friedriech’s ataxia, thereby causing the condition. In this first ever clinical trial involving patients with Friedreich’s ataxia and nicotinamide, the research teams tested the effect of increasing doses of this drug to determine how well it was tolerated and its safety profile. The patients received single and multiple doses of nicotinamide at doses much higher than used for vitamin supplementation. Nicotinamide was generally well tolerated and was shown to increase levels of frataxin protein to the levels found in carriers without symptoms of the condition, when taken daily for up to 2 months.

Finding a cure for Friedreich’s ataxia is what every researcher in the field dreams about. 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. Our results help us understand the key elements of how nicotinamide may work and are important for translating the research from the laboratory into a clinical treatment. However, given the exploratory nature of our investigation, our results should be interpreted with caution and require further substantiation from larger confirmatory studies before we can make our vision of a cure a reality.

Dr Vincenzo Libri

We are excited by the prospects of nicotinamide potentially being developed into a treatment but it is important to remember that we still need to conduct further trials to confirm the safety over a longer time and to see whether the increase in frataxin actually results in improvements for patients. We are extremely grateful to all the patients who have taken part in this important pilot trial.

Dr Paola Giunti

Dr Paola Giunti

Whether restoring frataxin levels will slow or halt the progress of the disease is still unclear, as the protein’s precise role in the disease is not yet fully understood. Yet as carriers do not suffer from symptoms, restoring frataxin levels to those seen in carriers is a significant step.

The trial was supported by the UK Medical Research Council, Ataxia UK, and the UK National Institute for Health Research (NIHR).

Further information

Vincenzo Libri, Cihangir Yandim,Stavros Athanasopoulos, Naomi Loyse, Theona Natisvili, Pui Pik Law, Ping Kei Chan, Tariq Mohammad, Marta Mauri, Kin Tung Tam, James Leiper, Sophie Piper, Aravind Ramesh, Michael H Parkinson, Les Huson, Paola Giunti, Prof Richard Festenstein. Epigenetic and neurological effects and safety of high-dose nicotinamide in patients with Friedreich's ataxia: an exploratory, open-label, dose-escalation study. Lancet. Available online May 1st 2014. DOI: 10.1016/S0140-6736(14)60382-2

David R Lynch, Kenneth H Fischbeck. Nicotinamide in Friedreich's ataxia: useful or not? Lancet. Available online May 1st 2014. DOI: 10.1016/S0140-6736(14)60573-0

UCL news

NIHR UCLH Biomedical Research Centre website

Ataxia UK

Ataxia Centre, UCL Institute of Neurology

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