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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.
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.
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