Vitamin B3 forms have recently emerged as potent boosters of energy metabolism, as they are precursors for NAD+, a molecular switch of metabolism between fasting and growth modes. As fasting has been shown to promote health and longevity in for example mice and worms, a variety of “NAD-boosters” are being developed. However, whether actual NAD+ deficiency exists in human disease, and whether NAD+ boosters could have curative effects, has remained elusive.
In the current publication, a collaborative team of investigators, led by academy professor Anu Suomalainen-Wartiovaara and academy research fellow Eija Pirinen, report lowered NAD+ levels in both blood and muscle of mitochondrial myopathy patients. The disease is characterized by progressive muscle weakness, exercise intolerance and cramps. Currently, no treatments that would slow down disease progression exist.
Our results are a proof-of-principle that NAD+ deficiency exists in humans, and that NAD+ boosters can delay progression of mitochondrial muscle disease. The study is a break-through in developing targeted therapy options for energy metabolic diseases.
Anu Suomalainen-Wartiovaara
Pirinen and colleagues report that niacin treatment efficiently increased blood NAD+ both in patients and healthy subjects. Niacin restored NAD+ in the muscle of the patients to the normal level, and improved strength of large muscles and mitochondrial oxidative capacity. Overall metabolism shifted towards that of normal subjects. The authors emphasize that niacin and NAD+ are efficient metabolic modifiers and should be used only if NAD-deficiency is detected. The results of this open pilot study revealed that niacin is a promising treatment option for mitochondrial myopathy.
Eija Pirinen, Mari Auranen, Nahid A. Khan, Virginia Brilhante, Niina Urho, Alberto Pessia, Antti Hakkarainen, Juho Kuula, Ulla Heinonen, Mark S. Schmidt, Kimmo Haimilahti, Päivi Piirilä, Nina Lundbom, Marja-Riitta Taskinen, Charles Brenner, Vidya Velagapudi, Kirsi H. Pietiläinen and Anu Suomalainen. Niacin cures systemic NAD+ deficiency and improves muscle performance in adult-onset mitochondrial myopathy. Cell Metabolism Epub 7 May (2020).
For more information, please contact:
Academy Professor Anu Suomalainen Wartiovaara, MD PhD
Chief Physician, Helsinki University Hospital
Stem Cells and Metabolism Research Program, University of Helsinki
Tel. +358 40 5936386, email: anu.wartiovaara@helsinki.fi, Twitter
Academy Research Fellow, Adjunct Professor Eija Pirinen, PhD
University of Helsinki
Research Program for Clinical and Molecular Metabolism University of Helsinki
Tel: +358 (0)50 4489 291, email: eija.pirinen@helsinki.fi, Twitter
The Sheekey Science Show features the "Niacin cures systemic NAD+ deficiency and improves muscle performance in adult-onset mitochondrial myopathy" by the Pirinen and Suomalainen-Wartiovaara research groups in their video.