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Review
. 2017 Nov;95(11):2236-2243.
doi: 10.1002/jnr.24111. Epub 2017 Jul 8.

Triheptanoin for the treatment of brain energy deficit: A 14-year experience

Fanny Mochel  1   2   3
Affiliations
Review

Triheptanoin for the treatment of brain energy deficit: A 14-year experience

Fanny Mochel. J Neurosci Res. 2017 Nov.

Abstract

Triheptanoin is an odd-chain triglyceride with anaplerotic properties-that is, replenishing the pool of metabolic intermediates in the Krebs cycle. Unlike even-chain fatty acids metabolized to acetyl-CoA only, triheptanoin can indeed provide both acetyl-CoA and propionyl-CoA, two key carbon sources for the Krebs cycle. Triheptanoin was initially used in patients with long-chain fatty acid oxidation disorders. The first demonstration of the possible benefit of triheptanoin for brain energy deficit came from a patient with pyruvate carboxylase deficiency, a severe metabolic disease that affects anaplerosis in the brain. In an open-label study, triheptanoin was then shown to decrease nonepileptic paroxysmal manifestations by 90% in patients with glucose transporter 1 deficiency syndrome, a disease that affects glucose transport into the brain. 31 P magnetic resonance spectroscopy studies also indicated that triheptanoin was able to correct bioenergetics in the brain of patients with Huntington disease, a neurodegenerative disease associated with brain energy deficit. Altogether, these studies indicate that triheptanoin can be a treatment for brain energy deficit related to altered anaplerosis and/or glucose metabolism. © 2017 Wiley Periodicals, Inc.

Keywords: GLUT1; Huntington disease; Krebs cycle; astrocytes; magnetic resonance spectroscopy.

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