Mitochondrial stroke-like episodes: The search for new therapies

Pharmacol Res. 2022 Jun:180:106228. doi: 10.1016/j.phrs.2022.106228. Epub 2022 Apr 21.

Abstract

A mitochondrial stroke-like event is an evolving subacute neurological syndrome linked to seizure activity and focal metabolic brain derangement in a genetically determined mitochondrial disorder. The acronym "MELAS" (mitochondrial encephalopathy associated with lactic acidosis and stroke-like lesions) identifies subjects with molecular, biochemical and/or histological evidence of mitochondrial disorder who experience stroke-like lesions. MELAS is a rare inherited mitochondrial disease linked to severe multiorgan involvement and stress-induced episodes of metabolic decompensation and lactic acidosis. Unfortunately, there are no etiopathogenetic therapies for stroke-like episodes to date, and the treatment is mainly based on anti-epileptic drugs and supportive therapies. This perspective opinion article discusses the current care standards for MELAS patients and revises current and innovative emerging therapies for mitochondrial stroke-like episodes.

Keywords: M.3243A>G; MELAS; MTTL1; POLG; Stroke; Stroke-like episodes; Stroke-like lesion.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acidosis, Lactic* / complications
  • DNA, Mitochondrial
  • Humans
  • MELAS Syndrome* / complications
  • MELAS Syndrome* / drug therapy
  • Mitochondrial Diseases*
  • Mutation
  • Stroke* / drug therapy

Substances

  • DNA, Mitochondrial