Abstract
The long-term effects of the sodium salt of dichloroacetic acid (DCA) were evaluated in four patients with mitochondrial encephalomyelopathy with lactic acidosis and stroke-like episodes (MELAS) carrying A3243G mutation. Oral administration of DCA in MELAS patients was followed for an average of 5 years 4 months. Serum levels of lactate and pyruvate were maintained at around 10 and 0.6 mg/dl, respectively. Serum levels of DCA were 40-136 microg/ml. Symptoms responding to treatment included persistent headache, abdominal pain, muscle weakness, and stroke-like episodes. In contrast, no improvements in mental status, deafness, short stature, or neuroelectrophysiological findings were observed. Adverse effects included mild liver dysfunction in all patients, hypocalcemia in three and peripheral neuropathy in one. None of these adverse events was severe enough to require discontinuation of treatment. To determine suitable indications for DCA therapy, analysis of many more patients who have undergone DCA administration is required.
References
Nov 26, 1992·The New England Journal of Medicine·P W StacpooleS Jenkinson
Aug 1, 1991·Neuropediatrics·T SaijoY Kuroda
Dec 13, 1990·Nature·Y GotoS Horai
Apr 1, 1989·European Journal of Pediatrics·D J ByrdD H Hunneman
Jan 1, 1986·Journal of Inherited Metabolic Disease·Y KurodaK Yamashita
Jan 1, 1987·Journal of Inherited Metabolic Disease·H Przyrembel
Jan 1, 1988·Annals of Internal Medicine·P W StacpooleE M Harman
Jun 1, 1995·Neurology·N De StefanoD L Arnold
Sep 1, 1993·The Journal of Pediatrics·P P TóthJ M Klein
Jan 1, 1996·Pediatric Neurology·W J Craigen
Dec 1, 1995·Journal of the Neurological Sciences·S KimuraA Nezu
Jan 1, 1996·Brain & Development·E NakagawaI Nonaka
Dec 18, 1996·Toxicology·A B DeAngeloG R Olson
Jan 1, 1997·Journal of the Neurological Sciences·J TakanashiH Niimi
Jun 1, 1997·Brain & Development·J TanakaS Okada
Jan 1, 1997·European Neurology·R S ChenN S Chu
Mar 4, 1998·Neurology·S SaitohM Imai
Aug 14, 1998·Environmental Health Perspectives·P W StacpooleM O James
Dec 16, 1998·Clinical Cardiology·J F LewisP Stacpoole
Dec 29, 1998·Toxicology·J Kato-WeinsteinR J Bull
May 12, 2000·Environmental Health Perspectives·C W Chen
Citations
May 27, 2010·Cancer Chemotherapy and Pharmacology·Dirk HesheClaudia Lanvers-Kaminsky
Jun 23, 2009·Breast Cancer Research and Treatment·Ramon C SunAnneke C Blackburn
May 27, 2010·European Journal of Neurology : the Official Journal of the European Federation of Neurological Societies·J Finsterer
Jun 1, 2006·CNS Drugs·Fernando Scaglia, Jennifer L Northrop
Sep 13, 2014·Breast Cancer Research and Treatment·Allison B HaugrudW Keith Miskimins
Oct 10, 2012·The Journal of Physiology·Joanne E MallinsonPaul L Greenhaff
Aug 21, 2009·European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society·Josef Finsterer
Feb 5, 2008·Toxicology·Ting LiJeffrey W Fisher
Feb 18, 2011·Biotechnology and Bioengineering·Ning Zhang, Andre F Palmer
Mar 12, 2010·Journal of Applied Toxicology : JAT·Ezdihar A HassounJessica Spildener
Nov 18, 2005·Mitochondrion·Kazutoshi NakanoMakiko Osawa
Jan 12, 2010·Molecular Genetics and Metabolism·Douglas S Kerr
Sep 4, 2008·British Journal of Cancer·E D MichelakisJ R Mackey
Apr 20, 2012·The Cochrane Database of Systematic Reviews·Gerald PfefferPatrick F Chinnery
Jun 26, 2007·Acta Neurologica Scandinavica·J Finsterer
Feb 15, 2008·Journal of Biochemical and Molecular Toxicology·Ezdihar A Hassoun, Soumyadeep Dey
May 22, 2008·Journal of Applied Toxicology : JAT·Ezdihar A Hassoun, Jatin Mehta
Dec 7, 2005·Medicine and Science in Sports and Exercise·Mark A Tarnopolsky, Sandeep Raha
Apr 11, 2006·Applied Physiology, Nutrition, and Metabolism = Physiologie Appliquée, Nutrition Et Métabolisme·Mark A Tarnopolsky
Nov 14, 2018·The Journal of Clinical Endocrinology and Metabolism·Rachel L BoalRobert McFarland
Jul 3, 2013·Nature Reviews. Neurology·Gerald PfefferPatrick F Chinnery
Sep 26, 2014·Molecular Biology Reports·Hanh NgoTom C Karagiannis
Apr 23, 2017·BMC Pharmacology & Toxicology·Amira El AremLotfi Achour
Aug 23, 2019·Cancers·Donatas StakišaitisMarta Maria Alonso
Jan 8, 2021·Metabolic Brain Disease·Wei WeiChanghao Sun