Arimoclomol: a potential therapy under development for ALS

Expert Opinion on Investigational Drugs
Veena LankaMerit Cudkowicz

Abstract

Arimoclomol, an amplifier of heat shock protein expression involved in cellular stress response, has emerged as a potential therapeutic candidate in amyotrophic lateral sclerosis (ALS) in recent years. Treatment with arimoclomol was reported to improve survival and muscle function in a mouse model of motor neuron disease. Several single- and multiple-dose safety studies have been completed in healthy control subjects. A 3-month Phase IIa study in people with ALS demonstrated safety at dosages up to 300 mg/day and another study is currently recruiting participants with familial ALS caused by mutations in the superoxide dismutase gene. We review the rationale for testing arimoclomol in sporadic and familial ALS in the context of available safety and pharmacokinetic data. Published and unpublished literature relative to the drug in the past two decades is discussed. The current review attempts to bring together our existing understanding of the actions of arimoclomol with the disease profile of ALS. The pharmacological profile of arimoclomol and the available preclinical data make it a promising therapeutic possibility in ALS.

References

Jan 1, 1986·Annual Review of Biochemistry·S Lindquist
Oct 1, 1987·Journal of Neurology, Neurosurgery, and Psychiatry·R J GuiloffS L Lightman
Apr 1, 1974·Archives of Neurology·J A BrodyC E Morris
Jul 1, 1994·Journal of the Neurological Sciences·S A SmithS P Ringel
Apr 1, 1994·Pharmacology, Biochemistry, and Behavior·E PoutiainenM Färkkilä
Dec 1, 1993·Clinical Pharmacology and Therapeutics·F H NorrisL Elias
Apr 1, 1996·Muscle & Nerve·R G MillerS P Ringel
Mar 1, 1996·Neuromuscular Disorders : NMD·R G MillerS Smith
Jul 1, 1997·Acta Neurologica Scandinavica·O GredalT S Jensen
Apr 24, 1999·BMJ : British Medical Journal·P J Shaw
Aug 10, 2000·Italian Journal of Neurological Sciences·A ChiòD Schiffer
Apr 11, 2001·Neurology·R G MillerUNKNOWN Western ALS Study Group
Jun 2, 2001·The New England Journal of Medicine·L P Rowland, N A Shneider
Jun 25, 2002·Annals of the New York Academy of Sciences·Maria KürthyKatalin Bíró
Jul 19, 2002·Chirality·Júlia VisyMiklós Simonyi
Dec 14, 2002·Journal of the Neurological Sciences·Cristina CidAlberto Alcazar
Mar 11, 2003·Nature Genetics·Imke PulsKenneth H Fischbeck
Aug 2, 2003·Biochemical and Biophysical Research Communications·Judit HargitaiDavid S Latchman
Aug 27, 2003·Neurology·M E CudkowiczUNKNOWN Northeast ALS Consortium
Nov 26, 2003·Neuropathology and Applied Neurobiology·J D WoodP J Shaw

❮ Previous
Next ❯

Citations

Aug 10, 2010·Cell Stress & Chaperones·Andrija FinkaPierre Goloubinoff
Dec 2, 2011·Nature Reviews. Drug Discovery·Daniel W NeefDennis J Thiele
Sep 10, 2010·Current Opinion in Rheumatology·Thomas E Lloyd
Dec 10, 2013·Acta Neuropathologica Communications·Darius Ebrahimi-FakhariLara Wahlster
Aug 28, 2013·Pharmacology & Therapeutics·Bernadett KalmarLinda Greensmith
Dec 3, 2014·Neurochemistry International·Mariarita GalbiatiAngelo Poletti
Feb 18, 2011·Expert Opinion on Emerging Drugs·Stefan M Gehrig, Gordon S Lynch
Nov 1, 2013·Drug Discovery Today·Bariş Genç, P Hande Özdinler
Apr 10, 2016·Clinica Chimica Acta; International Journal of Clinical Chemistry·Tarun KumarLaishram Rajendrakumar Singh
Jul 4, 2016·European Journal of Medicinal Chemistry·Vijay KumarFaizan Ahmad
Sep 9, 2016·Science Translational Medicine·Thomas KirkegaardFrances M Platt
Nov 22, 2017·Organic & Biomolecular Chemistry·Benjamin N AtkinsonPaul V Fish
Jul 9, 2010·Current Opinion in Neurology·Richard S Bedlack
Jul 19, 2013·Cellular and Molecular Life Sciences : CMLS·Rachna S PandyaXin Wang
Jul 30, 2020·British Journal of Pharmacology·Yvonne E KlinglLudo Van Den Bosch
Oct 6, 2020·Frontiers in Cellular Neuroscience·Erin N Lottes, Daniel N Cox
Mar 25, 2016·Science Translational Medicine·Mhoriam AhmedLinda Greensmith
Dec 20, 2020·Nature Reviews. Neurology·Matthew C KiernanMartin R Turner
Oct 2, 2021·Metabolic Brain Disease·Dominika SitarskaAgnieszka Ługowska

❮ Previous
Next ❯

Related Concepts

Related Feeds

Amyloid Lateral Sclerosis

Amyotrophic Lateral Sclerosis (ALS) is a progressive nervous system disease associated with the death of neurons that control voluntary muscles. Discover the latest research on ALS here.