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Expert Opinion on Investigational Drugs Volume 18, 2009 - Issue 12
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Drug Evaluations

Arimoclomol: a potential therapy under development for ALS

Veena Lanka Neurology Clinical Trial Unit, Bldg 149, 13th Street Cny, Room 2274, Charlestown, MA 02129, USA +1 617 726 9122; +1 617 643 3558; [email protected]
, MBBS,
Scott Wieland CytRx Corporation, 11726 San Vicente Blvd., Suite 650, Los Angeles, CA 90049, USA
, PhD,
Jack Barber CytRx Corporation, 11726 San Vicente Blvd., Suite 650, Los Angeles, CA 90049, USA
, PhD &
Merit Cudkowicz Neurology Clinical Trial Unit, Bldg 149, 13th Street Cny, Room 2274, Charlestown, MA 02129, USA +1 617 726 9122; +1 617 643 3558; [email protected]
, MD
Pages 1907-1918 | Published online: 25 Nov 2009

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Stefan M Gehrig & Gordon S Lynch. (2011) Emerging drugs for treating skeletal muscle injury and promoting muscle repair. Expert Opinion on Emerging Drugs 16:1, pages 163-182.
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Mhoriam Ahmed, Charlotte Spicer, Jasmine Harley, J. Paul Taylor, Michael Hanna, Rickie Patani & Linda Greensmith. (2023) Amplifying the Heat Shock Response Ameliorates ALS and FTD Pathology in Mouse and Human Models. Molecular Neurobiology 60:12, pages 6896-6915.
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Dominika Sitarska, Anna Tylki-Szymańska & Agnieszka Ługowska. (2021) Treatment trials in Niemann-Pick type C disease. Metabolic Brain Disease 36:8, pages 2215-2221.
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Yvonne E. Klingl, Donya Pakravan & Ludo Van Den Bosch. (2020) Opportunities for histone deacetylase inhibition in amyotrophic lateral sclerosis. British Journal of Pharmacology 178:6, pages 1353-1372.
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Cyril Jones Jagaraj, Sonam Parakh & Julie D. Atkin. (2021) Emerging Evidence Highlighting the Importance of Redox Dysregulation in the Pathogenesis of Amyotrophic Lateral Sclerosis (ALS). Frontiers in Cellular Neuroscience 14.
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Matthew C. Kiernan, Steve Vucic, Kevin Talbot, Christopher J. McDermott, Orla Hardiman, Jeremy M. Shefner, Ammar Al-Chalabi, William Huynh, Merit Cudkowicz, Paul Talman, Leonard H. Van den Berg, Thanuja Dharmadasa, Paul Wicks, Claire Reilly & Martin R. Turner. (2020) Improving clinical trial outcomes in amyotrophic lateral sclerosis. Nature Reviews Neurology 17:2, pages 104-118.
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Erin N. Lottes & Daniel N. Cox. (2020) Homeostatic Roles of the Proteostasis Network in Dendrites. Frontiers in Cellular Neuroscience 14.
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Rachel Kuta, Nancy Larochelle, Mario Fernandez, Arun Pal, Sandra Minotti, Michael Tibshirani, Kyle St. Louis, Benoit J. Gentil, Josephine N. Nalbantoglu, Andreas Hermann & Heather D. Durham. (2020) Depending on the stress, histone deacetylase inhibitors act as heat shock protein co-inducers in motor neurons and potentiate arimoclomol, exerting neuroprotection through multiple mechanisms in ALS models. Cell Stress and Chaperones 25:1, pages 173-191.
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Vijay Kumar, Tara Kashav & Md. Imtaiyaz Hassan. 2019. Pathology, Prevention and Therapeutics of Neurodegenerative Disease. Pathology, Prevention and Therapeutics of Neurodegenerative Disease 207 224 .
Cathrine K. Fog, Paola Zago, Erika Malini, Lukasz M. Solanko, Paolo Peruzzo, Claus Bornaes, Raffaella Magnoni, Arnela Mehmedbasic, Nikolaj H.T. Petersen, Bruno Bembi, Johannes F.M.G. Aerts, Andrea Dardis & Thomas Kirkegaard. (2018) The heat shock protein amplifier arimoclomol improves refolding, maturation and lysosomal activity of glucocerebrosidase. eBioMedicine 38, pages 142-153.
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Benjamin Guy Trist, Dominic James Hare & Kay Lorraine Double. (2018) A Proposed Mechanism for Neurodegeneration in Movement Disorders Characterized by Metal Dyshomeostasis and Oxidative Stress. Cell Chemical Biology 25:7, pages 807-816.
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Alberto Benussi, Maria Sofia Cotelli, Alessandro Padovani & Barbara Borroni. (2018) Recent neuroimaging, neurophysiological, and neuropathological advances for the understanding of NPC. F1000Research 7, pages 194.
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Francesca Bonomini, Gaia Favero, Valentina Trapletti & Rita Rezzani. 2018. HSP70 in Human Diseases and Disorders. HSP70 in Human Diseases and Disorders 223 245 .
Shambhunath Bose & Jungsook Cho. (2017) Targeting chaperones, heat shock factor-1, and unfolded protein response: Promising therapeutic approaches for neurodegenerative disorders. Ageing Research Reviews 35, pages 155-175.
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Benjamin N. Atkinson, Hannah L. Woodward, James Sipthorp & Paul V. Fish. (2017) Regioselective and enantiospecific synthesis of the HSP co-inducer arimoclomol from chiral glycidyl derivatives. Organic & Biomolecular Chemistry 15:46, pages 9794-9799.
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K. Ahmad, M.H. Baig, Girish Kumar Gupta, M.A. Kamal, Neelam Pathak & I. Choi. (2016) Identification of common therapeutic targets for selected neurodegenerative disorders: An in silico approach. Journal of Computational Science 17, pages 292-306.
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Vijay Kumar, Asimul Islam, Md. Imtaiyaz Hassan & Faizan Ahmad. (2016) Therapeutic progress in amyotrophic lateral sclerosis-beginning to learning. European Journal of Medicinal Chemistry 121, pages 903-917.
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Thomas KirkegaardJames GrayDavid A. PriestmanKerri-Lee WallomJennifer AtkinsOle Dines OlsenAlexander KleinSvetlana DrndarskiNikolaj H. T. PetersenLinda IngemannDavid A. SmithLauren MorrisClaus BornæsSigne Humle JørgensenIan WilliamsAnders HinsbyChristoph ArenzDavid BegleyMarja JäätteläFrances M. Platt. (2016) Heat shock protein–based therapy as a potential candidate for treating the sphingolipidoses. Science Translational Medicine 8:355.
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Tarun Kumar, Gurumayum Suraj Sharma & Laishram Rajendrakumar Singh. (2016) Homocystinuria: Therapeutic approach. Clinica Chimica Acta 458, pages 55-62.
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Mhoriam AhmedPedro M. MachadoAdrian MillerCharlotte SpicerLaura HerbelinJianghua HeJanelle NoelYunxia WangApril L. McVeyMamatha PasnoorPhilip GallagherJeffrey StatlandChing-Hua LuBernadett KalmarStefen BradyHuma SethiGeorge SamandourasMatt PartonJanice L. HoltonAnne WestonLucy CollinsonJ. Paul TaylorGiampietro SchiavoMichael G. HannaRichard J. BarohnMazen M. DimachkieLinda Greensmith. (2016) Targeting protein homeostasis in sporadic inclusion body myositis. Science Translational Medicine 8:331.
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Emma J. Duncan, Michael E. Cheetham, J. Paul Chapple & Jacqueline van der Spuy. 2015. The Networking of Chaperones by Co-chaperones. The Networking of Chaperones by Co-chaperones 243 273 .
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Barış Genç & P. Hande Özdinler. (2014) Moving forward in clinical trials for ALS: motor neurons lead the way please. Drug Discovery Today 19:4, pages 441-449.
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Bernadett Kalmar, Ching-Hua Lu & Linda Greensmith. (2014) The role of heat shock proteins in Amyotrophic Lateral Sclerosis: The therapeutic potential of Arimoclomol. Pharmacology & Therapeutics 141:1, pages 40-54.
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Darius Ebrahimi-Fakhari, Laiq-Jan Saidi & Lara Wahlster. (2013) Molecular chaperones and protein folding as therapeutic targets in Parkinson’s disease and other synucleinopathies. Acta Neuropathologica Communications 1:1.
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Rachna S. Pandya, Haining Zhu, Wei Li, Robert Bowser, Robert M. Friedlander & Xin Wang. (2013) Therapeutic neuroprotective agents for amyotrophic lateral sclerosis. Cellular and Molecular Life Sciences 70:24, pages 4729-4745.
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Tai Wang, Pablo C. Echeverría & Didier Picard. 2013. Inhibitors of Molecular Chaperones as Therapeutic Agents. Inhibitors of Molecular Chaperones as Therapeutic Agents 1 36 .
Bilal Malik, Niranjanan Nirmalananthan, Anna L. Gray, Albert R. La Spada, Michael G. Hanna & Linda Greensmith. (2013) Co-induction of the heat shock response ameliorates disease progression in a mouse model of human spinal and bulbar muscular atrophy: implications for therapy. Brain 136:3, pages 926-943.
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Ching-Hua Lu, Axel Petzold, Bernadett Kalmar, James Dick, Andrea Malaspina & Linda Greensmith. (2012) Plasma Neurofilament Heavy Chain Levels Correlate to Markers of Late Stage Disease Progression and Treatment Response in SOD1G93A Mice that Model ALS. PLoS ONE 7:7, pages e40998.
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Per Zetterström, Karin S. Graffmo, Peter M. Andersen, Thomas Brännström & Stefan L. Marklund. (2011) Proteins That Bind to Misfolded Mutant Superoxide Dismutase-1 in Spinal Cords from Transgenic Amyotrophic Lateral Sclerosis (ALS) Model Mice. Journal of Biological Chemistry 286:23, pages 20130-20136.
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Andrija Finka, Rayees U. H. Mattoo & Pierre Goloubinoff. (2010) Meta-analysis of heat- and chemically upregulated chaperone genes in plant and human cells. Cell Stress and Chaperones 16:1, pages 15-31.
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Thomas E Lloyd. (2010) Novel therapeutic approaches for inclusion body myositis. Current Opinion in Rheumatology 22:6, pages 658-664.
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Richard S Bedlack. (2010) Amyotrophic lateral sclerosis: current practice and future treatments. Current Opinion in Neurology 23:5, pages 524-529.
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Beatrice Nefussy & Vivian E. Drory. (2010) Moving toward a predictive and personalized clinical approach in amyotrophic lateral sclerosis: novel developments and future directions in diagnosis, genetics, pathogenesis and therapies. EPMA Journal 1:2, pages 329-341.
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