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Amyotrophic Lateral Sclerosis Volume 12, 2011 - Issue 2
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Original Article

Identification of compounds protective against G93A-SOD1 toxicity for the treatment of amyotrophic lateral sclerosis

Radhia Benmohamed Cambria Pharmaceuticals, Cambridge, Massachusetts
,
Anthony C. Arvanites Cambria Pharmaceuticals, Cambridge, Massachusetts; Harvard Stem Cell Institute, Harvard University, Cambridge, Massachusetts, USA
,
Jinho Kim Geriatric Research Education and Clinical Center, Bedford Veterans Administration Medical Center, Bedford, Massachusetts; Departments of Neurology, Laboratory Medicine and Pathology, and Psychiatry, Boston University School of Medicine, Boston, Massachusetts
,
Robert J. Ferrante Geriatric Research Education and Clinical Center, Bedford Veterans Administration Medical Center, Bedford, Massachusetts; Departments of Neurology, Laboratory Medicine and Pathology, and Psychiatry, Boston University School of Medicine, Boston, Massachusetts
,
Richard B. Silverman Department of Chemistry, Department of Molecular Biosciences, Center for Molecular Innovation and Drug Discovery, Chemistry of Life Processes Institute, Northwestern University, Evanston, Illinois
,
Richard I. Morimoto Department of Molecular Biosciences, Rice Institute for Biomedical Research, Northwestern University, Evanston, Illinois
&
Donald R. Kirsch Cambria Pharmaceuticals, Cambridge, MassachusettsCorrespondence[email protected]
 show all
Pages 87-96 | Received 15 Jun 2010, Accepted 07 Sep 2010, Published online: 12 Nov 2010

Citations (31)

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Read on this site (2)

Alexander McGown & Matthew John Stopford. (2018) High-throughput drug screens for amyotrophic lateral sclerosis drug discovery. Expert Opinion on Drug Discovery 13:11, pages 1015-1025.
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Marcie A Glicksman. (2011) The preclinical discovery of amyotrophic lateral sclerosis drugs. Expert Opinion on Drug Discovery 6:11, pages 1127-1138.
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Articles from other publishers (29)

S. Dharani, G. Kalaiarasi, Vincent M. Lynch, R. Shankar & R. Prabhakaran. (2023) Unpredicted coordination of hydrogen chloride in organoruthenium(II) pyrazolone complex: Synthesis, spectral and structural characterization. Inorganic Chemistry Communications 153, pages 110849.
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Mohamed F. Elmansy, Cory T. Reidl, Mizzanoor Rahaman, P. Hande Özdinler & Richard B. Silverman. (2023) Small molecules targeting different cellular pathologies for the treatment of amyotrophic lateral sclerosis. Medicinal Research Reviews.
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Ravichandran Manjupriya, Kamalanathan Pouthika, Gunabalan Madhumitha & Selvaraj Mohana Roopan. (2022) Biological aspects of nitrogen heterocycles for amyotrophic lateral sclerosis. Applied Microbiology and Biotechnology 107:1, pages 43-56.
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Barış Genç, Mukesh Gautam, Benjamin R. Helmold, Nuran Koçak, Aksu Günay, Gashaw M. Goshu, Richard B. Silverman & P. Hande Ozdinler. (2022) NU-9 improves health of hSOD1G93A mouse upper motor neurons in vitro, especially in combination with riluzole or edaravone. Scientific Reports 12:1.
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Dare E. George & Jetze J. Tepe. (2021) Advances in Proteasome Enhancement by Small Molecules. Biomolecules 11:12, pages 1789.
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Maria Elena Cicardi, Lara Marrone, Mimoun Azzouz & Davide Trotti. (2021) Proteostatic imbalance and protein spreading in amyotrophic lateral sclerosis. The EMBO Journal 40:10.
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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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Barış Genç, Mukesh Gautam, Öge Gözütok, Ina Dervishi, Santana Sanchez, Gashaw M. Goshu, Nuran Koçak, Edward Xie, Richard B. Silverman & P. Hande Özdinler. (2021) Improving mitochondria and ER stability helps eliminate upper motor neuron degeneration that occurs due to mSOD1 toxicity and TDP‐43 pathology. Clinical and Translational Medicine 11:2.
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Zefeng Zhao, Xufen Dai, Chenyang Li, Xiao Wang, Jiale Tian, Ying Feng, Jing Xie, Cong Ma, Zhuang Nie, Peinan Fan, Mingcheng Qian, Xirui He, Shaoping Wu, Yongmin Zhang & Xiaohui Zheng. (2020) Pyrazolone structural motif in medicinal chemistry: Retrospect and prospect. European Journal of Medicinal Chemistry 186, pages 111893.
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Evert Njomen & Jetze J. Tepe. (2019) Proteasome Activation as a New Therapeutic Approach To Target Proteotoxic Disorders. Journal of Medicinal Chemistry 62:14, pages 6469-6481.
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Cindy Gill, James P. Phelan, Theo Hatzipetros, Joshua D. Kidd, Valerie R. Tassinari, Beth Levine, Monica Z. Wang, Andrew Moreno, Kenneth Thompson, Marcel Maier, Jan Grimm, Alan Gill & Fernando G. Vieira. (2019) SOD1-positive aggregate accumulation in the CNS predicts slower disease progression and increased longevity in a mutant SOD1 mouse model of ALS. Scientific Reports 9:1.
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Victor Banerjee, Ofek Oren, Efrat Ben-Zeev, Ran Taube, Stanislav Engel & Niv Papo. (2017) A computational combinatorial approach identifies a protein inhibitor of superoxide dismutase 1 misfolding, aggregation, and cytotoxicity. Journal of Biological Chemistry 292:38, pages 15777-15788.
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Federica Rinaldi, Dario Motti, Laura Ferraiuolo & Brian K. Kaspar. (2017) High content analysis in amyotrophic lateral sclerosis. Molecular and Cellular Neuroscience 80, pages 180-191.
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Itsuki Anzai, Keisuke Toichi, Eiichi Tokuda, Atsushi Mukaiyama, Shuji Akiyama & Yoshiaki Furukawa. (2016) Screening of Drugs Inhibiting In vitro Oligomerization of Cu/Zn-Superoxide Dismutase with a Mutation Causing Amyotrophic Lateral Sclerosis. Frontiers in Molecular Biosciences 3.
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Yinan Zhang, Kevin Tianmeng Zhao, Susan G. Fox, Jinho Kim, Donald R. Kirsch, Robert J. Ferrante, Richard I. Morimoto & Richard B. Silverman. (2015) Tertiary Amine Pyrazolones and Their Salts as Inhibitors of Mutant Superoxide Dismutase 1-Dependent Protein Aggregation for the Treatment of Amyotrophic Lateral Sclerosis. Journal of Medicinal Chemistry 58:15, pages 5942-5949.
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Alf Håkon Lystad & Anne Simonsen. (2015) Assays to monitor aggrephagy. Methods 75, pages 112-119.
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Guoyao Xia, Radhia Benmohamed, Richard I. Morimoto, Donald R. Kirsch & Richard B. Silverman. (2014) Deuteration and fluorination of 1,3-bis(2-phenylethyl)pyrimidine-2,4,6(1H,3H,5H)-trione to improve its pharmacokinetic properties. Bioorganic & Medicinal Chemistry Letters 24:21, pages 5098-5101.
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Paul C. Trippier, Kevin Tianmeng Zhao, Susan G. Fox, Isaac T. Schiefer, Radhia Benmohamed, Jason Moran, Donald R. Kirsch, Richard I. Morimoto & Richard B. Silverman. (2014) Proteasome Activation is a Mechanism for Pyrazolone Small Molecules Displaying Therapeutic Potential in Amyotrophic Lateral Sclerosis. ACS Chemical Neuroscience 5:9, pages 823-829.
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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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Allison S Limpert, Margrith E Mattmann & Nicholas D P Cosford. (2013) Recent progress in the discovery of small molecules for the treatment of amyotrophic lateral sclerosis (ALS). Beilstein Journal of Organic Chemistry 9, pages 717-732.
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Yinan Zhang, Radhia Benmohamed, He Huang, Tian Chen, Cindy Voisine, Richard I. Morimoto, Donald R. Kirsch & Richard B. Silverman. (2013) Arylazanylpyrazolone Derivatives as Inhibitors of Mutant Superoxide Dismutase 1 Dependent Protein Aggregation for the Treatment of Amyotrophic Lateral Sclerosis. Journal of Medicinal Chemistry 56:6, pages 2665-2675.
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Lori Krim Gavrin, Rajiah Aldrin Denny & Eddine Saiah. (2012) Small Molecules That Target Protein Misfolding. Journal of Medicinal Chemistry 55:24, pages 10823-10843.
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Paul C. Trippier, Radhia Benmohamed, Donald R. Kirsch & Richard B. Silverman. (2012) Substituted pyrazolones require N2 hydrogen bond donating ability to protect against cytotoxicity from protein aggregation of mutant superoxide dismutase 1. Bioorganic & Medicinal Chemistry Letters 22:21, pages 6647-6650.
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Yinan Zhang, Radhia Benmohamed, Wei Zhang, Jinho Kim, Christina K. Edgerly, Yaoqiu Zhu, Richard I. Morimoto, Robert J. Ferrante, Donald R. Kirsch & Richard B. Silverman. (2012) Chiral Cyclohexane 1,3-Diones as Inhibitors of Mutant SOD1-Dependent Protein Aggregation for the Treatment of ALS. ACS Medicinal Chemistry Letters 3:7, pages 584-587.
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Tian Chen, Radhia Benmohamed, Jinho Kim, Karen Smith, Daniel Amante, Richard I. Morimoto, Donald R. Kirsch, Robert J. Ferrante & Richard B. Silverman. (2011) ADME-Guided Design and Synthesis of Aryloxanyl Pyrazolone Derivatives To Block Mutant Superoxide Dismutase 1 (SOD1) Cytotoxicity and Protein Aggregation: Potential Application for the Treatment of Amyotrophic Lateral Sclerosis. Journal of Medicinal Chemistry 55:1, pages 515-527.
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Wei Zhang, Radhia Benmohamed, Anthony C. Arvanites, Richard I. Morimoto, Robert J. Ferrante, Donald R. Kirsch & Richard B. Silverman. (2012) Cyclohexane 1,3-diones and their inhibition of mutant SOD1-dependent protein aggregation and toxicity in PC12 cells. Bioorganic & Medicinal Chemistry 20:2, pages 1029-1045.
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Frank O. Johnson, Yukun Yuan, Ravindra K. Hajela, Alisha Chitrakar, Dawn M. Parsell & William D. Atchison. (2011) Exposure to an Environmental Neurotoxicant Hastens the Onset of Amyotrophic Lateral Sclerosis-Like Phenotype in Human Cu 2+ /Zn 2+ Superoxide Dismutase 1 G93A Mice: Glutamate-Mediated Excitotoxicity . Journal of Pharmacology and Experimental Therapeutics 338:2, pages 518-527.
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Guoyao Xia, Radhia Benmohamed, Jinho Kim, Anthony C. Arvanites, Richard I. Morimoto, Robert J. Ferrante, Donald R. Kirsch & Richard B. Silverman. (2011) Pyrimidine-2,4,6-trione Derivatives and Their Inhibition of Mutant SOD1-Dependent Protein Aggregation. Toward a Treatment for Amyotrophic Lateral Sclerosis. Journal of Medicinal Chemistry 54:7, pages 2409-2421.
Crossref
Tian Chen, Radhia Benmohamed, Anthony C. Arvanites, Hantamalala Ralay Ranaivo, Richard I. Morimoto, Robert J. Ferrante, D. Martin Watterson, Donald R. Kirsch & Richard B. Silverman. (2011) Arylsulfanyl pyrazolones block mutant SOD1-G93A aggregation. Potential application for the treatment of amyotrophic lateral sclerosis. Bioorganic & Medicinal Chemistry 19:1, pages 613-622.
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