Literature DB >> 29331501

Incorporating upper motor neuron health in ALS drug discovery.

Ina Dervishi1, P Hande Ozdinler2.   

Abstract

Amyotrophic lateral sclerosis (ALS) is a complex disease, that affects the motor neuron circuitry. After consecutive failures in clinical trials for the past 20 years, edaravone was recently approved as the second drug for ALS. This generated excitement in the field revealed the need to improve preclinical assays for continued success. Here, we focus on the importance and relevance of upper motor neuron (UMN) pathology in ALS, and discuss how incorporation of UMN survival in preclinical assays will improve inclusion criteria for clinical trials and expedite the drug discovery effort in ALS and related motor neuron diseases.
Copyright © 2018 Elsevier Ltd. All rights reserved.

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Year:  2018        PMID： 29331501      PMCID： PMC5849515          DOI： 10.1016/j.drudis.2018.01.027

Source DB:  PubMed          Journal:  Drug Discov Today        ISSN： 1359-6446            Impact factor:   7.851


  67 in total

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Journal:  Exp Neurol       Date:  2004-02       Impact factor: 5.330

5.  C9orf72 BAC Transgenic Mice Display Typical Pathologic Features of ALS/FTD.

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Journal:  Neuron       Date:  2015-12-02       Impact factor: 17.173

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7.  A hexanucleotide repeat expansion in C9ORF72 is the cause of chromosome 9p21-linked ALS-FTD.

Authors:  Alan E Renton; Elisa Majounie; Adrian Waite; Javier Simón-Sánchez; Sara Rollinson; J Raphael Gibbs; Jennifer C Schymick; Hannu Laaksovirta; John C van Swieten; Liisa Myllykangas; Hannu Kalimo; Anders Paetau; Yevgeniya Abramzon; Anne M Remes; Alice Kaganovich; Sonja W Scholz; Jamie Duckworth; Jinhui Ding; Daniel W Harmer; Dena G Hernandez; Janel O Johnson; Kin Mok; Mina Ryten; Danyah Trabzuni; Rita J Guerreiro; Richard W Orrell; James Neal; Alex Murray; Justin Pearson; Iris E Jansen; David Sondervan; Harro Seelaar; Derek Blake; Kate Young; Nicola Halliwell; Janis Bennion Callister; Greg Toulson; Anna Richardson; Alex Gerhard; Julie Snowden; David Mann; David Neary; Michael A Nalls; Terhi Peuralinna; Lilja Jansson; Veli-Matti Isoviita; Anna-Lotta Kaivorinne; Maarit Hölttä-Vuori; Elina Ikonen; Raimo Sulkava; Michael Benatar; Joanne Wuu; Adriano Chiò; Gabriella Restagno; Giuseppe Borghero; Mario Sabatelli; David Heckerman; Ekaterina Rogaeva; Lorne Zinman; Jeffrey D Rothstein; Michael Sendtner; Carsten Drepper; Evan E Eichler; Can Alkan; Ziedulla Abdullaev; Svetlana D Pack; Amalia Dutra; Evgenia Pak; John Hardy; Andrew Singleton; Nigel M Williams; Peter Heutink; Stuart Pickering-Brown; Huw R Morris; Pentti J Tienari; Bryan J Traynor
Journal:  Neuron       Date:  2011-09-21       Impact factor: 17.173

8.  Therapeutic modulation of eIF2α phosphorylation rescues TDP-43 toxicity in amyotrophic lateral sclerosis disease models.

Authors:  Hyung-Jun Kim; Alya R Raphael; Eva S LaDow; Leeanne McGurk; Ross A Weber; John Q Trojanowski; Virginia M-Y Lee; Steven Finkbeiner; Aaron D Gitler; Nancy M Bonini
Journal:  Nat Genet       Date:  2013-12-15       Impact factor: 38.330

9.  Marked changes in dendritic structure and spine density precede significant neuronal death in vulnerable cortical pyramidal neuron populations in the SOD1(G93A) mouse model of amyotrophic lateral sclerosis.

Authors:  Matthew J Fogarty; Erica W H Mu; Peter G Noakes; Nickolas A Lavidis; Mark C Bellingham
Journal:  Acta Neuropathol Commun       Date:  2016-08-04       Impact factor: 7.801

Review 10.  ALS Clinical Trials Review: 20 Years of Failure. Are We Any Closer to Registering a New Treatment?

Authors:  Dmitry Petrov; Colin Mansfield; Alain Moussy; Olivier Hermine
Journal:  Front Aging Neurosci       Date:  2017-03-22       Impact factor: 5.750
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  6 in total

Review 1.  Complexity of Generating Mouse Models to Study the Upper Motor Neurons: Let Us Shift Focus from Mice to Neurons.

Authors:  Baris Genc; Oge Gozutok; P Hande Ozdinler
Journal:  Int J Mol Sci       Date:  2019-08-07       Impact factor: 5.923

Review 2.  Klotho Pathways, Myelination Disorders, Neurodegenerative Diseases, and Epigenetic Drugs.

Authors:  Walter H Moos; Douglas V Faller; Ioannis P Glavas; David N Harpp; Iphigenia Kanara; Anastasios N Mavrakis; Julie Pernokas; Mark Pernokas; Carl A Pinkert; Whitney R Powers; Konstantina Sampani; Kosta Steliou; Demetrios G Vavvas; Robert J Zamboni; Krishna Kodukula; Xiaohong Chen
Journal:  Biores Open Access       Date:  2020-03-31

3.  A multistationary loop model of ALS unveils critical molecular interactions involving mitochondria and glucose metabolism.

Authors:  Bruno Burlando; Marco Milanese; Giulia Giordano; Tiziana Bonifacino; Silvia Ravera; Franco Blanchini; Giambattista Bonanno
Journal:  PLoS One       Date:  2020-12-17       Impact factor: 3.240

4.  Improving mitochondria and ER stability helps eliminate upper motor neuron degeneration that occurs due to mSOD1 toxicity and TDP-43 pathology.

Authors:  Barış Genç; Mukesh Gautam; Öge Gözütok; Ina Dervishi; Santana Sanchez; Gashaw M Goshu; Nuran Koçak; Edward Xie; Richard B Silverman; P Hande Özdinler
Journal:  Clin Transl Med       Date:  2021-02

5.  NU-9 improves health of hSOD1G93A mouse upper motor neurons in vitro, especially in combination with riluzole or edaravone.

Authors:  Barış Genç; Mukesh Gautam; Benjamin R Helmold; Nuran Koçak; Aksu Günay; Gashaw M Goshu; Richard B Silverman; P Hande Ozdinler
Journal:  Sci Rep       Date:  2022-03-30       Impact factor: 4.379

6.  Mitochondrial dysregulation occurs early in ALS motor cortex with TDP-43 pathology and suggests maintaining NAD+ balance as a therapeutic strategy.

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  6 in total

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