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Literature DB >> 19019302

ALS drug development: reflections from the past and a way forward.

Abstract

Tremendous advances in our understanding of pathogenesis of amyotrophic lateral sclerosis (ALS) have provided a rich pipeline of drugs for clinical trialists. At least 32 unique compounds have been tested. Nevertheless, riluzole is currently the only treatment that prolongs survival. We present a critical overview of past clinical trials, how therapies are selected for testing in people, challenges with ALS clinical trial design and conduct, and ways to best move forward.

Entities: Chemical Disease Gene Species

Mesh：

Substances：

Year: 2008 PMID： 19019302 PMCID： PMC4514695 DOI： 10.1016/j.nurt.2008.08.002

Source DB: PubMed Journal: Neurotherapeutics ISSN： 1878-7479 Impact factor: 7.620

64 in total

1. A controlled trial of recombinant methionyl human BDNF in ALS: The BDNF Study Group (Phase III).

Authors:
Journal: Neurology Date: 1999-04-22 Impact factor: 9.910

2. An adverse property of a familial ALS-linked SOD1 mutation causes motor neuron disease characterized by vacuolar degeneration of mitochondria.

Authors: P C Wong; C A Pardo; D R Borchelt; M K Lee; N G Copeland; N A Jenkins; S S Sisodia; D W Cleveland; D L Price
Journal: Neuron Date: 1995-06 Impact factor: 17.173

3. Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomised trial.

Authors: Paul H Gordon; Dan H Moore; Robert G Miller; Julaine M Florence; Joseph L Verheijde; Carolyn Doorish; Joan F Hilton; G Mark Spitalny; Robert B MacArthur; Hiroshi Mitsumoto; Hans E Neville; Kevin Boylan; Tahseen Mozaffar; Jerry M Belsh; John Ravits; Richard S Bedlack; Michael C Graves; Leo F McCluskey; Richard J Barohn; Rup Tandan
Journal: Lancet Neurol Date: 2007-11-05 Impact factor: 44.182

4. VEGF is a modifier of amyotrophic lateral sclerosis in mice and humans and protects motoneurons against ischemic death.

Authors: Diether Lambrechts; Erik Storkebaum; Masafumi Morimoto; Jurgen Del-Favero; Frederik Desmet; Stefan L Marklund; Sabine Wyns; Vincent Thijs; Jörgen Andersson; Ingrid van Marion; Ammar Al-Chalabi; Stephanie Bornes; Rhiannon Musson; Valerie Hansen; Lars Beckman; Rolf Adolfsson; Hardev Singh Pall; Hervé Prats; Severine Vermeire; Paul Rutgeerts; Shigehiro Katayama; Takuya Awata; Nigel Leigh; Loïc Lang-Lazdunski; Mieke Dewerchin; Christopher Shaw; Lieve Moons; Robert Vlietinck; Karen E Morrison; Wim Robberecht; Christine Van Broeckhoven; Désiré Collen; Peter M Andersen; Peter Carmeliet
Journal: Nat Genet Date: 2003-08 Impact factor: 38.330

5. A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis.

Authors: M E Cudkowicz; J M Shefner; D A Schoenfeld; R H Brown; H Johnson; M Qureshi; M Jacobs; J D Rothstein; S H Appel; R M Pascuzzi; T D Heiman-Patterson; P D Donofrio; W S David; J A Russell; R Tandan; E P Pioro; K J Felice; J Rosenfeld; R N Mandler; G M Sachs; W G Bradley; E M Raynor; G D Baquis; J M Belsh; S Novella; J Goldstein; J Hulihan
Journal: Neurology Date: 2003-08-26 Impact factor: 9.910

6. Chronic inhibition of glutamate uptake produces a model of slow neurotoxicity.

Authors: J D Rothstein; L Jin; M Dykes-Hoberg; R W Kuncl
Journal: Proc Natl Acad Sci U S A Date: 1993-07-15 Impact factor: 11.205

7. Mitochondrial dysfunction in a cell culture model of familial amyotrophic lateral sclerosis.

Authors: Fiona M Menzies; Mark R Cookson; Robert W Taylor; Douglass M Turnbull; Zofia M A Chrzanowska-Lightowlers; Lichun Dong; Denise A Figlewicz; Pamela J Shaw
Journal: Brain Date: 2002-07 Impact factor: 13.501

8. Investigation of a null mutation of the CNTF gene in familial amyotrophic lateral sclerosis.

Authors: R W Orrell; A W King; R J Lane; J S de Belleroche
Journal: J Neurol Sci Date: 1995-10 Impact factor: 3.181

9. Paraoxonase promoter and intronic variants modify risk of sporadic amyotrophic lateral sclerosis.

Authors: Simon Cronin; Matthew J Greenway; Jochen H M Prehn; Orla Hardiman
Journal: J Neurol Neurosurg Psychiatry Date: 2007-09 Impact factor: 10.154

10. SOD1 mutants linked to amyotrophic lateral sclerosis selectively inactivate a glial glutamate transporter.

Authors: D Trotti; A Rolfs; N C Danbolt; R H Brown; M A Hediger
Journal: Nat Neurosci Date: 1999-05 Impact factor: 24.884

20 in total

Review 1. How can we improve clinical trials in amyotrophic lateral sclerosis?

Authors: Paul H Gordon; Vincent Meininger
Journal: Nat Rev Neurol Date: 2011-09-27 Impact factor: 42.937

2. Chemical-genetic screen identifies riluzole as an enhancer of Wnt/β-catenin signaling in melanoma.

Authors: Travis L Biechele; Nathan D Camp; Daniel M Fass; Rima M Kulikauskas; Nick C Robin; Bryan D White; Corinne M Taraska; Erin C Moore; Jeanot Muster; Rakesh Karmacharya; Stephen J Haggarty; Andy J Chien; Randall T Moon
Journal: Chem Biol Date: 2010-11-24

Review 3. Amyotrophic Lateral Sclerosis: An update for 2013 Clinical Features, Pathophysiology, Management and Therapeutic Trials.

Authors: Paul H Gordon
Journal: Aging Dis Date: 2013-10-01 Impact factor: 6.745

4. Utility of dissociated intrinsic hand muscle atrophy in the diagnosis of amyotrophic lateral sclerosis.

Authors: Parvathi Menon; Steve Vucic
Journal: J Vis Exp Date: 2014-03-04 Impact factor: 1.355

Review 5. Molecular pathways of motor neuron injury in amyotrophic lateral sclerosis.

Authors: Laura Ferraiuolo; Janine Kirby; Andrew J Grierson; Michael Sendtner; Pamela J Shaw
Journal: Nat Rev Neurol Date: 2011-11 Impact factor: 42.937

6. Proton MR spectroscopy-detectable major neurotransmitters of the brain: biology and possible clinical applications.

Authors: N Agarwal; P F Renshaw
Journal: AJNR Am J Neuroradiol Date: 2011-12-29 Impact factor: 3.825

7. Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS.

Authors: Kevin D Foust; Desirée L Salazar; Shibi Likhite; Laura Ferraiuolo; Dara Ditsworth; Hristelina Ilieva; Kathrin Meyer; Leah Schmelzer; Lyndsey Braun; Don W Cleveland; Brian K Kaspar
Journal: Mol Ther Date: 2013-09-06 Impact factor: 11.454