507
Views
64
CrossRef citations to date
0
Altmetric
REVIEW ARTICLE

Toward more efficient clinical trials for amyotrophic lateral sclerosis

, , , , , , , , , , , , , , , , , , & show all
Pages 259-265 | Received 03 Jul 2009, Accepted 21 Sep 2009, Published online: 04 Dec 2009

Keep up to date with the latest research on this topic with citation updates for this article.

Read on this site (9)

R Hergesheimer, D Lanznaster, P Vourc’h, Cr Andres, Se Bakkouche, S Beltran, H Blasco, P Corcia & P Couratier. (2020) Advances in disease-modifying pharmacotherapies for the treatment of amyotrophic lateral sclerosis. Expert Opinion on Pharmacotherapy 21:9, pages 1103-1110.
Read now
Pablo Villoslada & Lawrence Steinman. (2020) New targets and therapeutics for neuroprotection, remyelination and repair in multiple sclerosis. Expert Opinion on Investigational Drugs 29:5, pages 443-459.
Read now
Svitlana Garbuzova-Davis, Avery Thomson, Crupa Kurien, R. Douglas Shytle & Paul R. Sanberg. (2016) Potential new complication in drug therapy development for amyotrophic lateral sclerosis. Expert Review of Neurotherapeutics 16:12, pages 1397-1405.
Read now
Abigail DeLoach, Michael Cozart, Arianna Kiaei & Mahmoud Kiaei. (2015) A retrospective review of the progress in amyotrophic lateral sclerosis drug discovery over the last decade and a look at the latest strategies. Expert Opinion on Drug Discovery 10:10, pages 1099-1118.
Read now
Michael E. Bozik, Hiroshi Mitsumoto, Benjamin R. Brooks, Stacy A. Rudnicki, Dan H. Moore, Bing Zhang, Albert Ludolph, Merit E. Cudkowicz, Leonard H. van den Berg, James Mather, Thomas Petzinger$suffix/text()$suffix/text() & Donald Archibald. (2014) A post hoc analysis of subgroup outcomes and creatinine in the phase III clinical trial (EMPOWER) of dexpramipexole in ALS. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 15:5-6, pages 406-413.
Read now
Martin R. Turner, Robert Bowser, Lucie Bruijn, Luc Dupuis, Albert Ludolph, Michael McGrath, Giovanni Manfredi, Nicholas Maragakis, Robert G. Miller, Seth L. Pullman, Seward B. Rutkove, Pamela J. Shaw, Jeremy Shefner & Kenneth H. Fischbeck. (2013) Mechanisms, models and biomarkers in amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 14:sup1, pages 19-32.
Read now
Seward B. Rutkove, James B. Caress, Michael S. Cartwright, Ted M. Burns, Judy Warder, William S. David, Namita Goyal, Nicholas J. Maragakis, Lora Clawson, Michael Benatar, Sharon Usher, Khema R. Sharma, Shiva Gautam, Pushpa Narayanaswami, Elizabeth M. Raynor, Mary Lou Watson & Jeremy M. Shefner. (2012) Electrical impedance myography as a biomarker to assess ALS progression. Amyotrophic Lateral Sclerosis 13:5, pages 439-445.
Read now
Bharath Wootla, Aleksandar Denic, Arthur E Warrington & Moses Rodriguez. (2012) Need for a paradigm shift in therapeutic approaches to CNS injury. Expert Review of Neurotherapeutics 12:4, pages 409-420.
Read now
Magdalena Kuzma-Kozakiewicz & Hubert Kwiecinski. (2011) New therapeutic targets for amyotrophic lateral sclerosis. Expert Opinion on Therapeutic Targets 15:2, pages 127-143.
Read now

Articles from other publishers (55)

Anoopum S. Gupta, Siddharth Patel, Alan Premasiri & Fernando Vieira. (2023) At-home wearables and machine learning sensitively capture disease progression in amyotrophic lateral sclerosis. Nature Communications 14:1.
Crossref
Fernando G. Vieira, Subhashini Venugopalan, Alan S. Premasiri, Maeve McNally, Aren Jansen, Kevin McCloskey, Michael P. Brenner & Steven Perrin. (2022) A machine-learning based objective measure for ALS disease severity. npj Digital Medicine 5:1.
Crossref
Jonathan D. Glass & Christina N. Fournier. (2022) Unintended Consequences of Approving Unproven Treatments—Hope, Hype, or Harm?. JAMA Neurology 79:2, pages 117.
Crossref
Manfred Berres, Andreas U. Monsch & René Spiegel. (2021) Using historical data to facilitate clinical prevention trials in Alzheimer disease? An analysis of longitudinal MCI (mild cognitive impairment) data sets. Alzheimer's Research & Therapy 13:1.
Crossref
Eleonora Dalla Bella, Enrica Bersano, Giovanni Antonini, Giuseppe Borghero, Margherita Capasso, Claudia Caponnetto, Adriano Chiò, Massimo Corbo, Massimiliano Filosto, Fabio Giannini, Rossella Spataro, Christian Lunetta, Jessica Mandrioli, Sonia Messina, Maria Rosaria Monsurrò, Gabriele Mora, Nilo Riva, Romana Rizzi, Gabriele Siciliano, Vincenzo Silani, Isabella Simone, Gianni Sorarù, Valeria Tugnoli, Lorenzo Verriello, Paolo Volanti, Roberto Furlan, John M Nolan, Emmanuelle Abgueguen, Irene Tramacere & Giuseppe Lauria. (2021) The unfolded protein response in amyotrophic later sclerosis: results of a phase 2 trial. Brain 144:9, pages 2635-2647.
Crossref
Kristiana Salmon & Angela Genge. 2021. Spectrums of Amyotrophic Lateral Sclerosis. Spectrums of Amyotrophic Lateral Sclerosis 161 180 .
Steve Vucic, Julie Ryder, Linda Mekhael, Henderson RD, Susan Mathers, Merilee Needham, Schultz DW & Kiernan MC. (2020) Phase 2 randomized placebo controlled double blind study to assess the efficacy and safety of tecfidera in patients with amyotrophic lateral sclerosis (TEALS Study). Medicine 99:6, pages e18904.
Crossref
Masihuz Zaman, Asra Nasir Khan, Wahiduzzaman, Syed Mohammad Zakariya & Rizwan Hasan Khan. (2019) Protein misfolding, aggregation and mechanism of amyloid cytotoxicity: An overview and therapeutic strategies to inhibit aggregation. International Journal of Biological Macromolecules 134, pages 1022-1037.
Crossref
Leonard H. van den BergEric SorensonGary GronsethEric A. MacklinJinsy AndrewsRobert H. BalohMichael BenatarJames D. BerryAdriano Chio, Philippe CorciaAngela GengeAmelie K. GubitzCatherine Lomen-HoerthChristopher J. McDermottErik P. PioroJeffrey Rosenfeld, Vincenzo SilaniMartin R. TurnerMarkus WeberBenjamin Rix BrooksRobert G. MillerHiroshi Mitsumoto. (2019) Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials. Neurology 92:14.
Crossref
Stephanie R. ShepheardJoanne WuuMichell CardosoLuke WiklendtPhil G. DinningTim ChatawayDavid SchultzMichael BenatarMary-Louise Rogers. (2017) Urinary p75 ECD . Neurology 88:12, pages 1137-1143.
Crossref
Pablo Villoslada. (2016) Neuroprotective therapies for multiple sclerosis and other demyelinating diseases. Multiple Sclerosis and Demyelinating Disorders 1:1.
Crossref
Jonathan D. GlassVicki S. HertzbergNicholas M. BoulisJonathan RileyThais FedericiMeraida PolakJane BordeauChristina FournierKarl JoheTom HazelMerit CudkowiczNazem AtassiLawrence F. BorgesSeward B. RutkoveJayna DuellParag G. PatilStephen A. GoutmanEva L. Feldman. (2016) Transplantation of spinal cord–derived neural stem cells for ALS. Neurology 87:4, pages 392-400.
Crossref
R. Tortelli, M. Copetti, F. Panza, A. Fontana, R. Cortese, R. Capozzo, A. Introna, E. D'Errico, S. Zoccolella, S. Arcuti, D. Seripa, I. L. Simone & G. Logroscino. (2016) Time to generalization and prediction of survival in patients with amyotrophic lateral sclerosis: a retrospective observational study. European Journal of Neurology 23:6, pages 1117-1125.
Crossref
Christoph Neuwirth, Christian Burkhardt, James Alix, José Castro, Mamede de Carvalho, Malgorzata Gawel, Stephan Goedee, Julian Grosskreutz, Timothée Lenglet, Cristina Moglia, Taha Omer, Maarten Schrooten & Markus Weber. (2016) Quality Control of Motor Unit Number Index (MUNIX) Measurements in 6 Muscles in a Single-Subject “Round-Robin” Setup. PLOS ONE 11:5, pages e0153948.
Crossref
Steve Vucic. (2016) Motor Unit Number Index (MUNIX): A novel biomarker for ALS?. Clinical Neurophysiology 127:4, pages 1938-1939.
Crossref
Florian Krismer, Klaus Seppi, Nadia Stefanova & Gregor K. Wenning. (2016) Toward disease modification in multiple system atrophy: Pitfalls, bottlenecks, and possible remedies. Movement Disorders 31:2, pages 235-240.
Crossref
Sabrina Paganoni, Chafic Karam, Nanette Joyce, Richard Bedlack & Gregory T. Carter. (2015) Comprehensive rehabilitative care across the spectrum of amyotrophic lateral sclerosis. NeuroRehabilitation 37:1, pages 53-68.
Crossref
L. Galán Dávila, P. López Ruiz & J. Matías-Guiu Guía. (2015) Enfermedades de las motoneuronas. Medicine - Programa de Formación Médica Continuada Acreditado 11:78, pages 4678-4686.
Crossref
Neta Zach, David L. Ennist, Albert A. Taylor, Hagit Alon, Alexander Sherman, Robert Kueffner, Jason Walker, Ervin Sinani, Igor Katsovskiy, Merit Cudkowicz & Melanie L. Leitner. (2015) Being PRO-ACTive: What can a Clinical Trial Database Reveal About ALS?. Neurotherapeutics 12:2, pages 417-423.
Crossref
Erich Mohr. 2015. Re-Engineering Clinical Trials. Re-Engineering Clinical Trials 159 170 .
P. Hande Özdinler & Richard B. Silverman. (2014) Treatment of Amyotrophic Lateral Sclerosis: Lessons Learned from Many Failures. ACS Medicinal Chemistry Letters 5:11, pages 1179-1181.
Crossref
Nazem AtassiJames BerryAmy ShuiNeta ZachAlexander ShermanErvin SinaniJason WalkerIgor KatsovskiyDavid SchoenfeldMerit CudkowiczMelanie Leitner. (2014) The PRO-ACT database. Neurology 83:19, pages 1719-1725.
Crossref
Merit E Cudkowicz, Sarah Titus, Marianne Kearney, Hong Yu, Alexander Sherman, David Schoenfeld, Douglas Hayden, Amy Shui, Benjamin Brooks, Robin Conwit, Donna Felsenstein, David J Greenblatt, Myles Keroack, John T Kissel, Robert Miller, Jeffrey Rosenfeld, Jeffrey D Rothstein, Ericka Simpson, Nina Tolkoff-Rubin, Lorne Zinman & Jeremy M Shefner. (2014) Safety and efficacy of ceftriaxone for amyotrophic lateral sclerosis: a multi-stage, randomised, double-blind, placebo-controlled trial. The Lancet Neurology 13:11, pages 1083-1091.
Crossref
Sabrina Paganoni, Merit Cudkowicz & James D Berry. (2014) Outcome measures in amyotrophic lateral sclerosis clinical trials. Clinical Investigation 4:7, pages 605-618.
Crossref
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.
Crossref
Spencer Phillips HeyJonathan Kimmelman. (2014) The questionable use of unequal allocation in confirmatory trials. Neurology 82:1, pages 77-79.
Crossref
Ryoichi Nakamura, Naoki Atsuta, Hazuki Watanabe, Akihiro Hirakawa, Hirohisa Watanabe, Mizuki Ito, Jo Senda, Masahisa Katsuno, Fumiaki Tanaka, Yuishin Izumi, Mitsuya Morita, Kotaro Ogaki, Akira Taniguchi, Ikuko Aiba, Koichi Mizoguchi, Koichi Okamoto, Kazuko Hasegawa, Masashi Aoki, Akihiro Kawata, Koji Abe, Masaya Oda, Masaaki Konagaya, Takashi Imai, Masanori Nakagawa, Shoji Tsuji, Ryuji Kaji, Imaharu Nakano & Gen Sobue. (2013) Neck weakness is a potent prognostic factor in sporadic amyotrophic lateral sclerosis patients. Journal of Neurology, Neurosurgery & Psychiatry 84:12, pages 1365-1371.
Crossref
Andrew Eisen & Charles Krieger. (2013) Ethical considerations in the management of amyotrophic lateral sclerosis. Progress in Neurobiology 110, pages 45-53.
Crossref
Paul H Gordon. (2013) The murky path to drug discovery in ALS becomes clearer. The Lancet Neurology 12:11, pages 1037-1038.
Crossref
Merit E Cudkowicz, Leonard H van den Berg, Jeremy M Shefner, Hiroshi Mitsumoto, Jesus S Mora, Albert Ludolph, Orla Hardiman, Michael E Bozik, Evan W Ingersoll, Donald Archibald, Adam L Meyers, Yingwen Dong, Wildon R Farwell & Douglas A Kerr. (2013) Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial. The Lancet Neurology 12:11, pages 1059-1067.
Crossref
Jia Li, Minhee Sung & Seward B. Rutkove. (2013) Electrophysiologic Biomarkers for Assessing Disease Progression and the Effect of Riluzole in SOD1 G93A ALS Mice. PLoS ONE 8:6, pages e65976.
Crossref
Martin R Turner, Orla Hardiman, Michael Benatar, Benjamin R Brooks, Adriano Chio, Mamede de Carvalho, Paul G Ince, Cindy Lin, Robert G Miller, Hiroshi Mitsumoto, Garth Nicholson, John Ravits, Pamela J Shaw, Michael Swash, Kevin Talbot, Bryan J Traynor, Leonard H Van den Berg, Jan H Veldink, Steve Vucic & Matthew C Kiernan. (2013) Controversies and priorities in amyotrophic lateral sclerosis. The Lancet Neurology 12:3, pages 310-322.
Crossref
Alida Spalloni, Michele Nutini & Patrizia Longone. (2013) Role of the N-methyl-d-aspartate receptors complex in amyotrophic lateral sclerosis. Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease 1832:2, pages 312-322.
Crossref
Masahisa Katsuno, Hirohisa Watanabe, Fumiaki Tanaka & Gen Sobue. (2013) Translational research on disease‐modifying therapies for neurodegenerative diseases. Neurology and Clinical Neuroscience 1:1, pages 3-10.
Crossref
Matthew Gladman, Merit Cudkowicz & Lorne Zinman. (2012) Enhancing clinical trials in neurodegenerative disorders. Current Opinion in Neurology 25:6, pages 735-742.
Crossref
Mauro CozzolinoMaria Grazia PesaresiValeria GerbinoJulian GrosskreutzMaria Teresa Carrì. (2012) Amyotrophic Lateral Sclerosis: New Insights into Underlying Molecular Mechanisms and Opportunities for Therapeutic Intervention. Antioxidants & Redox Signaling 17:9, pages 1277-1330.
Crossref
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.
Crossref
Nazem Atassi, David Schoenfeld & Merit Cudkowicz. 2012. Clinical Trials in Neurology. Clinical Trials in Neurology 273 283 .
. 2012. Clinical Trials in Neurology. Clinical Trials in Neurology 215 308 .
. 2012. Clinical Trials in Neurology. Clinical Trials in Neurology 78 172 .
Benjamin Chee Chuan Cheah, Steve Vucic & Matthew Colm Kiernan. (2012) Potential utility of biomarkers in the diagnosis and treatment of amyotrophic lateral sclerosis. Clinical Investigation 2:1, pages 107-117.
Crossref
Masahisa Katsuno, Haruhiko Banno, Keisuke Suzuki, Atsushi Hashizume, Hiroaki Adachi, Fumiaki Tanaka & Gen Sobue. (2012) 1) Translational Research on Neurodegenerative Diseases. Nihon Naika Gakkai Zasshi 101:9, pages 2533-2538.
Crossref
Dan H Moore, Jonathan S Katz & Robert G Miller. (2011) A review of clinical trial designs in amyotrophic lateral sclerosis. Neurodegenerative Disease Management 1:6, pages 481-490.
Crossref
Merit Cudkowicz, Michael E Bozik, Evan W Ingersoll, Robert Miller, Hiroshi Mitsumoto, Jeremy Shefner, Dan H Moore, David Schoenfeld, James L Mather, Donald Archibald, Mary Sullivan, Craig Amburgey, Juliet Moritz & Valentin K Gribkoff. (2011) The effects of dexpramipexole (KNS-760704) in individuals with amyotrophic lateral sclerosis. Nature Medicine 17:12, pages 1652-1656.
Crossref
Lucy Lu Wang, Andrew J. Spieker, Jia Li & Seward B. Rutkove. (2011) Electrical impedance myography for monitoring motor neuron loss in the SOD1 G93A amyotrophic lateral sclerosis rat. Clinical Neurophysiology 122:12, pages 2505-2511.
Crossref
Orla Hardiman, Leonard H. van den Berg & Matthew C. Kiernan. (2011) Clinical diagnosis and management of amyotrophic lateral sclerosis. Nature Reviews Neurology 7:11, pages 639-649.
Crossref
Cinzia Volonté, Savina Apolloni, Maria Teresa Carrì & Nadia D'Ambrosi. (2011) ALS: Focus on purinergic signalling. Pharmacology & Therapeutics 132:1, pages 111-122.
Crossref
R.G. MillerD.H. MooreD.A. ForshewJ.S. KatzR.J. BarohnM. ValanM.B. BrombergK.L. GoslinM.C. GravesL.F. McCluskeyA.L. McVeyT. MozaffarJ.M. FlorenceA. PestronkM. RossE.P. SimpsonS.H. Appel. (2011) Phase II screening trial of lithium carbonate in amyotrophic lateral sclerosis. Neurology 77:10, pages 973-979.
Crossref
Peter D. DonofrioRichard Bedlack. (2011) Historical controls in ALS trials. Neurology 77:10, pages 936-937.
Crossref
Peter I. Joyce, Pietro Fratta, Elizabeth M. C. Fisher & Abraham Acevedo-Arozena. (2011) SOD1 and TDP-43 animal models of amyotrophic lateral sclerosis: recent advances in understanding disease toward the development of clinical treatments. Mammalian Genome 22:7-8, pages 420-448.
Crossref
Paul Wicks, Timothy E Vaughan, Michael P Massagli & James Heywood. (2011) Accelerated clinical discovery using self-reported patient data collected online and a patient-matching algorithm. Nature Biotechnology 29:5, pages 411-414.
Crossref
Richard S Bedlack. (2010) Amyotrophic lateral sclerosis: current practice and future treatments. Current Opinion in Neurology 23:5, pages 524-529.
Crossref
Jian-Ying Zhou, Leila Afjehi-Sadat, Seneshaw Asress, Duc M. Duong, Merit Cudkowicz, Jonathan D. Glass & Junmin Peng. (2010) Galectin-3 Is a Candidate Biomarker for Amyotrophic Lateral Sclerosis: Discovery by a Proteomics Approach. Journal of Proteome Research 9:10, pages 5133-5141.
Crossref
Gianfrancesco Goracci, Monica Ferrini & Vincenza Nardicchi. (2010) Low Molecular Weight Phospholipases A2 in Mammalian Brain and Neural Cells: Roles in Functions and Dysfunctions. Molecular Neurobiology 41:2-3, pages 274-289.
Crossref
Richard W Orrell, Russell JM Lane & Mark Ross. (2007) Antioxidant treatment for amyotrophic lateral sclerosis or motor neuron disease. Cochrane Database of Systematic Reviews 2011:6.
Crossref

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.