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Review
. 2024 Dec;70(6):1140-1150.
doi: 10.1002/mus.28244. Epub 2024 Sep 18.

Access for ALL in ALS: A large-scale, inclusive, collaborative consortium to unlock the molecular and genetic mechanisms of amyotrophic lateral sclerosis

James D Berry  1 , Sabrina Paganoni  1 , Matthew B Harms  2 , Neil Shneider  2 , Jinsy Andrews  2 , Timothy M Miller  3 , Suma Babu  1 , Alex V Sherman  1 , Brent T Harris  4 , Frank A Provenzano  2 , Hemali P Phatnani  2 , Jeremy Shefner  5 , Mark A Garret  1 , Shaffeeq S Ladha  5 , Amy Y Tsou  6 , Praveena Mohan  5 , Courtney Igne  1 ; ALL ALS Consortium; Robert Bowser  5
Affiliations
Review

Access for ALL in ALS: A large-scale, inclusive, collaborative consortium to unlock the molecular and genetic mechanisms of amyotrophic lateral sclerosis

James D Berry et al. Muscle Nerve. 2024 Dec.

Abstract

Recent progress in therapeutics for amyotrophic lateral sclerosis (ALS) has spurred development and imbued the field of ALS with hope for more breakthroughs, yet substantial scientific gaps persist. This unmet need remains a stark reminder that innovative paradigms are needed to invigorate ALS research. To move toward more informative, targeted, and personalized drug development, the National Institutes of Health (NIH) established a national ALS clinical research consortium called Access for ALL in ALS (ALL ALS). This new consortium is a multi-institutional effort that aims to organize the ALS clinical research landscape in the United States. ALL ALS is operating in partnership with several stakeholders to operationalize the recommendations of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS) Public Private Partnership. ALL ALS will provide a large-scale, centralized, and readily accessible infrastructure for the collection and storage of a wide range of data from people living with ALS (symptomatic cohort) or who may be at risk of developing ALS (asymptomatic ALS gene carriers). Importantly, ALL ALS is designed to encourage community engagement, equity, and inclusion. The consortium is prioritizing the enrollment of geographically, ethnoculturally, and socioeconomically diverse participants. Collected data include longitudinal clinical data and biofluids, genomic, and digital biomarkers that will be harmonized and linked to the central Accelerating Medicines Partnership for ALS (AMP ALS) portal for sharing with the research community. The aim of ALL ALS is to deliver a comprehensive, inclusive, open-science dataset to help researchers answer important scientific questions of clinical relevance in ALS.

Keywords: amyotrophic lateral sclerosis; asymptomatic gene carriers; biomarkers; natural history; observational study.

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Conflict of interest statement

Conflicts of Interest

James D. Berry reports research grants from Biogen, MT Pharma of America, Alexion, Rapa Therapeutics, the ALS Association, the Muscular Dystrophy Association, ALS One, ALS Finding a Cure, Tambourine, ALS Finding a Cure, DoD, NINDS, and reports personal consulting fees from Biogen, Clene Nanomedicine, MT Pharma of America, Janssen, Projects in Knowledge, Roon, and is a paid DSMB member for Sanofi.

Sabrina Paganoni reports research grants from Amylyx Therapeutics, Revalesio Corporation, Eledon, Alector, UCB, Biohaven, Clene Nanomedicine, Prilenia, Seelos, Calico, Denali, NIH, CDC, DoD, The ALS Association, the American Academy of Neurology, ALS Finding a Cure, the Salah Foundation, the Spastic Paraplegia Foundation, the Muscular Dystrophy Association, Tambourine and reports consulting fees from Amylyx, Arrowhead, Janssen, Medscape, and Cytokinetics.

Matthew Harms Biogen, Ionis, uniQure, Amylyx, Sarepta, ALS Association, ALS United, ALS Finding a Cure, Muscular Dystrophy Association, Target ALS.

Neil Shneider reports no disclosures.

Jinsy Andrews has served as a consultant for AL-S Pharma, Amylyx, Apellis, Biogen, Clene, Cytokinetics, Denali, Neurosense, Novartis, Akava, QurAlis, Sanofi, UCB and Wave Therapeutics; Received research funding to the institution from ALS Association, Amylyx, Biogen, Novartis, Cytokinetics, Biohaven/Clene/Ra Pharma/Prilenia/Seelos/Calico/Denali MGH Foundation [ALS Platform Trial], Corcept and NIH/NINDS.

Timothy Miller reports compensation received for consulting and licensing from Ionis, consulting from Bioge and Bioio, licensing from C2N and Honorarium from Denali.

Suma Babu has received research funding from, OrphAI therapeutics, Biogen, Denali Therapeutics, Novartis, Orion, Ionis, American Academy of Neurology, AANEM Foundation and Muscular Dystrophy Association and reports consulting fees from Biogen, OrphAI therapeutics and Medscape.

Alexander Sherman has received grants and contracts for clinical research projects sponsored by NIH, FDA, The ALS Association, ALS Finding a Cure Foundation, Biogen, Mitsubishi Tanabe Pharma America, Inc., and Amylyx Pharmaceuticals.

Brent Harris reports compensation received as a consultant for Dept of Justice and HHS, President for Neuropathology Consults, LLC, Consultant for Office of the Chief Medical Examiner of DC, Medical/Scientific Board member for non-profits (Manfull Fund, Neuroimmune Foundation)

Frank Provenzano reports compensation received as a consultant for Tesarakt.

Hemali Phatnani reports no conflicts of interest

Jeremy Shefner reports compensation received as a consultant from Amylyx, Annexon, Acurastem, Eikonizo, Uniqure, NeuroSense Therapeutics, Cytokinetics, Denali Therapeutics, GSK, Mitsubishi Tanabe Pharma America, Orthogonal, RRD, SwanBio, Novartis, Sanofi, PTC, Vertex, and EMD Serono; and research support from Amylyx, Biogen, Biotie Therapies (now Acorda Therapeutics), Cytokinetics, Mitsubishi Tanabe Pharma America, Alexion, MediciNova Inc, Ionis, Alector, and Orphazyme.

Mark Garret reports research support from Biogen, uniQure, NINDS, FDA, Target ALS, ALS Finding a Cure, and ALS Association.

Shaffeq Ladha reports consulting fees from Amylyx, Annexon, UCB Pharma, Genentech, Sanofi, Neurosense and Rapa Therapeutics

Amy Tsou reports no conflicts of interest

Praveena Mohan reports no conflicts of interest

Courtney Igne reports no conflicts of interest.

Robert Bowser reports research grants from Target ALS, NINDS, Bristol-Myers Squibb, and reports consulting fees from MT Pharma of America, Brainstorm, Eikonizo Therapeutics, Athira Pharma, Inc., Takeda, Neurosense Therapeutics, and AcuraStem.

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