Evidence map›Paper›PMID 36121612›Full record

ReviewDrugs2022

Pharmacotherapy for Amyotrophic Lateral Sclerosis: A Review of Approved and Upcoming Agents.

Stephen A Johnson, Ton Fang, Fabiola De Marchi, Dylan Neel, Donatienne Van Weehaeghe, James D Berry, Sabrina Paganoni

Abstract readReview
PubMed Publisher
In one paragraph

Review in Drugs, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
47citing papers in PubMed, 1 pooled it
10.3field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

47 citing papers in PubMed, 1 synthesis or guideline pooled it, 85 citations in OpenAlex.

  1. A systematic review and functionalFrontiers in neuroscience · 2025
    Pooled it
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors at 6 institutions in 3 countries.

Stephen A Johnson *Neurological Clinical Research Institute (NCRI), Healey & AMG Center for ALS, Massachusetts General Hospital, 165 Cambridge St, Suite 600, Boston, MA, 02114, USA.ORCID http://orcid.org/0000-0002-9058-4331
Ton Fang *University of Massachusetts Medical School, Worcester, MA, USA.ORCID http://orcid.org/0000-0003-2119-9015
Fabiola De Marchi *Department of Neurology, ALS Centre, Maggiore della Carità Hospital, Novara, Italy.ORCID http://orcid.org/0000-0003-0197-1880
Dylan NeelHarvard Medical School, Boston, MA, USA.ORCID http://orcid.org/0000-0001-6964-9842
Donatienne Van WeehaegheNuclear Medicine Subdivision, Department of Imaging and Pathology, University Hospital Leuven, Leuven, Belgium.ORCID http://orcid.org/0000-0001-6646-106X
James D BerryNeurological Clinical Research Institute (NCRI), Healey & AMG Center for ALS, Massachusetts General Hospital, 165 Cambridge St, Suite 600, Boston, MA, 02114, USA.ORCID http://orcid.org/0000-0003-0898-6081
Sabrina PaganoniNeurological Clinical Research Institute (NCRI), Healey & AMG Center for ALS, Massachusetts General Hospital, 165 Cambridge St, Suite 600, Boston, MA, 02114, USA. spaganoni@mgh.harvard.edu.ORCID http://orcid.org/0000-0003-0505-1168
Massachusetts General Hospital · USHarvard University · USKU Leuven · BESpaulding Rehabilitation Hospital · USUniversità degli Studi del Piemonte Orientale “Amedeo Avogadro” · ITUniversity of Massachusetts Chan Medical School · US

Funding

Medical Scientist Training ProgramT32GM007753 · NIGMS · HARVARD UNIVERSITY (MEDICAL SCHOOL) · PI WALENSKY, LOREN DAVID · 1985 to 2021
$50.0M
Medical Scientist Training ProgramT32GM144273 · NIGMS · HARVARD MEDICAL SCHOOL · PI David Shumway Jones, Jacqueline A. Lees · 2022 to 2026
$14.7M
NIGMS NIH HHS T32 GM007753
6 · The paper itself

Abstract

Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder involving loss of upper and lower motor neurons, with most cases ending in death within 3-5 years of onset. Several molecular and cellular pathways have been identified to cause ALS; however, treatments to stop or reverse disease progression are yet to be found. Riluzole, a neuroprotective agent offering only a modest survival benefit, has long been the sole disease-modifying therapy for ALS. Edaravone, which demonstrated statistically significant slowing of ALS disease progression, is gaining approval in an increasing number of countries since its first approval in 2015. Sodium phenylbutyrate and taurursodiol (PB-TURSO) was conditionally approved in Canada in 2022, having shown significant slowing of disease progression and prolonged survival. Most clinical trials have focused on testing small molecules affecting common cellular pathways in ALS: targeting glutamatergic, apoptotic, inflammatory, and oxidative stress mechanisms among others. More recently, clinical trials utilizing stem cell transplantation and other biologics have emerged. This rich and ever-growing pipeline of investigational products, along with innovative clinical trial designs, collaborative trial networks, and an engaged ALS community', provide renewed hope to finding a cure for ALS. This article reviews existing ALS therapies and the current clinical drug development pipeline.

Indexed as

Amyotrophic Lateral SclerosisBiological ProductsNeuroprotective AgentsClinical Trials as TopicDisease ProgressionEdaravoneHumansRiluzoleBiological ProductsEdaravoneNeuroprotective AgentsRiluzole

Identifiers

PMID36121612
OpenAlexW4296324533

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.