Evidence map›Paper›PMID 36543887›Full record

ReviewNature reviews. Drug discovery2023

Amyotrophic lateral sclerosis: a neurodegenerative disorder poised for successful therapeutic translation.

Richard J Mead, Ning Shan, H Joseph Reiser, Fiona Marshall, Pamela J Shaw

2 registry-linked trialsOpen access · bronzeAbstract readReview
In one paragraph

Review in Nature reviews. Drug discovery, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 277 papers, 7 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
277citing papers in PubMed, 7 pooled it
53.4field-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.

NCT06782958 phase1enrolling by invitationnot on this mapstarted 2024, after this paper: background citation

A Phase I Clinical Study to Evaluate the Safety, Tolerability, and Pharmacokinetic Characteristics of FHND1002 Granules After Single and Multiple Doses in Healthy Adult Volunteers

TypeinterventionalSponsorJiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd.Ran2024 to 2025Enrolled72ConditionsAmyotrophic Lateral Sclerosis (ALS)Arms50mg FHND1002, 100mg FHND1002, 150 mg FHND1002, 200 mg FHND1002(fasting), 250mg FHND1002
NCT07138014 phase2not yet recruitingnot on this mapstarted 2025, after this paper: background citation

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase II Study to Evaluate the Efficacy and Safety of FHND1002 Granules in Patients With Amyotrophic Lateral Sclerosis (ALS)

TypeinterventionalSponsorJiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd.Ran2025 to 2028Enrolled180ConditionsAmyotrophic Lateral Sclerosis (ALS)ArmsFHND1002 100mg, FHND1002 200mg, Placebo
3 · Its place in the literature

Who cites it

277 citing papers in PubMed, 7 syntheses or guidelines pooled it, 438 citations in OpenAlex.

  1. Pooled it
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  3. Exploring dysregulated miRNAs in ALS: implications for disease pathogenesis and early diagnosis.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025
    Pooled it
  4. A systematic review and functionalFrontiers in neuroscience · 2025
    Pooled it
  5. Pooled it
  6. Efficacy of pain management strategies in adults with Amyotrophic Lateral Sclerosis (ALS): A Systematic Review.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024
    Pooled it
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217 more citing papers are in PubMed but not listed here.

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

5 authors at 2 institutions in 1 country.

Richard J MeadSheffield Institute for Translational Neuroscience, Faculty of Medicine, Dentistry and Health, University of Sheffield, Sheffield, UK.ORCID http://orcid.org/0000-0002-3207-0068
Ning ShanAclipse Therapeutics, Radnor, PA, US.
H Joseph ReiserAclipse Therapeutics, Radnor, PA, US.ORCID http://orcid.org/0000-0001-5728-2879
Fiona MarshallMSD UK Discovery Centre, Merck, Sharp and Dohme (UK) Limited, London, UK.ORCID http://orcid.org/0000-0002-8681-005X
Pamela J ShawSheffield Institute for Translational Neuroscience, Faculty of Medicine, Dentistry and Health, University of Sheffield, Sheffield, UK. pamela.shaw@sheffield.ac.uk.ORCID http://orcid.org/0000-0002-8925-2567
e-Therapeutics (United Kingdom) · GBMSD (UK) Limited (United Kingdom) · GB

Funding

Medical Research Council MR/V027735/1
6 · The paper itself

Abstract

Amyotrophic lateral sclerosis (ALS) is a devastating disease caused by degeneration of motor neurons. As with all major neurodegenerative disorders, development of disease-modifying therapies has proven challenging for multiple reasons. Nevertheless, ALS is one of the few neurodegenerative diseases for which disease-modifying therapies are approved. Significant discoveries and advances have been made in ALS preclinical models, genetics, pathology, biomarkers, imaging and clinical readouts over the last 10-15 years. At the same time, novel therapeutic paradigms are being applied in areas of high unmet medical need, including neurodegenerative disorders. These developments have evolved our knowledge base, allowing identification of targeted candidate therapies for ALS with diverse mechanisms of action. In this Review, we discuss how this advanced knowledge, aligned with new approaches, can enable effective translation of therapeutic agents from preclinical studies through to clinical benefit for patients with ALS. We anticipate that this approach in ALS will also positively impact the field of drug discovery for neurodegenerative disorders more broadly.

Indexed as

Amyotrophic Lateral SclerosisBiomarkersDrug DiscoveryHumansMotor NeuronsBiomarkers

Identifiers

PMID36543887
PMCPMC9768794
OpenAlexW4312045033

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

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.