Evidence map›Paper›PMID 32043626›Full record

ReviewMedicinal research reviews2020

The clinical trial landscape in amyotrophic lateral sclerosis-Past, present, and future.

Heike J Wobst, Korrie L Mack, Dean G Brown, Nicholas J Brandon, James Shorter

Open access · greenAbstract readReview
In one paragraph

Review in Medicinal research reviews, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
41citing papers in PubMed, 2 pooled it
5.6field-weighted citation impact, top 3% 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

41 citing papers in PubMed, 2 syntheses or guidelines pooled it, 84 citations in OpenAlex.

  1. Multidisciplinary care in Amyotrophic Lateral Sclerosis: a systematic review and meta-analysis.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2021
    Pooled it
  2. Pooled it
  3. Article
  4. Introduction to the Supplement.Muscle & nerve · 2026
    Article
  5. Article
  6. Review
  7. Article
  8. Article
  9. Article
  10. Article
  11. Article
  12. Article
  13. Review
  14. Long Non-Coding RNAs: New Insights in Neurodegenerative Diseases.International journal of molecular sciences · 2024
    Review
  15. Review
  16. Review
  17. Article
  18. Article
  19. Review
  20. Review
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 3 institutions in 1 country.

Heike J WobstNeuroscience, BioPharmaceuticals R&D, AstraZeneca, Boston, Massachusetts.ORCID 0000-0002-9041-3241
Korrie L MackDepartment of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Dean G BrownHit Discovery, Discovery Sciences, BioPharmaceuticals R&D, AstraZeneca, Boston, Massachusetts.
Nicholas J BrandonNeuroscience, BioPharmaceuticals R&D, AstraZeneca, Boston, Massachusetts.ORCID 0000-0002-3917-0096
James ShorterDepartment of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.ORCID 0000-0001-5269-8533
Jnana Therapeutics (United States) · USMerck & Co., Inc., Rahway, NJ, USA (United States) · USUniversity of Pennsylvania · US

Funding

Defining the mechanistic basis of a prion disaggregaseR01GM099836 · NIGMS · UNIVERSITY OF PENNSYLVANIA · PI SHORTER, JAMES · 2013 to 2024
$3.9M
Programming human protein disaggregases against FTDR21AG065854 · NIA · UNIVERSITY OF PENNSYLVANIA · PI SHORTER, JAMES · 2020 to 2020
$446k
Isolating small-molecule enhancers of HtrA1, an alpha-synuclein disaggregaseR21NS102687 · NINDS · UNIVERSITY OF PENNSYLVANIA · PI SHORTER, JAMES · 2017 to 2018
$441k
Isolating small-molecule enhancers of the human alpha-synuclein disaggregase machineryR21AG061784 · NIA · UNIVERSITY OF PENNSYLVANIA · PI SHORTER, JAMES · 2019 to 2020
$441k
Exploring and enhancing Karyopherin beta-2 disaggregate activityR21NS090205 · NINDS · UNIVERSITY OF PENNSYLVANIA · PI SHORTER, JAMES · 2016 to 2017
$432k
NIA NIH HHS R21 AG061784NIA NIH HHS R21 AG065854NIGMS NIH HHS R01 GM099836NINDS NIH HHS R21 NS090205NINDS NIH HHS R21 NS102687
6 · The paper itself

Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease marked by progressive loss of muscle function. It is the most common adult-onset form of motor neuron disease, affecting about 16 000 people in the United States alone. The average survival is about 3 years. Only two interventional drugs, the antiglutamatergic small-molecule riluzole and the more recent antioxidant edaravone, have been approved for the treatment of ALS to date. Therapeutic strategies under investigation in clinical trials cover a range of different modalities and targets, and more than 70 different drugs have been tested in the clinic to date. Here, we summarize and classify interventional therapeutic strategies based on their molecular targets and phenotypic effects. We also discuss possible reasons for the failure of clinical trials in ALS and highlight emerging preclinical strategies that could provide a breakthrough in the battle against this relentless disease.

Indexed as

Clinical Trials as TopicAmyotrophic Lateral SclerosisAnti-Inflammatory AgentsAutophagyDrug ApprovalHumansImmunologic FactorsAnti-Inflammatory AgentsImmunologic FactorsALS preclinical strategiesamyotrophic lateral sclerosisclinical trialsedaravoneriluzole

Identifiers

PMID32043626
PMCPMC7417284
OpenAlexW3006375948

What OpenQuestion holds

Textmetadata
LicenceTDM
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.