Evidence map›Paper›PMID 36271285›Full record

ArticleScientific reports2022

Disease stages and therapeutic hypotheses in two decades of neurodegenerative disease clinical trials.

Meredith A Mortberg, Sonia M Vallabh, Eric Vallabh Minikel

Open access · goldAbstract read
In one paragraph

Article in Scientific reports, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
26citing papers in PubMed, 1 pooled it
8.7field-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

26 citing papers in PubMed, 1 synthesis or guideline pooled it, 32 citations in OpenAlex.

  1. Pooled it
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  9. In Vitro Models for Peripheral Nerve Regeneration.Advanced healthcare materials · 2024
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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

3 authors at 2 institutions in 1 country.

Meredith A MortbergMcCance Center for Brain Health and Department of Neurology, Massachusetts General Hospital, Boston, MA, 02114, USA.
Sonia M VallabhMcCance Center for Brain Health and Department of Neurology, Massachusetts General Hospital, Boston, MA, 02114, USA.
Eric Vallabh MinikelMcCance Center for Brain Health and Department of Neurology, Massachusetts General Hospital, Boston, MA, 02114, USA. eminikel@broadinstitute.org.
Massachusetts General Hospital · USBroad Institute · US

Funding

How Substrate Dosage Drives Prion Disease KineticsR01NS125255 · NINDS · BROAD INSTITUTE, INC. · PI Sonia Minikel Vallabh · 2022 to 2026
$2.7M
Biomarkers to enable ASO prevention trials in genetic prion disease carriersR21TR003040 · NCATS · MASSACHUSETTS GENERAL HOSPITAL · PI ARNOLD, STEVEN E · 2019 to 2020
$485k
NCATS NIH HHS R21 TR003040NIH HHS R01 NS125255
6 · The paper itself

Abstract

Neurodegenerative disease is increasingly prevalent and remains without disease-modifying therapies. Engaging the right target, at the right disease stage, could be an important determinant of success. We annotated targets and eligibility criteria for 3238 neurodegenerative disease trials registered at ClinicalTrials.gov from 2000 to 2020. Trials became more selective as the mean number of inclusion and exclusion criteria increased and eligible score ranges shrank. Despite a shift towards less impaired participants, only 2.7% of trials included pre-symptomatic individuals; these were depleted for drug trials and enriched for behavioral interventions. Sixteen novel, genetically supported therapeutic hypotheses tested in drug trials represent a small, non-increasing fraction of trials, and the mean lag from genetic association to first trial was 13 years. Though often linked to disease initiation, not progression, these targets were tested mostly at symptomatic disease stages. The potential for disease modification through early intervention against root molecular causes of disease remains largely unexplored.

Indexed as

Neurodegenerative DiseasesHumans

Identifiers

PMID36271285
PMCPMC9587287
OpenAlexW4307028181

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.