Evidence map›Paper›PMID 30400107›Full record

ArticleJournal of Parkinson's disease2019

A Proposed Roadmap for Parkinson's Disease Proof of Concept Clinical Trials Investigating Compounds Targeting Alpha-Synuclein.

Kalpana M Merchant, Jesse M Cedarbaum, Patrik Brundin, Kuldip D Dave, Jamie Eberling, Alberto J Espay, Samantha J Hutten, Monica Javidnia, Johan Luthman, Walter Maetzler and 5 more

Abstract read
In one paragraph

Article in Journal of Parkinson's disease, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
30citing papers in PubMed, 1 pooled it
–field-weighted citation impact
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

30 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Simplified quantification of [Journal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism · 2021
    Trial
  3. Trial
  4. Review
  5. Article
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  7. A framework for translating tauopathy therapeutics: Drug discovery to clinical trials.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2024
    Review
  8. Review
  9. Article
  10. Article
  11. Article
  12. Article
  13. Article
  14. A Mouse Model to Test Novel Therapeutics for Parkinson's Disease: an Update on the Thy1-aSyn ("line 61") Mice.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2023
    Review
  15. Article
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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

15 authors.

Kalpana M MerchantVincere Biosciences, Inc., and Department of Neurology, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
Jesse M CedarbaumBiogen, Cambridge, MA, USA.
Patrik BrundinVan Andel Research Institute, Center for Neurodegenerative Science, Grand Rapids, MI, USA.
Kuldip D DaveThe Michael J. Fox Foundation for Parkinson's Research, New York, NY, USA.
Jamie EberlingThe Michael J. Fox Foundation for Parkinson's Research, New York, NY, USA.
Alberto J EspayUC Gardner Center for Parkinson's Disease and Movement Disorders, University of Cincinnati, Cincinnati, OH, USA.
Samantha J HuttenThe Michael J. Fox Foundation for Parkinson's Research, New York, NY, USA.
Monica JavidniaCenter for Health and Technology, University of Rochester Medical Center, Rochester, New York, USA.
Johan LuthmanEisai, Woodcliff Lake, NJ, USA.
Walter MaetzlerDepartment of Neurology, University Hospital Schleswig-Holstein, Campus Kiel, Germany.
Liliana MenalledThe Michael J. Fox Foundation for Parkinson's Research, New York, NY, USA.
Alyssa N ReimerThe Michael J. Fox Foundation for Parkinson's Research, New York, NY, USA.
A Jon StoesslDjavad Mowafaghian Centre for Brain Health, Pacific Parkinson's Research Center, University of British Columbia, Vancouver, BC, Canada.
David M Weiner
The Michael J. Fox Foundation Alpha Synuclein Clinical Path Working Group

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The convergence of human molecular genetics and Lewy pathology of Parkinson's disease (PD) have led to a robust, clinical-stage pipeline of alpha-synuclein (α-syn)-targeted therapies that have the potential to slow or stop the progression of PD and other synucleinopathies. To facilitate the development of these and earlier stage investigational molecules, the Michael J. Fox Foundation for Parkinson's Research convened a group of leaders in the field of PD research from academia and industry, the Alpha-Synuclein Clinical Path Working Group. This group set out to develop recommendations on preclinical and clinical research that can de-risk the development of α-syn targeting therapies. This consensus white paper provides a translational framework, from the selection of animal models and associated end-points to decision-driving biomarkers as well as considerations for the design of clinical proof-of-concept studies. It also identifies current gaps in our biomarker toolkit and the status of the discovery and validation of α-syn-associated biomarkers that could help fill these gaps. Further, it highlights the importance of the emerging digital technology to supplement the capture and monitoring of clinical outcomes. Although the development of disease-modifying therapies targeting α-syn face profound challenges, we remain optimistic that meaningful strides will be made soon toward the identification and approval of disease-modifying therapeutics targeting α-syn.

Indexed as

BiomarkersClinical Trials as TopicDisease Models, AnimalGuidelines as TopicProof of Concept StudyTranslational Research, Biomedicalalpha-SynucleinAnimalsConsensusHumansParkinson DiseaseResearch Designalpha-SynucleinBiomarkersAlpha-synucleinanimal modelsbiomarkersdigital endpointsexperimental therapiesimmunotherapiesneuroimaging

Identifiers

PMID30400107
PMCPMC6398545

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.