Evidence map›Paper›PMID 39316411›Full record

ReviewAlzheimer's & dementia : the journal of the Alzheimer's Association2024

A framework for translating tauopathy therapeutics: Drug discovery to clinical trials.

Howard H Feldman, Jeffrey L Cummings, Adam L Boxer, Adam M Staffaroni, David S Knopman, Stacey J Sukoff Rizzo, Paul R Territo, Steven E Arnold, Clive Ballard, Dirk Beher and 23 more

Abstract readReview
In one paragraph

Review in Alzheimer's & dementia : the journal of the Alzheimer's Association, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed
–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

13 citing papers in PubMed.

  1. Review
  2. The Progress of Active Immunotherapy for Parkinson's Disease.International journal of molecular sciences · 2026
    Review
  3. Tau in Alzheimer's disease: Shaping the future patient journey.The journal of prevention of Alzheimer's disease · 2026
    Review
  4. Review
  5. Review
  6. Twenty years of therapeutic development in tauopathy mouse models: a scoping review.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2025
    Article
  7. Review
  8. The Clinical Trials Landscape for Alzheimer's Disease.CNS neuroscience & therapeutics · 2025
    Review
  9. Differences in the soluble and insoluble proteome between primary tauopathies.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2025
    Article
  10. Review
  11. Review
  12. Article
  13. A framework for translating tauopathy therapeutics: Drug discovery to clinical trials.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2024
    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

33 authors.

Howard H FeldmanDepartment of Neurosciences, University of California San Diego, La Jolla, California, USA.ORCID 0000-0002-9258-4538
Jeffrey L CummingsChambers-Grundy Center for Transformative Neuroscience, Department of Brain Health, School of Integrated Health Sciences, University of Nevada at Las Vegas, Las Vegas, Nevada, USA.
Adam L BoxerDepartment of Neurology, Memory and Aging Center, University of California San Francisco, San Francisco, California, USA.
Adam M StaffaroniDepartment of Neurology, Memory and Aging Center, University of California San Francisco, San Francisco, California, USA.
David S KnopmanDepartment of Neurology, Mayo Clinic, Rochester, Minnesota, USA.
Stacey J Sukoff RizzoAging Institute, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, USA.
Paul R TerritoDepartment of Medicine, Division of Clinical Pharmacology, Indiana University School of Medicine, Indianapolis, Indiana, USA.
Steven E ArnoldDepartment of Neurology, Harvard Medical School, Massachusetts General Hospital, Charlestown, Massachusetts, USA.
Clive BallardCollege of Medicine and Health, University of Exeter, Exeter, UK.
Dirk BeherAsceneuron, Lausanne, Switzerland.
Bradley F BoeveDepartment of Neurology, Mayo Clinic, Rochester, Minnesota, USA.
Penny A DacksThe Association for Frontotemporal Degeneration, King of Prussia, Pennsylvania, USA.
Kristophe DiazCurePSP, New York, New York, USA.
Colin EwenUCB Pharma, Slough, UK.
Brian FiskeThe Michael J. Fox Foundation for Parkinson's Research, New York, New York, USA.
M Isabel GonzalezDrug Discovery and Development Consultants, Cambridge, UK.
Glenn A HarrisRainwater Charitable Foundation, Fort Worth, Texas, USA.
Beth J HoffmanOrigami Therapeutics, San Diego, California, USA.
Terina N MartinezCritical Path Institute, Tucson, Arizona, USA.
Eric McDadeDepartment of Neurology, Washington University School of Medicine, St. Louis, Missouri, USA.
Laura K NisenbaumAlzheimer's Drug Discovery Foundation, New York, New York, USA.
Jose-Alberto PalmaNovartis Institutes for Biomedical Research, Cambridge, Massachusetts, USA.
Melanie QuintanaBerry Consultants, Austin, Texas, USA.
Gil D RabinoviciDepartment of Neurology, Memory and Aging Center, University of California San Francisco, San Francisco, California, USA.
Jonathan D RohrerDepartment of Neurodegenerative Disease, Dementia Research Centre, Queen Square Institute of Neurology, University College of London, London, UK.
Howard J RosenDepartment of Neurology, Memory and Aging Center, University of California San Francisco, San Francisco, California, USA.
Matthew D TroyerDenali Therapeutics, South San Francisco, California, USA.
Doo Yeon KimDepartment of Neurology, Genetics and Aging Research Unit, McCance Center for Brain Health, Mass General Institute for Neurodegenerative Disease, Massachusetts General Hospital, Charlestown, Massachusetts, USA.
Rudolph E TanziDepartment of Neurology, Genetics and Aging Research Unit, McCance Center for Brain Health, Mass General Institute for Neurodegenerative Disease, Massachusetts General Hospital, Charlestown, Massachusetts, USA.
Henrik ZetterbergDepartment of Psychiatry and Neurochemistry, Sahlgrenska Academy at the University of Gothenburg, Mölndal, Sweden.
Nick K ZiogasBiogen, Cambridge, Massachusetts, USA.
Patrick C MayADvantage Neuroscience Consulting LLC, Fort Wayne, Indiana, USA.
Amy RommelRainwater Charitable Foundation, Fort Worth, Texas, USA.

Funding

Imaging CoreU19AG032438 · NIA · WASHINGTON UNIVERSITY · PI BATEMAN, RANDALL J · 2010 to 2025
$53.9M
Renewal of Centers of Biomedical Research Excellence (COBRE) (Phase 2) CNTN - ResubmissionP20GM109025 · NIGMS · CLEVELAND CLINIC FOUNDATION · PI JESSICA KIRKLAND CALDWELL · 2015 to 2026
$22.8M
Alzheimer's Clinical Trial InnOvatioN (ACTION) InitiativeR35AG071476 · NIA · UNIVERSITY OF NEVADA LAS VEGAS · PI CUMMINGS, JEFFREY L. · 2021 to 2025
$2.9M
Alzheimer's Disease and Related Dementias Innovation Incubator (InnovaTor)R25AG083721 · NIA · UNIVERSITY OF NEVADA LAS VEGAS · PI JEFFREY L. CUMMINGS, Xue K Zhong · 2023 to 2026
$1.0M
NIA NIH HHS R35 AG071476NIA NIH HHS U19 AG032438NIGMS NIH HHS P20 GM109025Rainwater Charitable Foundation
6 · The paper itself

Abstract

The tauopathies are defined by pathological tau protein aggregates within a spectrum of clinically heterogeneous neurodegenerative diseases. The primary tauopathies meet the definition of rare diseases in the United States. There is no approved treatment for primary tauopathies. In this context, designing the most efficient development programs to translate promising targets and treatments from preclinical studies to early-phase clinical trials is vital. In September 2022, the Rainwater Charitable Foundation convened an international expert workshop focused on the translation of tauopathy therapeutics through early-phase trials. Our report on the workshop recommends a framework for principled drug development and a companion lexicon to facilitate communication focusing on reproducibility and achieving common elements. Topics include the selection of targets, drugs, biomarkers, participants, and study designs. The maturation of pharmacodynamic biomarkers to demonstrate target engagement and surrogate disease biomarkers is a crucial unmet need. HIGHLIGHTS: Experts provided a framework to translate therapeutics (discovery to clinical trials). Experts focused on the "5 Rights" (target, drug, biomarker, participants, trial). Current research on frontotemporal degeneration, progressive supranuclear palsy, and corticobasal syndrome therapeutics includes 32 trials (37% on biologics) Tau therapeutics are being tested in Alzheimer's disease; primary tauopathies have a large unmet need.

Indexed as

BiomarkersClinical Trials as TopicDrug DiscoveryTauopathiesDrug DevelopmentHumanstau ProteinsTranslational Research, BiomedicalBiomarkerstau Proteinsbiomarkersdevelopmentearly‐phase clinical trialspreclinicaltauopathytherapeutics

Identifiers

PMID39316411
PMCPMC11567863

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.