Evidence map›Paper›PMID 34718207›Full record

ReviewDrug discovery today2022

Knowledge-based approaches to drug discovery for rare diseases.

Vinicius M Alves, Daniel Korn, Vera Pervitsky, Andrew Thieme, Stephen J Capuzzi, Nancy Baker, Rada Chirkova, Sean Ekins, Eugene N Muratov, Anthony Hickey and 1 more

Abstract readReview
In one paragraph

Review in Drug discovery today, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.

0numbers the graph read from it
0cells of the map it votes in
19citing 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

19 citing papers in PubMed.

  1. Review
  2. Article
  3. Review
  4. Article
  5. Applications of artificial intelligence in drug discovery for neurological diseases.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2025
    Review
  6. Review
  7. Review
  8. Article
  9. Article
  10. Article
  11. Review
  12. Article
  13. Review
  14. Article
  15. Review
  16. Review
  17. Review
  18. Article
  19. 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

11 authors.

Vinicius M AlvesLaboratory for Molecular Modeling, Division of Chemical Biology and Medicinal Chemistry, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, USA; UNC Catalyst for Rare Diseases, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, USA.
Daniel KornLaboratory for Molecular Modeling, Division of Chemical Biology and Medicinal Chemistry, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, USA.
Vera PervitskyLaboratory for Molecular Modeling, Division of Chemical Biology and Medicinal Chemistry, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, USA.
Andrew ThiemeLaboratory for Molecular Modeling, Division of Chemical Biology and Medicinal Chemistry, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, USA.
Stephen J CapuzziLaboratory for Molecular Modeling, Division of Chemical Biology and Medicinal Chemistry, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, USA.
Nancy BakerParlezChem, 123 W Union Street, Hillsborough, NC 27278, USA.
Rada ChirkovaDepartment of Computer Science, North Carolina State University, Raleigh, NC 27695-8206, USA.
Sean EkinsCollaborations Pharmaceuticals Inc., 840 Main Campus Drive, Lab 3510, Raleigh, NC 27606, USA.
Eugene N MuratovLaboratory for Molecular Modeling, Division of Chemical Biology and Medicinal Chemistry, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, USA; Department of Pharmaceutical Sciences, Federal University of Paraiba, Joao Pessoa, PB, Brazil.
Anthony HickeyUNC Catalyst for Rare Diseases, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, USA. Electronic address: ahickey@unc.edu.
Alexander TropshaLaboratory for Molecular Modeling, Division of Chemical Biology and Medicinal Chemistry, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, USA. Electronic address: alex_tropsha@unc.edu.

Funding

ARAGORN: Autonomous Relay Agent for Generation Of Ranked NetworksOT2TR003441 · NCATS · UNIV OF NORTH CAROLINA CHAPEL HILL · PI TROPSHA, ALEXANDER · 2020 to 2024
$4.7M
Centralized assay datasets for modelling support of small drug discovery organizationsR44GM122196 · NIGMS · COLLABORATIONS PHARMACEUTICALS, INC. · PI EKINS, SEAN · 2018 to 2022
$3.3M
Drug Repurposing for Cancer Therapy: From Man to Molecules to ManU01CA207160 · NCI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI TROPSHA, ALEXANDER · 2016 to 2018
$1.2M
Enzyme Replacement Therapy For Infantile Onset Neuronal Ceroid LipofuscinosesR43NS107079 · NINDS · COLLABORATIONS PHARMACEUTICALS, INC. · PI COOPER, JONATHAN D, EKINS, SEAN · 2018 to 2019
$412k
Centralized assay datasets for modelling support of small drug discovery organizationsR43GM122196 · NIGMS · COLLABORATIONS PHARMACEUTICALS, INC. · PI EKINS, SEAN · 2017 to 2017
$150k
NCATS NIH HHS OT2 TR003441NCI NIH HHS U01 CA207160NIGMS NIH HHS R43 GM122196NIGMS NIH HHS R44 GM122196NINDS NIH HHS R43 NS107079
6 · The paper itself

Abstract

The conventional drug discovery pipeline has proven to be unsustainable for rare diseases. Herein, we discuss recent advances in biomedical knowledge mining applied to discovering therapeutics for rare diseases. We summarize current chemogenomics data of relevance to rare diseases and provide a perspective on the effectiveness of machine learning (ML) and biomedical knowledge graph mining in rare disease drug discovery. We illustrate the power of these methodologies using a chordoma case study. We expect that a broader application of knowledge graph mining and artificial intelligence (AI) approaches will expedite the discovery of viable drug candidates against both rare and common diseases.

Indexed as

Artificial IntelligenceRare DiseasesDrug DiscoveryHumansKnowledge BasesMachine LearningData miningDrug discoveryInformaticsKnowledge graphsRare diseases

Identifiers

PMID34718207
PMCPMC9124594

What Socratic 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 Socratic graph.