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Record W4378715818 · doi:10.1038/s41467-023-38668-2

From a drug repositioning to a structure-based drug design approach to tackle acute lymphoblastic leukemia

2023· article· en· W4378715818 on OpenAlexaff
Magalí Sáez‐Ayala, Laurent Hoffer, Sébastien Abel, Khaoula Ben Yaala, Benoit Sicard, Guillaume P. Andrieu, Mehdi Latiri, Emma K. Davison, Marco A. Ciufolini, Paul Brémond, E. Rebuffet, Philippe Roche, Carine Derviaux, Edwige Voisset, Camille Montersino, Rémy Castellano, Yves Collette, Vahid Asnafi, S. Betzi, Patrice Dubreuil, Sébastien Combes, Xavier Morelli

Bibliographic record

VenueNature Communications · 2023
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicBiochemical and Molecular Research
Canadian institutionsSimon Fraser UniversityOkanagan University CollegeUniversity of British Columbia, Okanagan CampusUniversity of British ColumbiaOntario Institute for Cancer Research
FundersSociété d'Accélération du Transfert de TechnologiesFrench Infrastructure for Integrated Structural BiologyH2020 Marie Skłodowska-Curie ActionsLigue Contre le CancerFondation de FranceFondation ARC pour la Recherche sur le CancerEuropean Synchrotron Radiation FacilityAgence Nationale de la RechercheNational Cancer InstituteInstitut National Du CancerUniversity of Auckland
KeywordsNucleotide salvageDrug discoveryDrugCancer researchLymphoblastic LeukemiaComputational biologyDrug developmentLeukemiaChemistryPharmacologyBiologyBiochemistryNucleotideGeneticsGene

Abstract

fetched live from OpenAlex

Cancer cells utilize the main de novo pathway and the alternative salvage pathway for deoxyribonucleotide biosynthesis to achieve adequate nucleotide pools. Deoxycytidine kinase is the rate-limiting enzyme of the salvage pathway and it has recently emerged as a target for anti-proliferative therapies for cancers where it is essential. Here, we present the development of a potent inhibitor applying an iterative multidisciplinary approach, which relies on computational design coupled with experimental evaluations. This strategy allows an acceleration of the hit-to-lead process by gradually implementing key chemical modifications to increase affinity and activity. Our lead compound, OR0642, is more than 1000 times more potent than its initial parent compound, masitinib, previously identified from a drug repositioning approach. OR0642 in combination with a physiological inhibitor of the de novo pathway doubled the survival rate in a human T-cell acute lymphoblastic leukemia patient-derived xenograft mouse model, demonstrating the proof-of-concept of this drug design strategy.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Bench or experimental · Consensus signal: Bench or experimental
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.023
Threshold uncertainty score0.724

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0000.001
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0010.001
Research integrity0.0000.000
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.017
GPT teacher head0.299
Teacher spread0.282 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designBench or experimental
Domainnot available
GenreEmpirical

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations13
Published2023
Admission routes1
Has abstractyes

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