Evidence map›Paper›PMID 40563807›Full record

ArticleBrain sciences2025

Integrated Workflow for Drug Repurposing in Glioblastoma: Computational Prediction and Preclinical Validation of Therapeutic Candidates.

Nazareno Gonzalez, Melanie Pérez Küper, Matías Garcia Fallit, Jorge A Peña Agudelo, Alejandro Nicola Candia, Maicol Suarez Velandia, Ana Clara Romero, Guillermo Videla Richardson, Marianela Candolfi

Abstract read
In one paragraph

Article in Brain sciences, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Review
  2. Article
  3. Article
  4. 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

9 authors.

Nazareno GonzalezInstituto de Investigaciones Biomédicas (INBIOMED, CONICET-UBA), Facultad de Medicina, Universidad de Buenos Aires, Buenos Aires C1121ABG, Argentina.ORCID 0000-0003-4013-3396
Melanie Pérez KüperInstituto de Investigaciones Biomédicas (INBIOMED, CONICET-UBA), Facultad de Medicina, Universidad de Buenos Aires, Buenos Aires C1121ABG, Argentina.ORCID 0009-0007-8155-9613
Matías Garcia FallitInstituto de Investigaciones Biomédicas (INBIOMED, CONICET-UBA), Facultad de Medicina, Universidad de Buenos Aires, Buenos Aires C1121ABG, Argentina.ORCID 0009-0005-1915-1604
Jorge A Peña AgudeloInstituto de Investigaciones Biomédicas (INBIOMED, CONICET-UBA), Facultad de Medicina, Universidad de Buenos Aires, Buenos Aires C1121ABG, Argentina.ORCID 0000-0003-3470-7824
Alejandro Nicola CandiaInstituto de Investigaciones Biomédicas (INBIOMED, CONICET-UBA), Facultad de Medicina, Universidad de Buenos Aires, Buenos Aires C1121ABG, Argentina.ORCID 0000-0002-0194-560X
Maicol Suarez VelandiaInstituto de Investigaciones Biomédicas (INBIOMED, CONICET-UBA), Facultad de Medicina, Universidad de Buenos Aires, Buenos Aires C1121ABG, Argentina.ORCID 0009-0009-4519-1166
Ana Clara RomeroInstituto de Investigaciones Biomédicas (INBIOMED, CONICET-UBA), Facultad de Medicina, Universidad de Buenos Aires, Buenos Aires C1121ABG, Argentina.
Guillermo Videla RichardsonFundación Para la Lucha Contra las Enfermedades Neurológicas de la Infancia (FLENI), Buenos Aires C1121A6B, Argentina.
Marianela CandolfiInstituto de Investigaciones Biomédicas (INBIOMED, CONICET-UBA), Facultad de Medicina, Universidad de Buenos Aires, Buenos Aires C1121ABG, Argentina.ORCID 0000-0002-0843-6568

Funding

Agencia Nacional de Promoción Científica y Tecnológica Fellowship to A.J.N.C, M.P.K. and N.GAgencia Nacional de Promoción Científica y Tecnológica PICT-2018-3088, PICT-2019-00117 and PICT-2022-11-00074 to M.CConsejo Interuniversitario Nacional Fellowship to M.G.F.Consejo Nacional de Investigaciones Científicas y Técnicas Fellowships to A.J.N.C, J.A.P.A. and M.G.FFundación Florencio Fiorini 2023 Cancer Research Award to M.CFundación Florencio Fiorini 2023 Research grant to N.G.Instituto Nacional del Cáncer Asistencia Financiera a Proyectos de Investigación en Cáncer IV to M.C.
6 · The paper itself

Abstract

backgroundGlioblastoma (GBM) remains a significant challenge in oncology due to its resistance to standard treatments including temozolomide. This study aimed to develop and validate an integrated model for predicting GBM sensitivity to alternative chemotherapeutics and identifying new drugs and combinations with therapeutic potential. RESEARCH DESIGN AND

methodsWe analyzed drug sensitivity data for 272 compounds from CancerRxTissue and employed in silico algorithms to assess blood-brain barrier permeability. The model was used to predict GBM sensitivity to various drugs, which was then validated using GBM cellular models. Alternative drugs targeting overexpressed and negative prognostic biomarkers in GBM were experimentally validated.

resultsThe model predicted that GBM is more sensitive to Etoposide and Cisplatin compared to Temozolomide, which was confirmed by experimental validation in GBM cells. We also identified novel drugs with high predicted sensitivity in GBM. Daporinad, a NAMPT inhibitor that permeates the blood-brain barrier was selected for further preclinical evaluation. This evaluation supported the in silico predictions of high potential efficacy and safety in GBM.

conclusionsOur findings using different cellular models suggest that this computational prediction model could constitute a valuable tool for drug repurposing in GBM and potentially in other tumors, which could accelerate the development of more effective cancer treatments.

Indexed as

blood-brain barriercombination therapyDaporinaddrug repurposingglioblastomaNAMPT inhibitorpersonalized medicinepredictive modeling

Identifiers

PMID40563807
PMCPMC12191016

What OpenQuestion holds

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LicenceCC BY
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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.