Tumour-agnostic drugs in paediatric cancers.


Journal

British journal of cancer
ISSN: 1532-1827
Titre abrégé: Br J Cancer
Pays: England
ID NLM: 0370635

Informations de publication

Date de publication:
05 2020
Historique:
received: 23 12 2019
accepted: 13 02 2020
revised: 03 02 2020
pubmed: 13 3 2020
medline: 23 12 2020
entrez: 13 3 2020
Statut: ppublish

Résumé

The recognition that new cancer drugs can be truly tumour-agnostic based on mechanism-of-action is important for paediatric cancers, where access to novel targeted therapies developed for adult indications has sometimes been problematic. The recently approved drug larotrectinib is an excellent case study of the development of a tumour-agnostic drug relevant to children.

Identifiants

pubmed: 32161367
doi: 10.1038/s41416-020-0770-5
pii: 10.1038/s41416-020-0770-5
pmc: PMC7217925
doi:

Substances chimiques

Antineoplastic Agents 0
Oncogene Proteins, Fusion 0
Pharmaceutical Preparations 0
Protein Kinase Inhibitors 0
Pyrazoles 0
Pyrimidines 0
larotrectinib PF9462I9HX

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1425-1427

Commentaires et corrections

Type : CommentIn
Type : CommentIn

Références

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doi: 10.1136/bmj.l6435
Hierro, C., Matos, I., Martin-Liberal, J., Ochoa de Olza, M. & Garralda, E. Agnostic-histology approval of new drugs in oncology: are we already there? Clin. Cancer Res. 25, 3210–3219 (2019).
doi: 10.1158/1078-0432.CCR-18-3694
Drilon, A. TRK inhibitors in TRK fusion-positive cancers. Ann. Oncol. 30(Supplement 8), viii23–viii30 (2019).
doi: 10.1093/annonc/mdz282
Albert, C. M., Davis, J. L., Federman, N., Casanova, M. & Laetsch, T. W. TRK fusion cancers in children: a clinical review and recommendations for screening. J. Clin. Oncol. 37, 513–524 (2019).
doi: 10.1200/JCO.18.00573
Pearson, A. D. J., Pfister, S. M., Baruchel, A., Bourquin, J. P., Casanova, M., Chesler, L. et al. From class waivers to precision medicine in paediatric oncology. Lancet Oncol. 18, e394–e404 (2017).
doi: 10.1016/S1470-2045(17)30442-4
Hong, T. D., Bauer, T. M., Lee, J. J., Dowlati, A., Brose, M. S., Farago, A. F. et al. Larotrectinib in adult patients with solid tumours:a multi-centre, open-label, phase I dose-escalation study. Ann. Oncol. 30, 325–331 (2019).
doi: 10.1093/annonc/mdy539
Laetsch, T. W., DuBois, S. G., Mascarenhas, L., Turpin, B., Federman, N., Albert, C. M. et al. Larotrectinib for paediatric solid tumours harbouring NTRK gene fusions: phase 1 results from a multicentre, open-label, phase 1/2 study. Lancet Oncol. 19, 705–714 (2018).
doi: 10.1016/S1470-2045(18)30119-0
George, S. L., Izquierdo, E., Campbell, J., Koutroumanidou, E., Proszek, P. & Jamal, S. A tailored molecular profiling programme for children with cancer to identify clinically actionable genetic alterations. Eur. J. Cancer 121, 224–235 (2019).
doi: 10.1016/j.ejca.2019.07.027
Mossé, Y. P., Lim, M. S., Voss, S. D., Wilner, K., Ruffner, K., Laliberte, J. et al. Safety and activity of crizotinib for paediatric patients with refractory solid tumours or anaplastic large-cell lymphoma: a Children’s Oncology Group phase 1 consortium study. Lancet Oncol. 14, 472–480 (2013).
doi: 10.1016/S1470-2045(13)70095-0
Vassal, G., Rousseau, R., Blanc, P., Moreno, L., Bode, G., Schwoch, S. et al. Creating a unique, multi-stakeholder paediatric oncology platform to improve drug development for children and adolescents with cancer. Eur. J. Cancer 51, 218–224 (2015).
doi: 10.1016/j.ejca.2014.10.029
Gaspar, N., Marshall, L. V., Binner, D., Herold, R., Rousseau, R., Blanc, P. et al. Joint adolescent-adult early phase clinical trials to improve access to new drugs for adolescents with cancer: proposals from the multi-stakeholder platform-ACCELERATE. Ann. Oncol. 29, 766–771 (2018).
doi: 10.1093/annonc/mdy002

Auteurs

Julia C Chisholm (JC)

Paediatric and Adolescent Oncology Drug Development Team, Children and Young People's Unit, Royal Marsden Hospital and Institute of Cancer Research, Sutton, UK. julia.chisholm@rmh.nhs.uk.

Fernando Carceller (F)

Paediatric and Adolescent Oncology Drug Development Team, Children and Young People's Unit, Royal Marsden Hospital and Institute of Cancer Research, Sutton, UK.

Lynley V Marshall (LV)

Paediatric and Adolescent Oncology Drug Development Team, Children and Young People's Unit, Royal Marsden Hospital and Institute of Cancer Research, Sutton, UK.

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