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Tumour-agnostic drugs in paediatric cancers

Julia C Chisholm1, Fernando Carceller2, Lynley V Marshall2

  • 1Paediatric 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.

British Journal of Cancer
|March 13, 2020
PubMed

Insights

Tumour-agnostic cancer drugs offer new hope for children. Larotrectinib, a targeted therapy, demonstrates the potential of this approach for paediatric cancers, addressing access challenges.

Area of Science:

  • Oncology
  • Paediatric Oncology
  • Pharmacology

Background:

  • Novel targeted cancer therapies are increasingly developed for adult indications.
  • Access to these targeted therapies can be challenging for paediatric cancer patients.
  • Tumour-agnostic drug development, based on mechanism-of-action, offers a promising alternative.

Purpose of the Study:

  • To highlight the importance of tumour-agnostic drug development for paediatric cancers.
  • To examine larotrectinib as a case study for a tumour-agnostic therapy in children.

Main Methods:

  • Review of the development and approval of larotrectinib.
  • Analysis of the implications of tumour-agnostic therapy for paediatric oncology.

Main Results:

  • Larotrectinib represents a successful example of a tumour-agnostic drug.
  • This approach can overcome limitations in accessing novel therapies for paediatric cancers.

Conclusions:

  • Tumour-agnostic drug development is crucial for advancing paediatric cancer treatment.
  • Larotrectinib's approval signifies a step forward in providing targeted therapies for children with specific genetic alterations.

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