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Targeted therapy for intractable cancer on the basis of molecular profiles: An open-label, phase II basket trial

Xiao-Dong Jiao1, Bao-Dong Qin1, Zhan Wang1

  • 1Department of Medical Oncology, Changzheng Hospital, Naval Medical University, Shanghai, China.

Frontiers in Oncology
|March 13, 2023
PubMed
Abstract

Insights

Molecular-guided targeted therapy shows clinical benefit for intractable cancers in Chinese patients. The study identified the frequency of tier II gene alterations, supporting this approach in basket trials.

Area of Science:

  • Oncology
  • Genetics
  • Pharmacology

Background:

  • Intractable cancers pose significant treatment challenges.
  • Molecular profiling aids in identifying actionable genetic alterations.
  • Targeted therapies offer personalized treatment strategies.

Purpose of the Study:

  • To evaluate the efficacy of molecular-guided targeted therapy for intractable cancers in the Chinese population.
  • To investigate the molecular epidemiology of druggable gene alterations in Chinese cancer patients.

Main Methods:

  • The Long March Pathway trial (NCT03239015) was a phase II basket study.
  • Molecular profiles of 520 intractable cancer patients were analyzed.
  • 27 patients with tier II gene alterations received targeted therapy.

Main Results:

  • Targeted therapy yielded an overall response rate (ORR) of 29.6% and a disease control rate (DCR) of 44.4%.
  • Median progression-free survival (PFS) was 4.67 months.
  • Tier II gene alterations were found in 17.7% of 17,841 Chinese pan-cancer patients, with higher frequencies in bladder, breast, and non-small cell lung cancer (NSCLC).

Conclusions:

  • Molecular-guided targeted therapy demonstrated clinical benefit in Chinese patients with intractable cancer.
  • The identified frequency of tier II gene alterations supports the use of molecular-guided targeted therapy within basket trial designs.
Keywords:
basket trialgene alterationintractable cancerprecision medicinetargeted therapy

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