Molecular information has been an important information in cancer treatment. For example, anti-Her2 therapy has been the standard treatment for Her2-positive breast cancer patients. However, genetic alternation occur in different frequencies in different cancer types. It is difficult to enroll enough patients with low incidence of targetable molecular alterations in clinical trials. Basket trial overcomes these obstacles by recruiting multiple types of cancer patients with the same genetic alternation. In a phase IIa (MyPathway) study, 230 patients with different advanced solid tumors harboring Her2, BRAF, EGFR or Hedgehog pathway alterations were treated with four targeted treatment, pertuzumab plus trastuzumab, erlotinib, vemurafenib, or vismodegib, respectivel. Results showed 52 (23%) patients of 14 different tumor types had objective responses. The disease control rate was 33.9% (78/230). The results demonstrated the efficacy of therapeutic strategy that treated patients across multiple tumor types by matching targeted therapy.

Source:Hainsworth JD, Meric-Bernstam F, Swanton C, et al. J Clin Oncol. 2018, 20;36(6):536-542.