Molecular analysis for refractory rare cancers: Sequencing battle continues - learnings for the MOSCATO-01 study

Background: For patients with metastatic rare cancers, treatments are limited. How systematic tumor sequencing can improve therapeutic possibilities in this population?Patients and methods: Patients with rare cancer were identified in the MOSCATO-01 trial. Patients' outcome was measured by progression-free survival (PFS) and overall survival (OS).Results: The most frequently identified histologic subypes were ovarian adenocarcinoma (N = 13), carcinoma of unknown primary (N = 11), and leiomyosarcoma (N = 10). Ninety-nine (39%) of them had at least one targetable cancer molecular alteration Forty-nine patients (50%) received the therapy proposed by the molecular tumor board, and 13 patients (26%, 95%CI 15-41%) achieved a PFS2/PFS1 > 1.3. The median PFS2 on matched treatment subgroup was 2.3 months (95% CI 1.8-3.6) and the median OS was 11.4 months (95% CI 9-15.5).Conclusions: The molecular screening of patients with refractory, metastatic rare cancers might increase the therapeutic options. Facilitating access strategy to molecular-driven clinical trials or agnostic-approved treat-ment is crucial.

Automated summary

Systematic tumor sequencing can potentially expand therapeutic options for patients with metastatic rare cancers, as shown in the MOSCATO-01 trial. Molecular screening identified targetable molecular alterations in a significant proportion of patients, leading to improved progression-free survival and overall survival.