Sarcomas are a diverse group of rare soft tissue and bone cancers, and amongst these, are the even rarer ultrarare sarcomas (URSs, defined as sarcomas with an incidence of ≤1 per 1,000,000).
There are 77 recognised types of ultrarare sarcomas and they account for 20% of sarcoma diagnoses. Given their individual scarcity, almost all URSs lack approved histology-driven treatments, and their management remains an area of unmet medical need.
Recently, the potential role for genomic testing to help guide therapy selection in URS was highlighted in an exciting publication from the phase 1 unit of the MD Anderson Cancer Centre. In the Sarcoma-Matched Biomarker Analysis (SAMBA-102) study, 94 patients with URSs underwent genomic sequencing with 33 (31%) subsequently treated on molecularly selected trials. Patients receiving matched treatments had higher response rates (24% vs 8.2%, p=0.024) and an improvement in overall survival (median OS 27.3 vs 13.4 months, p=0.29) demonstrating the importance of genomic sequencing to identify subsets more likely to benefit from molecular-targeted therapies.
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