Chronic myelomonocytic leukemia (CMML) is a rare clonal myeloid neoplasm. Preclinical studies have shown CMML to be sensitive to GM-GSF signaling and JAK inhibition. To this end, Hunter and colleagues conducted a phase I and II clinical trial testing ruxolitinib, a JAK1/2 inhibitor that downregulates GM-CSF signaling, in patients with CMML. Ruxolitinib was well tolerated in this patient population, and an overall response rate of 38% was observed. A parallel study of patient-derived xenografts from patients enrolled on the clinical trial corroborated the in-human findings. These results support further clinical study of ruxolitinib for the treatment of CMML.

In tumors with BRCA1/2 mutations, homologous repair deficiency causes inability to repair DNA double-strand breaks. PARP inhibitors (PARPi), such as olaparib, have proven to be effective in BRCA-mutated ovarian, breast, pancreatic and prostate cancer. van der Wijngaart and colleagues conducted a clinical trial of olaparib in patients with tumors harboring BRCA1/2...

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