- Chen, Anran;
- Kim, Beom-Jun;
- Mitra, Aparna;
- Vollert, Craig;
- Lei, Jonathan;
- Fandino, Diana;
- Anurag, Meenakshi;
- Holt, Matthew;
- Gou, Xuxu;
- Pilcher, Jacob;
- Goetz, Matthew;
- Northfelt, Donald;
- Hilsenbeck, Susan;
- Marshall, C;
- Hyer, Marc;
- Papp, Robert;
- Yin, Shou-Yun;
- De Angelis, Carmine;
- Schiff, Rachel;
- Fuqua, Suzanne;
- Ma, Cynthia;
- Foulds, Charles;
- Ellis, Matthew
Endocrine therapies (ET) with cyclin-dependent kinase 4/6 (CDK4/6) inhibition are the standard treatment for estrogen receptor-α-positive (ER+) breast cancer, however drug resistance is common. In this study, proteogenomic analyses of patient-derived xenografts (PDXs) from patients with 22 ER+ breast cancer demonstrated that protein kinase, membrane-associated tyrosine/threonine one (PKMYT1), a WEE1 homolog, is estradiol (E2) regulated in E2-dependent PDXs and constitutively expressed when growth is E2-independent. In clinical samples, high PKMYT1 mRNA levels associated with resistance to both ET and CDK4/6 inhibition. The PKMYT1 inhibitor lunresertib (RP-6306) with gemcitabine selectively and synergistically reduced the viability of ET and palbociclib-resistant ER+ breast cancer cells without functional p53. In vitro the combination increased DNA damage and apoptosis. In palbociclib-resistant, TP53 mutant PDX-derived organoids and PDXs, RP-6306 with low-dose gemcitabine induced greater tumor volume reduction compared to treatment with either single agent. Our study demonstrates the clinical potential of RP-6306 in combination with gemcitabine for ET and CDK4/6 inhibitor resistant TP53 mutant ER+ breast cancer.