Activating ESR1 mutations in hormone-resistant metastatic breast cancer

DR Robinson, YM Wu, P Vats, F Su, RJ Lonigro… - Nature …, 2013 - nature.com
DR Robinson, YM Wu, P Vats, F Su, RJ Lonigro, X Cao, S Kalyana-Sundaram, R Wang…
Nature genetics, 2013nature.com
Breast cancer is the most prevalent cancer in women, and over two-thirds of cases express
estrogen receptor-α (ER-α, encoded by ESR1). Through a prospective clinical sequencing
program for advanced cancers, we enrolled 11 patients with ER-positive metastatic breast
cancer. Whole-exome and transcriptome analysis showed that six cases harbored mutations
of ESR1 affecting its ligand-binding domain (LBD), all of whom had been treated with anti-
estrogens and estrogen deprivation therapies. A survey of The Cancer Genome Atlas …
Abstract
Breast cancer is the most prevalent cancer in women, and over two-thirds of cases express estrogen receptor-α (ER-α, encoded by ESR1). Through a prospective clinical sequencing program for advanced cancers, we enrolled 11 patients with ER-positive metastatic breast cancer. Whole-exome and transcriptome analysis showed that six cases harbored mutations of ESR1 affecting its ligand-binding domain (LBD), all of whom had been treated with anti-estrogens and estrogen deprivation therapies. A survey of The Cancer Genome Atlas (TCGA) identified four endometrial cancers with similar mutations of ESR1. The five new LBD-localized ESR1 mutations identified here (encoding p.Leu536Gln, p.Tyr537Ser, p.Tyr537Cys, p.Tyr537Asn and p.Asp538Gly) were shown to result in constitutive activity and continued responsiveness to anti-estrogen therapies in vitro. Taken together, these studies suggest that activating mutations in ESR1 are a key mechanism in acquired endocrine resistance in breast cancer therapy.
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