Homologous recombination deficiency and hemizygosity drive resistance in breast cancer.

Nature 2026
Open on PubMed

The co-occurrence of germline and somatic oncogenic alterations is frequently observed in breast cancer, yet their combined influence on tumour evolution and therapy resistance remains poorly defined. Through an integrated clinicogenomic analysis of more than 5,800 patients, we show that germline (g) pathogenic variants dictate the evolutionary trajectory of acquired resistance. We specifically find that gBRCA2-associated tumours are uniquely predisposed to develop acquired RB1 loss-of-function alterations, resulting in poor outcomes on standard-of-care frontline CDK4/6 inhibitor (CDK4/6i) combinations. This vulnerability is driven by a dual mechanism: baseline RB1 hemizygosity (heterozygous loss resulting in a single functional RB1 allele), which lowers the evolutionary barrier to biallelic inactivation, and ongoing homologous recombination deficiency, which promotes acquisition of RB1 loss-of-function alterations under the selective pressure of CDK4/6i. Preclinical models from gBRCA2 carriers showed near-uniform resistance to CDK4/6i, with consistent post-treatment Rb loss. Across multiple independent models and in our clinical data, PARP inhibition consistently outperformed CDK4/6i. Our findings suggest that prioritizing PARP inhibition in gBRCA2 carriers may intercept RB1-loss trajectories and delay resistance. More broadly, we establish a predictive framework for forecasting drug-resistant trajectories based on pre-treatment allelic configuration and mutational signatures.

14 Figures Extracted
Extended Data Fig. 1
Extended Data Fig. 1 PMC
CONSORT diagram and zygosity status. a , CONSORT diagram outlining inclusion criteria for germline–somatic analysis and CDK4/6i outcome analysis compa...
Fig. 1
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Germline–somatic interactions in breast cancer. a , Study schema. Enrichment analysis was performed to identify somatic alterations more prevalent in ...
Extended Data Fig. 2
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Germline–somatic interactions, stratified by zygosity status. a-e , Enrichment analysis of germline PVs compared to gWT. Analyses for g BRCA1 ( a ), ...
Extended Data Fig. 3
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Oncoprints of enriched germline–somatic interactions. Oncoprints showing somatic alteration types across patients (columns) and genes (rows). Somatic ...
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Clinical implications of g BRCA2 status. a , PFS for patients treated with first-line CDK4/6i + ET by g BRCA2 status. Patients with g BRCA2 ( n  = ...
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Impact of g BRCA2 status on therapeutic response across systemic modalities. a , Forest plot depicting the PFS by g BRCA2 status on the outcome of o...
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Dual effect of RB1 LOH and HRD mutagenesis on CDK4/6i resistance. a , RB1 and BRCA2 LOH in g BRCA2 ( n  = 63; top) versus gWT ( n  = 527; botto...
Extended Data Fig. 5
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Validation of concurrent RB1 and BRCA2 LOH in external g BRCA2 WES cohort. FACETS was performed on whole-exome sequencing of g BRCA2 breast canc...
Extended Data Fig. 6
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Progression-free and overall survival in PALOMA-3. PFS and OS results from the experimental arm (fulvestrant plus palbociclib) and the placebo arm of ...
Extended Data Fig. 7
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Association between preceding treatment and RB1 loss-of-function variant. Sequenced tumor samples from patients with HR+/HER2– MBC were categorized ...
Extended Data Fig. 8
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CONSORT diagram for case selection for first-line CDK4/6i + ET outcomes stratified by allele-specific copy number of RB1. ASCN analysis was performed...
Extended Data Fig. 9
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Implications of HRD signature on CDK4/6i PFS in g BRCA2 WT breast cancers. HRD inference was performed with an orthogonal method (HRD-IMPACT, see Me...
Fig. 4
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Mechanistic validation of Rb loss as a driver of CDK4/6i resistance in g BRCA2 tumours. a , PDXs derived from two metastatic deposits in a rapid auto...
Extended Data Fig. 10
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Multi-site genomic profiling from rapid autopsy of a g BRCA2 carrier. a , Oncoprint summarizing targeted sequencing (MSK-IMPACT) of tumors collected ...