Stereospecific lasofoxifene derivatives reveal the interplay between estrogen receptor alpha stability and antagonistic activity in ESR1 mutant breast cancer cells.
Hosfield, D.J., Weber, S., Li, N.S., Suavage, M., Joiner, C.F., Hancock, G.R., Sullivan, E.A., Ndukwe, E., Han, R., Cush, S., Laine, M., Mader, S.C., Greene, G.L., Fanning, S.W.(2022) Elife 11
- PubMed: 35575456 
- DOI: https://doi.org/10.7554/eLife.72512
- Primary Citation of Related Structures:  
6PSJ, 6V8T, 6VPF, 7KBS, 7UJ7, 7UJ8, 7UJC, 7UJF, 7UJM, 7UJO, 7UJW, 7UJY - PubMed Abstract: 
Chemical manipulation of estrogen receptor alpha ligand binding domain structural mobility tunes receptor lifetime and influences breast cancer therapeutic activities. Selective estrogen receptor modulators (SERMs) extend estrogen receptor alpha (ER¦Á) cellular lifetime/accumulation. They are antagonists in the breast but agonists in the uterine epithelium and/or in bone. Selective estrogen receptor degraders/downregulators (SERDs) reduce ER¦Á cellular lifetime/accumulation and are pure antagonists. Activating somatic ESR1 mutations Y537S and D538G enable resistance to first-line endocrine therapies. SERDs have shown significant activities in ESR1 mutant setting while few SERMs have been studied. To understand whether chemical manipulation of ER¦Á cellular lifetime and accumulation influences antagonistic activity, we studied a series of methylpyrollidine lasofoxifene (Laso) derivatives that maintained the drug's antagonistic activities while uniquely tuning ER¦Á cellular accumulation. These molecules were examined alongside a panel of antiestrogens in live cell assays of ER¦Á cellular accumulation, lifetime, SUMOylation, and transcriptional antagonism. High-resolution x-ray crystal structures of WT and Y537S ER¦Á ligand binding domain in complex with the methylated Laso derivatives or representative SERMs and SERDs show that molecules that favor a highly buried helix 12 antagonist conformation achieve the greatest transcriptional suppression activities in breast cancer cells harboring WT/Y537S ESR1 . Together these results show that chemical reduction of ER¦Á cellular lifetime is not necessarily the most crucial parameter for transcriptional antagonism in ESR1 mutated breast cancer cells. Importantly, our studies show how small chemical differences within a scaffold series can provide compounds with similar antagonistic activities, but with greatly different effects of the cellular lifetime of the ER¦Á, which is crucial for achieving desired SERM or SERD profiles.
Organizational Affiliation: 
Ben May Department for Cancer Research, University of Chicago, Chicago, United States.