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Wendt, J. R.

Publications and source records attributed to Wendt, J. R..

2 recordsLinked to original sources

Long-read, whole-genome sequencing and chemotherapy response of two patient-derived organoids from a TP53- and KRAS-mutant ovarian carcinoma

Patient-derived organoids (PDOs) have transformed translational cancer research, allowing tractable models that better represent clinical features than traditional immortalized cell lines. Here we describe two PDOs with differential responses to carboplatin derived from sequential ascites fluid collections from a patient with high-grade mullerian carcinoma, that could not be further subclassified on the omental biopsy. Uterine origin was clinically excluded by pelvic imaging/CT scan of the uterus and absence of vaginal bleeding. Successful derivation from independent collections enabled comparison of intra-patient heterogeneity across sequential ascites samples and demonstrates that PDO efficiency rate is at least partly patient-specific or tumor-dependent. We performed long-read whole genome sequencing on the two PDOs, OC104 and OC109, to better characterize the structural variant landscape while also obtaining information on single nucleotide variants and DNA methylation. In addition to confirming single nucleotide variants noted in clinical sequencing (TP53, KRAS, SPOP, PPP2R1A, KMT2D), we identified additional variants in TSC2, NCOR2, and CTNNA2 that are predicted to be likely pathogenic. The spectrum of mutations, particularly the coincident KRAS and TP53, highlighted unexpected overlap with ovarian mucinous carcinoma. We also identified larger insertions and deletions that result in non-synonymous variants in MUC5AC, TPRX1, and BMX, as well as four translocation events, including two that could not have been resolved with short-read sequencing. Differentially methylated promoters between the two PDOs include 201 oncogenes and tumor suppressor genes, with HNF1A, MSI2, and SETBP1 having methylation directions consistent with these genes roles in platinum response differences observed between the PDOs. Notably, the clonal nature of PDOs produced from two samples taken one week apart is important for the field to appreciate, particularly since they have clonal differences in platinum response. The temporal differences in clonality may indicate a limitation of low volume sampling, however may provide opportunity to longitudinally predict clinical outcomes. We also demonstrate the ability of long-read sequencing to add detail into the genomics and epigenetics of ovarian cancer.

cancer biology↗

Landscape of super-enhancers in small cell carcinoma of the ovary, hypercalcemic type and efficacy of targeting with natural product triptolide

PurposeSmall cell carcinoma of the ovary-hypercalcemic type (SCCOHT) is a rare form of ovarian cancer affecting young women and girls. SCCOHT is driven by loss of both SWI/SNF ATPases SMARCA4 and SMARCA2, having major effects on enhancer landscapes. Super-enhancers are a distinct subset of enhancer clusters frequently associated with oncogenes in cancer. Experimental DesignSCCOHT cell lines and PDX models were interrogated for super-enhancer landscape with H3K27ac CUT&RUN integrated with RNAseq data for associated oncogene analysis. IHC staining and drug efficacy studies in PDX models demonstrate clinical translatability. ResultsHere we discovered key distinctions between SWI/SNF chromatin occupancy following SMARCA4 restoration at enhancer vs. super-enhancer sites and characterized putative oncogene expression driven by super-enhancer activity. SCCOHT super-enhancer target genes were particularly enriched in developmental processes, most notably nervous system development. We found high sensitivity of SCCOHT cell lines to triptolide, a small molecule that targets the XPB subunit of the transcription factor II H (TFIIH) complex, found at super-enhancers. Triptolide inhibits expression of many super-enhancer associated genes, including oncogenes. Notably, SALL4 expression is significantly decreased following short triptolide treatment, and its RNA expression was high in SCCOHT tumors relative to other ovarian cancers. In SCCOHT patient-derived xenograft models, triptolide and its prodrug derivative minnelide are particularly effective in inhibiting tumor growth. ConclusionsThese results demonstrate the key oncogenic role of super-enhancer activity following epigenetic dysfunction in SCCOHT, which can be effectively targeted through inhibition of its functional components, such as TFIIH inhibition with triptolide. Statement of Translational RelevanceThis work identifies a potential therapeutic strategy for small cell carcinoma of the ovary-hypercalcemic type (SCCOHT), a rare and aggressive ovarian cancer affecting young women and children. This study highlights the role of the loss of SWI/SNF ATPase SMARCA4 in altering super-enhancers to promote high oncogene expression. We discovered that SCCOHT cells exhibited high sensitivity to triptolide, a small molecule derived from Tripterygium wilfordii, which targets the XPB subunit of the transcription factor II H (TFIIH) complex found at super-enhancers. Triptolide inhibits the expression of super-enhancer-associated genes, including oncogenes like SALL4, which is highly expressed in SCCOHT. Moreover, in SCCOHT patient-derived xenograft models, triptolide and its derivative minnelide effectively inhibited tumor growth. These findings suggest that targeting super-enhancer activity could be a promising therapeutic approach for SCCOHT, offering potential clinical benefits to patients who currently face limited treatment options and poor outcomes.

cancer biology↗