% IMPORTANT: The following is UTF-8 encoded. This means that in the presence % of non-ASCII characters, it will not work with BibTeX 0.99 or older. % Instead, you should use an up-to-date BibTeX implementation like “bibtex8” or % “biber”. @ARTICLE{Zech:301719, author = {H. B. Zech and P. Schafhausen and L. Ramke and J.-L. Velthaus and S. Kreutzfeldt$^*$ and D. Hübschmann$^*$ and K. Rothkamm and C. Bokemeyer and A. S. Hoffmann and S. Fröhling$^*$ and H. Glimm$^*$ and C. S. Betz and M. Kriegs and M. Christopeit}, title = {{L}ast {R}esort? {R}ationale for {C}omprehensive {M}olecular {A}nalysis in {T}reatment-{R}efractory {R}/{M} {HNSCC}: {A} {C}ase {R}eport of {R}emarkable {R}esponse to {S}acituzumab {G}ovitecan {F}ollowing {M}olecular and {F}unctional {C}haracterization.}, journal = {Biomedicines}, volume = {13}, number = {5}, issn = {2227-9059}, address = {Basel}, publisher = {MDPI}, reportid = {DKFZ-2025-01111}, pages = {1266}, year = {2025}, abstract = {Background/Objectives: In recurrent/metastatic head and neck squamous cell carcinoma (R/M HNSCC), the overall prognosis is poor, and systemic treatment options remain limited. While precision therapy approaches have revolutionized treatment strategies in several tumor types, molecularly informed therapies in R/M HNSCC are rare, primarily due to the low number of actionable genetic alterations identified through next-generation sequencing (NGS) panels. There is an urgent need to establish precision therapy approaches in R/M HNSCC using innovative predictive testing. Methods: We report the case of a 43-year-old patient with recurrent oral cancer who was extensively pretreated and comprehensively characterized using both descriptive and functional testing. Results: NGS revealed no targetable alterations. A tumor tissue slice radiosensitivity assay suggested radioresistance, arguing against re-irradiation. Kinome profiling identified upregulated Src-family kinases (SFK), and SFK inhibition reduced kinase activity in vitro. Most notably, mRNA analysis demonstrated high Trop-2 overexpression, confirmed by immunohistochemistry (3+ in $100\%$ of tumor cells). Following six cycles of the Trop-2-directed antibody-drug conjugate Sacituzumab govitecan (SG), the patient had an impressive clinical response. Conclusions: Tumor characterization beyond genetic profiling can identify novel treatment options in therapy-refractory HNSCC. This is the first report of 'real-world' data on promising antitumor efficacy of SG in a heavily pretreated oral cancer patient with Trop-2 overexpression. Consistent with the findings of the Basket TROPiCS-03 study, SG appears to be a promising novel therapy option for R/M HNSCC after failure of immunotherapy and chemotherapy, particularly in patients with Trop-2 overexpression.}, keywords = {Sacituzumab govitecan (Other) / Trop-2 (Other) / kinome (Other) / precision medicine (Other) / recurrent HNSCC (Other) / tumor models (Other) / tumor tissue slices (Other)}, cin = {B340 / HD01 / DD01}, ddc = {570}, cid = {I:(DE-He78)B340-20160331 / I:(DE-He78)HD01-20160331 / I:(DE-He78)DD01-20160331}, pnm = {312 - Funktionelle und strukturelle Genomforschung (POF4-312)}, pid = {G:(DE-HGF)POF4-312}, typ = {PUB:(DE-HGF)16}, pubmed = {pmid:40427091}, pmc = {pmc:PMC12108840}, doi = {10.3390/biomedicines13051266}, url = {https://inrepo02.dkfz.de/record/301719}, }