Identification of Novel Therapeutic Targets for Fibrolamellar Carcinoma Using Patient-Derived Xenografts and Direct-from-Patient Screening

利用患者来源的异种移植和直接从患者身上进行的筛选来识别纤维板层癌的新型治疗靶点

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作者:Gadi Lalazar, David Requena, Lavoisier Ramos-Espiritu, Denise Ng, Patrick D Bhola, Ype P de Jong, Ruisi Wang, Nicole J C Narayan, Bassem Shebl, Solomon Levin, Eleftherios Michailidis, Mohammad Kabbani, Koen O A Vercauteren, Arlene M Hurley, Benjamin A Farber, William J Hammond, James A Saltsman 3rd,

Significance

Therapeutics informed by genomics have not yielded effective therapies for FLC. A functional screen identified TOPO1, HDAC inhibitors, and napabucasin as efficacious and synergistic with inhibition of Bcl-xL. Validation on cells dissociated directly from patient tumors demonstrates the ability for functional precision medicine in a solid tumor.This article is highlighted in the In This Issue feature, p. 2355.

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