Terminating Oncogenic Signaling: Rational Design of a Molecular Inhibitor distorting and inactivating GTP bound to Oncogenic Mutated RAS Proteins.

09 December 2024, Version 1
This content is an early or alternative research output and has not been peer-reviewed by Cambridge University Press at the time of posting.

Abstract

Oncogenic mutations in RAS proteins, particularly within their GTP-binding domains, drive persistent activation of downstream signaling pathways, leading to uncontrolled cellular proliferation and various cancers, including pancreatic, colorectal, lung, and melanoma. These mutations impair GTP hydrolysis by making RAS proteins insensitive to GAP (GTPase-activating protein), maintaining their active state. This study introduces the design of a novel drug molecule targeting GTP-bound mutated RAS proteins. Key features include tert-butyl phenylsulfate as a core fragment due to its strong affinity for GTP, enhanced by electron-donating groups (EDGs) such as -NH2 and -OCH3 to improve binding efficiency. A benzyl-based protective group ensures selective reactivity, stability, and targeted activation exclusively in the presence of mutated RAS. Computational tools, including RDKit and SwissDock, were employed to assemble and optimize the drug molecule, yielding a structure that selectively interacts with GTP-bound RAS mutants, such as KRASG12C and NRASQ61K, defunctionalizing both the GTP and the mutant proteins. The proposed drug molecule shows significant affinity in molecular docking studies, with preliminary optimization ensuring stability and functionality. Future work involves in vitro assays, molecular dynamics simulations, ADMET profiling, and pharmacophore modeling to validate its efficacy and safety. This targeted approach holds promise for treating Ras-driven cancers with minimal off-target effects, marking a significant advancement in precision oncology.

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