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Updated: Jun 23, 2025

Assessing Cellular Target Engagement by SHP2 PTPN11 Phosphatase Inhibitors
Published on: July 17, 2020
Targeting SHP2 Cryptic Allosteric Sites for Effective Cancer Therapy
Ashfaq Ur Rehman1, Cizhang Zhao1, Yongxian Wu1
1Departments of Molecular Biology and Biochemistry, Chemical and Biomolecular Engineering, Materials Science and Engineering, and Biomedical Engineering, University of California, Irvine, CA 92697, USA.
Abstract:
SHP2, a pivotal component downstream of both receptor and non-receptor tyrosine kinases, has been underscored in the progression of various human cancers and neurodevelopmental disorders. Allosteric inhibitors have been proposed to regulate its autoinhibition. However, oncogenic mutations, such as E76K, convert SHP2 into its open state, wherein the catalytic cleft becomes fully exposed to its ligands. This study elucidates the dynamic properties of SHP2 structures across different states, with a focus on the effects of oncogenic mutation on two known binding sites of allosteric inhibitors. Through extensive modeling and simulations, we further identified an alternative allosteric binding pocket in solution structures. Additional analysis provides insights into the dynamics and stability of the potential site. In addition, multi-tier screening was deployed to identify potential binders targeting the potential site. Our efforts to identify a new allosteric site contribute to community-wide initiatives developing therapies using multiple allosteric inhibitors to target distinct pockets on SHP2, in the hope of potentially inhibiting or slowing tumor growth associated with SHP2.
Insights
This study reveals how cancer-driving mutations alter SHP2 protein structure. Researchers identified a new binding site for allosteric inhibitors to potentially slow tumor growth.
Area of Science:
- Biochemistry
- Molecular Biology
- Cancer Research
Background:
- SHP2 is crucial in signaling pathways implicated in cancer and neurodevelopmental disorders.
- Allosteric inhibitors aim to control SHP2 activity by targeting its regulatory sites.
- Oncogenic mutations, like E76K, lock SHP2 in an active conformation, exposing its catalytic cleft.
Purpose of the Study:
- To investigate the dynamic structural changes of SHP2 in different states, particularly in response to oncogenic mutations.
- To analyze the impact of mutations on known allosteric inhibitor binding sites.
- To discover novel allosteric binding pockets on SHP2.
Main Methods:
- Extensive molecular modeling and simulations were employed to study SHP2 dynamics.
- Analysis focused on the effects of oncogenic mutations on known allosteric inhibitor binding sites.
- Multi-tier screening was utilized to identify potential drug candidates for newly identified binding sites.
Main Results:
- The study elucidated the dynamic properties of SHP2 across various conformational states.
- Oncogenic mutations were shown to affect the accessibility and dynamics of known allosteric binding sites.
- A novel allosteric binding pocket in SHP2 solution structures was identified and characterized.
- Potential binders for this new site were identified through multi-tier screening.
Conclusions:
- Understanding SHP2's dynamic behavior and mutation effects is key for therapeutic development.
- The discovery of a new allosteric site offers opportunities for developing novel inhibitors.
- Targeting distinct SHP2 allosteric sites with multiple inhibitors could provide a strategy to combat SHP2-driven cancers.
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