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Small molecule stabilization of the KSR inactive state antagonizes oncogenic Ras signalling

Journal Article · · Nature (London)
DOI:https://doi.org/10.1038/nature19327· OSTI ID:1368230
 [1];  [1];  [1]
  1. The Icahn School of Medicine at Mount Sinai, New York, NY (United States)

Deregulation of the Ras–mitogen activated protein kinase (MAPK) pathway is an early event in many different cancers and a key driver of resistance to targeted therapies. Sustained signalling through this pathway is caused most often by mutations in K-Ras, which biochemically favours the stabilization of active RAF signalling complexes. Kinase suppressor of Ras (KSR) is a MAPK scaffold that is subject to allosteric regulation through dimerization with RAF. Direct targeting of KSR could have important therapeutic implications for cancer; however, testing this hypothesis has been difficult owing to a lack of small-molecule antagonists of KSR function. Guided by KSR mutations that selectively suppress oncogenic, but not wild-type, Ras signalling, we developed a class of compounds that stabilize a previously unrecognized inactive state of KSR. These compounds, exemplified by APS-2-79, modulate KSR-dependent MAPK signalling by antagonizing RAF heterodimerization as well as the conformational changes required for phosphorylation and activation of KSR-bound MEK (mitogen-activated protein kinase kinase). Furthermore, APS-2-79 increased the potency of several MEK inhibitors specifically within Ras-mutant cell lines by antagonizing release of negative feedback signalling, demonstrating the potential of targeting KSR to improve the efficacy of current MAPK inhibitors. In conclusion, these results reveal conformational switching in KSR as a druggable regulator of oncogenic Ras, and further suggest co-targeting of enzymatic and scaffolding activities within Ras–MAPK signalling complexes as a therapeutic strategy for overcoming Ras-driven cancers.

Research Organization:
Argonne National Laboratory (ANL), Argonne, IL (United States). Advanced Photon Source (APS)
Sponsoring Organization:
National Institutes of Health (NIH); Damon Runyon-Rachleff Foundation
OSTI ID:
1368230
Journal Information:
Nature (London), Journal Name: Nature (London) Journal Issue: 7618 Vol. 537; ISSN 0028-0836
Publisher:
Nature Publishing GroupCopyright Statement
Country of Publication:
United States
Language:
ENGLISH

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Cited By (13)

Substrate binding allosterically relieves autoinhibition of the TRIB1 pseudokinase posted_content May 2018
The evolving world of pseudoenzymes: proteins, prejudice and zombies journal November 2016
Homozygous KSR1 deletion attenuates morbidity but does not prevent tumor development in a mouse model of RAS-driven pancreatic cancer journal March 2018
MEK/ERK addiction in CNL/aCML journal November 2017
Stabilization of Sur8 via PKCα/δ degradation promotes transformation and migration of colorectal cancer cells journal December 2017
RAS: Striking at the Core of the Oncogenic Circuitry journal September 2019
Prospects for pharmacological targeting of pseudokinases journal March 2019
LncRNA NEAT1/let-7a-5p axis regulates the cisplatin resistance in nasopharyngeal carcinoma by targeting Rsf-1 and modulating the Ras-MAPK pathway journal February 2018
The PEAK family of pseudokinases, their role in cell signalling and cancer journal November 2019
Pseudokinases: a tribble‐edged sword journal October 2019
Evolutionarily conserved regulation of sleep by epidermal growth factor receptor signaling journal November 2019
Substrate binding allosterically relieves autoinhibition of the pseudokinase TRIB1 journal September 2018
Tracing the origin and evolution of pseudokinases across the tree of life journal April 2019

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