Exploiting RAS Nucleotide Cycling as a Strategy for Drugging RAS-Driven Cancers
Related Experiment Video
Updated: Jan 1, 2026

Identification of EGFR and RAS Inhibitors using Caenorhabditis elegans
Published on: October 5, 2020
Tyler E Mattox1, Xi Chen1,2, Yulia Y Maxuitenko1
1Drug Discovery Research Center, University of South Alabama Mitchell Cancer Institute, Mobile, AL 36604, USA.
Abstract:
Oncogenic mutations in RAS genes result in the elevation of cellular active RAS protein levels and increased signal propagation through downstream pathways that drive tumor cell proliferation and survival. These gain-of-function mutations drive over 30% of all human cancers, presenting promising therapeutic potential for RAS inhibitors. However, many have deemed RAS "undruggable" after nearly 40 years of failed drug discovery campaigns aimed at identifying a RAS inhibitor with clinical activity. Here we review RAS nucleotide cycling and the opportunities that RAS biochemistry presents for developing novel RAS inhibitory compounds. Additionally, compounds that have been identified to inhibit RAS by exploiting various aspects of RAS biology and biochemistry will be covered. Our current understanding of the biochemical properties of RAS, along with reports of direct-binding inhibitors, both provide insight on viable strategies for the discovery of novel clinical candidates with RAS inhibitory activity.
Insights
RAS mutations drive over 30% of cancers, but targeting RAS proteins has been challenging. This review explores RAS biochemistry and novel strategies for developing effective RAS inhibitors for cancer therapy.
Area of Science:
- Oncology
- Molecular Biology
- Drug Discovery
Background:
- Gain-of-function mutations in RAS genes elevate active RAS protein levels, driving cancer cell proliferation and survival.
- Over 30% of human cancers are driven by these mutations, highlighting RAS as a key therapeutic target.
- Despite decades of research, developing clinically active RAS inhibitors has proven difficult, leading to the "undruggable" designation.
Purpose of the Study:
- To review RAS nucleotide cycling and its biochemical properties.
- To identify opportunities for developing novel RAS inhibitory compounds.
- To cover existing compounds that inhibit RAS by exploiting its biology and biochemistry.
Main Methods:
- Review of existing scientific literature on RAS biochemistry and nucleotide cycling.
- Analysis of identified RAS-inhibiting compounds and their mechanisms of action.
- Synthesis of current understanding of RAS biochemical properties and direct-binding inhibitors.
Main Results:
- RAS biochemistry and nucleotide cycling present viable targets for drug development.
- Various compounds have been identified that inhibit RAS through different biochemical mechanisms.
- Current understanding provides insight into strategies for discovering novel clinical candidates.
Conclusions:
- RAS remains a critical target in oncology due to its role in numerous cancers.
- Exploiting RAS biochemistry offers promising avenues for developing effective RAS inhibitors.
- Continued research into RAS biology and inhibitor development is crucial for advancing cancer therapy.
Related Concept Videos
The Ras Gene
Ras is a...
Small GTPases - Ras and Rho
Three regulatory proteins control their activity:
Treatment Resistant Cancers
Targeted Cancer Therapies
There are several types of targeted therapies against...
Abnormal Proliferation
Inhibition of Cdk Activity

