Exploiting the PI3K/AKT pathway for cancer drug discovery
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Updated: Aug 8, 2026

Utilizing Functional Genomics Screening to Identify Potentially Novel Drug Targets in Cancer Cell Spheroid Cultures
Published on: December 26, 2016
Bryan T Hennessy1, Debra L Smith, Prahlad T Ram
1Department of Molecular Therapeutics, The University of Texas M.D. Anderson Cancer Center, 1515 Holcombe Blvd, Houston, TX 77030, USA.
Abstract:
Evolving studies with several different targeted therapeutic agents are demonstrating that patients with genomic alterations of the target, including amplification, translocation and mutation, are more likely to respond to the therapy. Recent studies indicate that numerous components of the phosphatidylinositol-3-kinase (PI3K)/AKT pathway are targeted by amplification, mutation and translocation more frequently than any other pathway in cancer patients, with resultant activation of the pathway. This warrants exploiting the PI3K/AKT pathway for cancer drug discovery.
Insights
Targeted therapies are more effective in cancer patients with specific genomic alterations. The phosphatidylinositol-3-kinase (PI3K)/AKT pathway is frequently altered, making it a promising target for new cancer drug discovery.
Area of Science:
- Oncology
- Molecular Biology
- Genomics
Background:
- Targeted therapeutic agents show increased efficacy in patients with specific genomic alterations.
- Genomic alterations include amplification, translocation, and mutation of therapeutic targets.
- These alterations predict a higher likelihood of patient response to targeted therapies.
Purpose of the Study:
- To highlight the significance of genomic alterations in predicting therapeutic response.
- To identify frequently altered molecular pathways in cancer.
- To propose the phosphatidylinositol-3-kinase (PI3K)/AKT pathway as a key target for cancer drug discovery.
Main Methods:
- Review of evolving studies on targeted therapeutic agents.
- Analysis of recent studies on molecular pathway alterations in cancer patients.
- Identification of frequently mutated, amplified, or translocated pathways.
Main Results:
- Genomic alterations in therapeutic targets correlate with improved treatment response.
- The phosphatidylinositol-3-kinase (PI3K)/AKT pathway exhibits frequent alterations (amplification, mutation, translocation) compared to other pathways.
- These alterations lead to the activation of the PI3K/AKT pathway.
Conclusions:
- The PI3K/AKT pathway's frequent alterations present a significant opportunity for cancer therapy.
- Exploiting the PI3K/AKT pathway is a rational strategy for developing novel cancer drugs.
- Targeted therapies informed by genomic profiling offer improved patient outcomes.
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