Plasma Metabolomic Changes following PI3K Inhibition as Pharmacodynamic Biomarkers: Preclinical Discovery to Phase I Trial Evaluation

PI3K plays a key role in cellular metabolism and cancer. Using a mass spectrometry-based metabolomics platform, we discovered that plasma concentrations of 26 metabolites, including amino acids, acylcarnitines, and phosphatidylcholines, were decreased in mice bearing PTEN-deficient tumors compared w...

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Main Authors: Ang, Joo Ern, Pandher, Rupinder, Ang, Joo Chew, Asad, Yasmin J, Henley, Alan T, Valenti, Melanie, Box, Gary, de, Haven Brandon Alexis, Baird, Richard, Friedman, Lori, Derynck, Mika, Vanhaesebroeck, Bart, Eccles, Suzanne A, Kaye, Stan B, Workman, Paul, de, Bono Johann S, Raynaud, Florence I
Other Authors: Antwerp X-ray Analysis, Electrochemistry and Speciation (AXES)
Language:English
Published: American Association for Cancer Research 2019
Online Access:https://demo7.dspace.org/handle/123456789/449
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Summary:PI3K plays a key role in cellular metabolism and cancer. Using a mass spectrometry-based metabolomics platform, we discovered that plasma concentrations of 26 metabolites, including amino acids, acylcarnitines, and phosphatidylcholines, were decreased in mice bearing PTEN-deficient tumors compared with non-tumor-bearing controls and in addition were increased following dosing with class I PI3K inhibitor pictilisib (GDC-0941). These candidate metabolomics biomarkers were evaluated in a phase I dose-escalation clinical trial of pictilisib. Time- and dose-dependent effects were observed in patients for 22 plasma metabolites. The changes exceeded baseline variability, resolved after drug washout, and were recapitulated on continuous dosing. Our study provides a link between modulation of the PI3K pathway and changes in the plasma metabolome and demonstrates that plasma metabolomics is a feasible and promising strategy for biomarker evaluation. Also, our findings provide additional support for an association between insulin resistance, branched-chain amino acids, and related metabolites following PI3K inhibition.