PLK1 Inhibitor Response Prediction via TCTP Phosphorylation in AML

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Solution Overview

Problem

There is a need for a method to enrich for the subset of subjects that have a greater likelihood of responding to PLK1 inhibitors, as existing methods do not effectively predict the efficacy of PLK1 inhibitors in cancer treatment.

Innovation Solution

Determine the phosphorylation of serine 46 of translational control tumor protein (TCTP) in cancer samples before and after exposure to the PLK1 inhibitor onvansertib, and select patients for treatment if phosphorylation is decreased, specifically for acute myeloid leukemia (AML).

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If PLK1 inhibitors are administered to cancer patients, then antitumor activity is achieved, but the ability to predict treatment efficacy is insufficient

Engineering Contradiction:
Improveprediction accuracy of treatment efficacyVSAvoidlack of predictive biomarker information
Core Design Contradiction:
ReliabilityVSLoss of information

Solution Approach 1:

The patent applies preliminary action by measuring TCTP phosphorylation status before administering PLK1 inhibitors to predict treatment response. This pre-treatment biomarker assessment allows clinicians to identify patients likely to respond to therapy, enabling informed treatment decisions before the actual therapeutic intervention occurs.

Inventive Principle:
Principle #10Preliminary action

Solution Approach 2:

The patent implements feedback by measuring changes in TCTP phosphorylation levels after PLK1 inhibitor administration and using this information to predict treatment efficacy. The phosphorylation status serves as a feedback signal that indicates whether the drug is effectively inhibiting its target, allowing for real-time assessment of treatment response.

Inventive Principle:
Principle #23Feedback

2Reliability

If TCTP phosphorylation is measured before and after onvansertib exposure, then treatment response can be predicted, but additional testing time and complexity are required

Engineering Contradiction:
Improveprediction accuracy of treatment responseVSAvoidtime required for phosphorylation assessment
Core Design Contradiction:
ReliabilityVSLoss of time

Solution Approach 1:

The patent measures TCTP phosphorylation status as a preliminary step before initiating full treatment, allowing for rapid identification of responsive patients. This upfront biomarker assessment prevents time loss by identifying non-responders early, avoiding prolonged ineffective treatment and subsequent treatment switching.

Inventive Principle:
Principle #10Preliminary action

Applied Scientific Principles

This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.

Function Achieved in This Case

This method allows for the early identification of patients likely to respond to onvansertib treatment by measuring the inhibition of TCTP phosphorylation, predicting treatment efficacy and enabling targeted therapy.

Implementation Method 1

Onvansertib (also known as PCM-075, NMS-1286937, NMS-937, 'compound of formula (1)' in U.S. Patent 8,927,530; IUPAC name 1-(2-hydroxyethyl)-8-{[5-(4-methylpiperazin-1-yl)-2-(trifluoromethoxy) phenyl] amino}- 4,5-dihydro-1H-pyrazolo[4,3-h] quinazoline-3-carboxamide) is the first PLK1 specific ATP competitive inhibitor administered by oral route to enter clinical trials with proven antitumor activity in different preclinical models

Methodology Applied
Scientific EffectATP competitive inhibition:

Implementation Method 2

determining phosphorylation of serine 46 of translational control tumor protein (TCTP) in a cancer sample obtained from a patient

Methodology Applied
Scientific EffectPhosphorylation:

Data Source

PatentEP3840741B1PLK1 target phosphorylation status and treatment of cancer with PLK1 inhibitors
Publication Date: 2025.07.16 CARDIFF ONCOLOGY INC
  • EP3840741B1 patent drawingFigure 1A~1B
  • EP3840741B1 patent drawingFigure 2A~2B
  • EP3840741B1 patent drawingFigure 2C

AI summary

Provided is a method comprising determining polo-like kinase 1 (PLK1) activity in a cancer in a patient by measuring phosphorylation of a PLK1 target (a) prior to treatment of (i) the patient or (ii) a cancer sample from the patient with a PLK1 inhibitor, and (b) after the treatment. Also provided is a method comprising determining polo-like kinase 1 (PLK1) activity in a cancer in a patient by measuring phosphorylation of a PLK1 target without treatment with a PLK1 inhibitor.