CFTR Modulators Attenuate Thrombocyte Activation

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Solution Overview

Problem

Current treatments for thrombosis, atherosclerosis, and related conditions associated with pathological thrombocyte activation are inadequate, leading to high morbidity and mortality rates, especially in cases like COVID-19.

Innovation Solution

The use of cystic fibrosis transmembrane conductance regulator (CFTR) modulators, such as ivacaftor, to treat and prevent pathological thrombocyte activation, aggregation, and adhesion, thereby addressing thrombosis and atherosclerosis.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If conventional treatments are used for thrombosis and atherosclerosis, then standard therapy is provided, but treatment effectiveness is inadequate leading to high morbidity and mortality

Engineering Contradiction:

Improvetreatment effectivenessVSAvoidmorbidity and mortality rates

Core Design Contradiction:

ReliabilityVSProductivity

Solution Approach 1:

The patent applies parameter changes by introducing a novel therapeutic agent (CFTR modulator) that targets a different physiological parameter (CFTR channel function in thrombocytes) compared to conventional treatments. This changes the mechanism of action from standard anticoagulant pathways to CFTR-mediated thrombocyte regulation, thereby improving treatment effectiveness and reducing morbidity/mortality associated with thrombotic events

Inventive Principle:

Principle #35Parameter changes

2Adaptability or versatility

If CFTR modulators are used to treat thrombosis, then thrombocyte activation is attenuated, but this is a novel application beyond cystic fibrosis treatment

Engineering Contradiction:

Improvedrug indication rangeVSAvoidtreatment proven efficacy

Core Design Contradiction:

Adaptability or versatilityVSReliability

Solution Approach 1:

The patent applies universality by demonstrating that CFTR modulators, originally developed for cystic fibrosis, have a second therapeutic indication for thrombotic diseases. The CFTR channel exists in multiple cell types including thrombocytes, and modulating this channel provides dual benefits: treating CF in eligible patients and preventing thrombotic events through attenuation of thrombocyte activation, adhesion, and aggregation

Inventive Principle:

Principle #6Universality (Multi-functionality)

Data Source

PatentUS20250177371A1CFTR modulators for the treatment of vascular disease

Publication Date: 2025.06.05 CHARITE UNIVERSITATMEDIZIN BERLIN

AI summary

Pharmaceutical compositions and the treatment and/or prevention of medical conditions are provided. The medical conditions including thrombosis, or atherosclerosis associated with pathological thrombocyte activation. A cystic fibrosis transmembrane conductance regulator (CFTR) modulator for use in the treatment and/or prevention of a medical condition including thrombosis, or a medical condition including atherosclerosis and pathological thrombocyte activation, in a human subject. The medical use and corresponding therapeutic methods of administering a CFTR modulator, such as ivacaftor, in the treatment and/or prevention of a medical condition pathological thrombocyte activation, adhesion and/or aggregation. A pharmaceutical composition is also provided including a CFTR modulator, such as ivacaftor.