Self-Reactive Arm Prodrugs for Enzymatic Release

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Solution Overview

Problem

Current prodrugs for anticancer agents lack specificity and stability, leading to non-selective destruction of both tumor and healthy tissues, resulting in severe side effects and limited therapeutic efficacy due to inefficient targeting and rapid metabolism.

Innovation Solution

A new self-reactive arm derived from phenol, capable of click chemistry reactions, is introduced to create prodrugs that can be specifically targeted to tumor tissues by enzymatic hydrolysis, ensuring rapid and controlled release of active compounds, enhancing bioavailability and reducing toxicity.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If conventional prodrugs are used to deliver anticancer agents, then the active compound can be transported to target tissues, but the prodrugs lack specificity and stability leading to non-selective destruction of healthy tissues and severe side effects

Engineering Contradiction:
Improvespecificity of prodrug deliveryVSAvoidtoxicity to healthy tissues
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The prodrug is segmented into distinct functional modules: a targeting moiety (folate) that directs the prodrug to tumor cells, a self-reactive arm (o-nitrobenzyl carbamate) that provides stability in circulation, and a labile group that enables enzyme-triggered release. This segmentation allows each component to perform its specific function optimally, achieving both stable transport and selective release.

Inventive Principle:
Principle #1Segmentation

Solution Approach 2:

The prodrug design implements local quality by concentrating the active anticancer agent at the tumor site through targeted delivery, while maintaining low toxicity in healthy tissues. The folate targeting moiety ensures accumulation at the tumor location, and the self-reactive arm maintains stability during circulation, creating a localized high concentration only where needed.

Inventive Principle:
Principle #3Local quality

2Productivity

If prodrugs are designed for targeted delivery, then bioavailability may be improved, but the prodrugs require rapid metabolism and release which compromises stability during administration

Engineering Contradiction:
Improverelease rate of active compoundVSAvoidstability of prodrug during administration
Core Design Contradiction:
ProductivityVSStability of the object's composition

Solution Approach 1:

The prodrug exhibits dynamic properties by being stable under physiological conditions during circulation, then rapidly transforming upon enzymatic trigger. The self-reactive arm maintains the prodrug in a stable inert form during administration, and upon encountering the specific enzyme at the target site, it dynamically converts to release the active compound, achieving both stability and rapid release.

Inventive Principle:
Principle #15Dynamics

Solution Approach 2:

The prodrug design utilizes parameter changes by maintaining stability at physiological pH and conditions during circulation, then undergoing rapid hydrolysis when triggered by specific enzymes at the tumor site. The self-reactive arm's chemical structure is designed to be stable under normal conditions but labile under enzymatic catalysis, allowing controlled transition from stable transport to rapid release.

Inventive Principle:
Principle #35Parameter changes

3Reliability

If conventional prodrugs are used, then active compounds can be administered, but they exhibit little selectivity towards tumor cells resulting in non-selective destruction

Engineering Contradiction:
Improveselectivity towards tumor cellsVSAvoidside effects from non-selective destruction
Core Design Contradiction:
ReliabilityVSObject-generated harmful factors

Solution Approach 1:

The prodrug design incorporates preliminary action by pre-attaching the folate targeting moiety to the active compound before administration. This ensures that the prodrug is pre-directed to tumor cells expressing folate receptors, allowing selective accumulation at the target site before the active compound is released, thereby preventing non-selective destruction of healthy tissues.

Inventive Principle:
Principle #10Preliminary action

4Speed

If self-reactive arms are designed for rapid release, then the active compound can be released immediately following elimination of the labile group, but the prodrug may lack sufficient stability for administration

Engineering Contradiction:
Improverelease speed of active compoundVSAvoidstability of prodrug structure
Core Design Contradiction:
SpeedVSStability of the object's composition

Solution Approach 1:

The self-reactive arm (o-nitrobenzyl carbamate) serves as an intermediary between the stable prodrug form and the active compound. It maintains stability during circulation as a protective carrier, and upon enzymatic trigger, it rapidly decomposes to release the active compound. The intermediary structure provides both stability when needed and rapid release when triggered.

Inventive Principle:
Principle #24Intermediary (Mediator)

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

The new prodrugs demonstrate improved stability and specificity, allowing for effective and rapid release of anticancer agents within targeted tissues, reducing side effects and improving therapeutic outcomes.

Implementation Method 1

Removal of the labile group leads, through intramolecular rearrangement of the self-reactive arm, to the release of the active compound

Methodology Applied
Scientific EffectEnzymatic hydrolysis: Hydrolysis

Implementation Method 2

A new self-reactive arm derived from phenol, capable of click chemistry reactions

Methodology Applied
Scientific EffectClick chemistry reaction: Chemical Bonding

Data Source

PatentEP2571528B1Self-reactive arms and prodrugs comprising same
Publication Date: 2018.01.31 CENT NAT DE LA RECH SCI (C N R S)
  • EP2571528B1 patent drawingFigure 1
  • EP2571528B1 patent drawingFigure 2
  • EP2571528B1 patent drawingFigure 3A~3B

AI summary

The present invention relates to a compound of general formula (I) in which: - X is OH, NH2, NHOH or RNH, wherein R may be a linear or branched, saturated or unsaturated, C1-C10 alkyl radical, - Y is H, or an electron-withdrawing group, in particular selected from NO2, CF3 or a halogen, - R1 and R2 are H or a linear or branched, saturated or unsaturated, C1-C10 alkyl radical, - F is a reactive functional group that can be activated by click chemistry.