MMP-Cleavable VDA Prodrugs for Selective Tumor Activation

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Solution Overview

Problem

Current vascular disrupting agents (VDAs) used in cancer treatment often have poor specificity for tumors over normal tissues and are toxic, with challenges in formulation due to water insolubility, limiting their clinical applicability.

Innovation Solution

Development of prodrugs with a matrix metalloproteinase (MMP) proteolytic cleavage site, specifically an Arg-Ser-Cit-Gly-Hof-Leu sequence, allowing selective release of VDAs like colchicine at tumor vasculature, enhancing therapeutic index and efficacy by activating only in tumor environments with overexpressed MMPs.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If vascular disrupting agents are administered systemically to achieve potent anti-cancer effects, then tumor cell killing is improved, but toxicity to normal tissues increases

Engineering Contradiction:
Improveanti-cancer efficacyVSAvoidtoxicity to normal tissues
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The VDA is segmented into a prodrug form that requires enzymatic activation. The parent VDA is linked to a peptide sequence containing an MMP cleavage site, creating a segmented structure that separates the active drug from its delivery vehicle. This segmentation allows the prodrug to be administered systemically with reduced toxicity while maintaining anti-cancer efficacy through selective activation at the tumor site.

Inventive Principle:
Principle #1Segmentation

Solution Approach 2:

An MMP-cleavable peptide sequence acts as an intermediary between the VDA and the tumor vasculature. This intermediary is designed to be stable in circulation but selectively cleaved by matrix metalloproteinases overexpressed at the tumor site, thereby mediating the selective release of the active VDA. The intermediary peptide sequence (e.g., RGDS or similar cell-adhesion motifs) facilitates targeted delivery while protecting normal tissues from direct drug exposure.

Inventive Principle:
Principle #24Intermediary (Mediator)

2Speed

If potent VDAs like colchicine are used to achieve rapid vascular collapse, then tumor necrosis is improved, but therapeutic index narrows due to high toxicity

Engineering Contradiction:
Improvevascular collapse speedVSAvoidtherapeutic index
Core Design Contradiction:
SpeedVSReliability

Solution Approach 1:

The VDA is converted into a prodrug form that accumulates at the tumor site before activation. The prodrug is administered systemically and selectively accumulates in the tumor vasculature through passive targeting or active uptake mechanisms. Only after accumulation at the target site does enzymatic activation occur, releasing the active VDA locally. This preliminary accumulation step ensures rapid vascular collapse at the tumor while avoiding systemic toxicity, thereby improving the therapeutic index.

Inventive Principle:
Principle #10Preliminary action

3Ease of manufacture

If VDAs are formulated for clinical evaluation, then water solubility must be improved, but formulation complexity increases

Engineering Contradiction:
Improveformulation feasibilityVSAvoidformulation complexity
Core Design Contradiction:
Ease of manufactureVSDevice complexity

Solution Approach 1:

The VDA is chemically modified by conjugation to a peptide sequence, fundamentally changing its physicochemical parameters. This conjugation improves water solubility and enables formulation as a stable prodrug suitable for clinical administration. The peptide-VDA conjugate can be formulated in standard pharmaceutical vehicles without requiring complex formulation strategies, thereby improving ease of manufacture while maintaining simplicity.

Inventive Principle:
Principle #35Parameter changes

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 approach increases tumor levels of the VDA while decreasing systemic levels, improving the therapeutic index and efficacy by targeting the tumor vasculature selectively, reducing toxicity and enhancing antitumor effects.

Implementation Method 1

a peptide comprising a matrix metalloproteinase (MMP) proteolytic cleavage site, wherein the VDA is an anti-cancer and tubulin binding agent

Methodology Applied
Scientific EffectProteolytic cleavage: Enzyme

Implementation Method 2

Colchicine and its analogues are potent VDAs causing haemorrhage and subsequent extensive necrosis in tumours (Tozer et al., Nat Rev Cancer, 5, 423-435 (2005)), as a direct consequence of tubulin binding and induction of microtubule depolymerisation

Methodology Applied
Scientific EffectTubulin binding:

Data Source

PatentEP2349344B1MMP activated vascular disrupting agents
Publication Date: 2018.12.05 ELLIPSES PHARMA LTD
  • EP2349344B1 patent drawingFigure 1A~2B
  • EP2349344B1 patent drawingFigure 3C~4D
  • EP2349344B1 patent drawingFigure 5~6F

AI summary

The present invention relates to prodrugs of vascular disrupting agents comprising a vascular disrupting agent (VDA) associated with a MMP proteolytic cleavage site and to the use of such prodrugs in the targeted treatment of cancer.