Cytidine Deaminase Inhibitors with Diazepinone Core for Acid Stability

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Solution Overview

Problem

Current cancer treatments, particularly those involving cytidine deaminase (CDA) inhibitors, face challenges such as acid instability and poor bioavailability, limiting their therapeutic effectiveness in inhibiting cytidine deaminase and enhancing the efficacy of cytidine-based anticancer drugs.

Innovation Solution

Development of new compounds, represented by formulas I to VIII, which are designed to inhibit cytidine deaminase with improved stability and bioavailability, allowing for their use in combination with non-decitabine CDA substrates like gemcitabine, ara-C, and 5-azacytidine to treat various cancers.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If cytidine deaminase inhibitors are used to enhance the efficacy of cytidine-based anticancer drugs, then the therapeutic activity is improved, but the compounds suffer from acid instability and poor bioavailability

Engineering Contradiction:
Improvetherapeutic activityVSAvoidacid stability
Core Design Contradiction:
ReliabilityVSStability of the object's composition

Solution Approach 1:

The patent modifies the chemical structure of cytidine deaminase inhibitors by changing parameters such as the sugar moiety (using 2'-deoxy-ribofuranosyl instead of ribofuranosyl), ring structure (diazepinone instead of pyrimidinone), and substituent groups (R1-R6). These parameter changes improve acid stability while maintaining or enhancing inhibitory activity against cytidine deaminase, thereby resolving the contradiction between therapeutic activity and acid stability.

Inventive Principle:
Principle #35Parameter changes

2Reliability

If cytidine deaminase inhibitors are used to enhance the efficacy of cytidine-based anticancer drugs, then the therapeutic activity is improved, but the compounds exhibit poor bioavailability

Engineering Contradiction:
Improvetherapeutic activityVSAvoidbioavailability
Core Design Contradiction:
ReliabilityVSQuantity of substance

Solution Approach 1:

The patent employs parameter changes by modifying molecular weight, lipophilicity (through fluorine substitution at R1/R2 positions), and hydrogen bonding capabilities (through hydroxyl and amino groups at R3-R6). These modifications enhance the compounds' ability to cross biological membranes and improve oral bioavailability while preserving their ability to inhibit cytidine deaminase and enhance cytidine-based drug efficacy.

Inventive Principle:
Principle #35Parameter changes

3Ease of operation

If traditional cytidine-based drugs are used alone, then the treatment is simple, but the drugs are rapidly deaminated by CDA into inactive metabolites, limiting their antineoplastic activity

Engineering Contradiction:
Improvetreatment simplicityVSAvoidantineoplastic activity
Core Design Contradiction:
Ease of operationVSReliability

Solution Approach 1:

The patent introduces cytidine deaminase inhibitors as intermediary compounds that co-administered with cytidine-based anticancer drugs. These inhibitors act as mediators by blocking the CDA enzyme that would otherwise rapidly deaminate the cytidine-based drugs into inactive metabolites. This intermediary approach maintains treatment simplicity while significantly enhancing and prolonging the antineoplastic activity of the cytidine-based drugs.

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

These compounds effectively inhibit cytidine deaminase, enhancing the therapeutic activity of cytidine-based drugs and improving their bioavailability, thereby offering a more potent approach to treating cancers like leukemia, lung cancer, and breast cancer.

Implementation Method 1

These compounds effectively inhibit cytidine deaminase, enhancing the therapeutic activity of cytidine-based drugs

Methodology Applied
Scientific EffectEnzyme inhibition: Enzyme

Data Source

PatentEP2417146B1(2'-deoxy-ribofuranosyl)-1,3,4,7-tetrahydro-(1,3)diazepin-2-one derivatives for treating cancer
Publication Date: 2016.07.13 OTSUKA PHARM CO LTD
  • EP2417146B1 patent drawingFigure 1
  • EP2417146B1 patent drawingFigure 2
  • EP2417146B1 patent drawingFigure 3

AI summary

Provided herein are compounds used to inhibit the deamination enzyme responsible for the inactivation of therapeutic compounds, and methods of using them.