HDAC Inhibitor AR-42 Suppresses Muscle Wasting in Cancer Cachexia

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Solution Overview

Problem

Current therapies are ineffective in preventing or halting the progression of cancer-related cachexia, a condition characterized by muscle wasting and weight loss in cancer patients, with no FDA-approved treatments available to manage muscle wasting in cachectic patients.

Innovation Solution

Administration of the HDAC class 1 and 2b inhibitor AR-42 to cancer patients to suppress cachexia, maintaining weight and reducing muscle wasting, independent of tumor load reduction, by downregulating pro-cachexia mediators and reprogramming cell metabolism.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Loss of substance

If HDAC inhibitors (e.g., Trichostatin A) are administered to suppress cachexia, then protein degradation may be reduced, but hyperacetylation can increase protein degradation leading to increased muscle wasting and cachexia

Engineering Contradiction:
Improvemuscle massVSAvoidtherapeutic effect consistency
Core Design Contradiction:
Loss of substanceVSReliability

Solution Approach 1:

The patent changes the chemical structure parameters of HDAC inhibitors by introducing specific substituents (halogen atoms at positions 2 and 4, methoxy group at position 3) on the benzamide core structure. This structural modification alters the biological activity parameters, producing inhibitors with improved selectivity and reduced side effects compared to conventional HDAC inhibitors like Trichostatin A.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent applies local quality modification by placing specific functional groups (halogen atoms and methoxy group) at specific positions on the benzamide molecule. This localized structural modification creates differential effects on HDAC isoforms, achieving selective inhibition that reduces harmful hyperacetylation while maintaining beneficial anti-cachexia effects.

Inventive Principle:
Principle #3Local quality

2Weight of moving object

If conventional nutritional support is provided to cachectic patients, then weight loss may be slowed, but it does not reverse skeletal muscle mass depletion

Engineering Contradiction:
Improvebody weightVSAvoidskeletal muscle mass
Core Design Contradiction:
Weight of moving objectVSLoss of substance

Solution Approach 1:

The patent introduces HDAC inhibitors as an intermediary substance that mediates between nutritional support and muscle mass preservation. The inhibitors act on the molecular level to modulate protein degradation pathways, working synergistically with nutritional support to achieve both weight maintenance and muscle mass preservation, whereas nutritional support alone only addresses weight but not muscle quality.

Inventive Principle:
Principle #24Intermediary (Mediator)

3Loss of substance

If HDAC inhibitors are used to treat cachexia, then muscle wasting may be reduced, but contradictory and conflicting results have been observed in different studies

Engineering Contradiction:
Improvemuscle wastingVSAvoidstudy result consistency
Core Design Contradiction:
Loss of substanceVSMeasurement precision

Solution Approach 1:

The patent modifies key parameters of HDAC inhibitors including molecular weight, lipophilicity, and enzymatic inhibition constants through strategic placement of halogen and methoxy substituents. These parameter changes produce compounds with optimized pharmacokinetic and pharmacodynamic properties, leading to more consistent and reliable therapeutic effects across different study conditions.

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

AR-42 effectively maintains skeletal muscle weight, reduces cachexia-induced adipose tissue loss, and prolongs survival in animal models of cancer cachexia, demonstrating its ability to suppress muscle wasting and improve survival rates without affecting tumor growth.

Implementation Method 1

Administration of the HDAC class 1 and 2b inhibitor AR-42 to suppress cachexia

Methodology Applied
Scientific EffectHDAC inhibition:

Data Source

PatentEP3071196B1HDAC inhibitors for suppressing cancer-related cachexia
Publication Date: 2019.03.27 OHIO STATE INNOVATION FOUND
  • EP3071196B1 patent drawingFigure 1A
  • EP3071196B1 patent drawingFigure 1B
  • EP3071196B1 patent drawingFigure 1C

AI summary

Methods of suppressing cachexia in a mammal with cancer comprising administering HDAC inhibitors are provided. Aspects include methods of administering an HDAC class 1 and 2b inhibitor in an amount effective to substantially maintain the mammal's weight compared to a mammal that does receive the HDAC class 1 and 2b inhibitor.