Glycogen Synthase Modulating Compounds for Polyglucosan Body Reduction
Find Innovative SolutionsGenerate Solutions
Solution Overview
Problem
Current treatments for glycogen storage disorders, particularly those involving polyglucosan body accumulation, lack effective compounds to reduce or prevent the deposition and accumulation of polyglucosan bodies, which are associated with various forms of glycogen storage diseases, including Adult Polyglucosan Body Disease and Lafora Disease, leading to neurodegenerative symptoms and tissue damage.
Innovation Solution
Development of specific compounds represented by Formulas I to IV, which include pharmacologically acceptable salts and tautomers, capable of reducing polyglucosan body content by modulating enzymes such as glycogen synthase and protein phosphatase-1, thereby addressing the accumulation of polyglucosan bodies in tissues.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If current treatments for glycogen storage disorders are used, then existing therapeutic options are maintained, but effective reduction or prevention of polyglucosan body accumulation is not achieved
Solution Approach 1:
The patent applies parameter changes by developing compounds with specific molecular structures (Formulas I-IV) that modify the biochemical parameters of glycogen metabolism. These compounds change the activity parameters of key enzymes (glycogen synthase, protein phosphatase-1) to reduce polyglucosan body accumulation, thereby improving treatment effectiveness while addressing the harmful accumulation issue
Solution Approach 2:
The patent uses small molecule compounds as intermediaries to mediate between the existing enzyme system and the desired therapeutic outcome. These compounds act as intermediaries that modulate enzyme activity (glycogen synthase and protein phosphatase-1) to achieve reduction of polyglucosan bodies, providing a bridge between current treatments and effective therapy
2Reliability
If compounds modulating glycogen metabolism enzymes are developed, then polyglucosan body content is reduced, but complexity of drug development increases
Solution Approach 1:
The patent applies segmentation by dividing the complex therapeutic goal into specific molecular targets (glycogen synthase and protein phosphatase-1). The compounds are designed with segmented structural features (Formulas I-IV with specific substituents R1-R9h) that can be independently optimized to achieve enzyme modulation while managing overall molecular complexity
Solution Approach 2:
The patent applies local quality by introducing specific substituent groups (R1-R9h) at localized positions on the core molecular structure. This allows selective modulation of enzyme interactions at specific sites while maintaining overall compound manageability, achieving therapeutic efficacy without excessive global complexity
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 compounds effectively decrease polyglucosan body content, showing therapeutic potential in treating glycogen storage diseases by inhibiting key enzymes involved in glycogen metabolism, thereby ameliorating clinical symptoms and preventing disease progression.
Implementation Method 1
capable of reducing polyglucosan body content by modulating enzymes such as glycogen synthase and protein phosphatase-1
Data Source
AI summary
Compounds represented by general Formulae I to IV as described herein are disclosed. Further disclosed are composition utilizing the herein disclosed compounds and using the same for the treatment of glycogen storage disorders.