Aminosterol Compounds for Alpha-Synuclein and PTP1B Modulation
Find Innovative SolutionsGenerate Solutions
Solution Overview
Problem
There is a need for novel aminosterol compounds and methods of using the same to address various human diseases and conditions, including neurodegenerative disorders, ischemic disorders, and regulatory phosphatase inhibition.
Innovation Solution
Development of aminosterol compounds with specific formulas and their pharmaceutically acceptable salts or solvates, formulated in various compositions for administration routes, targeting abnormal alpha-synuclein pathology and dopaminergic dysfunction, and inhibiting regulatory phosphatases like PTP1B to treat conditions such as Parkinson's disease, Alzheimer's disease, and cerebral ischemia.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If existing aminosterol compounds (e.g., squalamine, aminosterol 1436) are used, then diverse pharmacological activities are achieved, but novel therapeutic applications and improved efficacy are limited
Solution Approach 1:
The patent segments the existing aminosterol structure into core components (steroid nucleus, side chain, functional groups) and systematically modifies each segment to create novel compounds. This allows exploration of multiple therapeutic applications while maintaining the core pharmacological activity of aminosterols.
Solution Approach 2:
The patent applies parameter changes by modifying chemical parameters of aminosterol compounds including substitution patterns, functional groups, stereochemistry, and molecular weight. These parameter changes generate novel compounds with improved therapeutic efficacy and expanded adaptability to different disease states.
2Adaptability or versatility
If aminosterol compounds are developed for multiple disease indications, then versatility is improved, but complexity of compound characterization and validation increases
Solution Approach 1:
The patent designs aminosterol compounds with universal multi-functionality by targeting common pathological mechanisms across different diseases, particularly regulatory phosphatase inhibition and alpha-synuclein pathology. This allows single compounds to address multiple indications including Parkinson's disease, Alzheimer's disease, diabetes, and cancer without proportionally increasing characterization 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 aminosterol compounds effectively treat, prevent, or slow the progression of conditions related to abnormal alpha-synuclein pathology and dopaminergic dysfunction, including neurodegenerative diseases and ischemic disorders, by inhibiting PTP1B and increasing gene transcription, thereby providing therapeutic benefits.
Implementation Method 1
Aminosterol 1436 was shown to act within the nuclei of the hypothalamus involved in appetite and energy balance, and to reverse insulin resistance in both diet-induced and genetic models in mice and rats. The compound was shown to inhibit PTP1B, the phosphatase that turns off the activated insulin receptor
Implementation Method 2
regenerative repair of myocardial infarction and traumatic limb muscle injury by mobilizing stem cells in adult mice
Implementation Method 3
reduction in toxic alpha synuclein aggregate formation and an increase in lifespan in a C. elegans Parkinson's model
Data Source
Figure 1A
Figure 1B
Figure 1C
AI summary
The present application relates generally to novel aminosterol compounds, compositions comprising the same, and methods of making and using the novel aminosterol compounds and compositions.