Compound 1 GABA Modulation for Essential Tremor Treatment
Find Innovative SolutionsGenerate Solutions
Solution Overview
Problem
Current treatments for essential tremor do not effectively address the cerebellar dysfunction and GABA receptor imbalances implicated in the condition, leading to inadequate tremor reduction and impact on daily activities.
Innovation Solution
Administration of Compound 1 or its pharmaceutically acceptable salt, in doses ranging from 5 mg to 80 mg daily, to modulate GABA receptor function and reduce tremor amplitude and activities of daily living subscale scores in subjects with essential tremor.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If current treatments for essential tremor are used, then tremor reduction is achieved to some extent, but the treatment does not effectively address cerebellar dysfunction and GABA receptor imbalances
Solution Approach 1:
The patent applies parameter changes by modifying the chemical structure of progesterone to create Compound 1 with enhanced neuroactive steroid properties. This structural modification changes the pharmacological parameters to achieve better GABA receptor modulation and cerebellar dysfunction correction, directly addressing the inadequacy of current treatments while maintaining a focused mechanism of action.
Solution Approach 2:
The patent uses Compound 1 as an intermediary substance that mediates the treatment of essential tremor by specifically targeting GABA receptor imbalances and cerebellar dysfunction. This neuroactive steroid acts as a chemical mediator between the administration route and the therapeutic effect, providing a direct mechanism to address the underlying pathophysiology of ET.
2Productivity
If current treatments are used, then some tremor reduction is achieved, but inadequate tremor reduction occurs impacting daily activities
Solution Approach 1:
The patent achieves enhanced tremor reduction efficacy by changing the pharmacological parameters of the treatment through Compound 1 administration. The compound demonstrates superior GABA receptor modulation compared to current treatments, leading to more effective tremor control and improved daily activities functionality as measured by TETRAS scores.
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 treatment results in significant reductions in tremor amplitude, ranging from 5% to 75%, and improvements in activities of daily living scores, with plasma concentrations of Compound 1 achieving therapeutic levels for sustained efficacy.
Implementation Method 1
Administration of Compound 1 or its pharmaceutically acceptable salt, in doses ranging from 5 mg to 80 mg daily, to modulate GABA receptor function and reduce tremor amplitude and activities of daily living subscale scores in subjects with essential tremor
Data Source
AI summary
The present invention provides a method of treating essential tremor in a subject in need thereof comprising administering Compound 1or a pharmaceutically acceptable salt thereof, or a pharmaceutical composition comprising Compound 1 or a pharmaceutically acceptable salt thereof, wherein the subject experiences (a) a reduction in tremor amplitude of at least about 5% following treatment onset, (b) a reduction in TETRAS activities of daily living (ADL) subscale total score following treatment onset, or (c) both.