mTOR Inhibitor Compounds with Blood-Brain Barrier Permeability
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Solution Overview
Problem
Current treatments for epilepsy, Alzheimer's, Parkinson's, and other neurodegenerative diseases related to the mTOR pathway are inadequate, with existing antiepileptic drugs only controlling 70% of chronic epilepsy cases and no fundamental treatment methods available for drug-resistant epilepsy, and existing mTOR inhibitors facing challenges in crossing the blood-brain barrier.
Innovation Solution
Development of novel compounds represented by Chemical Formula 1, which inhibit mTORC1 activity and have high blood-brain barrier permeability, used in pharmaceutical compositions to treat brain diseases associated with the mTOR pathway, including epilepsy, Alzheimer's, and Parkinson's disease.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If existing mTOR inhibitors are used to treat brain diseases, then mTOR inhibition activity is achieved, but blood-brain barrier permeability is insufficient
Solution Approach 1:
The patent applies parameter changes by systematically modifying the chemical structure of mTOR inhibitors, specifically adjusting substituents at different positions (R1-R6 groups) to optimize the balance between mTOR inhibition activity and blood-brain barrier permeability. This involves changing molecular weight, lipophilicity, and other physicochemical parameters to enhance CNS penetration while maintaining therapeutic efficacy.
Solution Approach 2:
The invention creates composite chemical structures by combining different functional groups and molecular fragments that collectively provide both mTOR inhibition capability and improved blood-brain barrier penetration. The compound represents a composite molecular design integrating hydrophobic regions for membrane penetration with specific pharmacophores for mTOR binding.
2Reliability
If existing antiepileptic drugs are used to treat chronic epilepsy, then seizure control is achieved in 70% of cases, but no fundamental treatment is available for drug-resistant epilepsy
Solution Approach 1:
The patent extracts the core mechanism of epilepsy treatment by directly targeting the mTOR pathway, which is implicated in the pathophysiology of epilepsy and neurodegenerative diseases. This approach moves away from conventional antiepileptic mechanisms to address the underlying pathological process, potentially providing fundamental treatment for drug-resistant cases.
Solution Approach 2:
The invention creates a multi-functional therapeutic agent that can address multiple conditions (epilepsy, Alzheimer's, Parkinson's, and other neurodegenerative diseases) through a single mechanism of action targeting the mTOR pathway. This universal approach expands treatment coverage across different neurological disorders, including drug-resistant epilepsy cases.
Data Source
AI summary
The present invention relates to a novel compound as an mTOR inhibitor and a use thereof and, more specifically, to a novel compound represented by formula 1 that exhibits mTOR inhibitory activity and a pharmaceutical composition comprising same as an active ingredient for preventing or treating brain diseases associated with an mTOR pathway.