Immunophilin-Binding Compounds for CNS Drug Delivery Selectivity
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Solution Overview
Problem
Current therapeutic agents targeting mTOR face challenges in crossing the blood-brain barrier and are confounded by systemic toxicity and immunosuppression, limiting their effectiveness in treating CNS diseases.
Innovation Solution
Development of immunophilin-binding compounds, such as AB-LB1-RB1, which concentrate in circulating blood rather than the CNS, allowing for targeted delivery of anti-CNS disease drugs outside the CNS, thereby minimizing systemic side effects.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If mTOR inhibitors are administered to treat CNS diseases, then therapeutic efficacy is improved, but the ability to cross the blood-brain barrier is insufficient
Solution Approach 1:
The patent uses immunophilin-binding compounds as intermediary carriers that can cross the blood-brain barrier and deliver mTOR inhibitors to the CNS. The immunophilin-binding moiety acts as a mediator that facilitates blood-brain barrier penetration while the polar moiety carries the therapeutic agent, resolving the contradiction between maintaining therapeutic efficacy and achieving sufficient barrier penetration.
2Adaptability or versatility
If mTOR inhibitors are administered systemically, then therapeutic coverage is improved, but systemic toxicity increases
Solution Approach 1:
The patent creates a locally targeted delivery system where immunophilin-binding compounds concentrate the mTOR inhibitor delivery in specific tissues (CNS or peripheral tissues) rather than uniform systemic distribution. This allows therapeutic coverage where needed while minimizing toxicity in other systems, as the immunophilin-binding moiety directs localized accumulation.
3Reliability
If mTOR inhibitors are administered to treat CNS diseases, then disease treatment is improved, but immunosuppression occurs
Solution Approach 1:
The immunophilin-binding compound serves as a mediator that enables selective delivery of mTOR inhibitors to the CNS, reducing off-target effects in immune-privileged tissues. This intermediary approach allows effective CNS disease treatment while minimizing systemic immunosuppression by limiting the inhibitor's exposure to immune system components.
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
Enhances the delivery of mTOR inhibitors to the CNS while reducing systemic toxicity, providing effective treatment for CNS diseases like glioblastoma with improved safety profiles.
Implementation Method 1
Subsequent to administration to a subject, the concentration of the compound in circulating blood of the subject is greater than the concentration of the compound in the CNS of the subject
Data Source
AI summary
Described herein, inter alia, are immunophilin binding compounds and methods of treating CNS diseases, including co-administering outside the CNS of a subject an anti-CNS disease drug and a compound described herein.