NMDA Receptor Modulators for Sterol Synthesis Disorders
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Solution Overview
Problem
Current treatments for sterol synthesis disorders, such as Smith-Lemli-Opitz syndrome, lack effective modulators to normalize sterol levels and improve brain excitability, leading to persistent neurological and developmental issues.
Innovation Solution
Administration of NMDA receptor modulating compounds, specifically those like 24(S)-hydroxycholesterol, to normalize sterol concentrations and modulate brain excitability, combined with additional therapies like dietary cholesterol supplementation or statin treatment.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If current treatments for sterol synthesis disorders are used, then standard therapy is provided, but sterol levels remain abnormal and brain excitability is not improved
Solution Approach 1:
The patent applies parameter changes by administering NMDA receptor modulating compounds at specific dosages to alter the physiological parameters of sterol levels and brain excitability. This changes the biochemical parameters in the body to achieve normalization of sterol concentrations and improve neurological function in patients with sterol synthesis disorders.
Solution Approach 2:
The patent uses NMDA receptor modulating compounds as intermediary substances that mediate between the sterol synthesis disorder and the desired therapeutic outcome. These compounds act as mediators to regulate brain excitability and normalize sterol levels, bridging the gap between the pathological state and the desired physiological state.
2Reliability
If NMDA receptor modulating compounds are administered, then sterol levels are normalized and brain excitability is improved, but additional therapies are required
Solution Approach 1:
The patent merges multiple therapeutic approaches by combining NMDA receptor modulating compounds with additional therapies such as dietary cholesterol supplementation or statin treatment. This combination therapy integrates different treatment modalities to achieve synergistic effects in normalizing sterol levels and improving brain excitability.
Solution Approach 2:
The patent applies multi-functionality by using NMDA receptor modulating compounds that simultaneously normalize sterol levels and improve brain excitability. This single compound performs multiple therapeutic functions, reducing the need for separate treatments and simplifying the overall therapeutic approach.
3Ease of operation
If NMDA receptor modulating compounds are used, then brain excitability is modulated and neurological function improves, but treatment is needed for associated conditions
Solution Approach 1:
The patent applies multi-functionality by using NMDA receptor modulating compounds that simultaneously address multiple conditions including sterol synthesis disorders, autism spectrum disorders, and psychiatric disorders. This single therapeutic approach provides universal benefits across different neurological and psychiatric conditions.
Solution Approach 2:
The patent applies dynamics by using compounds that can dynamically modulate brain excitability based on the specific needs of different conditions. The NMDA receptor modulating compounds can adapt their effect to normalize sterol levels in sterol synthesis disorders while also providing therapeutic benefits for associated neurological and psychiatric conditions.
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 approach effectively normalizes sterol levels, improves cholesterol accumulation in tissues, and modulates brain excitability, providing therapeutic benefits for sterol synthesis disorders and associated conditions like autism and psychiatric disorders.
Implementation Method 1
NMDA receptors are ligand-gated ion channels that admit Ca2+ after binding of the neurotransmitters glutamate and glycine
Implementation Method 2
NMDA modulator compounds, e.g., neuroactive steroids such as pregnenolone sulfate (PS) have been shown to exert direct modulatory effects on several types of neurotransmitter receptors
Data Source
AI summary
Methods for treating a subject suffering from a sterol synthesis disorder or a sterol deficiency disorder, e.g., Smith-Lemli-Opitz syndrome, the method comprising administering to the subject an effective amount of an NMDA receptor modulating compound, are provided.