Leucine Therapy for Mobility and Cognitive Decline in LSDs
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Solution Overview
Problem
Current treatments for neurodegenerative diseases and lysosomal storage disorders (LSDs) are limited, with few curative options, and there is a need for therapies that can prevent or reduce symptoms such as impaired mobility and cognitive decline.
Innovation Solution
The use of leucine or its pharmaceutically acceptable salts for direct delivery in subjects to improve cognitive function and mobility, or treat neurodegenerative diseases and LSDs, through methods that include administering therapeutically effective amounts over durations ranging from at least 3 months to 5 years.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If current therapeutic approaches (enzyme replacement therapy, bone marrow transplantation) are used for LSDs, then some specific symptoms may be improved, but there are no general treatments that benefit all LSDs and curative options are limited
Solution Approach 1:
The patent applies universality by using a single compound (leucine or its pharmaceutically acceptable salts) to treat multiple different lysosomal storage disorders. The compound is designed to have broad-spectrum activity across various LSDs including Gaucher disease, Niemann-Pick disease, Tay-Sachs disease, and others, rather than requiring different treatments for each specific disorder.
Solution Approach 2:
The patent employs parameter changes by administering leucine at specific dosages (e.g., 10-50 mg/kg/day or higher) and for extended durations (at least 3 months to 5 years) to achieve therapeutic effects. The treatment parameters are optimized to delay or reverse disease progression across different LSD types.
2Reliability
If neurodegenerative diseases are left untreated, then disease progression continues leading to physical disability and mental deterioration, but existing treatments only improve quality of life without providing curative options
Solution Approach 1:
The patent applies self-service by utilizing leucine, which is a naturally occurring amino acid that the body can process. The compound leverages existing metabolic pathways and cellular mechanisms to achieve neuroprotective effects, reducing the need for complex external intervention systems while still providing substantial therapeutic benefit.
Solution Approach 2:
The patent employs a simple, readily available compound (leucine) that can be administered orally or via other straightforward routes. The treatment uses a basic amino acid that is inexpensive and easy to manufacture, rather than requiring complex biologics or sophisticated delivery systems.
3Duration of action of moving object
If leucine is administered for extended durations (at least 3 months to 5 years), then disease progression is delayed or reversed, but the treatment requires long-term commitment
Solution Approach 1:
The patent applies continuity of useful action by requiring sustained administration of leucine over extended periods (at least 3 months to 5 years). The treatment is designed to be continuous rather than intermittent, maintaining therapeutic levels in the body to progressively delay or reverse disease progression over time.
Data Source
AI summary
The present disclosure provides for treating neurodegenerative diseases and lysosomal storage disorders comprising administering leucine, or a pharmaceutically acceptable salt thereof for direct delivery in a subject in need thereof. The disclosure further provides for leucine, or a pharmaceutically acceptable salt thereof for direct delivery in a subject in need thereof, for example, in an elderly subject, to improve cognitive function, mobility, or cognitive function and mobility.