TUDCA Oral Therapy for Slowing ALS Functional Deterioration
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Solution Overview
Problem
Current treatments for amyotrophic lateral sclerosis (ALS) are inadequate in stopping or significantly slowing the neurodegenerative process, and there is a need for a well-tolerated treatment that can improve the quality of life of patients.
Innovation Solution
Administering tauroursodeoxycholic acid (TUDCA) or a pharmaceutically acceptable salt thereof, preferably orally, at a dose of at least 1.5 g per day, for at least 30 weeks, potentially combined with traditional treatments like riluzole, to slow the progression of ALS.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If traditional treatments like riluzole are used to treat ALS, then some disease modifying efficacy is achieved by prolonging survival, but the effect on functional deterioration is minimal and the degenerative process is not significantly slowed
Solution Approach 1:
The patent applies parameter changes by switching from glutamate release inhibition (riluzole mechanism) to mitochondrial dysfunction correction and apoptosis inhibition (TUDCA mechanism). This fundamental mechanism change enables simultaneous improvement in both survival and functional outcomes by addressing the root cause of neurodegeneration rather than just symptomatic management
Solution Approach 2:
TUDCA acts as an intermediary substance that mediates between mitochondrial dysfunction and neuronal cell death. By stabilizing mitochondrial function and inhibiting apoptosis pathways, TUDCA protects motor neurons from degeneration, thereby improving both survival and functional deterioration without directly inhibiting glutamate release
2Reliability
If higher doses of TUDCA are administered to achieve better therapeutic effect, then disease progression is slowed more effectively, but the risk of side effects and tolerance issues may increase
Solution Approach 1:
TUDCA leverages the body's existing bile acid transport systems and metabolic pathways for its delivery and elimination. By utilizing endogenous transport mechanisms, TUDCA achieves effective tissue distribution without requiring complex delivery systems or high doses, thereby minimizing side effects while maintaining therapeutic efficacy
Solution Approach 2:
The patent optimizes the dosage parameter to achieve the minimum effective concentration that provides therapeutic benefit while avoiding toxicity. By carefully controlling the dose and administration frequency, the treatment maximizes therapeutic effect on mitochondrial function and apoptosis inhibition while minimizing potential side effects
Data Source
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AI summary
It is disclosed a method for the treatment of neurodegenerative disorders, such as ALS, Alzheimer's disease, Parkinson's disease, Huntington's disease and/or retinitis pigmentosa, which method comprises administering to a patient tauroursodeoxycholic acid or a pharmaceutically acceptable salt thereof.