Injectable Trehalose Delivery for Protein Aggregation Diseases
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Solution Overview
Problem
Current therapeutic strategies for myopathies and neurodegenerative disorders associated with abnormal protein aggregation, such as oculopharyngeal muscular dystrophy (OPMD), lack effective treatments that can halt or reverse the progression of the disease, and existing methods like oral trehalose administration are hindered by significant degradation in the gastrointestinal tract, limiting bioavailability.
Innovation Solution
Parenteral administration of trehalose, specifically through intravenous routes, to bypass intestinal metabolism and achieve higher bioavailability and therapeutic efficacy in treating diseases characterized by abnormal protein aggregation, including OPMD, by delivering trehalose directly to muscle and neuronal tissues.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Ease of operation
If oral trehalose administration is used, then the treatment approach is simple and non-invasive, but significant degradation in the gastrointestinal tract limits bioavailability and therapeutic efficacy
Solution Approach 1:
The patent uses parenteral administration (intravenous, intramuscular, or subcutaneous injection) as an intermediary route to deliver trehalose directly into the systemic circulation, bypassing the gastrointestinal tract and its degrading enzymes. This mediator approach eliminates the degradation problem while maintaining effective drug delivery to target tissues.
Solution Approach 2:
Instead of administering trehalose orally and hoping it survives gastrointestinal degradation, the patent inverts the approach by using parenteral administration to deliver trehalose directly into the bloodstream. This reversal of the administration route completely avoids the degradation pathway while achieving systemic distribution.
2Reliability
If parenteral administration is used, then bioavailability and plasma concentrations are significantly improved, but the treatment becomes more invasive and complex
Solution Approach 1:
The patent changes the administration route parameter from oral to parenteral (intravenous, intramuscular, or subcutaneous). This parameter change fundamentally alters the pharmacokinetic profile, achieving high and sustained plasma concentrations of trehalose that are necessary for therapeutic efficacy in protein aggregation diseases.
3Object-affected harmful factors
If oral trehalose is administered, then the treatment is well-tolerated with established safety, but the therapeutic effect is limited due to gastrointestinal degradation
Solution Approach 1:
Parenteral administration serves as an intermediary pathway that bypasses the harmful gastrointestinal degradation environment. By injecting trehalose directly into the systemic circulation, the treatment avoids the harmful factors in the GI tract while ensuring adequate therapeutic doses reach the target tissues.
Data Source
AI summary
Disclosed is a method of treatment of a disease associated with abnormal protein aggregation comprising parenterally administering pharmaceutical formulations comprising trehalose. Also disclosed is an injectable aqueous pharmaceutical formulation comprising a therapeutically effective amount of trehalose.