Intravenous Trehalose Formulation for MPS Symptom Relief
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Solution Overview
Problem
Current treatments for mucopolysaccharidoses, such as hematopoietic stem cell transplant and enzyme replacement therapy, are invasive, expensive, and provide uneven enzyme distribution, leaving certain areas untreated, necessitating a less invasive and more effective treatment option.
Innovation Solution
Administering an intravenous aqueous pharmaceutical formulation comprising substantially purified trehalose with specific pH, osmolality, and contaminant levels, optionally with a trehalase inhibitor, over a defined duration, to treat and alleviate symptoms of mucopolysaccharidoses.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If enzyme replacement therapy is used, then enzyme activity is restored, but distribution is uneven leaving bones, lungs, and brain untreated
Solution Approach 1:
The patent uses trehalose as an intermediary substance that facilitates uniform enzyme distribution throughout the body, particularly to hard-to-reach areas like bones, lungs, and brain. The trehalose formulation acts as a carrier or mediator that enables the enzyme replacement therapy to reach all target tissues effectively.
Solution Approach 2:
The patent modifies the physical and chemical parameters of the enzyme formulation by combining it with trehalose, creating a new formulation with improved distribution properties. This parameter change enables the enzyme to be delivered uniformly across different tissue types and anatomical regions.
2Reliability
If hematopoietic stem cell transplant is performed, then enzyme production is restored, but the procedure is invasive and expensive
Solution Approach 1:
The patent employs a disposable enzyme replacement formulation that provides temporary but effective enzyme activity without requiring permanent implantation or complex surgical procedures. The trehalose-based formulation can be administered repeatedly via simple routes, replacing the need for invasive stem cell transplantation.
Solution Approach 2:
The patent replaces the mechanical/surgical approach of stem cell transplantation with a chemical/pharmacological approach using trehalose-based enzyme formulation. This substitution eliminates the need for invasive procedures while achieving the same therapeutic goal of restoring enzyme function.
3Reliability
If conventional enzyme replacement therapy is administered, then treatment is provided, but access to advanced medical care facilities is required
Solution Approach 1:
The trehalose-based formulation is designed to be self-administerable or easily administrable without requiring specialized medical facilities or advanced equipment. The formulation's stability and ease of administration enable treatment to be delivered in simpler settings, reducing dependency on advanced medical care infrastructure.
Data Source
AI summary
The present disclosure relates to methods of treating or alleviating one or more symptoms of mucopolysaccharidoses in a subject, via administering a trehalose formulation to the subject.