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Current and Emerging Therapies for Mucopolysaccharidoses.
1Institute for Inborn Errors of Metabolism and Department of Paediatrics, Paracelsus Medical University, Salzburg, Austria. florian.lagler@pmu.ac.at.
Handbook of Experimental Pharmacology
|August 28, 2019
Summary
Mucopolysaccharidoses (MPSs) treatments like enzyme replacement and stem cell therapy improve outcomes but don't cure. New therapies aim to overcome limitations like immune reactions and poor tissue drug exposure for better MPS management.
Area of Science:
- Biochemistry
- Genetics
- Pharmacology
Background:
- Mucopolysaccharidoses (MPSs) result from enzyme deficiencies impairing glycosaminoglycan (GAG) degradation.
- Current treatments, enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT), are the standard of care for MPS.
- Despite improvements, ERT and HSCT do not offer a complete cure for MPS.
Purpose of the Study:
- To review the current landscape of MPS therapies.
- To highlight the limitations of existing treatments.
- To discuss innovative therapeutic strategies for MPS.
Main Methods:
- Review of existing literature on MPS treatments.
- Analysis of limitations associated with current ERT and HSCT.
- Exploration of novel therapeutic approaches.
Main Results:
- Existing MPS therapies face challenges including immune reactions and insufficient drug distribution.
- Innovative strategies involve small molecules, encapsulated cells, and gene therapy.
- New enzyme formulations aim to cross biological barriers like the blood-brain barrier.
Conclusions:
- Novel therapeutic strategies hold promise for overcoming current treatment limitations in MPS.
- Further development is needed to address challenges in drug delivery and immune response.
- These advancements aim to provide more effective and potentially curative treatments for MPS.
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