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Rapid One-step Enzymatic Synthesis and All-aqueous Purification of Trehalose Analogues
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Effects of Trehalose Administration in Patients with Mucopolysaccharidosis Type III

Moein Mobini1, Shabnam Radbakhsh2,3, Francyne Kubaski4,5,6

  • 1Faculty of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran.

Current Medicinal Chemistry
|April 11, 2023
PubMed
Abstract

Insights

Trehalose treatment improved quality of life and certain biomarkers in patients with Mucopolysaccharidosis type III (MPS III). This study suggests trehalose may be a beneficial therapeutic agent for MPS III, showing positive effects on health-related quality of life and liver enzymes.

Area of Science:

  • Biochemistry
  • Genetics
  • Neurology

Background:

  • Mucopolysaccharidosis type III (MPS III) is a rare genetic lysosomal storage disease affecting the central nervous system.
  • It results from enzyme deficiencies impairing glycosaminoglycan (GAG) breakdown.
  • Trehalose is being investigated as a potential therapy for MPS III neuropathology.

Purpose of the Study:

  • To evaluate the efficacy of trehalose treatment in patients diagnosed with MPS IIIA and MPS IIIB.
  • To assess changes in quality of life, cognitive function, and specific biomarkers following trehalose administration.

Main Methods:

  • A single-arm, open-label study involving five MPS III patients.
  • Intravenous trehalose (15 g/week) administered for 12 weeks.
  • Evaluations included quality of life scores, cognitive assessments, serum biomarkers, and organ imaging.

Main Results:

  • Health-related quality of life scores (TAPQOL) showed improvement in all patients.
  • Serum GAG levels decreased, and liver enzymes (ALT, AST) were significantly reduced post-treatment.
  • Antioxidant status improved, indicated by decreased pro-oxidant-antioxidant balance and increased GPX activity.
  • Spleen size decreased, while liver volume remained unchanged.

Conclusions:

  • Three months of trehalose administration led to improvements in health-related quality of life for MPS III patients.
  • Positive changes were observed in serum biomarkers, including GAGs, liver aminotransferases, and antioxidant status.
  • Trehalose demonstrated potential as a therapeutic agent for MPS III, impacting key disease indicators and organ measurements.
Keywords:
Lysosomal storage disease (LSD)aspartate transaminase (AST).mucopolysaccharidosis IIIAmucopolysaccharidosis IIIBsanfilippo syndrometrehalose

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