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Published on: May 18, 2017
Attempted enzyme replacement using human amnion membrane implantations in mucopolysaccharidoses
J Muenzer1, E F Neufeld, G Constantopoulos
1Human Genetics Branch, National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892.
Journal of Inherited Metabolic Disease
|January 1, 1992
Summary
Human amnion membrane implantation did not effectively treat mucopolysaccharidoses (MPS). While some joint mobility improved, enzyme levels and urinary glycosaminoglycan excretion showed no significant long-term benefit.
Area of Science:
- Biomedical Engineering
- Regenerative Medicine
- Lysosomal Storage Disorders
Background:
- Mucopolysaccharidoses (MPS) are a group of rare genetic disorders.
- Enzyme replacement therapy is a potential treatment strategy for MPS.
- Human amnion membrane implantation has been explored as a novel approach for enzyme delivery.
Purpose of the Study:
- To evaluate the efficacy of human amnion membrane implantation for enzyme replacement in patients with mucopolysaccharidoses.
- To assess changes in clinical status, enzyme activity, and biochemical markers post-implantation.
Main Methods:
- Subcutaneous implantation of human amnion membranes in 19 MPS patients (Types I, II, and III).
- Systematic clinical evaluation of joint mobility before and 6 months after implantation.
- Serial measurements of lysosomal enzyme activity in serum and white blood cells.
- Monitoring of urinary glycosaminoglycan excretion.
- Histopathological analysis of implantation sites.
Main Results:
- No significant increase in deficient lysosomal enzyme activity was observed.
- Transient decrease in urinary glycosaminoglycan excretion in 2 out of 10 patients.
- Significant improvement in overall joint mobility, particularly shoulder extension and hip internal rotation.
- Histological examination revealed foreign-body reaction, fibrosis, and no viable amnion tissue at 6 months.
Conclusions:
- Human amnion membrane implantation is not an effective therapy for mucopolysaccharidoses.
- The observed improvements in joint mobility may not be directly attributable to enzyme replacement.
- Further research is needed to explore alternative and effective treatment strategies for MPS.
