A therapeutic trial of amniotic epithelial cell implantation in patients with lysosomal storage diseases.
To determine whether allografts of normal amniotic epithelium might provide a nonimmunogenic cellular source of exogenous lysosomal enzymes, subcutaneous implants of amniotic epithelium were performed in six children with clinically advanced storage diseases. The clinical and the biochemical status of each patient was observed for several weeks after amniotic epithelial cell implantation (AECI). Serial studies of blood samples from each patient in the post-AECI period did not demonstrate any increase in levels of deficient lysosomal hydrolase. In two patients, quantitative urinary excretion of substrate was also studied and did not show consistent alterations after AECI. No patient had objective improvement in clinical or neurodevelopmental status following AECI. Two patients died with progressive disease at 2 1/2 and 3 1/2 mo after AECI; no residual amniotic epithelium was found at postmortem examination. Four patients are alive with progressive disease at 6-14 mo after AECI. We conclude that allografts of normal human amnion do not provide sufficient replacement hydrolases for clinical or biochemical improvement in lysosomal storage diseases.
D000581:Amidohydrolases / Q000172:deficiency; D000650:Amnion / Q000166:cytology / Q000637:transplantation*; D055572:Ceramidases; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D004847:Epithelial Cells; D004848:Epithelium / Q000637:transplantation; D005260:Female; D005733:Gangliosidoses / Q000628:therapy; D006801:Humans; D006867:Hydrolases / Q000378:metabolism; D007223:Infant; D007966:Leukodystrophy, Metachromatic / Q000628:therapy; D008297:Male; D008661:Metabolism, Inborn Errors / Q000628:therapy*; D009083:Mucopolysaccharidoses / Q000628:therapy
D006867:Hydrolases; D000581:Amidohydrolases; D055572:Ceramidases