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Immune response hinders therapy for lysosomal storage diseases
Katherine P. Ponder
Katherine P. Ponder
Published July 24, 2008
Citation Information: J Clin Invest. 2008;118(8):2686-2689. https://doi.org/10.1172/JCI36521.
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Immune response hinders therapy for lysosomal storage diseases

Abstract

Enzyme replacement therapy (ERT) for the lysosomal storage disease mucopolysaccharidosis I (MPS I) involves i.v. injection of α-l-iduronidase, which can be taken up by cells throughout the body. While a significant immune response to ERT has been shown in patients with MPS I, little is known about what effect anti-enzyme antibodies have on treatment efficacy. In this issue of the JCI, Dickson et al. demonstrate that anti-enzyme antibodies inhibit enzyme uptake and substantially limit the therapeutic efficacy of ERT in canines with MPS I (see the related article beginning on page 2868). Furthermore, the induction of immune tolerance — via oral delivery of cyclosporine A and azathioprine for two months at the time of initiation of ERT with recombinant human α-l-iduronidase — improved enzyme uptake in organs. Therefore, transient immunosuppression may enhance ERT for lysosomal storage diseases.

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Katherine P. Ponder

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