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Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease.

ABSTRACT: Approximately 35-40% of patients with classic infantile Pompe disease treated with enzyme replacement therapy (ERT) develop high, sustained antibody titers against the therapeutic enzyme alglucosidase alfa, which abrogates the treatment efficacy. Induction of antigen-specific immune tolerance would greatly enhance ERT for these patients. Here we show that a short-course treatment with non-depleting anti-CD4 monoclonal antibody successfully induced long-term ERT-specific immune tolerance in Pompe disease mice. Our data suggest an effective adjuvant therapy to ERT.

SUBMITTER: Sun B

PROVIDER: S-EPMC5121343 | biostudies-literature | 2014

REPOSITORIES: biostudies-literature

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Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease.

Sun Baodong B Banugaria Suhrad G SG Prater Sean N SN Patel Trusha T TT Fredrickson Keri K Ringler Douglas J DJ de Fougerolles Antonin A Rosenberg Amy S AS Waldmann Herman H Kishnani Priya S PS

Molecular genetics and metabolism reports 20141012

Approximately 35-40% of patients with classic infantile Pompe disease treated with enzyme replacement therapy (ERT) develop high, sustained antibody titers against the therapeutic enzyme alglucosidase alfa, which abrogates the treatment efficacy. Induction of antigen-specific immune tolerance would greatly enhance ERT for these patients. Here we show that a short-course treatment with non-depleting anti-CD4 monoclonal antibody successfully induced long-term ERT-specific immune tolerance in Pompe ...[more]

PMID: 27896120

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Improved efficacy of a next-generation ERT in murine Pompe disease.

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Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction.

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First-in-human phase 1 study of IT1208, a defucosylated humanized anti-CD4 depleting antibody, in patients with advanced solid tumors.

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| S-EPMC6657210 | biostudies-literature

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