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

• Published in: Molecular genetics and metabolism reports Vol. 1; no. C; pp. 446 - 450
• Main Authors: Sun, Baodong, Banugaria, Suhrad G, Prater, Sean N, Patel, Trusha T, Fredrickson, Keri, Ringler, Douglas J, de Fougerolles, Antonin, Rosenberg, Amy S, Waldmann, Herman, Kishnani, Priya S
• Format: Journal Article
• Language: English
• Published: United States Elsevier 01-01-2014
• Subjects: Anti-CD4 antibody; Antigen-specific immune tolerance; Enzyme replacement therapy; Pompe disease; Short Communication; Pompe disease; IPD, infantile Pompe disease; Anti-CD4 antibody; MTX, methotrexate; Enzyme replacement therapy; CRIM, cross-reacting immunologic material; ITI, immune tolerance induction; ERT, enzyme replacement therapy; Antigen-specific immune tolerance; GAA, acid-α-glucosidase
• ISSN: 2214-4269; 2214-4269
• DOI: 10.1016/j.ymgmr.2014.08.005

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.