Patent ID: 11944628
Assignee: DUKE UNIVERSITY
Field: Pharmaceuticals (Chemistry)
Classification: CPC A | IPC A

Claim 0:
1. A method of inducing immune tolerance in a human subject suffering from Pompe disease, the method comprising:
(i) administering via infusion to a human treatment-naive subject a therapeutically effective amount of recombinant human alglucosidase alfa (rhGAA) as enzyme replacement therapy (ERT), and
(ii) administering to the subject an immune tolerance induction protocol,
wherein the immune tolerance induction protocol does not comprise administration of intravenous gamma globulin (IVIG), rituximab, or bortezomib prior to step (i);
wherein the immune tolerance induction protocol consists of a three-day cycle of methotrexate,
wherein the first day of the three-day cycle of methotrexate is the day of rhGAA administration,
wherein a dose of methotrexate comprises about 0.1 mg/kg body weight to about 0.6 mg/kg body weight per day,
wherein the methotrexate is administered either orally about one hour before the rhGAA administration or subcutaneously about 15 minutes before the rhGAA administration; and

(iii) repeating (i) and (ii) at least three times so that the subject begins a three-day cycle of methotrexate with each of the first four rhGAA infusions;
wherein, by following step (ii) and step (iii), immune tolerance to rhGAA is induced in the subject.