Biodistribution and pharmacodynamics of recombinant human alpha-l-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations

Molecular Genetics and Metabolism

Volumn 103, Issue 3, 2011, Pages 268-274

  Vite, Charles H ^a   Wang, Ping ^a   Patel, Reema T ^a   Walton, Raquel M ^a   Walkley, Steven U ^b   Sellers, Rani S ^b   Ellinwood, N Matthew ^c   Cheng, Alphonsus S ^d   White, Joleen T ^d   O'Neill, Charles A ^d   Haskins, Mark ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b ALBERT EINSTEIN COLLEGE OF MEDICINE   (United States)

^c IOWA STATE UNIVERSITY   (United States)

^d BIOMARIN PHARMACEUTICAL INC   (United States)

Author keywords

Alpha l iduronidase; Enzyme replacement therapy; Feline; Intrathecal; Lysosomal storage disease; Mucopolysaccharidosis

Indexed keywords

BETA N ACETYLHEXOSAMINIDASE; CHOLESTEROL; GANGLIOSIDE GD2; GANGLIOSIDE GD3; GLYCOSAMINOGLYCAN; LARONIDASE; PLACEBO;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DRUG ACTIVITY; DRUG BIOAVAILABILITY; DRUG BRAIN LEVEL; DRUG EFFECT; DRUG EFFICACY; DRUG SAFETY; ENZYME ACTIVITY; ENZYME REPLACEMENT; EXPERIMENTAL CAT; FEMALE; HURLER SYNDROME; LIMB WEAKNESS; MALE; NEUROLOGIC DISEASE; NONHUMAN; PHARMACODYNAMICS; PRIORITY JOURNAL;

ANIMALS; ANTIBODIES; BRAIN; CATS; ENZYME REPLACEMENT THERAPY; FEMALE; GLYCOSAMINOGLYCANS; HEXOSAMINIDASES; HUMANS; IDURONIDASE; INJECTIONS, SPINAL; MALE; MUCOPOLYSACCHARIDOSIS I; RECOMBINANT PROTEINS;

ANIMALIA; FELIDAE;

EID: 79958064659     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2011.03.011     Document Type: Article

References (22)

1. Muenzer J., Wraith J., Clarke L. Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics 2009, 123:19-29.
2. Wraith E., Hopwood J., Fuller M., Meikle P., Brooks D. Laronidase treatment of mucopolysaccharidosis I. BioDrugs 2005, 19:1-7.
3. Kakkis E., Muenzer J., Tiller G., Waber L., Belmont J., Passage M., Izykowski B., Phillips J., Doroshow R., Walot I., Hoft R., Neufeld E. Enzyme-replacement therapy in mucopolysaccharidosis I. N. Engl. J. Med. 2001, 344:182-188.
4. Wraith J., Clarke L., Beck M., Kolodny E., Pastores G., Muenzer J., Rapoport D., Berger K., Swiedler S., Kakkis E., Braakman T., Chadbourne E., Walton-Bowen K., Cox G. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-l-iduronidase (laronidase). J. Pediatr. 2004, 144:581-588.
5. Wraith E. The first 5years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I. Expert Opin. Pharmacother. 2005, 6:489-506.
6. Sifuentes M., Doroshow R., Hoft R., Mason G., Walot I., Diament M., Okazaki S., Huff K., Cox G., Swiedler S., Kakkis E. A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6years. Mol. Genet. Metab. 2007, 90:171-180.
7. Tandon V., Williamson J., Cowie R., Wraith J. Spinal problems in mucopolysaccharidosis I (Hurler syndrome). J. Bone Joint Surg. Br. 1996, 78:938-944.
8. Kachur E., Del Maestro R. Mucopolysaccharidoses and spinal cord compression: case report and review of the literature with implications of bone marrow transplantation. Neurosurgery 2000, 47:223-228.
9. Kakkis E., McEntee M., Vogler C., Le S., Levy B., Belichenko P., Mobley W., Dickson P., Hanson S., Passage M. Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS1. Mol. Genet. Metab. 2004, 83:163-174.
10. Dickson P., Hanson S., McEntee M., Vite C., Vogler C., Mlikotic A., Chen A., Ponder K., Haskins M., Tippin B., Le S., Passage M., Guerra C., Dierenfeld A., Jens J., Snella E., Kan S., Ellinwood N. Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I. Mol. Genet. Metab. 2010, 101:115-122.
11. Munoz-Rojas M., Vieira T., Costa R., Fagondes S., John A., Jardim L., Vedolin L., Raymundo M., Dickson P., Kakkis E., Giugliani R. Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression. Am. J. Med. Genet. A 2008, 146A:2538-2544.
12. Tolar J., Dickson P., Orchard P. Combined intrathecal iduronidase, intravenous iduronidase, and transplantation as therapy for Hurler syndrome. Mol. Genet. Metab. 2009, 96:S43.
13. Dickson P., Naylor D., Mlikotic A., Victoroff A., Chen A., Passage M. Intrathecal recombinant human α-l-iduronidase alleviates spinal cord compression symptoms and is well tolerated in attenuated MPS I patients. Mol. Genet. Metab. 2007, 93:247.
14. Haskins M., Aguirre G., Jezyk P., Desnick R., Patterson D. The pathology of the feline model of mucopolysaccharidosis I. Am. J. Pathol. 1983, 112:27-36.
15. Kakkis E., Schuchman E., He X., Wan Q., Kania S., Wiemelt S., Hasson C., O'Malley T., Weil M., Aguirre G., Brown D., Haskins M. Enzyme replacement therapy in feline mucopolysaccharidosis I. Mol. Genet. Metab. 2001, 72:199-208.
16. Haskins M., Jezyk P., Patterson D. Mucopolysaccharide storage disease in 3 families of cats with arylsulfatase B deficiency: leukocyte studies and carrier identification. Pediatr. Res. 1979, 13:1203-1210.
17. Zervas M., Dobrenis K., Walkley S. Neurons in Niemann-Pick disease type C accumulate gangliosides as well as unesterified cholesterol and undergo dendritic and axonal alterations. J. Neuropathol. Exp. Neurol. 2001, 60:49-64.
18. McGlynn R., Dobrenis K., Walkley S. Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders. J. Comp. Neurol. 2004, 480:415-426.
19. Shull R., Kakkis E., McEntee M., Kania S., Jonas A., Neufeld E. Enzyme replacement in a canine model of Hurler syndrome. Proc. Natl Acad. Sci. U.S.A. 1994, 91:12937-12941.
20. Dickson P., McEntee M., Vogler C., Le S., Levy B., Peinovich M., Hanson S., Passage M.M., Kakkis E. Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid. Mol. Genet. Metab. 2007, 91:61-68.
21. Taccone A., Tortori Donati, Marzoli P.A., Dell'Acqua A., Gatti R., Leone D. Mucopolysaccharidosis: thickening of dura mater at the craniocervical junction and other CT/MRI findings. Pediatr. Radio 1993, 23:349-352.
22. Schwartz G., Cohen E. Hydrocephalus in Maroteaux-Lamy syndrome. Arch. Ophthalmol. 1998, 116:400.