Safe and Efficient Drug for Hunter Syndrome

  • Author: Anar Murphy
  • Published: 11 September 2014
  • Copyright: Wiley-VCH Verlag GmbH & Co. KGaA, Weinheim
  • Source / Publisher: Biotechnology & Bioengineering/John Wiley & Sons
thumbnail image: Safe and Efficient Drug for Hunter Syndrome

Mucopolysaccharidosis II (MPS II) or Hunter syndrome is an X-linked inherited lysosomal storage disease. This serious genetic disorder interferes with the body's ability to break down and recycle specific mucopolysaccharides. It is caused by a defective or missing enzyme, iduronate-2-sulfatase (IDS). IDS is a large molecule unable to pass the brain-blood barrier (BBB), making enzyme replacement therapies impossible.

To solve this issue, William Pardridge, ArmaGen Technologies, Inc., Calabasas, CA, USA, and colleagues reengineered IDS by fusing it with a monoclonal antibody against the human insulin receptor (HIRMAb). HIRMAb has an innate ability to cross the BBB via receptor-mediated transport on the endogenous BBB insulin receptor, making it a perfect molecular Trojan horse to shuttle IDS from blood into brain.
The researchers conducted the first safety pharmacology and pharmacokinetics study of the HIRMAb-IDS fusion protein in juvenile male Rhesus monkeys by infusing them with it intravenously on a weekly basis for 26 days at 3–30 mg/kg doses.

Continuous monitoring of the plasma clearance, pharmacokinetic profile and immunogenicity of the HIRMaB-IDS in the tested animals demonstrated that the new drug is safe and efficient for use in primates. This makes it suitable for future clinical trials.


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