Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I

for the Mucopolysaccharidosis I Intrathecal Research Collaborative

Research output: Contribution to journalArticle

21 Scopus citations

Abstract

Enzyme replacement therapy with laronidase (recombinant human alpha- l-iduronidase) is successfully used to treat patients with mucopolysaccharidosis type I (MPS I). However, the intravenously-administered enzyme is not expected to treat or prevent neurological deterioration. As MPS I patients suffer from spinal cord compression due in part to thickened spinal meninges, we undertook a phase I clinical trial of lumbar intrathecal laronidase in MPS I subjects age 8. years and older with symptomatic (primarily cervical) spinal cord compression. The study faced significant challenges, including a heterogeneous patient population, difficulty recruiting subjects despite an international collaborative effort, and an inability to include a placebo-controlled design due to ethical concerns. Nine serious adverse events occurred in the subjects. All subjects reported improvement in symptomatology and showed improved neurological examinations, but objective outcome measures did not demonstrate change. Despite limitations, we demonstrated the safety of this approach to treating neurological disease due to MPS I.

Original languageEnglish
Pages (from-to)69-74
Number of pages6
JournalMolecular genetics and metabolism
Volume116
Issue number1-2
DOIs
StatePublished - Sep 1 2015
Externally publishedYes

Keywords

  • Alpha-l-iduronidase
  • Enzyme replacement therapy
  • Hurler
  • Intrathecal
  • Lysosomal storage disease
  • Scheie

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