Intranasal ERT in LSDs may bypass BBB

Posted by & filed under Part 16: LYSOSOMAL DISORDERS, Part 30: MULTISYSTEM INBORN ERRORS OF DEVELOPMENT, Treatment.

According to the study by Wolf et al. intranasal administration of α-l-iduronidase (IDUA) enzyme may be able to bypass the blood-brain barrier. This appears to be the case in IDUA-deficient mice after a single intranasal treatment with concentrated Aldurazyme®. Promising results in mice were also noted after intranasal treatment with an adeno-associated virus (AAV) vector expressing human IDUA. These experiments raise the possibility of non-invasive treatments for lysosomal storage disorders.

Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration. Wolf et al. Mol Genet Metab. 2012 May;106(1):131-4. PMID: 22420937 http://dx.doi.org/10.1016/j.ymgme.2012.02.006

posted by Yannis Trakadis MD, MSc

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