Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
| Status: | Archived |
|---|---|
| Conditions: | Orthopedic, Endocrine, Metabolic |
| Therapuetic Areas: | Endocrinology, Pharmacology / Toxicology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 7/1/2011 |
A Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I
The investigators are studying the use of enzyme replacement therapy into the spinal fluid
for treatment of spinal cord compression in the Hurler-Scheie and Scheie forms of
mucopolysaccharidosis I (MPS I).
Enzyme replacement therapy (ERT) has been developed for mucopolysaccharidosis I (MPS I), a
lysosomal storage disorder. ERT helps many physical ailments due to the disease, but does
not treat the central nervous system, due to inability to cross the blood brain barrier.
Our purpose is to test delivery of ERT to the spinal fluid via intrathecal injection in
patients with MPS I. In this pilot study, we will use recombinant human α-L-iduronidase
administered intrathecally once per month for four months to individuals with the
Hurler-Scheie and Scheie forms of MPS I and spinal cord compression. If successful,
intrathecal delivery could represent a practical, straightforward method of treating central
nervous system disease due to lysosomal storage.
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