| Autor: |
Smith RA; Center for Neurologic Study, San Diego, CA 92121, USA., Balis FM, Ott KH, Elsberry DD, Sherman MR, Saifer MG |
| Jazyk: |
angličtina |
| Zdroj: |
Journal of the neurological sciences [J Neurol Sci] 1995 May; Vol. 129 Suppl, pp. 13-8. |
| DOI: |
10.1016/0022-510x(95)00051-3 |
| Abstrakt: |
The discovery of mutations in the gene for Cu/Zn superoxide dismutase (SOD) in some cases of familial amyotrophic lateral sclerosis (FALS) provides a rationale for enzyme replacement therapy. The inability of SOD to cross the blood-brain barrier motivated this study of the safety, tolerability and pharmacokinetics of bovine SOD (bSOD) administered into the CSF of rhesus monkeys and one late-stage, SOD-deficient FALS patient. Kinetic analyses in the patient indicated that intracerebroventricular (i.c.v.) administration, but not lumbar administration, delivered bSOD to the entire CSF pathway. Daily bolus i.c.v. injections (32 mg/day) and continuous i.c.v. infusion (30 mg/day) were well tolerated by the patient. During the period of daily bolus injections, the patient's performance on manual muscle tests was nearly stable, in contrast with the rapid decline before and after that period. These results justify further investigation of bSOD therapy in SOD-deficient FALS patients. |
| Databáze: |
MEDLINE |
| Externí odkaz: |
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