ProSavin is an experimental drug believed to be of use in the treatment of Parkinson's disease. It is administered to the striatum in the brain, inducing production of dopamine.[1]
It is manufactured by Oxford BioMedica. Results from a Phase I/II clinical trial were published in the Lancet[2] and showed safety, but little efficacy.[3] ProSavin was superseded by AXO-Lenti-PD (OXB-102), an optimized version of the drug.[4]
Mechanism of action edit
Prosavin uses Oxford BioMedica's Lentivector delivery system to transfer three genes, aromatic amino acid dopa decarboxylase, tyrosine hydroxylase and GTP-cyclohydrolase 1, to the striatum in the brain, reprogramming transduced cells to secrete dopamine.[5][6]
See also edit
References edit
- ^ Oxford BioMedica. Drug Information Page. Retrieved on March 29, 2007.
- ^ Palfi, Stéphane; Gurruchaga, Jean Marc; Ralph, G. Scott; Lepetit, Helene; Lavisse, Sonia; Buttery, Philip C.; Watts, Colin; Miskin, James; Kelleher, Michelle; Deeley, Sarah; Iwamuro, Hirokazu (2014-03-29). "Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial". The Lancet. 383 (9923): 1138–1146. doi:10.1016/S0140-6736(13)61939-X. ISSN 0140-6736. PMID 24412048. S2CID 4993549.
- ^ Communications, Maggie Kuhl Director Research & CEO (28 January 2014). "ProSavin Trial Results: Once Again, a Gene Therapy Approach to Parkinson's Yields Encouraging Safety Data but Modest Efficacy | Parkinson's Disease". www.michaeljfox.org. Retrieved 2021-04-09.
- ^ Wexler, Marisa (20 August 2019). "Parkinson's Gene Therapy in Clinical Trial, AXO-Lenti-PD, Safe And Effective in Monkey Model of Disease, Study Says". Retrieved 2021-04-09.
- ^ "Positive results in Phase I/II trial". OxfordBiomedica. 19 November 2008.
- ^ "Prosavin". OxfordBiomedica. Archived from the original on 16 March 2014.
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