Previous open-label trials testing glial cell line-derived neurotrophic factor (GDNF) family ligands in Parkinson disease have shown promising clinical effects. However, in placebo-controlled trials, the treatments have failed. A new randomized placebo-controlled trial of intraputamenal delivery of GDNF designed to resolve this conundrum has again failed to do just that.
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References
Evans, J. R. & Barker, R. A. Neurotrophic factors as a therapeutic target for Parkinson’s disease. Expert Opin. Ther. Targets 12, 437–447 (2008).
Kordower, J. H. In vivo gene delivery of glial cell line-derived neurotrophic factor for Parkinson’s disease. Ann. Neurol. 53 (Suppl. 3), 120–132 (2003).
Lang, A. E. et al. Randomized controlled trial of intraputamenal glial cell line-derived neurotrophic factor infusion in Parkinson disease. Ann. Neurol. 59, 459–466 (2006).
Lang, A. E. et al. GDNF in treatment of Parkinson’s disease: response to editorial. Lancet Neurol. 5, 200–202 (2006).
Warren Olanow, C. et al. Gene delivery of neurturin to putamen and substantia nigra in Parkinson disease: a double-blind, randomized, controlled trial. Ann. Neurol. 78, 248–257 (2015).
Whone, A. et al. Randomized trial of intermittent intraputamenal glial cell line-derived neurotrophic factor in Parkinson’s disease. Brain 142, 512–525 (2019).
Whone, A. L. et al. Extended treatment with glial cell line-derived neurotrophic factor in Parkinson’s disease. J. Parkinsons Dis. https://doi.org/10.3233/JPD-191576 (2019).
Decressac, M. et al. α-Synuclein-induced down-regulation of Nurr1 disrupts GDNF signaling in nigral dopamine neurons. Sci. Transl Med. 4, 163ra156 (2012).
Patel, N. K. et al. Benefits of putaminal GDNF infusion in Parkinson disease are maintained after GDNF cessation. Neurology 81, 1176–1178 (2013).
Kordower, J. H. et al. Disease duration and the integrity of the nigrostriatal system in Parkinson’s disease. Brain 136, 2419–2431 (2013).
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A.K. is the owner of the company Kirkeby Cell Therapy holding patents for the development of cell therapies for Parkinson disease (PD). A.K. and R.A.B. are consultants for Novo Nordisk A/S on the development of cell therapies for PD. R.A.B. also works as a consultant on cell-based therapies for Fujifilm Cellular Dynamics, Cellino and Living Cell Technologies.
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The Parkinson’s Drug Trial: A Miracle Cure?: https://www.bbc.co.uk/programmes/m00031cb
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Kirkeby, A., Barker, R.A. Parkinson disease and growth factors — is GDNF good enough?. Nat Rev Neurol 15, 312–314 (2019). https://doi.org/10.1038/s41582-019-0180-6
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DOI: https://doi.org/10.1038/s41582-019-0180-6
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