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Neurobiol Dis. 2019 Oct;130:104530. doi: 10.1016/j.nbd.2019.104530. Epub 2019 Jul 10.

Cysteamine as a novel disease-modifying compound for Parkinson's disease: Over a decade of research supporting a clinical trial.

Author information

1
Département de Psychiatrie & Neurosciences, Faculté de Médecine, Université Laval, Québec G1V 0A6, QC, Canada; Centre de Recherche du CHU de Québec, Québec G1V 4G2, QC, Canada. Electronic address: francesca.cicchetti@crchudequebec.ulaval.ca.
2
Département de Psychiatrie & Neurosciences, Faculté de Médecine, Université Laval, Québec G1V 0A6, QC, Canada; Centre de Recherche du CHU de Québec, Québec G1V 4G2, QC, Canada.

Abstract

To date, medical and surgical interventions offered to patients with Parkinson's disease (PD) serve only to manage clinical symptoms; they have not shown the capacity to halt nor reverse degenerative processes. There is therefore an urgent need to identify and/or develop therapeutic strategies that will demonstrate 'disease modifying' capacities. The molecule cystamine, and its reduced form cysteamine, act via a number of pathways determined to be critical to the pathogenesis of PD. In particular, cystamine is capable of crossing the blood-brain barrier, and both agents (cystamine and cysteamine) can promote the secretion of neurotrophic factors, inhibit oxidative stress, reduce inflammatory responses and importantly, have already been trialed in humans for a number of other clinical indications. In the last decade, our laboratory has accumulated compelling evidence that both cystamine and cysteamine can halt, and even reverse, ongoing neurodegenerative processes in a number of different models of PD, and as such, should now be taken forward to clinical trials in PD.

PMID:
31301344
DOI:
10.1016/j.nbd.2019.104530

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