Cystamine and analogues for the treatment of Parkinson's disease
Parkinson’s disease is an important neurodegenerative disorder for which there is currently no treatment that can halt or reverse degenerative processes. In fact, medical interventions and therapeutic options available to patients can, at best, manage certain clinical symptoms, and new therapeutic strategies are critically needed.
In the last decade, Prof Cicchetti and colleagues have accumulated compelling evidence that cystamine (and its metabolite cysteamine), a molecule with anti-inflammatory and anti-apoptotic properties, has potent effects against various aspects of Parkinson's disease. The research team at Université Laval has shown that cystamine can cross the blood-brain-barrier, a feature which carries enormous clinical implications given the rarity of drugs with such properties and hence, can prevent cell death in part by increasing the levels of brain derived neurotrophic factors (BDNF). The neuroprotective effect of cystamine itself, and its reduced metabolite cysteamine, can halt and reverse ongoing neurodegenerative processes in toxin-induced animal models and therefore could offer the first disease-modifying therapeutic option for Parkinson's disease. Importantly, cystamine has previously been reported to inhibit tranglutaminase, an enzyme contributing to the formation of insoluble protein aggregates that have been observed in the brains of patients with Parkinson's, as well as Alzheimer's and Huntington’s diseases.
Cysteamine bitartrate is currently marketed in Europe and in the United States for the treatment of nephropathic cystinosis under the trade name Cystagon®. However, patient compliance with orally-administered Cystagon® is sometimes challenging due to gastrointestinal adverse effects.
To pursue the search for a novel therapeutic option for Parkinson's disease, SOVAR and Université Laval are seeking for a partner for chemical derivation of cystamine/cysteamine, co-development of lead candidate(s) or formulation(s) better tolerated by mice (for in vivo evaluation) and humans, licensing, or commercialization of this technology.
The laboratory of Prof Cicchetti is fully capable of performing in vitro, in vivo (in mice and rats), and neurocomportmental evaluation of compounds in animal models of Parkinson's disease. They have also been involved in a number of clinical studies analyzing blood samples and post-mortem brain tissue.