To date, despite numerous active efforts, there are no drugs available for the cure of the neurodegenerative diseases collectively referred as prion and prion-like disorders. Symptomatic treatment is the only available option, including the administration to patients of antipsychotics, such as quetiapine and clonazepam, to treat myoclonus, and of selective serotonin re-uptake inhibitors (SSRIs) to treat depression. Indeed, through this invention, we are presenting a new class of small molecules acting as SERPINA3 inhibitors, which are able to reduce PrPSc accumulation in prion-infected cell lines, thereby representing a new therapeutic treatment against prion and prion-like diseases. The development of specific inhibitors of SERPINA3 with an activity in the nanomolar range is expected.
The characterization of structural models of the binding of the small molecules with the protein should allow the identification of novel inhibitors with improved potency.
So far there are no effective therapies for prion diseases. The proposed technology aims at developing novel molecules with improved efficacy on these devastating diseases. The extended European search report, issued on 24th Novembre, 2018 by EPO, has already shown the complete novelty of the Technology proposed.
Compounds for use in the therapeutic treatment of a prion or prion-like disease (including Parkinson and Alzheimer's Diseases) and to pharmaceutical compositions comprising these compounds. The project is being developed at SISSA. Thus far the research team has characterized the system in vitro and partially in vivo. Further developments are expected in the coming months also depending on the identification of appropriate partnership.