Prof. Sandro Sonnino, University of Milan; Dr Elena Chiricozzi, University of Milan
Counteract the motor symptomatology typical of Parkinson's disease and consequentially improving patients' clinical conditions and quality of life
The invention relates to a small molecule (having an oligosaccharide structure) able to pass the blood-brain barrier remaining unchanged. Data reported in the text shows that the molecule has, as main effect, the biochemical and functional recovery of dopaminergic neurons (in vitro test results). The administration of the oligosaccharide to Parkinson mouse model allowed the observation of the repair of neurons and of a consequent reduction in the motor symptoms typical of Parkinson's disease (data obtained in vivo). According to its activity of neuron damage repair, this oligosaccharide could be used for reducing the symptomatology of several neurodegenerative diseases, for example in the treatment of Alzheimer's disease and Multiple Sclerosis or in reducing the symptoms related to the aging process, improving the quality of life of people affected by this kind of pathologies or of the elderly.
The technology is available for Joint Development Agreement, Licensing or Ownership Assignment agreements.
The molecule efficacy tests have been made in vivo (on genetic Parkinson mouse model, recapitulating the human sporadic Parkinson’s disease). Pharmacokinetic information (tissues/organs distribution, doses, metabolic stability, routes of administration, time points) have been obtained in vivo (using wild type mice) Blood brain barrier penetrability has been characterized using an in vitro model of human brain like endothelial cells. Pharmacology safety has been done in vivo using mouse embryo culture methods.
Italian patent application filed on July 2018.
1. Chiricozzi E, et al. (2017) Role of the GM1 ganglioside oligosaccharide portion in the TrkA-dependent neurite sprouting in neuroblastoma cells J. Neurochem 143:645-659
2. Lunghi G, et al. (2018) GM1 neuroprotective properties are related to GM1 oligosaccharide FEBS Open Bio, 8:14-018 doi:10.1002/2211-5463.12453
3. Maggioni M, et al. (2018) Charaterization of GM1 oligosaccharide transport across the blood-brain-barrier FEBS Open Bio, 8:14-017 doi:10.1002/2211-5463.12453 4. Chiricozzi E, et al. (2019) The neuroprotective role of the GM1 oligosaccharide, II3Neu5Ac-Gg4 in neuroblastoma cells Mol. Neurobiol submitted January 2019