The pioneering method was developed by Italian researchers from Perugia University and Casa Sollievo della Sofferenza Research Institute.
The experimental basis for an innovative therapeutic approach to Huntington disease, published in the international scientific journal CNS Neuroscience & Therapeutics, opens new perspectives to the treatment of this rare, neurodegenerative, hereditary disease, thanks to a team of Italian researchers, coordinated by Giovanni Luca and Riccardo Calafiore, School of Medicine, University of Perugia and Ferdinando Squitieri, Head of the Huntington and Rare Diseases Unit at Casa Sollievo della Sofferenza Research Hospital and Mendel Institute of Human Genetics in Rome.
"The research was conducted on animal models: it opens the door to future innovative therapeutic approaches, with potential future application to patients” says Luigi Frati, Scientific Director of Neuromed in Pozzilli.
Sertoli cells from pig's testes exert immunomodulatory, anti-inflammatory and trophic properties. Usually, these cells show nutritive and protective physiological functions in spermatogenesis. Once obtained from pigs, they have been included in microscopic capsules, under strict protection from infectious agents.
“This is a very exciting strategy”, Ferdinando Squitieri states, “because such sodium alginate microscopic capsules have already been approved for human use”.
The microencapsulated cells were then injected into the Huntington mice belly, within the peritoneum, a strategy largely less invasive than in the nervous system. The result was the improvement of the clinical condition of treated mice that showed a significant extension of their life span, together with the reduction in the levels of pro-inflammatory molecules in the brain. "The microcapsules containing Sertoli cells - the researchers state - act as a biological microbrewery that from the peritoneal cavity releases factors which, by a systemic route, can reach the sick parts, without the need for local injections in the nervous system and without requiring pharmacological immunosuppression. Two great advantages that make particularly promising the treatment protocol”.
The application of tissues transplantated from animals bred in special conditions, such as pigs, to humans could be an achievable and not so far away goal. "Casa Sollievo della Sofferenza (CSS) Research Hospital mission is to combat hereditary diseases also with regenerative medicine and innovative therapies," Angelo Vescovi, CSS Scientific Director, states. “The results of this research, along with those recently obtained for other devastating neurodegenerative diseases, such as ALS, may open new important scenarios in the treatment of these severe – and up to now uncurable – diseases”.
In the picture: the microcapsules containing Sertoli cells used in this study. The microscopic capsules have a size of about 0.5 mm in diameter .