A new therapeutic approach has been successfully tested for the first time in the world: it involves the infusion of healthy liver stem cells in infants suffering from severe genetic metabolic disorders.
The new therapeutic strategy – a sort of non–invasive “mini–transplantation” – was conducted in Italy, at the Regina Margherita Hospital in Turin, by the team of Dr. Marco Spada, Director of Paediatrics and Regional Centre for the Treatment of Metabolic Diseases, in collaboration with other healthcare professionals and institutes involved in the experimentation.
The study concerns «infants born with serious genetic diseases – they generally lack a protein for metabolism – and who risk neonatal coma: for this reason they must be stabilized in the first days of life and then have to undergo a liver transplant, which can be performed not before the sixth month», explains Dr. Spada. Three newborn babies have already undergone the treatment successfully: «Through this trial, which took place before the 3rd month of life, we were able to stabilize them. And the transplant can take place when they are older,» continues Dr. Spada.
The study was published in the renowned journal Stem Cell Reviews and Reports, and reports on the excellent results obtained in Phase I of the procedure, infusing healthy liver stem cells directly into the liver of the three newborns using an ultrasound-guided needle.
The metabolic diseases that affect newborns, putting their life and growth at risk, are about three hundred. For this reason, clinicians plan to continue with the testing of this new therapy and to start the Phase II study, which will allow to involve other facilities and healthcare professionals throughout Europe and, above all, to assess the effects of stem cell treatment on a larger sample of patients.
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