Fondazione REVERT ONLUS

Brain stem cell transplantation represents one of the potentially effective therapies against neurodegenerative diseases such as ALS.

Prof. Angelo Vescovi with his research team has developed a technique of isolation and culture of human brain stem cells coming exclusively from fetal tissue taken from fetuses that died of natural causes following the same authorization and certification procedure of the donation. voluntary organ. The cells are produced under GMP (Good Manufacturing Practice) according to the Italian and European legislation, therefore they are in all respects a drug and can also be used in the clinical setting.

Numerous preclinical studies have been conducted to verify and validate the therapeutic potential of these cells. The researches developed using an animal model of ALS (SOD-1 transgenic rats) have shown in particular that, following the intraspinal administration of brain stem cells, there is an improvement in the course of the disease, a slowdown in the functional loss of motor neurons and a increased survival of animals. These favorable symptomatic outcomes are accompanied by a delay in the accumulation of histopathological markers of ALS. In fact, in treated rats, there is a reduction in the inflammatory process at the level of the anterior horns of the spinal cord, site of cell transplantation, attributable to a pleiotropic mode of action by the cells themselves. The results obtained thus paved the way for clinical experimentation and a Phase I clinical trial was in fact completed.

Thanks to the support of the Revert Onlus Foundation, 18 patients with ALS underwent treatment with brain stem cells. During the trial, no adverse events emerged, thus confirming the feasibility and safety of stem cell transplantation.

To investigate the therapeutic potential of these cells, an experimental protocol is now being designed to access Phase II of the clinical trial.