The fight against neurological diseases is moving ahead

Print Pdf


NEW STUDY DISCOVERS HOW TO OBTAIN HUMAN BRAIN STEM CELLS FROM THE SKIN. The research was published in the scientific journal Cell Death and Disease

Rome, September 24, 2018 – the IRCCS Casa Sollievo della Sofferenza and the Italian non-profit association Revert Onlus, which supports pre-clinical and clinical research on brain stem cells and is the Italian benchmark for the development of innovative therapies for genetic and neurodegenerative diseases, announce the publication of a study which demonstrates how, by starting from a skin sample, it is possible to obtain a fundamental tool in the fight against brain diseases: human brain stem cells suitable for auto-transplant. The research was conducted at the IRCCS Casa Sollievo della Sofferenza by Dr. Jessica Rosati and Prof. Angelo Luigi Vescovi, with the collaboration of the Fondazione Cellule Staminali [Stem Cell Foundation] in Terni and the Bicocca University of Milan.

The study, entitled: “Establishment of stable iPS-derived human neural stem cell lines suitable for cell therapieswas published in the prestigious scientific journal Cell Death and Disease, part of the series Springer Nature.

The project solves the long-standing problem of identifying an easily accessible source of human brain stem cells for the treatment of neurological diseases. This approach replaces the use of cerebral cells of fetal origin and solves the problem of post-transplant rejection. The results obtained have shown that it is possible to produce, directly from the skin of a patient, brain stem cells identical to the clinical-grade fetal cerebral stem cells, certified by AIFA and currently used by Prof. Vescovi's team in phase I and II trials on patients with amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS).

This study thus describes a technology immediately applicable in clinical trials, thanks to which skin cells are extracted by means of a simple skin biopsy, then reprogrammed and rendered embryonic-like (induced pluripotent stem or iPS cells) using the method that earned Prof. Shinya Yamanaka the Nobel Prize.

Thanks to the discovery reported here it is thus possible to:

-          Expand and store in a cell bank, reprogrammed human cells for each individual patient, with a method that does not alter cellular genetic material and does not use viruses or dangerous molecules. These cells are kept for decades and remain available to the donor at all times.

-          Differentiate the reprogrammed cells into brain stem cells, using the same technique certified for clinical use, currently applied for fetal cerebral stem cells used in completed or ongoing clinical trials on ALS and MS.

-          Certify the entire process in accordance with the European Medicines Agency (EMA/AIFA) and Food and Drug Administration (FDA) regulations and use them for clinical transplantation in humans, generating all types of brain cells.

-          As cells are transplanted into the patient from whom they are initially taken as skin, this is an out-and-out autologous transplant that avoids the use of immunosuppressant drugs since the risk of rejection is, in fact, zero.

The therapeutic development potential of these new cells is, therefore, enormous. There is now an unlimited number of human, autologous brain cells reproducible both in their production process and therapeutic effects, unimaginable until a short time ago. To date, the application of cell therapy in neurodegenerative and neurological fields has been seriously limited by at least 3 main factors: 1) the limited availability of cells; 2) the poor reproducibility of the methods used to produce them; 3) the rejection due to the allogeneic nature of brain fetal cells. The publication of this paper obviates these problems and will make it possible to extend the application of cellular therapy for brain diseases to cohorts of patients and pathologies much broader than possible up till now.

The Revert Onlus Association, founded by Monsignor Vincenzo Paglia, current President of the Pontifical Academy for Life, was founded in 2003 with the aim finding a cure for neurodegenerative diseases which, at the moment, leave no room for hope in a cure. For this purpose, it has undertaken to fund, promote and encourage research on brain stem cells and clinical trials on humans.

Since July 1991, the Ospedale Casa Sollievo della Sofferenza has been recognized by the Ministry of Health as a Scientific Institute for Research, Hospitalization and Health Care (IRCCS). The original decree for "genetic and hereditary-family diseases” was extended in 2012 to also recognize "genetic diseases, innovative therapies and regenerative medicine”. The clinical and preclinical research of the Institute is therefore mainly focused within this category.

Revert Onlus Administration Office -