An important goal, all Italian. It is the one achieved with the publication of the results of the study – the first in the world – conducted by transplanting neuronal stem cells into people with multiple sclerosis, the precursors of those that the disease damages. The results, in addition to confirming the safety of the treatment, show a reduction in brain atrophy in patients treated with the highest number of neural stem cells and a change in the CSF profile in a pro-regenerative sense after treatment. In other words, stem cells could help counteract the disease by protecting nervous tissue, and could also play an anti-inflammatory and supportive role in the nervous system of these patients.
Multiple sclerosis: first study with neural stem cells at the start
by LETIZIA GABAGLIO
Neuronal stem cells to protect nervous tissue
If it is true that the one presented today on the pages of Nature Medicine is a pioneering study of neuronal stem cells against multiple sclerosis – also supported thanks to funds made available by the Italian Multiple Sclerosis Association (AISM), through its foundation, FISM – research in the field is at least twenty years old. Twenty years in which preclinical studies on animal models, first mice and then monkeys, have indicated that neuronal stem cells, once transplanted into the host, help the tissue to self-regenerate, as he tells Salute Gianvito Martino, scientific director of the IRCCS San Raffaele Hospital in Milan and head of the study. “These cells do not help replace the lost ones – as the paradigm of the first research on stem cells wanted – because once transplanted they do not differentiate into neurons, but produce neuroprotective substances”.
Multiple sclerosis, from stem cells a hope to delay disability
by Anna Lisa Bonfranceschi
After the encouraging results in animal models, a few years ago Martino’s team started a clinical study to understand if this new mode of action, combined with the innovative delivery mechanism – never before had neuronal cells been inoculated into the cerebrospinal fluid – could also work in humans, on a small sample of patients. Twelve in all those enrolled since 2017 in the STEMS (neural STEM cells in Multiple Sclerosis) study, all with progressive forms of the disease, with walking disorders, who received neuronal stem transplants at different doses. “The first objective of the study was to understand whether the procedure was safe and well tolerated in patients, and so it was – continues the expert – after a few months we collected samples of cerebrospinal fluid and observed the presence of trophic factors, neuroprotective and anti-inflammatory”. But not only that: patients who received higher doses of cells observed a lower rate of brain atrophy, after two years. “This is a fundamental achievement, which confirms what has been observed in animal models and which gives us reason to continue on this path”.
A new therapeutic strategy on the horizon
The idea now is to understand whether, as observed in animal models, following transplantation both the neurons and the cells that produce myelin – the target of the uncontrolled response in multiple sclerosis – work better, producing clinically observable results. The concept of protection is in fact vast when it comes to the nervous system, explains Martino, and has to do with the idea of supporting, helping to repair damaged tissue. “Neurons don’t necessarily have to die to not function at their best, they can only be damaged. In this sense, the presence of trophic factors can give them the necessary support to restore their function, recover them”. In fact, self-regenerating them. “If future studies demonstrate their therapeutic potential, neuronal stem cells could be used both to act on the inflammatory aspects linked to the disease and to promote neuroprotection”, imagines Martino: “Not necessarily only in the progressive forms, even if these are those in which we need more therapeutic solutions”. And where much of the research today on multiple sclerosis is concentrated, not only with the search for new therapies, as he adds Albert Battaglia, president of the Italian Multiple Sclerosis Foundation (FISM): “In the last twenty-five years a lot has changed in multiple sclerosis, a lot is also being done for the progressive forms. The most serious people have innovative answers to deal with symptoms and gain quality of life, including through rehabilitation. We will continue to develop this research with stem cells and other innovative projects to move towards a world free from multiple sclerosis”.