For the first time, neural stem cells have been used on patients with an “advanced-stage progressive” form of the disease. Here are the results of the study by researchers at San Raffaele in Milan: however, further steps will be required before being able to consider it a therapy
A cell therapy based on neural stem cells opens up new perspectives for the treatment of advanced stage progressive multiple sclerosis.
This is indicated by the results, published in the journal Nature Medicine of the STEMS study, which began in 2017 and coordinated by Gianvito Martino, scientific director of the San Raffaele Hospital in Milan.
The treatment consists of ainfusion of these cells via lumbar puncture into the cerebrospinal fluid. Animal models had already demonstrated the ability of neural stem cells to be able to reach, once infused, brain and spinal cord lesions and promote neuroprotection and repair mechanisms, releasing immunomodulatory and pro-regenerative substances.
However, the study in question for the first time, it has made it possible to evaluate the effects on humans.
I study
The trial involved 12 people with progressive multiple sclerosis and high disability who had already received, without satisfactory results, the treatments currently available.
The patients, divided into 4 groups, received, with a single lumbar puncture, an increasing number of cells, from about 50 million to 500 million.
«The results demonstrated, in addition to the safety and tolerability of the therapy, a significant reduction in brain tissue lossevaluated by magnetic resonance in the two years following the treatment (the first occurred in 2017), in the group that received the largest number of neural stem cells.
In confirmation of the finding, the analysis of the cerebrospinal fluid showed a change in its composition demonstrating a enrichment in terms of growth factors and neuroprotective substancesexplains Angela Genchi, researcher of the Neuroimmunology laboratory and first author of the publication.
The scientific foundations
Neural stem cells had never been used before in patients with multiple sclerosis and, unlike the hematopoietic ones (used in relapsing-remitting forms of the disease but ineffective in the progressive forms) and the mesenchymal ones (which did not give benefits in patients with progressive multiple sclerosis), had shown in the preclinical studies conducted in the laboratory that they could have a high pro-regenerative potential. The scientific foundations of this trial were in fact laid by a series of works by the group directed by Gianvito Martino which, between 2003 and 2009, demonstrated the efficacy of neural stem cells in several experimental models of multiple sclerosis. «The one achieved is an important goal, even if it represents only the first stage of the clinical-experimental path that leads to a real therapy. My first thought goes, above all, to the sick people and their families who have supported our research in all these years, certainly dramatic from the point of view of public health, with patience, hope, dedication and sacrifice. We wouldn’t have gotten this far without their contributions.”
It’s not a therapy yet: the next steps
“However, the road taken is still long”, underlines Professor Martino. «The published data are not yet sufficient to consider this opportunity, in fact, as a real therapy, the next step will be to proceed with a new experimental clinical study involving a larger group of patients, with the aim of demonstrating, on the one hand, the efficacy of neural stem cells in blocking the progression of the disease, on the other hand, their ability to promote the regeneration of damaged areas of the nervous system. The ultimate goal, which is the great challenge we decided to face 20 years ago, is to develop an innovative and effective therapy for people with progressive forms of MS who have, to date, limited therapeutic options”.
The cells used for the transplant were of fetal origin and were prepared in collaboration with the Stefano Verri Cell Therapy Laboratory, supported by the Matilde Tettamanti and Menotti De Marchi Onlus Foundation and with the IRCCS Ca’ Granda Ospedale Maggiore Policlinico Foundation. The research was conducted thanks to the support of various partners, in particular the Italian Multiple Sclerosis Association (AISM) and its Foundation (FISM), and of the Cariplo Foundation, the Friends of the Multiple Sclerosis Center Association (ACeSM), BMW Italy, the Maria Committee Letizia Verga Onlus.
January 9, 2023 (change January 9, 2023 | 5:18 pm)
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