Secondary progressive multiple sclerosis, new treatment perspectives – WWN

Secondary progressive multiple sclerosis, new treatment perspectives – WWN


Of Maria Giovanna Faiella

Even though this is a phase 1 clinical trial, the results of brain stem cell transplantation in 15 patients are promising. Professor Vescovi: «Phase 2 of the study is underway»

New prospects for treatment with stem cells in patients with Multiple sclerosis
secondary progressive, an advanced form of the disease characterized by persistent disability that progresses gradually over time and which currently has no effective therapies. A phase 1 clinical trial demonstrated that the brain stem cell transplant the
n 15 patients is safe, very well tolerated and with possible long-lasting and protective effects from further brain damage, thus paving the way for phase 2 of the research. The study, coordinated by the IRCCS Casa Sollievo della Sofferenza Hospital of San Giovanni Rotondo and conceived by Professor Angelo Vescovi of the University of Milan – Bicocca, Scientific Director of the IRCCS itself, as well as President of the National Bioethics Committee, in collaboration with Professor Stefano Pluchino of the University of Cambridge, was published in the scientific journal Cell Stem Cell .

I study

The clinical trial of phase 1, to determine safety, feasibility and tolerability of the treatment, involved 15 patients with advanced secondary progressive multiple sclerosis, with high disability (in a wheelchair), who received the brain stem cell transplant directly into the lateral ventricles of the brain.
The cells used are free from ethical problems as they are isolated from fetuses who died of natural causes.
Furthermore, as Professor Vescovi clarifies: «In phase 1, for ethical reasons, patients are enrolled who do not have an alternative treatment and are in complex conditions, in the case of secondary progressive multiple sclerosis
there are no effective therapies and, above all, it is one very advanced stage of the diseasesince nerve fibers tend to die.”
The patients who participated in the study

they were then monitored for 12 months and, in this period, no deaths or serious adverse events due to treatment were recorded; the side effects were modest, temporary and in any case reversible. Furthermore, the patients they showed no increase in the degree of disability or worsening of symptomstherefore there was substantial stability of the pathology.

The comment

Even if it is a phase 1 study, the results give rise to hope, as Professor Vescovi reports: «The result is solid: we found in the patients treated a loss of brain volume, which leads us to hypothesize a reduction in inflammationand it is in line with what we had observed in animals, i.e. a shutdown of the inflammatory process.
The researchers measured, in the cerebrospinal fluid (which bathes the brain and its cavities) of transplanted patients, the variations in the levels of some molecules linked to the mechanisms through which the nervous tissue uses and metabolizes fatty acids, also to produce energy. It was observed that the greater the dose of stem cells transplanted, the greater the levels of fatty acids detected in the patients’ cerebrospinal fluid. We didn’t expect this increase in “energy production”, therefore in energy metabolism, which depended on the injection of stem cells and lasted 12 months (the observed period) – says Professor Vescovi -. While we are obviously cautious as this is phase 1 of the study, these results, associated with a clinical picture of patients did not worsen, give us hope for phase 2, also thanks to the availability of the stem cells already produced and stored in the GMP workshop (authorized by the Italian drug agency AIFA) of Casa Sollievo della Sofferenza, thanks to which – the only case in the world – the availability of the cellular drug will no longer be a problem. Today transplanting new brain cells into the patient’s brain, as if it were a drug, is one clinical reality» underlines the expert.

Single donor, why it’s important

Another relevant fact is that, dto a single donorit was possible to obtain a virtually unlimited number of these stem cells.
Professor Vescovi explains: «A huge problem in many studies is that, every time stem cell transplants are performed, they are from a new donor. Our technique allows di produce cells for thousands of patients
from a single donor . This means that in the subsequent phase 2 and 3 studies – but also in other trials – the same cells for treatments».
The AOSP Santa Maria di Terni, the University of Colorado and the Cantonal Hospital Authority based in Lugano, Switzerland, where the evaluation of the radiological images was centralized, also participated in the experiment. The study, partly supported by the CARIT Foundation (Terni), and supported by the Puglia Region, was started on the initiative of Monsignor Vincenzo Paglia, president of the Pontifical Academy for Life of the Vatican State.

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November 27, 2023 (changed November 27, 2023 | 7:30 pm)



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