NO relapse and no new lesions in any of the patients. These are the encouraging results coming from a study on the efficacy of hematopoietic stem cell transplantation in patients with multiple sclerosis. A confirmation of their effectiveness, yes, but above all of the importance of selecting well the patients suitable for receiving this treatment. Because stem cells work, with effects that are maintained even in the long term, but they cannot be a therapy for everyone.
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Stem cell transplantation
Matilde Inglese, professor of Neurology at the University of Genoa and head of the Multiple Sclerosis Center, reiterated this, commenting on the study data presented during the 2022 Forum of the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) in Florida – Irccs San Martino Hospital. Inglese has been studying the role of hematopoietic stem cell transplantation in this disease for some time: “Even if it is a treatment born for some forms of cancer, it has been applied in the field of autoimmune diseases since the mid-nineties”, she remembers. And since then doctors and researchers have put together a series of data to be able to understand which patients can benefit most from it. “Today we know that we cannot offer it to everyone, because hematopoietic stem cell transplantation has a challenging risk profile, especially due to the chemotherapy that is administered to patients, with risks of infection and even death in some cases, although we have managed to significantly lower mortality over the years “.
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The results of the study
In autologous hematopoietic stem cell transplantation, in fact, the cells are taken from the patient himself and then re-infused, after a chemotherapy treatment to eliminate the crazed cells that support the inflammation and the abnormal immune response. The transplanted stem cells can thus contribute to forming a new, more tolerant defense system. Those presented in Florida are the results related to its use in about seventy patients with multiple sclerosis (most with relapsing-remitting forms) followed in Ottawa, Canada, with a follow-up ranging from a few months up to twenty years. The results, as anticipated, confirm its effectiveness: no relapses or new lesions observed, and no need to resort to other drugs. But there is more: the best results were observed for patients with a lower degree of disability, confirming how important it is to select patients for stem cell transplants, so as to mitigate the risks and maximize the benefits of therapy. , points out the researcher. Which reminds the importance of the center that administers the treatment: it must be a qualified and experienced blood center.
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The risks
“We are talking about a therapy that is certainly not routine – explains Inglese – but practiced only in some cases, such as those of patients with aggressive disease, that is to say with very high inflammation activity, with new lesions and new clinical relapses and which do not improve with approved highly effective therapies. The research presented in Florida is in line with what has been observed in recent times, also in our study published in Neurology on the long-term efficacy of hematopoietic stem cell transplants ”. The risk profile was also confirmed, with a mortality below 3% in the Canadian study (and even lower in the English and colleagues study).
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The next study
What is rather lacking now, the researcher goes on, are the results from controlled studies, in which transplantation is compared with drug therapies currently considered to be highly effective. “This is what we will do in the Net-MS multicenter study, funded by the Italian Multiple Sclerosis Foundation, on patients in whom the first line of treatment has failed”. The study will hopefully start within the year, after the delays accumulated due to the pandemic, concludes Inglese.