Hematologists from researching major advances to cure leukemia

Research has opened up new avenues for the treatment of blood cancers. This is what emerged from the presentation of the sixteenth ‘National Day for the Fight against Leukemia, Lymphoma and Myeloma’ on 21 June, presented today by AIL. “The research – explains Alessandro Vannucchi, professor of Hematology at the University of Florence, and head of the Crimm, Research and Innovation Center for myeloproliferative diseases – has made it possible to make significant progress in various areas: increasing awareness of the disease, improving of diagnostic approaches using the discoveries of gene mutations (such as Jak2, Mpl and Carl, and many others) and the development of risk models that allow to identify the most serious cases requiring stem cell transplantation. of genes associated with the disease to favor the development of drugs, the Jak2 inhibitors of which the parent is ruxolitinib, and a second drug, fedratinib, has recently been approved. These therapies – underlines Vannucchi – have been shown to reduce, up to normalize, the volume of the spleen and get to the total regression of symptoms; the quality of life has improved greatly and they begin to have solid scientific evidence also on the favorable impact on lengthening life “.” In the last 20 years the greatest results – explains Marco Vignetti, president of the Gimema Franco Mandelli Foundation and national vice president of Ail – are linked to the development of targeted drugs , target therapy. They are drugs that derive from years of research that have made it possible to discover genetic mutations of the diseased cell that caused the disease. Some blood cells in the multiplication activity make a mistake that is not recognized in time and eliminated. This leads to the creation of groups of identical cells, the cell clones, which cause the disease which is usually characterized by an uncontrolled proliferation of cells “.” Research – Vignetti points out – has identified the mutation and started to create drugs that only affect the mutation, sparing the other cells. This has led to great results, one for all being chronic myeloid leukemia (CML) therapy. Today, Lmc is considered treatable, with a good quality of life and with a duration comparable to that of a healthy person. Although with fewer results – adds Vignetti – ‘target’ drugs have also been developed in essential thrombocythemia and polycythemia vera, which are used for example to reduce the size of the spleen. They are not decisive, but they can be of great help in case allogeneic transplantation is necessary, as the patient is in much better conditions to be able to deal with it “.” Myeloproliferative neoplasms, rare tumors affecting the bone marrow such as chronic myeloid leukemia , polycythemia vera, essential thrombocythemia and myelofibrosis – explains the president of Ail, Sergio Amadori – are chronic indolent diseases, difficult diagnoses often occur by chance and treatments thus begin with years of delay and therefore risks for patients “.” Today, the knowledge of the genetic basis of MPN – underlines Amadori – has made it possible to develop molecules capable of specifically inhibiting the action of the genes responsible for the disease, paving the way for a new treatment approach based on molecular diagnostics. These patients have a better chance of controlling the disease even in the long term “.