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Blood cancers, the most common and what are the new treatments

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Seven out of ten people, considering all possible ones oncohematological diseases, survive or have fully recovered ten years after diagnosis. But science doesn’t stop. It is always necessary to find new solutions to deal with these pathologies, especially in the most serious forms, through successive lines of treatment.

What is happening? The experts are gathered in Milan, for the National Congress of the Italian Society of Hematology (SIE) precisely to design the future.

Identikit of enemies

Tumors of the blood more frequent are lymphomas, leukemias and multiple myeloma, which each year in Italy register about 30 thousand new cases, of which over 13 thousand are non-Hodgkin’s lymphomas – explains Paolo Corradini, President of SIE -. The ‘world‘ of hematology has changed profoundly in the last two years, today we enter the post covid era. And the search for our country opens up new paths.

An all-Italian study of 190 patients with aggressive non-Hodgkin’s lymphoma showed that 40% recover thanks to cell therapy with CAR T. A result obtained with a single infusion, therefore without the need for maintenance therapy, in people without any therapeutic alternative. CAR T’s are based on the patient’s genetically engineered lymphocytes. The procedure is complex and includes various phases: from the collection of T lymphocytes from the blood, to their engineering and expansion in the laboratory, up to their reinfusion into the patient ”.

“Thanks to this approach – continues Corradini -, significant results are being highlighted in people who have exhausted all therapeutic alternatives even in multiple myeloma. The disease leads to 5,800 new cases in Italy every year. In the KarMMa study of 128 heavily pretreated patients, the overall response rate reached 73%. Median overall survival exceeded two years. Very important results, if we consider that for these patients with multiple myeloma without alternative treatment, before the arrival of CAR T, the median life expectancy was between 6 and 9 months “.

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Warning: this does not mean that “trained” lymphocytes are the only weapon to try to give strength to the immune system of the organism committed to fighting the tumor. Just think of bispecific antibodies, which are revolutionizing the treatment of acute lymphoblastic leukemia, a rare cancer of lymphocytes, which causes almost 800 new diagnoses every year in our country.

“However, it is the most frequent in the pediatric age: it represents 80% of leukemias and about 25% of all cancers in under 14s – explains Pier Luigi Zinzani, President of the Training Activities Commission of the SIE -. The incidence peaks between the ages of 2 and 5 and then declines with increasing age. Two Italian studies can change the history of the disease.

In the first, in 149 patients, thanks to the combination of the chemotherapy sequence with a bispecific antibody, a complete response of 90% was shown and the molecular remission increased from 73% to 96% after the addition of the antibody. This last parameter indicates the impossibility of visualizing a minimal amount of diseased cells with a molecular test.

The second study demonstrates that it is possible to treat the disease without chemotherapy by combining targeted therapy with bispecific antibody. Remission was achieved in 98% of cases. The bispecific antibody is the basis of a new treatment principle, an innovative form of immunotherapy: it creates a ‘bridge’ between two different proteins, the receptor expressed on the surface of T cells and the one on the surface of B cells. In this way the immune system is stimulated to recognize cancer cells and fight them “.

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The future in the genes

Gene therapy also opens up new fronts in beta thalassemia. “In Italy, one of the most affected countries in the world, there are about 7,000 patients with this genetic, hereditary disease, caused by a defect in the production of hemoglobin, the protein responsible for transporting oxygen throughout the body – underlines Emanuele Angelucci, Vice President of SIE -.

They are forced to undergo regular blood transfusions at intervals of 2-3 weeks throughout their life. And they take iron chelation therapy every day, to limit the accumulation of iron in organs such as the heart, liver and pancreas.

An international study, presented by Franco Locatelli, President of the Superior Health Council, showed that gene therapy is able to obtain independence from transfusion needs in these patients. In this study, transfusion independence was achieved in 35 of 44 patients undergoing gene therapy.

Before gene therapy becomes really available, various aspects will have to be defined, including the problem of the sustainability of very expensive innovative therapies, outlining new models to guarantee access to all patients ”.

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