What they are, how they work and when are CAR-T treatments used

What they are, how they work and when are CAR-T treatments used

CAR-T are the new frontier of personalized medicine in the field of cancer, but it must be clarified who, when and how it can be administered

Think how nice. An army of “good guys”, prepared in the laboratory for the challenge with cancer starting from the patient’s own cells, ready to take sides against the cells of a tumor. In short, a tailor-made defense. Well, if you believe that this is a dream you are wrong. This represents the CAR-T: today there are already three approved therapies of this type and in Italy as many as 30 centers can administer these treatments.

But when are they really needed? To clarify, the AIL (Italian Association for the Fight against Leukemia, Lymphomas and Myeloma) takes care of launching an information campaign entitled CAR-T – Future Destination. If it is true that these engineered cells are the future of many treatments, in fact, it is undeniable that there are still several questions that need to be answered: what are the actual benefits of CAR-T? What forms of cancer can they cure? Who are the patients who can benefit from it? Where are they administered? How are side effects managed? And how to make them sustainable for the Health Service, in light of their costs?

Really important results

These therapies have been shown to ensure complete remission rates of up to 82% for acute lymphoblastic leukemia, the most frequent cancer in children, between 40 and over 50% for two very aggressive non-Hodgkin’s lymphomas (diffuse lymphoma large B-cell and primary mediastinal lymphoma) and a major improvement in survival (2 years for 51% of patients) in myeloma.

Use against other haematological diseases and solid tumors are also currently being studied. “The arrival of CAR-T has opened new perspectives for some haematological diseases, has triggered great expectations among patients and their families and has aroused great enthusiasm even among us haematologists: but correct, timely and transparent information is needed to clarify doubts and uncertainties about such an innovative therapy – explains Sergio Amadori, AIL National President. The CAR-T weapon is there and it is effective, but at the moment it is not a weapon for everyone, it is indicated only for certain types of haematological diseases and only for patients with adequate qualifications, in authorized centers “.

How do they work

CAR-T (Chimeric Antigen Receptor T cell therapies), are the new frontier of personalized medicine in the field of cancer and represent a therapeutic option in those patients in whom the previous standard therapeutic strategies (chemotherapy and hematopoietic stem cell transplantation) have failed: the T lymphocytes taken from the patient’s blood are “armed” in such a way as to express the CAR receptor on their surface which helps them to recognize the malignant cells and kill them, to then be reinfused into the patient himself.

“CAR-T can be considered at this time as the therapy for otherwise incurable patients – says Alessandro Rambaldi, Full Professor of Hematology, University of Milan, Director of the Department of Oncology-Hematology Papa Giovanni XXIII Hospital in Bergamo – these therapies combine at least three fundamental aspects: they are intelligent therapies, as they identify a target expressed by tumor cells, sparing the patient’s non-sick tissues; they are immunotherapies, therefore non-pharmacological therapies capable of modifying the mechanisms of resistance to drugs that cancer cells implement in the most advanced stages of the tumor disease itself; third aspect, is that it is an immunotherapy based on cells, which can therefore remain for a long time in the patient’s organism in which they are infused, guaranteeing a prolonged mechanism of action and able to reactivate each time the disease reappears “.

Much remains to be understood and studied with respect to the safety and efficacy of these treatments, the use of which is associated with the risk of sometimes serious events, the cytokine release syndrome, which is caused by an excessive immune response due to infusion of modified T lymphocytes. In addition, reduction of B lymphocytes and antibodies and persistence of late cytopenia may occur. Acute criticalities can be remedied with a good organizational and training system and with close contact with intensive care. Chronic criticalities must make use of a good pathology team that leverages dedicated hematologists. For this reason, CAR-T therapies can be administered in a limited number of pediatric and adult hematology and onco-hematology centers, highly specialized for the treatment of leukemia and lymphomas, with specific requirements.

Category: Health
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