Three thousand people are affected by chronic lymphocytic leukemia every year: the majority are over 70 years old and often have more than one pathology. This is why it is essential that therapies are easy to take and manage in the long term. And this is also why it is important to approve the reimbursement by the Italian Medicines Agency (Aifa) of a new formulation in small tablets of acalabrutinib, a targeted therapy that has contributed to changing the treatment of this blood cancer. Now, thanks to the volume halved compared to previous capsules, the medicine will be much easier to swallow. Not only that: it can be taken with or without food, or with agents that reduce gastric acidity (such as proton pump inhibitors) without compromising absorption. It may seem like a small thing, but it is actually an innovation that makes a difference to the quality of life of people who find themselves having to take multiple medications.
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The efficacy obviously remains the same as the previous formulation, as has been demonstrated in a clinical study, but in this way it will also be possible to improve adherence and expand the number of patients who will be able to benefit from this treatment: the novelty in fact concerns both new patients and those already under treatment.
What is chronic lymphocytic leukemia
Chronic lymphocytic leukemia is the most frequent blood cancer in the adult population in the Western world. It is caused by the proliferation of B lymphocytes, which have acquired specific molecular alterations and generate an immunodeficiency condition that predisposes to infections: these grow in excessive numbers in the blood, bone marrow and lymphatic tissues, which consequently increase in volume. This proliferation “takes away space” for red blood cells and platelets, which decrease, which can lead to anemia and a tendency to have hemorrhages and hematomas.
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Diagnosis and active surveillance
Usually the disease is discovered by chance, because the symptoms – anemia, swollen lymph nodes, thrombocytopenia, tiredness, night sweats, involuntary weight loss – appear late. “In approximately 55-60% of cases, it is not necessary to start treatment immediately – he explains Gianluca Gaidano, Professor of Blood Diseases at the Department of Translational Medicine of the University of Eastern Piedmont and Director of the Complex University Management Structure of Hematology of the Azienda Ospedaliero-Universitaria Maggiore della Carità of Novara – Several years can pass before the appearance of evident symptoms. Treatments are necessary when white blood cells tend to grow very rapidly or when red blood cell and platelet values fall below alert levels. Even very large lymph nodes or spleen require therapeutic intervention.”
A cure without chemo
The first step to take before establishing the most suitable therapy is to see which molecular alterations are present. “The treatment of chronic lymphocytic leukemia has changed a lot and no longer includes chemotherapy in any case – continues the expert – There are instead various targeted therapies – from BTK inhibitors to BCL2 inhibitors – which can be associated with monoclonal antibodies. Acalabrutinib belongs to the class of BTK inhibitors, which has revolutionized the treatment of the disease.”
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Acalabrutinib: efficacy and safety data
The latest data on this drug come from the phase III ELEVATE-TN study, conducted on 535 previously untreated patients, and were presented last December at the American Hematology Congress (ASH, American Society of Hematology Annual Meeting): the 78th % and 62% of patients treated with acalabrutinib plus obinutuzumab or acalabrutinib monotherapy, respectively, were alive without disease progression, compared to 17% of patients treated with the chemo-immunotherapy combination. And in the phase III ASCEND study, conducted on 310 patients with relapsed or refractory chronic lymphocytic leukemia, acalabrutinib reduced the risk of disease progression or death by 69% compared to the control arm (rituximab combined with idelalisib or bendamustine). Furthermore, no new adverse effects were detected compared to those already known.
Quality of life among the primary objectives
“In addition to the symptoms, we must always consider the emotional, social and functional consequences of living with a chronic pathology. Hence the increasingly central role of quality of life – he states Rosalba Barbieri, Vice President of AIL (Italian Association against Leukemia, Lymphoma and Myeloma) -. Even those who present symptoms and therefore require treatment can now lead a normal life thanks to innovative therapies. A need and a right of patients is rapid and uniform access to innovation throughout the national territory. Furthermore, if we really want to talk about personalized therapy, we need to take charge of the person and not just the disease, a new model that takes into account not only the clinical dimension, but also the social and psychological needs”.
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“Patients’ quality of life is a priority for AstraZeneca’s research – he concludes Paola Morosini, Medical Affairs Head Oncology AstraZeneca -. Acalabrutinib is a new generation molecule, born from years of research, and is now available in the tablet formulation, which thanks to the innovations we have talked about will allow a greater number of patients to benefit from this therapy. Our extensive development program includes more than 25 clinical trials involving monotherapies and combination therapies across different forms of hematological malignancies.”