Milan, June 26. (beraking latest news Salute) – New hope for patients with chronic myeloid leukemia, a rare blood cancer that affects around 100,000 people worldwide and almost 9,000 in Italy, who are forced to change their therapy because they are now resistant or intolerant of the one they are following. This need is now met by asciminib (Scemblix*), a drug in tablet form aimed at the ‘molecular switch’ of the disease, but equipped with an innovative mechanism of action that makes it more effective and tolerated. Developed by the Swiss Novartis, the treatment is also available in our country, reimbursed by the National Health Service.
Chronic myeloid leukemia (CML) accounts for 15% of all leukemia cases and affects men 1.2-1.7 times more than women. It is caused by the uncontrolled proliferation of bone marrow stem cells and in over 90% of cases it is characterized by an acquired genetic alteration known as the Philadelphia chromosome. Tyrosine kinase inhibitors (Tki) are the drugs used to treat it, but “still today many second-line patients develop resistance or intolerance to the therapy”, explains Fabrizio Pane, full professor at the Federico II University of Naples, director Unit of Hematology and Bone marrow Transplants Aou Federico II of Naples. It is therefore “important to make new therapeutic options available in the third line, which are effective, well tolerated and capable of guaranteeing a good quality of life”. An element which, according to an international survey conducted in 11 countries including Italy, is “the first objective to be achieved with therapy” for one in two patients. Asciminib fits into this scenario, indicated for CML patients with Philadelphia chromosome positive in chronic phase (CML-Cp Ph+), with resistance or intolerance to at least two previous TKIs.
“Asciminib is the progenitor of a new class of drugs, the Stamp inhibitors (Specifically targeting the Abl myristoyl pocket) – explains Fausto Castagnetti, associate professor of the University of Bologna, ‘Seràgnoli’ Hematology Institute, Irccs University Hospital of Bologna – Pur belonging to the Tki class, asciminib differs in a novel mechanism of action: unlike the other Tkis, it does not bind to the Atp-ase site of the Bcr-Abl1 tyrosine kinase, but to the myristoyl site in the kinase domain of Bcr-Abl1 . This binding has the advantage of being highly specific and stabilizing the Bcr-Abl protein in its closed, inactive conformation, arresting tumor progression with minimal side effects.” Tolerability is “a very important aspect – the specialist remarks – given the risks to which patients may be exposed due to the long years of treatment and the frequent presence of other comorbidities”.
“The efficacy of asciminib – underlines Castagnetti – is confirmed by data from the phase III Ascembl pivotal study which showed, after 24 weeks of treatment, an almost double major molecular response rate (25.5% versus 13.2%). compared to another tyrosine kinase inhibitor. Efficacy also confirmed at 96 weeks, with a greater molecular response rate of 37.6% for asciminib against 15.8% for the other Tki”.
For the new drug there is also positive evidence in long-term treatment: “The phase I trial – reports Massimo Breccia, associate professor of La Sapienza University of Rome, Hematology Policlinico Umberto I of Rome – evaluated for a median duration of 4 years the effects of asciminib in heavily pre-treated patients and demonstrated its long-term safety, tolerability and efficacy: not only did the majority of patients (70%) remain on asciminib treatment, but two-thirds achieved major molecular response or the deep molecular response, two parameters that indicate the state of disease activity”.
Even “real world data”, continues the expert, “confirm the potential role of asciminib as a standard of care for patients with chronic phase CML who had previously received at least two lines of treatment with Tki” : the results obtained on “patients treated with asciminib in the compassionate use program conducted in Italy between 2019 and 2023, and recently presented at the European Hematology Association congress, highlighted the efficacy of asciminib in maintaining or improving molecular response and its good tolerability profile”.
“For over twenty years we have been at the forefront of transforming the therapeutic paradigm in the field of chronic myeloid leukemia and asciminib is only the latest testimony of our commitment to support people living with this rare blood cancer – says Paola Coco, CSO & Medical Affairs Head Im Novartis Italia – Also in this case we wanted to start from the unsatisfied needs of patients to develop a new therapeutic option capable of transforming their treatment path”.
A new way to combat CML promoted by insiders, as certified by a survey by the Gimema Foundation for the promotion and development of scientific research on hematological diseases, according to which 9 out of 10 Italian hematologists would use asciminib for patients who no longer respond at the first lines of treatment; and practically everyone, precisely because of its tolerability, considers it a drug suitable for the elderly. For CML, the average age at diagnosis is around 60-67 years.
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