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Ponatinib is effective in chronic myeloid leukemia patients, according to study

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Ponatinib is effective in chronic myeloid leukemia patients

A study published in the New England Journal of Medicine shows that a new oral drug, ponatinib, is effective in patients with chronic myeloid leukemia (CML) and Philadelphia chromosome positive acute lymphoblastic lymphoma (Ph+ ALL). The study was conducted by researchers at Huntsman Cancer Institute (HCI).

Chronic myeloid leukemia is a lymphoproliferative disease associated with a characteristic chromosomal translocation called the Philadelphia chromosome. In other words, CML is a cancer of white blood cells. Depending on the stage of the disease, there are several phases: chronic phase, accelerated phase and blast phase. Many patients are in the chronic phase at diagnosis, that is often asymptomatic. Untreated it progresses to accelerated phase of the disease, which is characterized by increased number of blasts, decreased platelet count, splenomegaly etc. The last stage, blast phase, is rapidly progressive and is characterized by more than 20% myeloblasts in the bone marrow. CML can affect people of all ages and has an incidence of 1-2 per 100 000 inhabitants. Leukemia causes are not fully known but there are incriminated several factors. Most commonly leukemia is associated with exposure to ionizing radiation.

Chronic Myeloid Leukemia

Chronic Myeloid Leukemia

Leukemia symptoms are due to uncontrolled proliferation of white blood cells such as asthenia, fatigue, bone pain, splenomegaly, fever or low fever etc. Usually people with leukemia appear pale, due to anemia, have multiple bruises (thrombocytopenia) or infections (neutropenia). It  may happen that leukemia  be discovered by laboratory tests because sometimes has an asymptomatic evolution.

CML  prognosis is good with treatment, survival at 5 years is approximately 80-85%. In 2001, FDA approved imatinib mesylate, a tyrosine-kinase Bcr-Abl inhibitors, which had good responses, imatinib becoming  the first-line treatment for CML. Then two more drugs were approved: nilotinib and dasatanib, in order to increase response and to overcome imatinib resistance that occurs in some patients.

Studies show that 20-30% of patients develop resistance to treatment and this is the main reason for treatment failure. Another treatment option is bone marrow transplantation but it requires several conditions: a compatible donor,  immunosuppressive treatment after transplantation  etc.

Now researchers at Huntsman Cancer Institute (HCI) found that ponatinib has good results in patients with resistant  CML and Philadelphia chromosome positive acute lymphoblast lymphoma (Ph + ALL). Phase 1 clinical trial, in which 81 patients were included, showed that ponatinib gives meaningful responses even in those with advanced disease. Deininger, a senior author on the study who leads an ongoing ponatinib trial at Huntsman Cancer Institute, said:
“Ponatinib is arguably the most potent and broadest BCR-ABL1 available thus far, covering even the T315I mutant, which is completely resistant against all approved TKIs”.