Results from a breakthrough study on a potential drug to treat blood cancers were recently announced, revealing that ONO/GS-4059, a BTK inhibitor drug, had impressive efficacy in the treatment of patients diagnosed with Chronic Lymphocytic Leukemia and Non-Hodgkin’s Lymphoma. The research paper, entitled “A Phase I clinical trial of the selective BTK inhibitor ONO/GS-4059 in relapsed and refractory mature B-cell malignancies,” was published in the journal Blood.
The Phase I first-in-man ongoing study included 90 patients from centers across the UK and France, with different relapsed/refractory B-cell malignancies, including Chronic Lymphocytic Leukemia (CLL), Non-Hodgkin’s Lymphomas (Mantle Cell Lymphoma) and diffuse large B-cell lymphoma (DLBCL). The dose escalating study, which experimented several once-a-day and twice-a-day drug dosage regimens, was initiated in January 2012 and most patients are still in the study three years later. The treatment was especially successful in Chronic Lymphocytic Leukemia patients. Importantly, treatment with ONO/GS-4059 was well-tolerated, with 75% of adverse events registered as weak /mild, and some patients affected by more serious events recovering spontaneously during ongoing therapy.
The results are considered very encouraging, especially because the drug improved the lives of patients who had failed chemotherapy treatments and had no other available therapy. According to one of the patients, “After just 48 hours of taking this tablet is was like turning the lights on.”
Dr. Harriet Walter from the Department of Cancer Studies at the University of Leicester and one of the authors of the study, said in a press release, “These patients were confronted with a cruel reality: they had failed multiple chemotherapy lines and there were no other treatment options available for them. This drug has changed their lives; from desperate and tired they are now leading a normal and really active life. This is hugely rewarding and encouraging.”
The researchers plan to test this drug in combination with other agents in a trial that will open for recruitment in Leicester, UK. In parallel, scientists at the Hematological Research Institute will continue to study the drug and its mechanisms to overcome a potential resistance developed by the human organism.