First clinical trials of novel drug for aggressive leukemia treatment  

Blood oncological disease (leukemia) called non-Hodgkin's lymphoma (NHL) and chronic lymphocytic leukemia (CLL) are extremely aggressive and resistant to any treatment. Against these, several chemotherapy treatment types are used being more or less effective. But at the same time, patients rapidly develop treatment resistance and unexpected adverse events of intoxication. A novel drug voiding these adverse events has been successfully clinically studied in patients.

While studying all the molecular processes that accompany the emergence and development of CLL and NHL, scientists from several European countries, revealed that the leading role in uncontrolled malignant blood cancer cells’ developing and multiplication belongs to BTK (specific protein), located on the cancer cells’ surface. After several years of research, a special inhibitor dramatically suppressing the BTK protein and the cancer cells’ growth and division was developed and called ONO / GS-4059. Scientists have developed a drug containing an inhibitor of that kind. After preclinical studies in lab animals, new drug was first clinically studied in more than 120 patients of different ages and sexes diagnosed with CLL or NHL. Some of the patients have already been treated with chemotherapy; however, this did not prevent the disease relapse after the initial treatment and caused severe intoxication. Other patients have not previously received any anti-cancer therapy. The results of the clinical trial of the ONO/GS-4059 inhibitor containing drug formulated as a tablet for oral administration revealed significant suppression of the blood cancer cells’ growth of and did not cause adverse intoxication.

Developers of new anticancer drug are now close to finish its introduction into clinical practice to treat patients with severe blood oncological diseases