Blood Cancer Drug Successful in First Clinical Trial

Novel drug shows promise in chronic lymphocytic leukemia and non-Hodgkin lymphoma refractory or resistant to current chemotherapies.

A new drug in development for the treatment of specific blood cancers recently completed its first clinical trial on human patients with encouraging results.

An international clinical trial led by researchers from the Ernest and Helen Scott Haematological Research Institute at the University of Leicester and from the Leicester Royal Infirmary evaluated the novel inhibitor ONO/GS-4059 in patients with chronic lymphocytic leukemia and in patients with non-Hodgkin lymphoma refractory or resistant to current chemotherapies.

"We are dedicated to offer the best treatment options to our patients and the development of targeted therapies that increase the chance of therapeutic success and at the same time avoid toxicities generally observed in chemotherapies, is the most exciting progress in cancer research,” said Martin Dyer, professor of Haemato-Oncology at the University of Leicester.

The drug targets the BTK protein that is vital to the survival and proliferation of tumor cells.

The trial began in January 2012 with 90 patients enrolled at different centers in the United Kingdom and France. ONO/GS-4059 produced the best response in patients with chronic lymphocytic leukemia, with the majority of these patients still on the drug after 3 years without notable toxicities, according to researchers.

"These patients were confronted with a cruel reality: they had failed multiple chemotherapy lines and there were no other treatment options available for them,” said Dr Harriet Walter of the Department of Cancer Studies at the University of Leicester. “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 results of the study, recently published in the journal Blood, pave the way for future research on ONO/GS-4059 in combination with additional targeted therapies.