Researchers announced a breakthrough advance in the results of a world-first international clinical trial of a new drug to treat particular blood cancers. The clinical trial, a first-in-man study, looked at the efficacy of a new inhibitor, ONO/GS-4059, for the treatment of chronic lymphocytic leukemia and non-Hodgkin lymphoma that is refractory or resistant to current chemotherapies. The findings were published in Blood (doi:10.1182/blood-2015-08-664086).

“This drug has changed patients’ lives; from desperate and tired, they are now leading a normal and really active life. This is hugely rewarding and encouraging,” said study leader Dr Harriet Walter, MRCP, MBChB, of the University of Leicester in the United Kingdom.

ONO/GS-4059 targets Bruton’s tyrosine kinase (BTK), a protein essential for the survival and proliferation of the tumor cells.

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This study opened in January 2012 and 90 patients from different centers in the United Kingdom and France were enrolled, with 28 coming from Leicester. The study was a first-in-man dose escalation study in relapsed/refractory B cell malignancies.

Results concluded that ONO-4059 induced clinically durable responses in the patients without significant drug-related toxicities. Researchers also reported that “the selectivity of ONO/GS-4059 should confer advantages in combination therapies.”

Patients with chronic lymphocytic leukemia showed the best response and most of them are still on the study after 3 years, remarkably without notable toxicities.

“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 Walter. The success story of this drug has paved the way for its future development in combination studies, which will be opening to recruitment soon.

Asking one participant on the trial about the drug, the patient said, “After just 48 hours of taking this tablet is was like turning the lights on.”

The next step is now to improve on these results. A further study using this drug in combination with additional targeted agents is shortly to open in Leicester with the aim of achieving cure.