A new blood cancer drug that targets a protein essential for growth of tumour cells has been found to be effective in patients resistant to current chemotherapies, a world-first clinical trial has shown.
In the first-in-human study, the researchers from the University of Leicester and Leicester's Hospitals in UK looked at the efficacy of a new inhibitor, ONO/GS-4059, in the treatment of Chronic Lymphocytic Leukemia and Non-Hodgkin Lymphoma patients resistant to current chemotherapies.
ONO/GS-4059 targets BTK, a protein essential for the survival and proliferation of the tumour cells.
The study started in January 2012 and 90 patients were enrolled in different centres in the UK and in France.
Patients with Chronic Lymphocytic Leukemia showed the best response and most of them are still on the study after 3 years, and 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 Harriet Walter, from 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," Walter said.
"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 at the University of Leicester and Honorary Consultant Physician in the Department of Haematology at Leicester Royal Infirmary.
The next step is now to see how best we can improve on these outstanding results, the researchers said.
The study was published in the journal Blood.
