A breakthrough advance in the results of the world-first clinical trial with actual patients of a new drug to treat particular blood cancers has been announced by researchers from the University of Leicester and Leicester’s Hospitals.

Results of the international clinical trial, led by Dr Harriet Walter and Professor Martin Dyer from the Ernest and Helen Scott Haematological Research Institute at the University of Leicester and from the Leicester Royal Infirmary, are published in the journal Blood.

This clinical trial, a first-in-man study, looked at the efficacy of a new inhibitor, ONO/GS-4059, in the treatment of Chronic Lymphocytic Leukaemia and Non-Hodgkin Lymphoma patients refractory or resistant to current chemotherapies.

ONO/GS-4059 targets BTK, a protein essential for the survival and proliferation of the tumour cells.

Chronic Lymphocytic Leukaemia

This study opened in January 2012 and 90 patients were enrolled in different centres in the UK and in France, with 28 coming from Leicester. Patients with Chronic Lymphocytic Leukaemia showed the best response and most of them are still on the study after 3 years, and remarkably without notable toxicities.

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

Dr Harriet Walter, from the Department of Cancer Studies at the University of Leicester, said:

“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 next step is now to see how best we can improve on these outstanding 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.

In parallel with the clinical development of the drug, our team of scientists at the Haematological Research Institute are studying how this drug is working and how to overcome potential resistance.

H. S. Walter et al. A phase I clinical trial of the selective BTK inhibitor ONO/GS-4059 in relapsed and refractory mature B-cell malignancies Blood (2015). DOI: 10.1182/blood-2015-08-664086

Image: Wellcome Images

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