World’s first blood cancer drug trial yields positive results

A world-first clinical trial with actual patients has demonstrated the potential of a new drug in treating particular blood cancers.

Researchers from the University of Leicester and Leicester’s Hospitals have announced a breakthrough advance in the results of a new inhibitor, called ONO/GS-4059 targets BTK, which is a protein essential for the survival and proliferation of the tumour cells. The clinical trial, a first-in-man study, looked at the efficacy of the protein in the treatment of chronic lymphocytic leukaemia and Non-Hodgkin Lymphoma patients who are resistant to current chemotherapies.

The study, which started in January 2012, involved 90 patients in different centres in the UK and France, with 28 coming from Leicester. Among the participants, patients with chronic lymphocytic leukaemia showed the best response, and most of them are still on the study after three 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. 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,” said Dr Harriet Walter from the Department of Cancer Studies at the University of Leicester.

One of the patients on the trial said, “After just 48 hours of taking this tablet is was like turning the lights on.”

According to the researchers, their next step is to see how best they 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, the team of scientists at the Haematological Research Institute is studying how the drug works and how to overcome potential resistance.

The international clinical study, which is detailed in the journal Blood, was led by 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.

According to the Leukaemia Foundation, 34 people in Australia are diagnosed every day with leukaemia, lymphoma, myeloma or a related blood disorder. Leukaemias are cancers that affect the blood and bone marrow, which start in the bone marrow where developing blood cells, usually white cells, undergo a malignant change. Meanwhile, lymphomas are a type of blood cancer that affects the lymphatic system. Lymphomas occur when developing lymphocytes undergo a malignant change and multiply in an uncontrolled way.

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