The first trial, which starts this month, will give local patients with Polycythaemia Vera (PV) and Essential Thrombocythaemia (ET) access to a promising new drug called Ruxolitinib if they cannot tolerate or are not responding to the standard treatment.
The treatment is only available because the Queen Elizabeth Hospital is one of 13 centres in the TAP network (TAP), which connects blood cancer trials at Birmingham with other hospitals across the UK and is coordinated from a central hub at the University of Birmingham. The collaborative nature of the network increases efficiency and ensures that potentially life-saving drugs are proven and made available as soon as possible.
PV and ET are blood disorders that cause blood clotting and bleeding. Current treatment usually includes blood removal and chemotherapy drugs like hydroxycarbamide which reduce blood clotting. Around 20% of patients with PV and ET currently cannot tolerate or do not respond to treatment. The only options available to these patients involve drugs that carry a significantly increased risk of severe side effects such as leukaemia.
Ruxolitinib is a type of drug known as a ‘JAK 1 and 2 inhibitor’, which works by targeting a common abnormality in these conditions. Nearly all of patients with PV and half of patients with ET have a mutation in the JAK2 gene that affects normal blood cell production and all patients have increased activation of JAK 2 whether they have the mutation or not. Ruxolitinib inhibits the activation of JAK 2 and a related enzyme called JAK 1. It has been shown in trials of related conditions to considerably improve abnormal blood counts and symptoms, leading to improved quality of life.
Leukaemia & Lymphoma Research Clinical Trials Coordinator, Professor Charlie Craddock, Director of the Centre for Clinical Haematology run by the University of Birmingham and University Hospitals Birmingham NHS Trust, said: “This is the first clinical trial to enter the Trials Acceleration Programme in Birmingham. Ruxolitinib has shown considerable promise that it could improve quality of life and life-expectancy for a significant number of patients with these serious, but relatively rare, blood disorders. The opening of six new clinical trials this year will give local patients with a range of blood cancers new options if current treatments fail or are unsuitable.”
Notes to Editors
For more information or to arrange an interview with Professor Craddock, please contact Jenni Ameghino, Press Officer, University of Birmingham, 0121 415 8134. Mobile 07768 924156