Southampton leads first national trial of chemotherapy free treatment for a rare blood cancer

Researchers at the University of Southampton have been awarded a major international research grant to lead the first UK-wide study of a fully oral treatment for a rare type of blood cancer.

It’s hoped the treatment could mean better outcomes for patients with Hairy Cell Leukaemia in the future and see them avoid the side-effects often caused by traditional chemotherapy.

The trial is led by Professor Francesco Forconi, Professor of Haematology at the University of Southampton, and coordinated by the Southampton Clinical Trials Unit. The team has been awarded $1.75m from The Hairy Cell Leukemia Foundation and Blood Cancer United , through the HCL2030 funding initiative.

A chemotherapy free approach

Hairy Cell Leukaemia (HCL) is a rare type of chronic blood cancer, named after the hair-like growths that can be seen on the cancer cells under a microscope. These abnormal white blood cells, called B lymphocytes, which have not developed properly, build up in the body preventing the production of healthy white blood cells which are vital for fighting infections.

“Around 230 people a year in the UK are diagnosed with this cancer*, making it a rare cancer,” says Professor Forconi. “While chemotherapy is often effective initially, it can have unpleasant side-effects and long term toxicities for patients. Many patients with Hairy Cell Leukaemia will later relapse and see the cancer come back, and at this point there are limited treatment options for them.”

Targeting the biology of the disease

Hairy Cell Leukaemia is driven by abnormal activation of B cell receptor signalling and over expression of a protein, called BCL2, that prevents cancer cells from dying.

The MAYFLOWER-HCL trial will investigate two oral treatments** that directly target both pathways simultaneously, halting the growth of the abnormal B cells and triggering cancer cell death. It builds on years of laboratory and clinical research by the University’s Cancer B cell Group.

Professor Forconi continues: “This trial is designed to move the field decisively away from the more toxic chemotherapy treatments, targeting instead the fundamental mechanisms that allow tumour B cells to survive.”

UK wide collaboration

MAYFLOWER-HCL is the first multicentre clinical trial ever designed in the UK for Hairy Cell Leukaemia.

The trial will be coordinated by the Southampton Clinical Trials Unit (SCTU), led by Professor Gareth Griffiths, which has world-leading expertise in blood cancer trials. It will recruit patients across five major UK university hospitals***, ensuring national access for this rare cancer.

“Because Hairy Cell Leukaemia is rare, large national trials are often difficult to deliver,” says Professor Griffiths. “This award places Southampton at the centre of a UK wide collaborative effort and positions the trial to influence future international treatment standards.

“It also reflects the national drive to improve treatments and survival for rare cancers which is outlined in the new Rare Cancers Bill that recently came into UK law. As a leading UK trials unit, we are making sure we are playing our part in helping to accelerate this vital research in areas of unmet need for cancer patients.”

A milestone for rare cancer research

With the major funding grant secured, the trial is now in set-up and aims to open to patient recruitment by the end of the year.

“This trial is the first step in our long-term goal to provide better, kinder treatments for patients with HCL,” Professor Forconi adds. “Our hope is to not only control the disease, potentially leading to remission, but also improve patient’s quality of life while on treatment.

“This is a landmark moment for patients with Hairy Cell Leukaemia in the UK. It reflects both the strength of collaboration across NHS and academic partners, and Southampton’s growing role in leading innovative, patient-focused cancer trials.”

MAYFLOWER-HCL is a phase II national trial in relapsed or refractory Hairy Cell Leukaemia. The drugs for the trial are provided by BeOne Medicines, and funding is provided by the Hairy Cell Leukaemia Foundation and Blood Cancer United and through the HCL2030 Initiative. The trial is run by the Southampton Clinical Trials Unit and sponsored by University Hospital Southampton.