Southampton studies change blood cancer treatment
New research into a cancer drug is changing the way a type of leukaemia is treated.
Two studies, led by Professor Francesco Forconi, have shown venetoclax can help treat patients with Chronic Lymphocytic Leukaemia (CLL).
As a result, the National Institute for Health and Care Excellence (NICE) has now approved the use of venetoclax, in combination with another treatment, for NHS patients with CLL.
Prof Forconi is part of the University of Southampton’s Centre for Cancer Immunology. He is a haematologist at University Hospital Southampton (UHS).
Patients are already receiving the treatment at UHS, as part of a new service.
Standalone treatment
Venetoclax is a type of targeted cancer drug called a cancer growth blocker. It blocks certain proteins in cancer cells (BCL2 proteins) that help them grow and survive. By doing this, it is able to kill and slow down the growth of cancer cells. It is not considered a type of chemotherapy.
B-cell receptor-associated kinase inhibitors (BCRi) are a class of drugs that target specific enzymes involved in cancer growth. They are used to treat cancer, but can be toxic if taken over a long time, which they usually are.
In the first study, published in Lancet Oncology, the researchers assessed venetoclax as a single treatment for patients with CLL whose cancer had returned or stopped responding to treatment.
In total, 258 CLL patients were given venetoclax for just over two years. Of these patients, 67 had been previously treated with BCRis. They were then followed up for another two years - the longest follow-up for any trial evaluating venetoclax as a standalone treatment.
In the group not previously treated with BCRis, 35% achieved complete remission, where the cancer was no longer detectable. In those previously treated with BCRis, it was 27%.
Despite this promising outcome, some patients experienced side effects. These included low levels of neutrophils (a type of white blood cell) and pneumonia.
Combined treatment
The second study, published in the New England Journal of Medicine, looked at combining venetoclax with the BCRi ibrutinib. This was compared against the standard chemoimmunotherapy treatment of fludarabine–cyclophosphamide–rituximab (FCR).
More than 500 patients with untreated CLL were randomly assigned receive either ibrutinib and venetoclax or FCR. They took the treatment for up to six years, or until the cancer was undetectable.
The results showed patients in the ibrutinib–venetoclax group had much better outcomes than those receiving FCR. They had a lower risk of death, and 58 percent stopped the treatment after three years because their cancer was no longer present.
New service for patients
The team has implemented a new service for minimal-residual disease assessment within Prof Forconi’s clinic at Southampton General Hospital. This service uses venetoclax, in combination with other treatments, to help patients end their cancer treatment.
Prof Forconi said: “Thousands of people are diagnosed with CLL each year, and there are a number of drugs which have helped patients to fight this disease. However, they can be toxic, and some patients do not respond for long. Finding new ways of eradicating this cancer before the drugs become toxic is important, so we can enable more people to get their life back, free from this disease.
“These studies showed the potential venetoclax could have in patents with CLL, and we are proud to have implemented our new service, which is already helping people.
“The way we treat cancer has changed so much since I came to Southampton 10 years ago. We are now about to deliver treatment approaches which are completely chemotherapy-free and use drugs that target the main biological pillars of CLL. This will mean less toxicity and more efficacy for our patients.”