A new therapy to treat chronic myeloid leukaemia developed by Novartis has received a National Institute of Care Excellence (NICE) final draft recommendation.  The Pharma Times reports that the treatment, known as Scemblix, or asciminib, will shortly be made available to eligible patients in England and Wales.

Chronic myeloid leukaemia (CML) is a slow-progressing cancer that affects the white blood cells. It can be a serious and life-threatening condition, which tends to affect older people over the age of 65, although it can affect people of all ages. Currently, about 70% of men and 75% of women live for at least five years after their diagnosis. 

The condition is caused by a mutation in the stem cells produced in the bone marrow. There are often no symptoms in the early stages, but eventually patients may notice weight loss, night sweats, swelling on the left side of the stomach, shortness of breath, pallor, high temperature, bruising and bleeding easily, and more frequent infections. 

The main treatment is currently the use of tyrosine kinase inhibitors, which slow the progress of the cancer. There are about 830 new cases of the disease each year in England and Wales, and patients require treatment for the rest of their lives. In some cases, they may also develop a resistance or intolerance to the treatment.

David FitzGerald, Trustee at CML Support UK, said: “Though outcomes for CML patients have improved, challenges in treatment have continued to have an impact on patients’ quality of life, together with the potential to develop resistance or intolerance to their current treatment.”

He added: “For this reason, we welcome the addition of asciminib to the treatment pathway, offering a new option for third-line therapy for eligible patients living with CML.”

Currently, the only potential cure for the disease is a stem cell transplant, but this is a very intensive process, and not all patients are suitable candidates for the treatment. It is usually only carried out as a last resort, when all other treatments have failed. Patients need an extended stay in hospital, of up to one month, in order to recover fully. 

Stem cell transplants also rely on a suitable donor being found, as the stem cells need to have a genetic marker that is identical, or a very close match, to that of the patient. Ideally, it should be a brother or sister, or another close relative. 

Dr Dragana Milojkovic, Professor of Practice at Imperial College London said: “Over the past two decades, TKIs have been the mainstay of treatment for CML, however, over time patients often have to switch their treatment due to intolerance or resistance.”

She added: “A significant number of patients on second-line TKI treatment fail to achieve a satisfactory response after two years and are at risk of disease progression.” 

The new therapy, asciminib, offers an alternative treatment pathway that may lead to extended life spans, or an improved quality of life, for many oncology patients.

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