Leukaemia Care successfully campaigns for new leukaemia treatment on behalf of CML patients

Leukaemia Care successfully campaigns for the National Institute for Health and Care Excellence (NICE) to approve asciminib on the NHS in England for chronic myeloid leukaemia (CML) patients after 2 or more tyrosine kinase inhibitors (TKIs).

What’s the news and how was Leukaemia Care involved? 

We are pleased to say that a new treatment, asciminib, has been approved for NHS use in chronic myeloid leukaemia (CML) patients after 2 or more tyrosine kinase inhibitors (TKIs).

Leukaemia Care submitted a written response to NICE advocating for the approval of asciminib before the decision had been made. We did so by drawing on relevant data and patient experiences we gathered to adequately represent patient views. 

However, Leukaemia Care would like to see this treatment be more widely available for patients in the future, such as it being an option that doctors and patients can use earlier on, without a patient needing to try as many as 2 previous drugs beforehand.   

Which patients are able to access this treatment?

This treatment will only be available to CML patients who have already tried two or more other TKIs. 

There are two types of CML. The vast majority of people with CML have CML because of a chromosome change that caused their cells to become cancerous. This chromosome is called a Philadelphia chromosome. Asciminib, the newly approved treatment, will only be used to treat adult patients who have chronic-phase Philadelphia chromosome-positive CML. 

Some CML patients also have a genetic mutation in their CML cells called the T315i mutation. These patients will not be eligible for this treatment.

Why is the approval of this treatment important for patients? 

We know that some of the existing TKI treatments don’t suit everyone. Many patients who take TKIs go through multiple different ones to find the balance between the drug’s effectiveness and mangeable side effects. Additionally, some patients become resistant to certain TKIs over time. This is why having as many options as possible for CML patients is important. Asciminib is another TKI treatment option and its approval means that patients are less likely to run out of options if they are unable to have the other TKIs. This was one of the key points in Leukaemia Care’s written response to NICE and one of NICE’s key decision making factors. 

What are the other benefits of the treatment to patients? 

Not only would asciminib have a positive impact on a patient’s quality of life by virtue of the fact it is another option for those who cannot have other TKIs, but it also has been shown to be more effective and have fewer side effects when compared to bosutinib in a clinical trial. The effectiveness of asciminib refers to the number of MMR (major molecular responses) that patients achieved. Achieving MMR predicts a CML-specific survival close to 100%, as disease progression is uncommon once MMR is achieved. Additionally, the fewer severe side effects mean that patients are less likely to face treatment discontinuation with asciminib. 

How does the treatment work?

Usual treatment for CML in this setting includes the TKIs bosutinib, ponatinib, dasatinib or nilotinib. All TKIs treat CML by binding to the protein BCR-ABL. This is the protein that is made by the Philadelphia chromosome and causes the CML cells to multiply. By binding to the BCL-ABL protein, the TKI stops it working and hence stops CML cells from growing. While asciminib is a TKI, it targets a different binding site than all other TKIs (the myristoyl binding site, rather than the ATP binding site). Some CML cells can edit the BCL-ABL protein in a way that stops the TKI binding as well, causing the TKI to stop working. Because asciminib binds to a different part of the protein, it even works on CML cells with the protein that has changed in a way that stops the other TKIs working (resistance to TKIs).

If you have any further questions about the drug announcement, our Advocacy and Policy and Public Affairs team is here to help. Email them at advocacy@leukaemiacare.org.uk.

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