Clinical update

New treatment possibility for some cancer patients

Larotrectinib is approved by NICE as an option for NTRK fusion-positive solid tumours
Picture shows woman cancer patient lying in a hospital bed.

Larotrectinib is approved by NICE as an option for NTRK fusion-positive solid tumours

Essential facts

Larotrectinib is one of a new breed of cancer drugs designed to target specific changes in the DNA of cancer cells, rather than where the disease is growing in the body. This means patients, including children, with various cancer types may benefit from its use.

It is particularly useful for those patients at an advanced stage of cancer, when other options are limited or have been exhausted. The treatment aims to shrink tumours, potentially enough to make surgery a possibility for patients whose cancer could not otherwise be treated in this way.

Whats new?

After funding was initially rejected in January this year, larotrectinib was approved for NHS use in England in April, following successful negotiations between NHS England, the National Institute

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Larotrectinib is approved by NICE as an option for NTRK fusion-positive solid tumours

Picture shows woman cancer patient lying in a hospital bed.
Picture: iStock

Essential facts

Larotrectinib is one of a new breed of cancer drugs designed to target specific changes in the DNA of cancer cells, rather than where the disease is growing in the body. This means patients, including children, with various cancer types may benefit from its use.

It is particularly useful for those patients at an advanced stage of cancer, when other options are limited or have been exhausted. The treatment aims to shrink tumours, potentially enough to make surgery a possibility for patients whose cancer could not otherwise be treated in this way. 

What’s new?

After funding was initially rejected in January this year, larotrectinib was approved for NHS use in England in April, following successful negotiations between NHS England, the National Institute for Health and Care Excellence (NICE) and the manufacturer, with a new price agreed.

In its final appraisal document, NICE says the drug is recommended as an option for treating neurotrophic tyrosine receptor kinase (NTRK) fusion-positive solid tumours in adults and children, if the disease is locally advanced or metastatic, or surgery could cause severe health problems, and there are no satisfactory treatment options.

NTRK gene fusions are commonly seen in some rare cancers – examples are mammary analogue secretory carcinoma and infantile fibrosarcoma – and occasionally in the more common cancers.

Now that larotrectinib is covered by the Cancer Drugs Fund (CDF), the NHS will pay for it for a limited time while data are collected. If its use is shown to be cost-effective, it will become available permanently. CDF drugs are usually also made available to patients in Wales and Northern Ireland. Scotland has a separate system and has yet to consider larotrectinib.

How you can help your patient

Cancer nursing specialists need to be aware of this new treatment possibility for their patients. According to NICE, between 600 and 700 people in England have solid tumours with NTRK gene fusions

A proportion of these patients who have no satisfactory treatment options will be eligible for treatment within the first year that larotrectinib is available on the CDF. Patients need to be tested for their suitability.  

For adults, the recommended dose is 100mg of larotrectinib twice daily, until disease progression or unacceptable toxicity occurs. For children, dosing is based on body surface area, with a recommended 100mg/m² of larotrectinib twice daily, with a maximum of 100mg per dose until disease progression or unacceptable toxicity occurs.

Expert comment

Picture of Helen Stradling, a sarcoma specialist nurse and support line lead for charity Sarcoma UKHelen Stradling, sarcoma specialist nurse and support line lead for charity Sarcoma UK

‘This is fantastic news. For sarcoma patients in particular, the approval of larotrectinib marks the first new drug available for them since regorafenib in autumn 2017. Crucially, it sets an important precedent for using this type of treatment that targets specific genetic abnormalities.

‘We know that novel treatments for sarcoma patients are few and far between, so larotrectinib is a welcome addition to the available therapies, alongside a number of other tumour types.

‘The drug will only benefit people who have the specific NTRK gene mutation, no matter their tumour type or location. Genetic testing will need to be undertaken, but hopefully, with the rolling out of the Genomic Medicine Service, patients will soon find out if they have this particular fusion.

‘This means that for those who could benefit it can be in their armoury of treatment options.’


Lynne Pearce is a health journalist

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