‘Cutting-edge’ cancer drug given approval for use in the NHS

‘Cutting-edge’ drug that can be used to treat rare cancers in children by targeting cells according to their genetic make-up is to be made available on the NHS

  • Larotrectinib is a tumour agnostic drug that targets cells regardless of origin
  • It is thought to work on all solid tumours meaning it can help in rare cancers
  • Its wide rollout requires extensive testing but is being delayed by coronavirus 
  • Learn more about how to help people impacted by COVID

A new ‘cutting-edge’ cancer drug for children and young people is being made available on the NHS after the service signed a deal with drug maker Bayer. 

Larotrectinib is a so-called ‘tumour agnostic’ medicine, meaning it targets cancerous cells according to their genetic make-up, rather than where they are in the body.

The drug is thought to work on all solid tumours meaning it could be used to fight rare forms of cancer where other treatments have previously failed.

It will be rolled out slowly to the most in need patients first as it will require testing and monitoring before it is made more widely available.

At the moment labs required for testing people taking this new drug are being used to look for cases of COVID-19. 

Larotrectinib is a so-called ‘tumour agnostic’ medicine, meaning it targets cancerous cells according to their genetic make-up, rather than where they are in the body 

The NHS says all relevant patients will be given the ‘personal cancer treatment’ medication as soon as capacity is available in these labs. 

The goal is to test up to 100,000 patients per year for the genetic change that this drug helps with – but coronavirus is delaying this rollout. 

‘Because of the crisis equipment and people have been diverted from Genomic Laboratory Hubs to support testing for coronavirus,’ says Peter Johnson, clinical director for cancer at NHS England. 

‘As soon as we can, we will introduce the capacity to continue rolling this out in a phased way to a much wider population of people for whom conventional treatment has not been successful.’ 

It has been confirmed to work on neurotrophic tyrosine receptor kinase (NTRK) gene fusions – a condition found in rarer forms of the disease.

NTRK fusions are thought to encourage tumour growth and occur in less than 1 per cent of more common cancers.

Children, teenagers and young adults with rare cancers are thought to be the most likely to benefit from the new Bayer drug.

NHS England chief executive Sir Simon Stevens praised staff working on other conditions during the coronavirus pandemic. 

‘Cancer treatment must be a priority, and that will be advanced by the first in a new generation of drugs that can cure previously untreatable tumours,’ he said.

‘This exciting new breakthrough in cancer treatment is the latest example of how the NHS leads the way in the new era of personalised cancer care’.

He said this was happening even while the health service was pulling out all the stops to respond to coronavirus.

‘The benefits for patients, in particular children, of being able to treat many different types of cancers with one drug is potentially huge, helping them to lead longer, healthier lives.’

The goal is to test up to 100,000 patients per year for the genetic change that this drug helps with - but coronavirus is delaying this rollout

The goal is to test up to 100,000 patients per year for the genetic change that this drug helps with – but coronavirus is delaying this rollout 

The National Institute for Health and Care Excellence (NICE) said it was ‘pleased’ to approve the ‘cutting-edge therapy’ for use in the NHS.

Larotrectinib was rejected for funding when it was first proposed in January but Bayer and NHS England have negotiated a new price, making it viable.

The approval is for use over a time limited period while more data is collected from NHS patients to see how effective it is and how it should be used.

Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, said these therapies can be used to treat tumours with rare genetic mutations regardless of where they originate. 

‘The clinical evidence is usually based on extremely small sample sizes, requiring novel approaches to testing them in clinical trials and translation into models of assessment for potential value in NHS practice.’ 

New ‘tumour agnostic drugs’ can be used to treat any type of cancer regardless of its origins 

Tumour agnostic drugs are part of a broader approach to treatment called personalised care.

Research has expanded here in recent years due to developments in genomic sequencing technologies.

A tumour agnostic drug can treat any type of cancer, regardless of its origins in the body or the tissue.

Most cancer treatments are developed to treat a cancer that has developed in a specific organ or tissue, like breast cancer or lung cancer. 

But tumour agnostic drugs treats any kind of cancer as long as the cancer has the specific molecular alteration targeted by the drug. 

Findings out the molecular features  of a tumour can help doctors decide which treatments may be best for an individual with cancer, regardless of where the cancer is located or how it looks under the microscope. 

These drugs show promise in clinical trials, with tumours responding in two-thirds to three-quarters of the cancers tested, according to NHS England.

Targetted therapies already exist, such as Pembrolizumab (Keytruda). But these don’t have the potential to treat a wide range of cancers like tumour agnostic drugs do.

It gives hope to patients with rare or difficult to treat cancers.