For the first time, an innovative new class of cancer drug that can treat a wide array of tumors has been approved for use in Europe. Tumor-agnostic drugs are independent of where the cancer is growing in the body as long as it has an explicit genetic abnormality inside.
UK doctors testing the new drugs said they were “a really exciting thing”. They believe the approach had the potential to cure more patients without side-effects. The cancer drug that has been approved is called larotrectinib.
Two-year-old Charlotte Stevenson from Belfast was one of the first patients to benefit from this new revolutionary medication. She was detected with infantile fibrosarcoma, a cancer of the connective tissues in the body. She was treated with larotrectinib as part of a clinical trial at the Royal Marsden Sutton, in London, for the past year.
Her mum, Esther, said: “We knew that our options were limited [so] we decided to give it a try and are so glad that we did. We have been able to watch Charlotte develop and grow at a rapid rate, making up for lost time in so many ways and amazing us all with her energy and enthusiasm for life. She can now have a relatively normal life and, best of all, the drug has had an incredible impact on the tumour.”
Charlotte’s tumor was produced by a genetic abnormality called an NTRK gene fusion. A part of her DNA inadvertently merged with another and the variation in the blueprint for her body led to the growth of her cancer.
But such NTRK gene fusions are not unique to sarcomas as they can also manifest in some brain, kidney, thyroid and other cancers.
Dr. Julia Chisholm, a children’s cancer consultant at the Royal Marsden Hospital, told the BBC, “It is a really exciting thing, as is it works across a range of cancers. It’s not confined to one”.
NTRK mutations are comparatively rare nevertheless other targeted therapies are in development. This symbolizes a move away from typically treating a “breast cancer” or “bowel cancer” or a “lung cancer” and towards more precision medicine that exploits the genetic make-up of each patient’s tumor.
Dr. Chisholm told the BBC, “The beauty is it targets the abnormality. There are a number of biochemical pathways that are common in many different tumour types. I think this is the way things are going and this is about better outcomes, curing more patients and producing kinder treatments with reduced side-effects.”
However, this approval by European regulators does not imply it will be instantly available for patients in the UK.
Earlier this year, NHS England labeled tumor-agnostic drugs as a “revolutionary” and “exciting new breakthrough” in cancer and said arrangements were underway to ensure patients were given access to them.
NHS England chief executive Simon Stevens said at the time, “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”.
Prof Charles Swanton, Cancer Research UK’s chief clinician, said the drugs were “exciting”.
He added, “The NHS will need to ensure the right genomic testing is available across the country to identify patients who could benefit so it’s good that the NHS is already thinking about how to get this to patients with cancer as soon as possible.”
Dr. Brendon Gray, from the drug company Bayer that developed larotrectinib, said, “As the first tumour-agnostic medicine approved in Europe, larotrectinib represents a real shift in cancer treatment.”