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Shifting Targets for Cancer Treatment By Marc Astick

Part of the REAL SCIENCE DOWNLOAD, a monthly series exploring exciting topics in the world of healthcare. Because #thisisrealscience and we are #poweredbycuriosity.

Cancer treatment – where are we now? 
Think of three people you know. If you are based in the UK, chances are that one of them will develop some form of cancer in their lifetime.1 In 2018 alone, there were more than 18 million new cases of cancer in the world,2 which is a terrifying statistic. On the upside, over the last 40 years, the 10-year survival rate for all cancers in the UK has more than doubled.3 This increase is in no small part due to improvements in the availability and effectiveness of systemic treatments (treatments that spread throughout the body to treat cancer cells wherever they are).
However, neither all systemic therapies nor all cancers are created equal. For instance, survival rates can be widely different for different types of cancer, varying from 2% – 98%.3 Each type of cancer comes with its own, well-established treatment guideline; so far, treatment regimens have been dictated by the origin, location and level of spread (among other factors) and treatments have been designed to target specific cancers. 

How is the landscape evolving? 
Yet, this landscape may be about to change. This month the European Medicines agency, and specifically the Committee for Medicinal Products for Human Use have returned a positive opinion, granting conditional marketing authorisation for larotrectinib. These are exciting news as larotrectinib represents the first ‘histology-independent’ treatment to be recommended to treat cancer based upon its molecular signature rather than its place of origin in the body. More specifically, larotrectinib is intended for the treatment of patients with solid tumours that display a neurotrophic tyrosine receptor kinase (NTRK) gene fusion.4
This type of fusion occurs when the NTRK gene fuses with an unrelated gene; this can lead to the production of a new TRK-fusion protein, which in turn, can drive the development of tumours. NTRK gene fusion is rare, but can occur anywhere in the body and at any age. Larotrectinib acts by blocking these proteins and inhibiting cancer growth.5 It is recommended for patients who have such a gene fusion and have no other viable treatment options.4
So what is the potential impact of this news?
This type of cancer treatment represents a potential new and exciting model for systemic therapy, where a drug is designed based upon molecular signatures – a so-called agnostic therapy. For patients, this brings a shift in the way that cancers may be treated, where the treatment available to them may not be limited by the origin or location of their tumour. This move may help push the survival rate of patients with solid tumours higher and open the way for treating cancer differently!

  1. NHS. Cancer.  Available from Last accessed August 2019;

  2. WHO. International Agency for Research on Cancer. Available from Last accessed August 2019;

  3. Cancer Research UK. Cancer survival statistics. Available from Last accessed August 2019;

  4. European Medicines agency. Available from Last accessed August 2019;

  5. Bayer. What is TRK fusion cancer and how can we detect it? Available from Last accessed August 2019.