For a long time, researchers have been working on it all over the world, but it’s finally arrived. It is the first tumor-agnostic drug treatment and is called Larotrectinib (the active ingredient of the drug).

Before we dig into the details, we must specify that the current cancer drugs are all tumor-specific, or for a very small class of tumors because chemotherapy acting directly on the cells.

Larotrectnib, on the other hand, is said to be tumor-agnostic because it doesn’t act on the cells of a specific tumor, but on the gene modification of the Neurotrophic Tyrosine Receptor Kinase gene (NTRK).

What are the advantages of Larotrectnib?

Undoubtedly, the fact that such a drug can act on several tumors that have the same genetic origin regardless of where the tumor originated. Since the modification of the NTRK gene (known in biological jargon as “gene fusion”) is the basis of at least 29 types of solid tumors, including those of the lung, thyroid, melanoma, stromal gastrointestinal tumors; colon, soft tissue sarcomas, gliomas and glioblastomas, salivary gland tumors, and childhood fibrosarcoma.

Another great advantage is that larotrectnib can be taken orally.

The drug was approved in the United States by the Food and Drug Administration (FDA) in November 2018, with an accelerated procedure, due to its clear efficacy and duration of response. It’s also been approved in Brazil and Canada, and approval procedures are underway in other states, based on specific local regulations.

Its use is for the treatment of adult and pediatric patients with NTRK gene fusion cancer, and for whom no satisfactory alternative treatment is available — or who have demonstrated tumor progression following treatment.

The European Medicines Agency has now recommended the marketing authorization of larotrectnib in Europe. This means that the drug’s manufacturer, Bayer, must register larotrectnib within the European countries where it’s to be marketed, and will therefore soon be available in Italy.

Can larotrectnib be used in all cases of solid tumor?

It should be noted that tumors with TRK gene fusion are rare (i.e. only few lung tumors, for example, have gene fusion), so before proceeding with drug therapy, the physician must first ascertain with a DNA molecular diagnostic investigation whether larotrectnib is actually useful; otherwise, he or she must proceed with other drugs.

Regardless of the efficacy of larotrectnib, the most important news here is that the efficacy of a drug that selectively acts on tumors with specific genome alterations has been approved, regardless of the site of origin of the tumor. This will soon lead to new drugs that are also effective on those tumors that are currently poorly treated or do not respond well to current therapies. Moreover, the clinical use of tumor-agnostic drugs represents an additional step forward for precision medicine.

Being a brand new therapy, there are not a lot of videos that provide further explanation. However, I include this video that tells — in three minutes — the story of a little girl whose life was saved by larotrectnib. Seeing the images from 1:28, everything becomes clear.

This post is also available in: Italiano


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