Pharmaceutical research in Italy, once flourishing, has all-but disappeared … but it’s more accurate to say “almost”.

We’re used to associate the introduction of an innovative drug with the United States or Switzerland — or one of a very few other countries, but even the little pharmaceutical industry left in Italy is able to produce very important results.

We’re now talking about the “made in Italy” drug Entrectinib, a molecule born in the laboratories of the Nerviano Research Center and whose clinical development was initiated at the Niguarda Cancer Center and the National Cancer Institute, both in Milan.

But what’s so exceptional about the development of this drug? First of all, as mentioned, it was completed developed in Italy, and then the first researcher is a biologist, Elena Ardini, and finally it’s a clear example of Precision Medicine.

But let’s see in better detail what all this is really about. The drug, which is administered orally, has a targeted action toward a series of molecular targets (i.e. genes with strange names: NTRK/1/2/3, ROS1 and ALK). These genes, if altered, lead to the development of very serious tumors such as non-small cell lung cancer, rectal colon cancer, salivary gland carcinoma, melanoma and renal cancer.

Entrectinib, as for its characteristic of acting in a targeted manner, it’s a clear example of Precision Medicine. It is also able to cross the blood-brain barrier and therefore. it’s also effective on those tumors that lead to brain metastases. In one case studied during the clinical trial, it was shown that brain metastases disappeared completely, while the patient was on therapy for small cell lung cancer. A truly amazing result and a great success.

The study is still in its initial phase; two Phase I clinical trials were carried out (the first step in the clinical development of a drug). However, the results obtained on the 25 cases treated are very comforting, as 79% responded to the treatment, also showing an excellent tolerability profile (i.e. very few side-effects).

Entrectinib has already received two important awards; one from the Food and Drug Administration (FDA) as “Breakthrough Therapy”, and from the European Medicine Agency (EMA), as a “Priority Medicine” (PRIME) — a very clear example of an innovative and effective drug. The reason for these two awards also lies in the fact that Entrectinib is not effective on a particular tumor, but is effective on all those tumors that have a molecular alteration in the NTRK1/2/3, ROS1 and ALK genes.

In short, there are all the prerequisites for which a new drug will soon be put on the market that will provide new hope to patients with lung cancer, and not to small cells, colorectal cancer, salivary gland carcinoma, melanoma and renal cancer!

Unfortunately, the “made in Italy” drug will not be marketed by an Italian company, but by an American company; Ignyta, which would have been the icing on the cake.

This post is also available in: Italiano


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