Posted by Michael Wonder on 22 Feb 2018
Developing anti-cancer drugs in orphan molecular entities — a paradigm under construction
22 February 2018 - Genomic characterisation of cancers has shown that some oncogenic alterations occur at very low frequency and are shared across tumour types.
For example, NTRK translocations mediate malignant transformation and are observed in less than 1% of cancers and in more than 20 cancer types.1 The genes NTRK1, NTRK2, and NTRK3 encode for the tropomyosin receptor kinase (TRK) proteins TRKA, TRKB, and TRKC, respectively.
In this week's issue of the New England Journal of Medicine, Drilon et al. report a pooled analysis of three prospective clinical trials testing larotrectinib, a TRK inhibitor, in 55 patients.
