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. 2014 Feb;24(100):114–119. doi: 10.1016/j.gde.2013.12.002

Table 1.

FDA-approved targeted cancer drugs in clinical use that are dependent for activity on the presence of a genomic alteration in the patient's tumour. Approved small molecule inhibitors and antibodies targeting specific drug-sensitizing mutations in human cancers. Although these mutations dramatically affect the likelihood of a given patient responding to a particular therapy, it is pertinent to note that in many cases these mutations are only present in a subset of that specific tumour type. Identification of these subgroups using next-generation sequencing technologies will become increasingly important for the management of cancer patients in the clinic

Tumour Gene (mutation) Prevalence of gene alteration (%) FDA-approved drug Year approved Therapeutic target Response rate in mutant tumours (%) Study
Chronic myeloid leukaemia BCR-ABL (translocation) >95 Imatinib 2001 ABL1 >95 Druker et al. [4]
Gastrointestinal stromal tumour KIT (mutation), PDGFRA (mutation) 85 (KIT), 5–8 (PDGFRA) Imatinib 2002 KIT, PDGFRA >80 Verweij et al. [8]
Non-small cell lung cancer EGFR (mutation) 10 Gefitinib, erlotinib 2003, 2004 EGFR 70 Mok et al. [6]
Chronic myeloid leukaemia (imatinib-resistant) BCR-ABL (translocation) >95 Dasatanib 2006 ABL1 >90 Talpaz et al. [7]
Breast cancer–node +ve HER2 amplification 15–20 Trastuzunab 2006 ERBB2 HR 0.48 Perez et al. [13]
Melanoma BRAF (mutation) 40–70 Vemurafenib 2011 BRAF >50 Chapman et al. [20]
Non-small cell lung cancer EML4-ALK (translocation) 2-7 Crizotinib 2011 ALK 57 Kwak et al. [5]
Melanoma BRAF (mutation) 40–70 Debrafenib 2013 BRAF 52 Hauschild et al. [10]
Melanoma BRAF (mutation) 40–70 Trametinib 2013 MEK1 22 Flaherty et al. [11]
Non-small cell lung cancer EGFR (mutation) 10 Afatinib 2013 EGFR/ERBB2 50 Yang et al. [12]
Breast cancer (metastatic) HER2 amplification 15–20 Trastuzumab 2013 ERBB2 44 Verma et al. [14]

Abbreviations: KIT, v-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homologue; PDGFRA, platelet-derived growth factor receptor; alpha polypeptide.

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