with an increased focus on precision medicine, a revolutionary new approach to cancer treatment, patient care and drug development has emerged – tumour agnostic therapies.

tumour agnostic therapy is a type of therapy that uses drugs or other substances to treat cancer based on the cancer’s genetic and molecular features without regard to the cancer type or where the cancer started in the body.

while agnostic is actually a misnomer – agnostic from greek means ‘lacking in knowledge’ – whereas with these approaches there is comprehensive information about the tumour, and very gnostic [from greek gnostos meaning known].

tumour agnostic therapies target specific mutations or genomic alterations [biomarkers] regardless of tumour site of origin. pan-tumour approaches signal an important new paradigm in cancer care.

in canada, pembrolizumab was granted approval for the treatment of patients with unresectable or metastatic, microsatellite instability–high [MSI-H] or mismatch repair deficient [dMMR] solid tumours and subsequently followed by the approval of larotrectinib and entrectinib for solid tumours with neurotrophic receptor tyrosine kinase (NTRK) gene fusion.

at least 10 further tumour agnostic therapies are in development, based on a range of genetic mutations, including mutations in the RET gene, found in 2.21% of all cancers, and mutations in the neuregulin 1 gene [NRG1], which is found across solid tumours in lung, pancreas and breast tissue. 

tumour agnostic indications create a new way of thinking about treatment, targeting patients based on a highly specific rare driver mutation [for example, present in less than 1 percent of solid tumours] rather than on tumour type. this approach has the potential to completely change the way patients are treated, but there are questions about how our regulatory and health systems would need to adapt to this new paradigm. 

could this change also mean a change in the way patient groups function?