Role_of_microbiome_in_rare_cancers_in_Australia. Role_of_microbiome_in_rare_cancers_in_Australia

Combination immune checkpoint inhibitor (ICI) therapy with anti-CTLA-4 and anti-PD-1 blockade has demonstrated significant clinical activity across tumour types. Rare cancer patients have limited treatment options due to the scarcity of studies of novel treatment options, so they are often offered chemotherapies untested in their disease. ONJ2016-001/CA209-538 is a prospective, multicentre clinical trial of combination ICI (ipilimumab and nivolumab) in patients with advanced rare cancers, including biliary tract, adrenocortical, rare gynaecological and neuroendocrine cancers (https://clinicaltrials.gov/ct2/show/NCT02923934). The prespecified primary outcome measure is clinical benefit rate (complete response + partial response + stable disease >3 months) by RECIST 1.1. The prespecified secondary outcome measure is to identify common predictive biomarkers or signature in responding patient irrespective of rare tumour type. We hypothesise that the gut microbiome may be associated with cancer subtype, and may be predictive of clinical benefit to ICI in these patients with rare cancers. This study involves generation and analysis of stool DNA sequencing data, to pair with other biological and clinical data set, in order to potentially identify combinatorial biomarkers that associate with clinical benefit or other clinical efficacy / toxicity metric. This data is part of a pre-publication release. For information on the proper use of pre-publication data shared by the Wellcome Trust Sanger Institute (including details of any publication moratoria), please see http://www.sanger.ac.uk/datasharing/