Exceptional Responders (BIG 16-04)

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Identify breast cancer patients with a truly remarkable clinical response to anticancer treatments and characterise their tumours molecularly

Exceptional Responders is an international academic programme led by BIG Headquarters that seeks to identify breast cancer patients with an exceptionally favorable, unexpected drug response, and to characterise their tumours molecularly.

Thanks to tumour sequencing, the identification and molecular characterisation of patients with exceptional sensitivities to anticancer therapies has emerged as a valuable way to understand and decode the mechanisms of drug response.

The hope is to discover clinically useful biomarkers, which will potentially lead to new clinical trials. It could enable scientists to revive drugs that have failed to demonstrate their benefit in past clinical trials by refining their target population, or accelerate the development of promising combination therapies based on new drug sensitivities.

Because BIG is a large, multi-continental network of 59 collaborative research groups and thousands of hospitals where a huge number of breast cancer patients are treated, its member physicians collectively encounter many exceptional responders.

This programme aims to provide physicians around the world with the necessary resources to undertake the molecular characterisation of patients with truly remarkable responses, as well as to gather and disseminate the findings in order to advance research more rapidly and efficiently.

Physicians from the BIG network signify through a secured IT platform that they have identified an “Exceptional Responder” candidate among their patients, and submit basic, non-identifiable information such as disease subtype, prior treatments and nature of the exceptional response.

Based on this information, a panel of experts selects the 10 patients with the most unusual drug responses that will undergo molecular analysis.

Pre-collected tumour tissue and blood samples of the participating patients will be analysed by a central laboratory (exome sequencing, RNA sequencing and ad hoc molecular analyses).

This will provide a detailed view of the genetic make-up of these tumours, helping physicians to identify the molecular factors behind exceptional response, and providing valuable insights into the mechanisms of drug sensitivity.

The treating physician will be involved throughout the whole process, including the discussion of the molecular results.

10 patients with invasive breast cancer, early stage or metastatic, who show an exceptional response to treatment are currently enrolled in the study.

The programme is coordinated by BIG Headquarters in collaboration with 16 academic research groups from the BIG network.

The programme was launched in 2018.

16 academic member groups from the BIG network are taking part in the programme, which allows to reach investigators from many institutions in over 20 countries in Europe, Asia and Latin America.

This programme is made possible thanks to a grant from the Breast Cancer Research Foundation (BCRF).