Category: Study Design
Labels: rationale
Related questions: NA
Creation date and Author: Unknown, October 23th 2024
Last Update and Author: Merry K., June 12th 2025
Therapeutics for cancer remain a major challenge in medicine and are the concerns of most pharmaceutical companies and academic laboratories. Worldwide, approximately 20 million new cancer cases and almost 10.0 million cancer deaths occurred in 2020, reaching the second cause of mortality, and the incidence rate rises every year in all countries. It appears that some cancers show an extremely poor prognosis and do not benefit from any advanced therapeutics that leads to the major challenge in oncology drug discovery: the identification of tumor vulnerabilities that can lead to novel therapeutic targets, and linking these vulnerabilities to specific patient populations that are likely to benefit from pharmacological inhibition of these targets.
In regards to a five-year relative survival rate (described as the percentage of patients with a disease alive five years after the disease is diagnosed), less than 10% of patients suffering from those highly aggressive cancers actually survive, the Outliers. Survival rate varies among the population and is hardly correlated as a function of age, sex, ethnicity, environment as well as the background history of the patient.
If one could understand the reasons behind this exceptional resistance, one could probably open the way to new medicines or repurposing strategies. These patients are usually not included into large-scale studies because they are not representative, but also their small numbers make them unavailable for significant analysis.
Analyzing and understanding Outliers experiencing an exceptional and overall survival (OS) > 3-5 years after advanced stage diagnosis, may help identify new oncology targets and open the way to new medicines.
Very few studies were engaged to understand why and how some patients survive after having a dismal prognosis. This might be due to the small number of cases of a specific cancer in each individual institution. To avoid these obstacles, we propose to build large cohorts with the help of well-recognized Cancer Centers in more than 50 countries.
The objective of our research is to build, thanks to a multicenter approach, these large cohorts of cancer survivors to conduct the first extended and coordinated study between cancer centers and unravel the molecular mechanisms behind this aberrant survival.
Our scientific approach aims at finding biological signatures that are common to our cohorts and associated with patients with unexpected survival compared to patients with standard survival that could enable potential future therapeutic approaches.
This study will focus on three cohorts among the most aggressive cancers: metastatic pancreatic ductal adenocarcinoma (PDAC), glioblastoma IDH wild type (GBM-IDHwt), extensive small cell lung cancer (SCLC).
These three types of cancer account respectively for 90% of all pancreatic cancers, 50% of brain tumors and 13% of lung cancers and constitute a rising burden for all healthcare systems.