A collaborative hematology team based at Oncopole in Toulouse, France, is poised to lead a significant national study aimed at improving treatment outcomes for acute myeloid leukemia (AML). The initiative has secured approximately €2 million in funding and is expected to launch at the end of 2026.
AML is an aggressive cancer affecting the bone marrow and blood, primarily impacting adults between the ages of 18 and 60. Approximately 3,000 new cases are diagnosed annually in France. While treatments, typically involving intensive chemotherapy and sometimes bone marrow transplantation, can achieve remission in roughly half of patients, therapeutic advancements have been limited in recent years compared to other blood cancers.
The team, led by Professor Christian Recher at the Toulouse University Hospital, builds upon the foundation laid by the ongoing BIG-1 clinical trial (2015-2025). The newly funded BIG-2 program will enroll 1,300 patients across France – a substantial number given the incidence of AML – and aims to refine treatment strategies and improve long-term survival.
Standardizing Practices and Reducing Chemotherapy Doses
Professor Recher explains that the BIG-1 trial already yielded important benefits. “BIG-1 had already made it possible to standardize practices, which makes it possible to guarantee each patient the same level of expertise and access to therapeutic innovations in the face of this complex disease. Thanks to this protocol, we have also shown that by reducing the doses of chemotherapy, we do not do less well, on the contrary.” This finding challenges the historical trend in hematology of escalating chemotherapy doses in an attempt to maximize response rates in severe cases.
The BIG-2 trial will incorporate novel therapeutic agents alongside existing treatments, with the goal of improving both relapse-free survival and overall survival rates. A key focus will also be on earlier and more precise detection of relapse.
Detecting Minimal Residual Disease with Advanced Technologies
“New technologies will make it possible to analyze the level of remission after the first phase of treatment,” Professor Recher states. “If we better detect residual cancer cells that the biologist cannot detect with the naked eye, we improve the prevention of relapse.” Current methods of assessing remission often rely on morphological analysis, which can only detect cancer cells when they are present at relatively high levels (one in 200). BIG-2 will utilize more sensitive techniques like flow cytometry and molecular biology to identify minimal residual disease (MRD) – as few as one cancer cell in 10,000 or even 100,000.
For patients identified as having MRD after initial treatment, therapy will be intensified. Conversely, patients with no detectable residual disease will be monitored closely, with the aim of detecting any relapse before it becomes clinically apparent, allowing for earlier intervention.
The study benefits from funding of €1,991,000 from the General Directorate for Healthcare Supply as part of the INCa’s “Hospital Clinical Research Program in Cancer – PHRC-K 2025” call for projects.
Recent advances in understanding the molecular underpinnings of AML have led to the development of targeted therapies, including BCL2 inhibitors, FLT3 inhibitors, and IDH inhibitors. , twelve agents have been approved for treating specific AML subtypes. However, achieving optimal outcomes in this complex and heterogeneous disease requires specialized expertise and access to a range of therapies delivered under rigorous supportive care conditions.
Beyond the BIG-2 trial, initiatives like Klineo, a French medtech startup, are working to improve access to clinical trials for cancer patients. Klineo’s platform helps patients identify relevant trials in France and connect directly with participating centers, providing real-time updates and notifications. Founded in 2021, Klineo secured €2 million in funding in to expand its services and reach patients across Europe and the United States.
The ongoing research and collaborative efforts, such as those spearheaded by Professor Recher’s team in Toulouse, represent a concerted push to improve outcomes for individuals battling AML and to translate scientific discoveries into tangible benefits for patients.
