ALL-TARGET (Precision medicine randomized trial for patients with relapsed or refractory T-cell Acute Lymphoblastic Leukemia based on a functional approach)

This is an international project funded by the ATTRACT Iniative (Accelerate Together Rare Cancer Treatment) aiming to accelerate drug development for rare cancers.

From a previous work, the research already derived targeted therapeutic options (TTO) based on the combination of inhibitory molecules targeting most of the molecular alterations observed in T-ALL. This approach has been validated in vitro on leukemic cells from patients and in vivo in murine models mimicking the human leukemia.
We built and validated a drug testing assay based on fresh leukemic cells. A clinical decision can be made within a week based on the results of the assay and by integrating other information from conventional assays (NGS, immunophenotyping). The proposed molecules are already prescribed in other diseases with a known safety profile.
The principles of the ALL-TARGET project are currently tested in an observatory study in France. Patients diagnosed with a relapse/refractory T-ALL (R/R T-ALL) are proposed to have a centralized drug testing. The assay is, informative in 76% of the cases. Based on the individual results from each sample of leukemic cells, a validation committee meets every 2 weeks (and more frequently if needed) in order to derive therapeutic proposals based on off-label use of targeted medications. From January 2023, 40 patients were discussed and 25 started a targeted therapeutic option based on their drug testing results.
The ALL-TARGET project aims to extend and validate this first experience to other countries in Europe.