GIVING ACCESS TO INNOVATIVE TREATMENTS FOR CHILDREN IN RELAPSE OR WITH THERAPEUTIC FAILURE, FOR ANY TYPE OF CANCER (UNIQUE IN THE WORLD)
This trial has been co-financed with the Children without Cancer races of 2015 and 2019.
ESMART – European Proof-of-concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed of Refractory Tumors in children
About Precision Molecular Medicine
Research projects, some of which have been funded by Imagine for Margo, have helped to better understand the biological mechanisms behind the development and progression of childhood cancers. These mechanisms, as well as the genes involved, vary from patient to patient. Each tumor is unique, has its own molecular profile, and contains specific alterations. For example, the MAPPYACTS project, also led by Dr. Birgit Geoerger, showed that 70% of patients have targetable molecular abnormalities. Unfortunately, only 30% of them could benefit from a treatment targeting these abnormalities. To address this gap, Dr. Birgit Geoerger developed a unique and innovative trial, AcSé-ESMART.
About AcSé-ESMART trial
AcSé-ESMART is a phase I and II trial, unique in the world in pediatrics, that focuses on all cancers and leukemias in children who are relapsed or treatment-resistant. The principle of this trial is to offer multiple therapeutic options with innovative treatments, usually prescribed in combination, to target molecular abnormalities frequently found in these tumors. Treatment options are offered to children based on the molecular and immunological analyses of their tumor or leukemia, mostly conducted in MAPPYACTS as well as in other European programs. This trial significantly accelerates the access of children who are relapsed or treatment-resistant to innovative treatments, regardless of their cancer type.
To date, AcSé-ESMART has included 252 patients in 16 treatment arms targeting 17 molecular targets. Some of these arms are now closed due to the lack of effectiveness of the attempted therapeutic combination. However, others have opened over the years.
This large-scale program is led by Gustave Roussy within the framework of the AcSé program of the French National Cancer Institute (INCa).
Project Summary
- Sponsor: Gustave Roussy
- Principal Investigator: Dr. Birgit Geoerger
- Program Duration: 2016 – 2028
- Recruitment: 252 patients
- Countries Involved: France, Spain, United Kingdom, Netherlands, in the process of opening in Germany, Denmark, and Italy
- Funding by Imagine for Margo: €1,832,000
- Other Co-funders: The French National Cancer Institute (INCa) and the ARC Foundation
- Publications :
- The European MAPPYACTS Trial: Precision Medicine Program in Pediatric and Adolescent Patients with Recurrent Malignancies – PubMed (nih.gov)
- Phase II and biomarker study of programmed cell death protein 1 inhibitor nivolumab and metronomic cyclophosphamide in paediatric relapsed/refractory solid tumours: Arm G of AcSé-ESMART, a trial of the European Innovative Therapies for Children With Cancer Consortium – PubMed (nih.gov)
- Phase I or II Study of Ribociclib in Combination With Topotecan-Temozolomide or Everolimus in Children With Advanced Malignancies: Arms A and B of the AcSé-ESMART Trial – PubMed (nih.gov)
- First-in-child phase I/II study of the dual mTORC1/2 inhibitor vistusertib (AZD2014) as monotherapy and in combination with topotecan-temozolomide in children with advanced malignancies: arms E and F of the AcSé-ESMART trial – PubMed (nih.gov)
- AcSé-ESMART, a European precision cancer medicine proof-of-concept platform trial – PubMed (nih.gov)
French Professor Gilles Vassal, ITCC President, talks about ESMART at launch (interview in 2016)
