The research

The programs that we cofinance

Since the creation of the association in 2011, we have allocated MORE THAN 20 MILLION EUROS to 61 translational and clinical research programs in France and Europe to accelerate research against childhood cancer. Clinical projects have benefited over 7,200 children and have tested 119 new therapies, some of which have proven to be highly effective (Biomede, Tag-N-Track, BEACON, AcSé-ESMART, etc.).

Our strategy consists of funding fundamental and translational research programs to better understand the causes of childhood cancer and resistance to treatments, as well as early phase clinical trials that allow children who suffer relapse or therapeutic failure to benefit from innovative personalized treatments, thanks to precision medicine.

Since 2012, the programs that we have co-funded have come from recommendations from experts in pediatric oncology: the European consortium ITCC *, the SFCE ** and, since 2020, the scientific committee of Fight Kids Cancer.

* Innovative Therapies for Children with Cancer
** Société Française de lutte contre les Cancers et leucémies de l’Enfant et de l’adolescent

OUR MOST PROMISING ADVANCES AGAINST CHILDHOOD CANCERS

Accelerating precision medicine. At the inception of Imagine for Margo, unfortunately, when frontline treatments failed, few innovative therapeutic solutions were available for children. Since 2016, Imagine for Margo has supported major sequencing programs for every child with relapsed cancer (MAPPYACTS) or at high risk (MICCHADO). More than 3,600 children have benefited from this program, now integrated into the France Genomic Medicine 2025 framework. These programs have identified numerous molecular targets, for two-thirds of which unfortunately no drugs currently exist. The specialist committee analyzing children’s molecular profiles can guide their patients to the AcSé-ESMART program, a clinical platform accelerating the development of precision medicine, which has included 254 children with high-risk and relapsed cancers.

Tag-N-TRACK: the first major success of precision medicine. Thanks to the MAPPYACTS project, it was possible to identify all children with the NTRK anomaly within their tumor and provide them with a new drug: larotrectinib, which has a 94% response rate, no side effects, and is administered as a syrup twice daily.

Our first victories against Diffuse Intrinsic Pontine Glioma (DIPG). The BIOMEDE program is the largest clinical trial conducted on this disease, which typically allows only a few months of life expectancy after diagnosis. This trial has improved the understanding and treatment of this disease. For the first time worldwide, 8 children are long-term survivors of this disease. This project continues through BIOMEDE 2, the PATOI clinical trial, and a program led by Dr. David Castel.

Life expectancy for children with Neuroblastoma is improving! Thanks to the BEACON study, and subsequently BEACON Immuno, it is now possible to better treat children with relapsed or refractory neuroblastoma. These new treatment protocols, one of which has become the standard in the UK, have improved long-term survival for children. However, we need to go even further, particularly through a better understanding of the disease (DigiTwins), prevention of relapses (Prevention of Neuroblastoma Relapse), and more effective and targeted treatments (RESTRAIN).

We are accelerating Immunotherapy. Although these projects are recent, some have begun to show promise. The NIVO-ALCL project improves survival for children with resistant forms of lymphoma using an immunotherapy drug, Nivolumab (response rate: 50%). We also fund projects that enhance the effectiveness of CAR T-cell therapies, particularly against solid tumors (Imagine, University of Nantes, GLO-BNHL, CARBEMED).

Access to innovative therapies is now secured through SACHA. This study, unique in Europe, collects all efficacy and toxicity data on new drugs prescribed under early or compassionate access. More than 779 patients who received 79 therapies have been included in this study, recognized by the French National Authority for Health (HAS) and the National Agency for the Safety of Medicines and Health Products (ANSM), and deployed internationally since 2023.