On Thursday, September 12, 2024, we organized an evening dedicated to the 10th anniversary of our collaboration with Gustave Roussy to accelerate research against pediatric cancers. Researchers, pediatricians, donors, and volunteers gathered to celebrate 10 years of innovations benefiting children and adolescents with cancer. In total, over 9 million euros have been allocated by Imagine for Margo to projects carried out by Gustave Roussy. Attendees at this event were able to discover research programs that improve the understanding and treatment of each tumor for each patient, thus contributing to the acceleration of precision medicine.
Accelerating access to precision molecular medicine for all children affected by relapsed cancers.
Several research projects funded by Imagine for Margo have enhanced the understanding of the biological mechanisms underlying the development and progression of childhood cancers. These mechanisms and the involved genes vary from patient to patient. Therefore, each tumor is unique, has its own molecular profile, and contains alterations specific to it. To this end, the MAPPYACTS program was launched in 2017 to offer relapsed children sequencing of their cancer and to identify all the factors involved and the alterations that could be targeted with the help of specific medications.
MAPPYACTS (MoleculAr Profiling for Pediatric and Young Adult Cancer Treatment Stratification)
MAPPYACTS is a program aimed at conducting high-throughput molecular analysis and immunological assessment of tumors and leukemias in relapsed or treatment-failure children to guide them towards innovative treatments. This involves sequencing all the genes expressed in the tumor or leukemia to find, among other things, the anomalies that could be targeted by new medications, thus including them, for example, in clinical trials such as AcSé-ESMART.
Sequencing has been conducted on nearly 800 French, Spanish, Danish, Italian, and Irish children and adolescents, half of whom were under 13 years old. In the 2022 MAPPYACTS publication, of the 436 patients whose data could be analyzed, 356 (or 70%) presented at least one genetic alteration potentially targetable by treatment. Among them, 107 received at least one therapy specifically targeting their alteration (30%); for the remaining 70%, unfortunately, no dedicated treatment exists.
This large study has demonstrated its robustness and feasibility in routine practice, allowing patient care within the France Médecine Génomique 2025 (FMG 2025) program. Thus, in 2023, Imagine for Margo participated in the rollout of MAPPYACTS 2, which includes patients who have undergone complete sequencing of their high-risk tumor. The team in charge of this trial provides patients and their oncologists with therapeutic recommendations. This committee collects all complete clinical and molecular data from patients registered in FMG2025 or in equivalent international programs, facilitating patients’ access to innovative clinical trials. Therefore, the number one objective of this new program is to improve the survival of these patients whose prognosis is critical. This second phase of the MAPPYACTS project aims to include 1,800 patients in France and 500 patients in international programs.
MAPPYACTS has also generated essential data for research. Since the program’s inception, 35 new research projects have been validated by a scientific committee shared by Gustave Roussy and the Institut Curie, advancing research in various fields: analysis of genetic predispositions, evaluation of treatment responses, study of immune profiling, comparison with liquid biopsies, studies of treatment responses, and analysis of equitable access to new treatments.
Finally, this program, like MICCHADO (Institut Curie), has generated a vast amount of data that must be stored, analyzed, and shared easily. Thus, Imagine for Margo has also supported the BIG DATA program of the ITCC (420,000 euros). This ambitious project involves aggregating all genomic, immunological, and clinical data from several European research programs conducted in ITCC (notably MAPPYACTS, MICCHADO, as well as those funded under the FMG 2025 program). With the help of bioinformaticians, the aim is to store, structure, and aggregate all the data, knowing that a molecular profile of a child’s tumor represents 100 GB. As a result, this data has been accessible for free since July 2024. Additionally, this project uses machine learning and artificial intelligence tools to generate new knowledge to better understand pediatric cancers and find new therapeutic targets for developing new medications specific to childhood cancers.
AcSé-ESMART: a clinical platform to accelerate progress
Since nearly 70% of the genetic alterations identified in MAPPYACTS remain orphaned, meaning there are no medications to target them, it was necessary to establish a clinical trial unlike any other: AcSé-ESMART. This phase I and II trial covers all cancers and leukemias in children experiencing relapse or treatment failure. The principle of this trial is to offer several therapeutic options with innovative treatments, typically prescribed in combination, to target molecular anomalies 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 within MAPPYACTS and other European programs.
To date, AcSé-ESMART has included 252 patients across 17 treatment arms that have targeted 17 molecular targets. Some of these arms are now closed due to a lack of efficacy of the attempted therapeutic combination. However, others have opened over the years. This large-scale program is led by Gustave Roussy as part of the AcSé program of the National Cancer Institute (INCa).
Brain Cancers in Children and Adolescents
Central nervous system tumors are the most common solid tumors in children. They account for nearly 30% of tumors, just ahead of leukemias. More than 3,000 children are affected each year in Europe, with around 500 per year in France. Generally, these cancers are classified into two categories based on their microscopic morphology: low-grade tumors (benign) and high-grade tumors (malignant). Among the latter, gliomas, including certain forms such as midline gliomas or infiltrating brainstem gliomas, are common, very aggressive, and have a very poor prognosis. Indeed, these tumors are infiltrative and located in vital brain areas, preventing any surgical intervention.
At the creation of Imagine for Margo, our knowledge was still insufficient to attempt targeted therapeutic approaches that would complement radiotherapy, which unfortunately remains inadequate. Thus, over 10 years, Imagine for Margo, through the ITCC and the SFCE, has chosen to fund numerous projects on brain tumors, many of which are conducted at Gustave Roussy.
Vinilo: A New Therapeutic Combination for Low-Grade Pediatric Gliomas
Low-grade gliomas are usually treated with chemotherapy (Vinblastine), which in most cases allows them to regress or stabilize. However, after stopping chemotherapy, in more than 2 out of 3 cases, the tumor reappears.
The European ITCC network selected this phase I and II project in 2012 for its ability to test the effectiveness of a combination of vinblastine with a new treatment for these pediatric tumors. It is also the very first research project that was funded by Imagine for Margo.
The goal of this trial is to test a combination of two drugs to stabilize the tumor: vinblastine combined with nilotinib. These molecules prevent the establishment and maintenance of the blood vessel network created by tumors, which is essential for their growth.
Nilotinib is a new medication never before used in children. The study recruited 109 children and adolescents and did not report any particular side effects. However, inclusions had to be halted because patients treated with vinblastine alone showed significantly longer progression-free survival than those receiving the therapeutic combination.
The BIOMEDE Adventure
BIOMEDE is the most ambitious clinical trial offered to children with infiltrating brainstem glioma. To address the grim prognosis of this disease, Imagine for Margo decided to support Dr. Jacques Grill and his team based at Gustave Roussy (€1,605,000 since 2014) for this promising trial that has changed clinical practices for this cancer.
Early Victories Thanks to BIOMEDE 1
Conducted between 2014 and 2019, the first phase of BIOMEDE demonstrated the utility of biopsy at diagnosis as a standard, thereby allowing for the systematic molecular profiling of high-grade gliomas in each child. Based on the results, Dr. Jacques Grill and his team compared the efficacy of three medications (erlotinib, dasatinib, and everolimus), combined with radiation therapy. This first phase involved 70 centers located in 9 European countries, Australia, and New Zealand, and included 233 patients. This randomized phase 3 trial is the largest ever conducted for infiltrating brainstem glioma. The toxicity profile and slight improvement in median overall survival obtained during the study led to the retention of everolimus as the reference treatment for the second part of the trial, BIOMEDE 2. This treatment allowed, for the first time in the world, the saving of the first children from this cancer, including young Lucas.
“Lucas is a young patient from Belgium who came to France to participate in the BIOMEDE 1 trial. The treatment that was randomized for him was everolimus. His disease responded remarkably well to the treatment: his neurological condition returned to normal, and the radiological signs of the tumor disappeared with normalization of the appearance of the brainstem outside the biopsy pathway. After 5 years of treatment, which I hesitated to stop, he confessed to me that he was no longer regularly taking his medication. We then all decided together to stop the treatment. That was a year ago, and everything continues to go well since then. We continue to monitor his MRI, which remains unchanged. He is the only patient with a biopsy-proven infiltrating brainstem glioma who is in complete remission. We believe we have understood why and are now trying to reproduce what happened naturally in Lucas with medications in the cells of other children in the lab.” Dr. Jacques Grill
This success, along with the results obtained from the molecular profiling of tumors collected through biopsy, has allowed for the correlation between certain mutations and the severity of these gliomas. Consequently, at the end of this project, in addition to BIOMEDE 2, new research projects are emerging (see below) to find therapies that correspond even more closely to these specificities.
BIOMEDE IA: Artificial Intelligence in the Service of Research
Whole-genome sequencing of tumors, medical follow-up, and imaging analyses of patients included in the BIOMEDE trial have provided a vast amount of data, the analysis of which is essential for a better understanding and treatment of infiltrating brainstem glioma. This is the objective of BIOMEDE IA, which uses artificial intelligence to analyze this information obtained after the biopsy of each tumor. Algorithms allow for the identification and matching of certain factors that are undetectable by human analysis.
BIOMEDE 2: Accelerating the Cure for This Cancer
This trial, which opened on September 23, 2022, aims to compare the therapeutic effect of ONC201 to everolimus, the reference treatment, still in combination with radiation therapy. Opened in 10 European countries, this trial plans to include 368 adult and child patients over 4 years. ONC201 is the first medication of a new class of anticancer drugs, targeting the energy metabolism of cancer cells and has shown, in the United States, signs of efficacy in some patients relapsing from a malignant midline brain tumor. This efficacy is due to the presence of a mutation within the tumor, H3K27M, which will be detected through tumor sequencing from a biopsy.
This trial should take place in 40 European centers in France, Great Britain, Italy, Germany, Austria, Spain, Switzerland, Denmark, and the Netherlands.
Exploration of Genetic Alterations Identified in BIOMEDE
Malignant gliomas in children are not at all similar to malignant gliomas in adults. This is primarily due to very specific mutations in these pediatric tumors that have never been described in other cancers. These mutations focus on genes that regulate DNA organization and its expression. Furthermore, these alterations make the DNA even more vulnerable, generating secondary mutations that also need to be explored. These discoveries, particularly enabled by BIOMEDE, are redirecting therapeutic research toward these alterations, which Imagine for Margo, notably through Fight Kids Cancer, has chosen to support.
- PATOI – Phase I/II Study of Combined Inhibition of PARP and Topoisomerase Type 1 in Malignant Brain Tumors (Dr. Samuel Abbou) : Poly (ADP-ribose) polymerase inhibitors (PARPi) are drugs developed to treat cancers caused by specific genetic mutations. PARPi work by preventing cells from repairing themselves, leading to the death of these cancerous cells. It has been shown that the combination of PARPi with chemotherapy improves outcomes in several cancers and is currently being studied for use in pediatric cancers, such as brain tumors. The aim of this project is to study NIRAPARIB, a type of PARPi capable of crossing the blood-brain barrier, in combination with IRINOTECAN, a chemotherapy agent used for several brain tumors. The first part of the study will focus on defining a well-tolerated dose and administration schedule. The second will evaluate the efficacy of this new treatment. This study will be part of the AcSé-ESMART platform study and was selected in 2023 for its excellence by the Fight Kids Cancer scientific committee. The study plans to enroll 58 patients over a period of 2 to 3 years in 7 European countries. If this study shows encouraging results, this new therapeutic combination will be used for other pediatric tumors. Funding from Fight Kids Cancer: €513,499 (including €186,169 financed by Imagine for Margo).
- Targeted Approach to DNA Repair in H3.3 Mutants of High-Grade Pediatric Gliomas (Dr. Béatrice Rondinelli) : High-grade pediatric gliomas frequently express mutated forms of a protein called histone H3.3, which has established functions in DNA damage repair. These cancer cells therefore exhibit a repair defect for DNA anomalies, partially compensated by the polynucleotide kinase-phosphatase (PNKP), making it a prime target in pediatric gliomas. Initially, the team will analyze the role of this protein in H3.3 mutant glioma cells by characterizing the genetic determinants and the mechanism underlying the anti-cancer effect of targeting PNKP. Current chemotherapy and radiotherapy treatments do not completely and durably kill tumor cells. Therefore, the combination of these treatments with PNKP targeting will soon be evaluated to reduce the proliferation of cancerous cells in pediatric gliomas. Secondly, the team will leverage preclinical models to predict treatment efficacy in affected children. This work is expected to stimulate the development of specific PNKP inhibitors and pave the way for new phase I clinical trials aimed at better treating this grim prognosis disease. This project was selected for its excellence by the SFCE scientific committee in 2023. Imagine for Margo supports this project with €40,000.
- Analysis of Tumors from a New Subtype of Diffuse Midline Glioma, H3 K27 and MAPK Co-Altered, and Modeling Their Treatment Response (Dr. David Castel) : The WHO classification recently categorized midline gliomas, with the most emblematic pediatric form being infiltrating brainstem glioma, based on a common biological anomaly (H3K27M). This mutation prevents tumor cells from differentiating, keeping them in a stem cell state, allowing them to resist treatments and disseminate through the central nervous system. In addition to the H3K27M mutation, other anomalies may accompany it, altering the tumor’s behavior. Researchers from Dr. Jacques Grill’s team, including Dr. David Castel, have identified a subgroup of these diffuse midline gliomas that activate a particular signaling pathway (MAP kinases; via a mutation of BRAF or FGFR1). This subtype of diffuse midline glioma is less aggressive than classical forms, and there are even patients who survive more than five years after diagnosis. By analyzing the gene expression profile of these specific gliomas, many treatment pathways have been identified and must now be explored. Dr. Castel’s team will investigate this second line of mutation (BRAF or FGFR1) and how it interferes with the oncogenic process developed in cells carrying the initial alteration, H3K27M. This knowledge could lead to the development of new treatments targeting these various alterations, which represent up to 20% of these aggressive gliomas. They will then develop cellular and murine models to interrogate the biology of these particular tumors and their response to several promising therapeutic avenues. These results will be complemented by an analysis of the efficacy of ONC201 and ONC206, of which ONC201 is currently being tested in the BIOMEDE 2 trial. This project was selected for its scientific qualities by the SFCE scientific committee in 2024 and is supported by Imagine for Margo with €89,000.
Improve Our Understanding of Sarcomas and Osteosarcomas and the Survival of Patients Affected by These Cancers
For many years, the life expectancy of children and adolescents affected by these cancers has progressed very little. This makes these cancers, which are usually metastatic at diagnosis, a significant unmet need for children, for whom we must accelerate the development of new innovative drugs. Imagine for Margo has selected numerous projects focused on these diseases and has worked to ensure that the 2026 call for projects from Fight Kids Cancer is dedicated to research on these conditions.
Sample Collection to Establish a Biological and Genomic Research Network on Osteosarcomas in France
Osteosarcoma is the most common malignant bone tumor in adolescents and young adults (approximately 100 to 150 new patients per year in France, with a cure rate of 70%).
The majority of these osteosarcomas (70%) occur in young people aged 10 to 25 years. Additionally, in 10 to 20% of cases, metastases are present at diagnosis. Of these, 85 to 90% are located in the lungs, and others may subsequently occur in a different bone than the one initially affected.
The goal of the project was to define and characterize subclasses of osteosarcoma at diagnosis based on genetic and biological profiles, considering the tumor cell and its bone and immune microenvironment. This project has allowed for the optimal use of the significant genetic and biological data from the collection of tumor, blood, and urine samples generated prospectively during the OS2006 protocol. This database enables other research teams to assess the clinical relevance of their preclinical data.
This project allows for the exploration of new targeted therapeutic avenues for patients (according to the osteosarcoma subclass) and has created a database usable by all researchers.
This project has enabled:
- The creation of the OsteoBioNetwork Database to store, share, and visualize all biological and genomic data generated by the various research teams on the OS2006 cohort (434 patients).
- The retrieval of additional data from other databases.
- The integration of this collection into cBioPortal.
- The establishment of a rights management system within the database and the integration of cleaned and consolidated biological data into OsteoBioNetwork.
- The launch of the “BoOST-DataS” project, which allows OsteoBioNetwork to expand throughout France and collect data on osteosarcomas, particularly from MAPPYACTS or MICCHADO.
Identification of New Therapeutic Approaches for Desmoplastic Round Cell Tumors (Sarcoma) Based on the Destabilization of Abnormal Fusion Proteins
Ewing’s sarcoma is the second most common malignant bone tumor after osteosarcoma in adolescents and young adults (70% of cases are between 5 and 25 years old). Ewing’s sarcoma affects approximately 80 to 100 new patients per year in France. The current treatment for these tumors relies on chemotherapy (combining several drugs), radiotherapy, and surgery. Unfortunately, relapses are almost systematic when surgery has not been able to remove the entire tumor, and little therapeutic progress has been made in recent years.
The event leading to desmoplastic round cell tumors is well known. It involves the abnormal joining of two chromosomes, resulting in the formation of a fusion protein (EWS-WT1).
This project aims to identify new therapeutic approaches for this form of sarcoma by searching for drugs that can either prevent the production of the abnormal protein or promote its degradation within the cell. To do this, the team has proceeded as follows:
- Create a “tagged” protein, meaning fluorescent, so that its quantity can be measured through fluorescence measurement.
- Evaluate, among over 1500 drugs, which ones reduce the fluorescence of the cells. 79 have shown signals of efficacy, notably PARP and ATR inhibitors, opening therapeutic avenues to explore via in vivo models. These have been generated using biopsies taken from patients followed at the Curie Institute or Gustave Roussy. The creation of such models is crucial for future research, as there are currently very few for Ewing’s sarcoma.
The leader of this project, Dr. Sophie Postel-Vinay, received the first Fight Kids Cancer & St Baldrick’s Foundation Areci Innovation Award at the 5th International SIOPE Congress held on May 15, 2024. Dr. Sophie Postel-Vinay was recognized for her significant capacity to innovate in pediatric cancers and the high quality of her research and publications. The funding of 1 million euros will allow her, over four years, to develop innovative therapeutic approaches to better treat children with sarcoma.
REGO-INTER-EWING 1: Phase Ib Study of the Combination of Regorafenib and Chemotherapy for Patients with Multimetastatic Ewing Sarcoma
The effectiveness of new treatments for Ewing’s sarcoma has been disappointing over the past few decades. Furthermore, no new drug has been successfully introduced as a first-line treatment. Among the drugs tested, initial clinical data suggest that strategies using multi-targeted tyrosine kinase inhibitors (TKIs), which have anti-angiogenic activities, are among the most effective.
Several TKIs have been and are currently being tested as monotherapy in patients with relapsed or refractory Ewing’s sarcoma, with encouraging results in phase II trials. Among them, regorafenib has shown promising activity in relapsed Ewing’s sarcoma. However, this drug has never been combined with the intensive chemotherapy regimen VDC/IE (which includes vincristine, doxorubicin, cyclophosphamide, ifosfamide, and etoposide). Therefore, this combination must be evaluated to avoid reducing the dose of the current standard treatment and thus maintain its effectiveness.
Rego-Inter-Ewing 1 is a clinical trial designed to test the feasibility of regorafenib with conventional chemotherapy. It consists of a phase Ib that only recruits patients with multimetastatic Ewing sarcoma, representing the highest unmet medical need in pediatric cancers (2-year event-free survival: 33%, similar to patients who are relapsed/refractory).
Accelerating the Development of Immunotherapy for Pediatric Cancers
In its early days, immunotherapy, which involves boosting the patient’s anti-tumor immune response, was primarily used to combat metastatic adult melanoma, on which this therapy proved effective. Thanks to its excellent results and advances in research, immunotherapy is now increasingly recommended early in the management of the disease and for a growing number of cancers: melanoma, lung, liver, breast, kidney, bladder cancers, or in cases of Hodgkin lymphoma (a type of white blood cell cancer). Immunotherapy is becoming a promising future solution. Thus, we must increasingly prescribe these therapies for children, even though the obstacles are greater than in adults. “Unlike adult cancers that respond to immunotherapy, pediatric cancers have few T cells and therefore respond poorly to current immunotherapies. We need to adapt and evaluate new specific immunotherapy strategies for children.” Pr Véronique MINARD COLIN, pediatric oncologist at Gustave Roussy.
NIVO-ALCL: Evaluation of Nivolumab for Anaplastic Large Cell Lymphoma
Non-Hodgkin lymphoma (NHL) in children is a cancer that originates in lymphocytes and is distinguished into several categories. Anaplastic large cell lymphoma (ALCL) is more common in young children and usually originates in T lymphocytes. This disease can appear in lymph nodes in the neck or other parts of the body (skin, lungs, bones, digestive system, for example). At the time of diagnosis, ALCL often has already spread to other parts of the body and causes multiple symptoms. The main treatment for childhood forms is chemotherapy, and the 5-year event-free survival rate is approximately 70%. 25% of children relapse after first-line treatments and must therefore undergo sometimes very toxic treatments, particularly in the case of allogeneic transplantation, which are not always effective.
NIVO-ALCL is a phase 2 immunotherapy trial evaluating the effectiveness of Nivolumab in children and adult patients with ALCL positive for the ALK alteration, in relapse or refractory. ALK (Anaplastic Lymphoma Kinase) is a genetic alteration that causes the cell to lose its ability to control its proliferation. Nivolumab is an antibody that binds to the PD-1 receptor present on the surface of the patient’s T lymphocytes. This immunotherapy will prevent the tumor cell from recognizing this protein and thus increase the ability of T lymphocytes to destroy it.
The objective of this program is to evaluate the effectiveness of Nivolumab to avoid allogeneic bone marrow transplantation. This trial involved two cohorts (23 patients). Patients received treatment including chemotherapy combined with Nivolumab and were reviewed by doctors 8, 16, and 24 weeks after entering the study for radiological evaluation, then every 12 to 16 weeks until the end of treatment. Finally, anti-ALK antibody levels were measured at 8, 24, and 52 weeks, indicating the effectiveness of Nivolumab in stimulating the patient’s immune system against the tumor. Patients will be followed for 3 years after the end of the study treatment.
Result: Significant anti-tumor activity was observed in patients (several are in complete remission), preventing them from undergoing allogeneic bone marrow transplantation.
Our New Fight Kids Cancer Projects 2024 to Boost Immunotherapy for Children with Brain Cancer
MiMicsKids: Reflecting Microglia-Cancer Cell Interactions to Improve Immune Response Against Gliomas
As previously mentioned, pediatric midline gliomas are associated with extremely low survival rates. Therefore, it is urgent to develop new treatments, particularly immunotherapy, which could prove to be very effective. Microglial cells, which are macrophages located in the brain, are among the most abundant immune cells in these tumors and are often associated with resistance to treatments. Targeting these cells could lead to decisive advances. This project proposes to use 3D culture models derived from patient tumors (immune-tumoroids and neuronal organoids containing microglial cells).
These tools will be used as a platform to optimize immunotherapeutic approaches and discover new targets. For these treatments, the team will compare molecular factors between “responding” and “non-responding” organoids to identify new mechanisms of treatment resistance and predictive biomarkers of treatment response. This knowledge will lead to the design of new therapeutic combinations that will be tested, in a final phase, on the models established during this study.
The ultimate goal is to make these new preclinical models available to the scientific community, to create a powerful platform for conducting studies on the immune system and glioma cells, and to discover and test new drugs more effectively to increase survival for pediatric patients with these aggressive brain tumors.
ELICIT: Early-Phase Clinical Trials Platform in Immuno-Oncology for Pediatric Malignant Gliomas
Except for a few very specific cases, immunotherapy approaches have not fulfilled their promises for pediatric solid tumors. Two main explanations can be put forward: the lack of information on the immunological context of brain tumors and the complexity of conducting efficacy trials with these agents.
The goal of this innovative project is to establish a platform to accelerate the transition from phase 1 data to phase 2 trials to convincingly boost the effectiveness of these treatments. The infrastructure of this platform will provide 1) a strategy to develop these agents within and outside of the ITCC (defining the patient population to maximize efficacy evaluation), 2) appropriate control populations, including synthetic arms, and 3) immune and radiological references that will allow comparisons between trials and facilitate phase 2 trials.
Additionally, this platform will start with an innovative trial combining immune checkpoint blockers with MEK inhibitors. The platform will also provide preliminary phase 1 data collection and establish two phase 2 trials: one with CAR T cells and the other with oncolytic viruses, which destroy tumors and further activate the patient’s immune system. This large program will allow for the development of other innovative immuno-oncology approaches in the future.
Structuring Clinical Practices Better: The SACHA Project Secures Access to Innovative Therapies
France is a pioneer in Europe in the development of new drugs in pediatric oncology. For example, from 2015 to 2018, more than 2,200 patients were included in 34 clinical trials of the ITCC. However, despite this strong momentum, the therapeutic offering remains insufficient, and pediatric oncologists and hematologists prescribe innovative drugs either through early access or compassionate use, or off-label for drugs already authorized for adults. For all these medications, safety and efficacy data are very rarely collected.
To secure access to these innovative therapies outside of clinical trials, the New Medicines Committee of the SFCE has established a prospective observational cohort study to monitor the use of innovative molecules in oncology and hematology for children, adolescents, and young adults who are in therapeutic failure or relapse and not eligible for a clinical trial. This is the SACHA program, which involves prospective collection of toxicity and efficacy data for these innovative therapies.
The objectives of the SACHA study are to:
- Frame and secure access for children, adolescents, and young adults in therapeutic failure and not eligible for a clinical trial to innovative molecules
- Frame off-label prescriptions and early access requests
- Secure the use of medications through an organized follow-up of patients
- Evaluate the tolerance and efficacy of these treatments under real-world usage conditions
- Draft recommendations
International Deployment
SACHA International builds on the experience of the French pilot, whose success has confirmed the need for this type of registry. SACHA International is an initiative of the Consortium for Innovative Therapies in Children with Cancer (ITCC) and plans to include 500 patients per year for three years across several European countries, as well as in Australia and New Zealand.
The consortium agreement was signed in May 2023 between France, Spain, the United Kingdom, Ireland, Denmark, and Austria (with the first patient included outside France on June 14, 2023).
