Since our creation in 2011, we have increased fundraising actions to finance research projects aimed at better understanding and improving treatments for childhood cancer. We have allocated more than 20 million euros to French and European researchers, allowing them to carry out more than 50 research projects which have benefited more than 3,000 children. These have been carefully selected because of their excellence and their potential to improve care, as well as accelerate the development of new therapies, such as precision medicine, immunotherapy, CAR T-cells, etc.
BETTER UNDERSTANDING CHILDREN’S CANCERS
Understanding the factors and mechanisms linked to the appearance and development of childhood cancers and their resistance to first-line treatments is essential. In recent years, we have provided significant financial support to projects such as MICCHADO, MAPPYACTS and BIOMEDE, aimed at identifying all the molecular, genetic and even immunological factors involved in the most serious pediatric cancers.
The MICCHADO study, led by Dr. Gudrun Schliermacher (Institut Curie), included more than 512 patients and made it possible to determine all the factors specifically linked to high-risk cancers at the time of diagnosis in order to allow children to benefit from a precision medicine treatment. In addition to tumor DNA, researchers also analyze circulating tumor DNA. This analysis, carried out from a simple blood test, offers the advantage of monitoring the evolution of the tumor over time and adapting treatments, if necessary.
It is also crucial to better understand the mechanisms of resistance to treatments that tumors develop. For this, the team of Professor Ory (University of Nantes) recently demonstrated the activation of specific genetic regions in osteosarcoma cells resistant to doxorubicin, a chemotherapy commonly used for this bone cancer. These genetic domains promote the production of proteins that hinder the entry of the drug into the cell, thus inhibiting its therapeutic effect. His team showed that targeting these genetic sequences restores the sensitivity of these cells to doxorubicin, thus opening a promising therapeutic avenue that will need to be explored clinically.
BETTER CARE OF PEDIATRIC CANCERS
This year was also marked by major progress in the treatment of high-risk pediatric cancers.
- An abnormal protein, NTRK, is involved in pediatric and adult cancers, notably fibrosarcomas and brain tumors. Dr. Daniel Orbach (Institut Curie) tested a new drug in syrup form, which specifically targets this anomaly, and obtained spectacular results. “The tumors (fibrosarcomas) shrink visibly,” said Dr. Daniel Orbach. An international collaboration has been set up to detect NTRK in brain tumors, with the hope that this drug could be just as effective there.
- The “BIOMEDE” program on infiltrative gliomas of the brainstem, led by Dr. Jacques Grill (Gustave Roussy) and supported by Imagine for Margo since 2014 (1.6 million euros allocated), has produced unexpected results. For the first time, eight children are now the first long-term survivors of this cancer with a poor prognosis, and the sequencing of their tumor has made it possible to understand the reasons for this success, thus opening up promising perspectives.
- Finally, Imagine for Margo has participated in financing the BEACON project since its beginnings. This project aims to test several therapies on high-risk neuroblastoma, which represents 10% of solid tumors in children under 15 years of age and is generally diagnosed at a metastatic stage. In 2018, Dr. Moreno, project leader at the University of Birmingham, decided to test the effectiveness of the combination of chemotherapy and Bevacizumab, a therapeutic antibody that neutralizes the growth of blood vessels necessary for tumor progression. In total, 160 young people aged 1 to 21 from 11 European countries, including France, benefited from this treatment. The chances of recovery for patients who received the combination therapy have greatly increased and this new therapy has now become the standard treatment in the United Kingdom.
SECURING ACCESS TO INNOVATIVE TREATMENTS FOR CHILDhood CANCER
Pediatricians may be required to prescribe innovative drugs when children cannot be included in a clinical trial. At that time, they can receive an innovative treatment, within the framework of a Temporary Authorization for Use (ATU) or outside the Marketing Authorization for medicines already authorized for adults. Until now, the data of these patients remained within the hospital perimeter and were never centralized. With the aim of securing access to these innovative therapies, Dr. Pablo Berlanga (Gustave Roussy) launched the SACHA program, which collects data on the toxicity and effectiveness of these treatments in 31 French centers and, since this year , internationally. Ultimately, more than 650 patients who had received 51 new medications were recorded, and recommendations were established for some of them. SACHA was published this year and was recognized as a source of usable data by the French High Authority of Health (HAS).
THE 2024 HORIZON FOR IMAGINE FOR MARGO
In 2020, Imagine for Margo co-created the European Fight Kids Cancer consortium. This initiative, also supported by other European associations (Kick Cancer, Kriibskrank Kanner, Kika and Cris), makes it possible to pool funds in order to finance the most innovative, very large-scale research programs in Europe. The latter are selected for their scientific excellence and their ability to provide answers or concrete solutions for patients by an independent scientific committee.
This year, the Fight Kids Cancer call for projects is dedicated to brain tumors, which represent nearly 30% of pediatric cancers and for which therapeutic advances have been rare over the last 30 years. In total, more than 3,000 children in Europe, including 500 in France, are diagnosed with these cancers every year for which progress is more than expected by families.
Do you also want to advance the cause of childhood cancer? Find out how to help Imagine for Margo here. You can also donate directly here.
