We support the research project of Professor Marie-Anne Debily (Gustave Roussy)
Professor Marie-Anne Debily is a researcher at Gustave Roussy in the only laboratory in France entirely dedicated to pediatric gliomas (co-directed by Dr. David Castel and Dr. Jacques Grill, a pediatric oncologist at Gustave Roussy, who is leading the only international comparative clinical trial devoted to brainstem and midline gliomas). This team focuses in particular on diffuse midline gliomas (DMG), tumors still considered incurable.
Largely supported by philanthropy, the work carried out by this team has contributed to advancing knowledge about these tumors. In particular, it has established that DMG is not a single disease, but rather encompasses four different subtypes, as has since been recognized by the World Health Organization in its new classification of central nervous system tumors.
Despite numerous clinical trials, the survival of these children has not been significantly improved over the past 70 years.
Professor Marie-Anne Debily established a research project in the laboratory aimed at identifying, without preconceived notions, all the vulnerabilities of DMG cells in vitro, using a methodology that allows for the silencing of genes one by one. This work led to the identification, in 2019, of a protein vital for the survival of diffuse intrinsic pontine glioma (DIPG) tumor cells, but much less essential for normal cells. In preclinical models, the inhibition of this protein resulted in the highly efficient and specific death of cancer cells. The researchers then focused on better understanding the role of this protein, about which little knowledge was available, particularly because it had never been shown to be directly involved in any pathology.
The teams at Gustave Roussy are now working on developing a treatment to block this protein, a true Achilles' heel of DMG, in order to open a new therapeutic avenue that could mark a turning point in the management of pediatric brain cancers.
Drug development is a long and expensive process, including identifying the partners of this protein in DMG cells, determining the region of the protein crucial for its activity and which will need to be targeted by the drug, designing and testing a large number of molecules targeting the protein in our preclinical models, and optimizing the formulation of the selected molecule to allow its penetration into the brain, maximize its effectiveness and prolong its effect.
Diffuse infiltrating gliomas of the brainstem are considered a rare disease, affecting approximately 70 children per year in France, which limits the investment of the pharmaceutical industry in the development of such drugs and makes your support in this project essential.
