Pursuing new treatments for Neuroblastoma children

Dear Cure First and Project Neuroblastoma supporters,

Last month, I had the amazing opportunity to present my work at the Annual Meeting of the American Association of Cancer Research (AACR) in Chicago where 20,000 scientists came together to present their most up-to-date research. My poster: “Pediatric Cancer: organoid patient derived cells guide personalized neuroblastoma treatments” was well received and resulted in fruitful discussions that will help bring this exciting project to the finish line. This was also a great opportunity to catch up with our collaborator Dr. John Maris from Children’s Hospital of Philadelphia, who was enthusiastic about our results as well.

Our initial results with the Informer Set drug library (320 compounds) highlighted that each sample was unique as well as and the cancer pathways involved in neuroblastoma (NB).  Scientific rigor requires to repeat the screens, this time with an updated, clinically relevant library (PARIS library: 120 FDA approved drugs). Indeed, I found additional NB patient derived samples to the one mentioned in our January report, which are extremely sensitive to the same targeted agent, that is FDA approved for ovarian and breast cancer. Whereas these targeted agents’ sensitivity in one of our model is well explained by mutations in the DNA repair pathway, the other samples responding to them don’t have any alterations in this pathway. This is a great example of how functional drug screening is an effective approach for the discovery of drug responses overcoming what cannot yet be explained by genomic data alone. The data analysis was carried on with the help of an App made by our analyst Hallie Swan, where all the drug screenings results are incorporated and available to browse.  

We confirmed the original pathological diagnosis using immune-histochemical staining and FISH (Fluorescence in Situ Hybridization) for the MYCN status. This data was generated thanks to an amazing collaboration with Chantal Pauli MD at the Institute of Pathology and Molecular Pathology of Zurich (Switzerland).

The next step to successfully bring our findings into the clinic is the validation with an in vivo study, in other words, in living mice. We designed a research study where mice with NB tumors will be treated with inhibitors to the DNA repair pathway to test their efficacy. This in vivo study is needed to eventually initiate a clinical trial and ultimately make this treatment available for children with NB. In order to proceed with this ambitious plan, we need your help: we are launching a fundraising campaign to collect the necessary funds to make the in vivo study possible. Please help us by donating and spreading the word about our work on new, targeted treatments for neuroblastoma. By contributing to this project, you are advancing novel, less toxic and more effective treatments in the fight against neuroblastoma.

We look forward to reporting our results to you and advancing a cure for this devastating childhood cancer.

Thanks for all your extensive support,