What is the challenge?
Despite intensive treatments involving months of chemotherapy, surgery and consolidation therapy with life threatening side effects and long-term disabilities, the survival today of children with high-risk NB is less than 40%.
Cure First's approach to this problem employs powerful, robotics-based high-throughput screening that applies small RNA molecules, which are able to inhibit gene function and identify targeted treatments. The most effective and biologically relevant gene targets were further validated with large drug libraries that include targeted FDA approved drugs, in NB organoids derived from individual children. While we plan to continue testing for thousands of new possible combinations with retinoic acid, which is a relatively non-toxic drug already in use, we also want to bring our findings closer to a clinical trial. Our results show that a class of drugs approved for other cancer types, could be significantly beneficial for NB as well, although we need to validate this with a mouse study.
We have already published promising results for this approach (Toyoshima et al. PNAS, 2012; Cermelli et al, CSH Perspectives in Medicine, 2014; Grandori and Kemp, Future Oncology, 2013). Link to publications.
Including the 2017 paper validating the screening method with collaborator C. Pauli
Our goal is to give children afflicted by NB and their families the chance for a happy life. We will translate the approach we take for NB to other cancers, and use the power of robotics, genomics and computers to identify personalized, targeted and less-toxic treatments for hundreds of childhood cancers.
Funds are needed to make the next step possible, an in-vivo mouse study. Please consider donating to support our Neuroblastoma Project either by giving directly to Cure First where your donation will be deposited into the NB fund or to our Global Giving Page, where you can browse through our quarterly reports and have more info about the fundraising campaign.
Fund this Project Through Global Giving
Proceeds from your donation will help fund Cure First's continuing efforts to discover new drug targets for children with neuroblastoma.