Neuroblastoma Fundraiser
Project Overview
Cure First raised funds to drive the research of high-throughput technologies and screening to precisely identify characteristics of the cancerous tissue and pinpoint therapies specific to individual neuroblastoma (NB) patients. In the past 2 years, thanks to generous donations, we have continued to meet our goals and push forward the next generation of healthcare.
What Was the Challenge?
JOHN MARIS, MD, of The Children's Hospital of Philadelphia Cancer Center, discusses the collaboration with Cure First to advance research on personalized and targeted treatments for pediatric NB. The generous support of the Soupy for Loopy Foundation has provided for the screening and analysis of ten live NB samples.
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
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.
Why Did We Need Funds?
Funds were needed to make the next step possible, an in-vivo mouse study. Sponsors helped support our Neuroblastoma Project either by giving directly to Cure First where their donations were deposited into the NB fund or to our Global Giving Page, where they could browse through our quarterly reports and obtain more info about the fundraising campaign.