New Cures for Children with Neuroblastoma

Summary

Cure First is tackling neuroblastoma (NB), a childhood cancer for which survival is poor and current treatments are life threatening, and which has not attracted the focus of the pharmaceutical industry. We are raising funds to drive the research that will apply high-throughput technologies and screening to precisely identify therapies for individual NB patients and the characteristics of the cancerous tissue in each of them.

What is the issue, problem, or 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 then 40%. Pharmaceutical R&D does not focus on NB because the number of cases does not meet their criteria for commercial development. We are raising funds to drive the research to apply new technologies to precisely identify therapies specific to individual NB patients.

How will this project solve this problem?

Working closely with physicians who are seeking improved therapies for their NB patients, Cure First will identify novel, targeted and less-toxic treatments. We employ powerful, robotics-based high throughput screening and the ability to inhibit gene function with small RNA molecules to identify targeted treatments in NB cells derived from individual childrens. We will test for thousands of new possible combinations with retinoic acid, which is a relatively non-toxic drug already in use.

Potential Long Term Impact

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. Our goal is to give children afflicted by NB and their families the chance for a happy life. 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).