Pursuing new treatments for Neuroblastoma children

by Cure First Vetted since 2015

Summary

Cure First is tackling neuroblastoma (NB), a childhood cancer for which survival is poor and current treatments are overshadowed by side effects. Despite this, NB has not attracted the focus of the pharmaceutical industry. Recently we were able to establish patient-derived organoids and to apply high-throughput robotic tools to highlight drug sensitivities. We are now raising funds to finance an in vivo study which will help validate our findings and prepare them for a clinical trial.

$90,000
total goal
$72,469
remaining
115
donors
0
monthly donors
3
years

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%. Pharmaceuticals do not focus on NB because of the low number of cases. In the past 2 years we have met our goals: we established NB organoids from patient-derived cells and we identified safe targeted agents. Now we need funds to validate these results in vivo.

Solution

Working closely with physicians who are seeking improved therapies for their NB patients, Cure First is identifying novel, targeted and less-toxic treatments. By employing powerful, robotics-based high-throughput screening, we have identified safe targeted treatments in NB cells derived from children, using an FDA-approved drug library. We are planning to validate these results in vivo to demonstrate the efficacy of this class of drugs which are approved for other cancer types in living animals.

Long-Term Impact

We will translate the approach we use for NB to other cancers, and use the power of robotics, genomics, and computers to identify personalized, targeted and less-toxic treatments for childhood cancers. However to make our findings available for children they need to be validated in vivo. The proof that an NB tumor will shrink if treated with our drug in mice as well as it did in vitro, will attract the interest of pharmaceuticals to start clinical trials focused on neuroblastoma patients.

Additional Documentation

This project has provided additional documentation in a Microsoft Word file (projdoc.doc).

Resources

http:/​/​www.curefirst.org/​
Dr. Grandori, Cure First founder
Study describing new drug target for neuroblastoma
Cures for cancer hidden in plain sight?

Organization Information

Cure First

Location: Seattle, WA - USA
Website: http:/​/​www.curefirst.org/​
Project Leader:
Carla Grandori
Seattle, WA United States

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