Pursuing new treatments for Neuroblastoma children

GLOBAL GIVING SEPTEMBER 2019 REPORT

Dear Cure First and Project Neuroblastoma Supporters,

Our progress on finding new treatments for children with Neuroblastoma (NB) continues as we focus on re-purposing existing targeted drugs used in adults with other cancer types as well as drugs currently under experimentation.  Our work to date indicates that indeed existing FDA-approved drugs could prove effective in treating this terrible childhood cancer and their use in potential novel combinations could enhance their effectiveness even further.

Cure First’s utilization of patient-derived tumor organoids also continues. These “mini-cancers in a dish,” which maintain the heterogeneity and genomic profile of the original tumor from one child’s body, can reveal significant drug responses which match a respective organoid’s genomic features. We are also able to record novel drug sensitivities which cannot be anticipated by genomics alone.  The process basically allows us to perform “in vitro” trials. It is far less costly than working with avatar mice and allows the opportunity to test hundreds if not thousands of drug combinations on cancer cells from a single individual.

Over the summer, our work was focused on the validation of our findings on multiple samples of tumor-organoids derived from various patients with NB. We are working hard to assemble all the data and figures from this project for a future publication so that the scientific and biomedical community can be aware of how cancer medicines developed for adults can be beneficial to children with neuroblastoma.

None of this progress would have been achieved without your help and your support.

Thanks to you, we have raised more than $22,500. And we recently received a challenge match of $10,000! As you know, cancer research is expensive. The reagents and materials which keep NB cells alive for testing are costly.  This year our fundraising target is $100,000 dollars. While we are already 1/10^th of the way there, we hope you can help us achieve our goal so that we can continue to validate new specific medicines for neuroblastoma before proposing these drugs for clinical trials.

Thank you again for your interest in our research which is devoted to improving the quality of life for children with high-risk neuroblastoma.,

Rachele Rosati, Lead Scientist, MS, and the Cure First Team