By Rachele Rosati | Cure First Research Fellow
Within the past few months, I have advanced the neuroblastoma (NB) project targeting the identification of novel drug targets that can be used in combination with retinoic acid (RA). RA is currently one of the more effective and less toxic compounds used in the treatment of children with NB. Although high-risk NB is still difficult to treat, the addition of RA in the consolidation phase of therapy has given good results, significantly improving the rate of survival. However, there is more we can do. Cure First believes that by using high-throughput screening (HTS), technology that employs robotic tools, we can accelerate the discovery of new RA combination therapies.
Thus far, I have optimized the crucial methods of culturing and transfecting NB cell lines with siRNA molecules targeting hundreds of genes, which are potential future drug targets. I use siRNA technology to silence each individual gene and measure NB cell survival its absence. This allows me to select candidates that will enhance the effectiveness of RA when they are used together in combination. Our ultimate goal is to eliminate the risk of recurrence during RA therapy. Through this functional genomic approach, we will also be able to learn a great deal about the driving pathways and potential vulnerabilities of NB.
Finally, we are pleased to announce that our director, Dr. Carla Grandori, will soon launch a collaborative project with Dr. John Maris. Dr. Maris is a world expert physician and researcher on NB. We plan to test at least 10 patient-derived NB samples with a collection of hundreds of novel drugs. I will execute this project, by culturing the NB cells in vitro and carrying out the high throughput screening starting next fall.
I am looking forward to these exciting new projects!! I want to thank you for supporting our NB research project through Global Giving, and I hope that my work will lead to identifying less toxic and more effective drugs for children affected by NB.
Rachele Rosati, Cure First Research Fellow
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