Pursuing new treatments for Neuroblastoma children

Dear Cure First and Project Neuroblastoma Supporters,

In collaboration with Dr. John Maris’s laboratory at the Children’s Hospital of Philadelphia (CHOP), the in vivo validation of our drug candidate -- which I established on a patient-derived model at Cure First-- has been performed. The study employed a total of 10 mice implanted with a neuroblastoma (NB) tumor: 5 mice were treated with a placebo (as control) and 5 mice were treated with our drug candidate, an oral drug given every day.

The drug candidate slowed the growth of the NB tumor, validating our initial finding. However, two facts must be considered before this type of drug should be proposed for the clinic:

• 1) This class of targeted drugs is mainly used as adjuvant therapy or in combination with other chemotherapeutic drugs in the clinic.
• 2) Because of its mechanism of action, this type of drug needs to be given for a prolonged time.

These considerations led to the in vitro combinatorial experiment that I have just completed. Using a low concentration of the new targeted drug and the speed and accuracy of robotics, I tested a library of more than 120 different drugs on a patient-derived model of NB to find combinations which might increase the efficacy of this inhibitor. The results indeed identified several existing and FDA-approved drugs that increased the efficacy of the new drug candidate in NB cells.

With your support, we hope to test the new drug combination in avatar mice with patient-derived neuroblastoma, a necessary step before proposing it for clinical consideration.

Thank you for your priceless support of our research which is aimed at improving high risk neuroblastoma prognosis and quality of life.

Rachele Rosati, MS, and the Cure First team

The accompanying graph illustrates that the drug candidate in combo with three different drugs highlighted in blue, is diminishing the viability of the NB cells.