Childhood Cancer

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PI-3 kinase as a novel and attractive target in pediatric cancer and neuroblastoma

Emory University
Donald Durden
Grant Type: 
Innovation Grants
Year Awarded: 
Type of Childhood Cancer: 
Brain Tumors, Medulloblastoma, Neuroblastoma
Project Description: 

1. Layman's Title for your Research Project

"A new therapeutic paradigm for the treatment of neuroblastoma and other pediatric cancers"

2. The type of childhood cancer your project focuses on and facts about it

The major focus of our research efforts is the application of this new form of therapy to neuroblastoma and medulloblastoma however other pediatric cancers will be sensitive to this therapy.

3.  Research Objectives:

What do you hope to achieve through your research and what will it mean to the world of childhood cancer if your project is successful?

To test different neuroblastoma and medulloblastoma cell lines for sensitivity in vitro and in vivo to our new drug, SF1126. We will examine synergy with other commonly used agents to treat these pediatric cancers e.g. chemotherapy and radiation. If successful, this will be a new drug class to treat childhood cancer.

4.  Explain in simple terms how you will conduct your research, for example,  is it lab based, is it a clinical trial, are children directly involved?

Our research is laboratory based. We will test our new drug against pediatric cancer cell lines and primary isolates of human tumors for sensitivity against our new drug, a panPI-3 kinase inhibitor. We will use genetic models for Neuroblastoma to further prove our hypothesis that this signaling pathway will be an important target for cancer therapy.

5.  What is innovative about the approach you are taking?

The drug, termed SF1126 is one of the first drugs of its type used in human cancer clinical trials. SF1126 was the first PI-3 kinase inhibitor introduced into adult Phase I clinical trials. We hope to apply it to pediatric cancers in the future.

6.  If there is collaboration, international or national, who is it with, and how will you work together?

We are collaborating with various scientist in the USA and Canada to enrich our analysis of this drug. For example we collaborate with Dr. David Kaplan, Toronto Sick Childrens to test sensitivity of NB stem cells to this new agent.