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Pharmacogenomics and Drug Screening Lead to a Novel Targeted Therapy with Potent and Specific Activity Against Mouse Models of MYCN-amplified Neuroblastoma

Virginia Commonwealth University
Anthony Faber, MD & Yael Mosse, MD (CHOP)
Grant Type: 
Innovation Grants
Year Awarded: 
Type of Childhood Cancer: 
Project Description: 


Neuroblastoma is the second deadliest cancer in children. This is largely attributed to high-risk neuroblastoma, which often has an abnormality known as MYCN amplification. Targeted therapies are newer cancer therapies that have already revolutionized how cancer is treated. Despite all the successes of targeted therapies, there have been no proven targeted therapy options for MYCN amplified neuroblastoma.

Project Goal:

In pursuit of a new targeted therapy for these patients, we have developed a combination therapy using two drugs, one termed ABT-199, and a second termed MLN8237. We have found that they act together to kill MYCN amplified neuroblastoma cells in laboratory experiments. This therapy is effective specifically in MYCN amplified neuroblastoma. Excitingly, in mouse models of MYCN amplified neuroblastoma where therapies are tested to determine whether they may be expected to have activity in clinical trials with human subjects, the combination therapy have shrunk tumors and in some instances cured mice of any measurable cancer. Equally excitingly, this therapy has induced no signs of toxicity with the mice in these preliminary experiments, suggesting that this therapy may be tolerated by the pediatric population with this devastating cancer. We plan on leveraging this grant to further develop this therapy and test it in more mouse models. As we have significant experience in clinical trials for pediatric patients with neuroblastoma, this collaboration yields the ideal components to bring this therapy into clinical trial testing.

Co-funded by: 
Cure4Cam Childhood Cancer Foundation
I Care I Cure