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A phase 2 clinical trial has found that combining a novel compound called alisertib with chemotherapy has anti-tumor activity in children with high-risk, relapsed neuroblastoma. The New Approaches to Neuroblastoma Therapy (NANT) consortium study was led by Araz Marachelian, MD, MS, of the Children’s Center for Cancer and Blood Diseases at Children’s Hospital Los Angeles and Steven G. DuBois, MD, of the Dana-Farber Cancer Institute. Results were recently published online in the journal Clinical Cancer Research.

In the multicenter trial, alisertib was combined with two common chemotherapy agents: irinotecan and temozolomide. Alisertib is an experimental compound that targets and inhibits a particular enzyme–called an Aurora A kinase–which neuroblastoma tumors often use to grow.

Neuroblastoma is the second most common solid tumor in children (after brain tumors). The cancer arises in immature nerve cells called neuroblasts, and it typically affects children ages 5 or younger. Nearly half of patients are diagnosed with high-risk, metastatic disease, which has a 50 percent mortality rate.

The NANT study–a follow-up to an earlier phase 1 trial–evaluated the combination therapy in a group of 20 patients with high-risk neuroblastoma that had relapsed or had not responded to standard treatment. Of those patients, 21 percent had

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