Both preclinical and clinical studies involving pediatric solid and CNS tumors have shown an aberration of mTOR pathway. In addition, these studies have demonstrated that inhibition of the mTOR pathway has significant efficacy impeding growth of these tumors.

A phase 1 study has been initiated with the Children’s Oncology Group in which pediatric patients with recurrent or refractory solid tumors including CNS tumors, are treated with a combination of ABI-009 + temozomide + irinotecan (NCT02975882).

This study includes patients with osteosarcoma, Ewing sarcoma, rhabdomyosarcoma, medulloblastoma, gliomas etc.