Phase I Vinblastine and Sirolimus in Pediatric Patients With Recurrent or Refractory Solid Tumors Including CNS Tumors
Project Title: Phase I Vinblastine and Sirolimus in Pediatric Patients With Recurrent or
Refractory Solid Tumors Including CNS Tumors
Researcher: Sylvain Baruchel, MD
Institution: Hospital for Sick Children, Toronto
Study Type: Phase I clinical trial
Project Summary
Researchers identified one drug that was very effective in killing neuroblastoma cells. The drug is called Sirolimus, also known as rapamycin, and is currently approved for preventing rejection in patients with organ transplants. This work led to the first use of this drug in children with solid tumors. In this phase 1 clinical trial, children received rapamycin and another cancer drug known as vinblastine because the two combined drugs were shown to be even more effective against pediatric tumors.
Impact
The results were published in the January 2014 issue of Pediatric Blood Cancer and showed a partial response in one patient and stable disease in three children. The result of this study led other researchers to investigate the next generation of these inhibitors in other clinical trials.