Project Update Summaries 2014
RTF Driving the Creation of Two New Cutting-Edge Immunotherapy Treatments for Neuroblastoma
The Ronan Thompson Foundation is extremely proud to partner with Solving Kids' Cancer to help bring two new next-generation treatment options to children currently battling neuroblastoma. Both clinical trials are from the emerging new treatment category, immunotherapy, that harnesses the child's own immune system to fight their cancer. In one trial that will open to children at the Children's Hospital of Philadelphia and Primary Children's Hospital Salt Lake City, the researchers will collect the child's own immune cells (T-cells) and genetically re-engineer them to attack neuroblastoma while recruiting other immune cells to do the same. The T-cells are temporarily modified so children can have multiple treatments and ensure safety. This technology is based on the same platform first developed for childhood leukemia which is demonstrating dramatic early success, and the same research team will conduct the neuroblastoma trial.
Our second trial will use a therapeutic cancer vaccine that has shown significant early promise in an initial study (sustained remissions after relapse). The phase 1 clinical trial is built on an earlier protocol and will use a vaccine that targets the GD2 and GD3 antigens on the surface of neuroblastoma cells, and include an immune adjuvant given intravenously, called beta-glucan, which stimulates the immune response to the vaccine.
Significantly, this trial also marks the first effort to provide access of promising treatment options to children in other countries who currently have to travel to the U.S. to enroll on cutting-edge clinical trials after relapse. The low-toxicity vaccine was developed by researchers at Memorial Sloan-Kettering Cancer Center (MSKCC) in New York City, which is participating in the trial alongside Bristol Children’s Hospital in the UK, and the Hospital Sant Joan de Déu Barcelona, in Spain. In addition, the trial is the first-of-its kind to have international cancer centers collaborate on an immunotherapeutic for pediatrics and have international pediatric cancer charities collaborate to fund it.