Neuroblastoma is the most common solid tumor cancer in children and is challenging to treat regardless of existing therapies that are available. Dr. Andras Heczey, assistant professor of pediatrics - hematology and oncology, and Dr. Leonid Metelitsa, professor of pediatrics - oncology at Baylor College of Medicine, have received a $1.5 million grant from Alex’s Lemonade Stand Foundation to conduct a Phase 1, first-in-human clinical trial of a new form of immunotherapy to treat neuroblastoma using native and engineered properties of natural killer T-cells (NKTs).

“Our goal is to find a safe option to effectively treat neuroblastoma, one of the most prevalent and deadly types of cancer in children. NKTs can naturally traffic to the tumor site and suppress tumor growth indirectly by attacking tumor-supportive macrophages. In addition, we armed NKTs with a genetically engineered protein called chimeric antigen receptor (CAR) that enables them to directly kill neuroblastoma cells,” Heczey said. “Thus, CAR NKTs can eliminate tumors by targeting both tumor cells and tumor-supportive macrophages. There is tremendous promise in this therapy, but we need to determine how to best implement it for patients.”

The key aims of the clinical trial are to establish the maximum tolerated dose and to measure the antitumor and immunological activities of the therapy. These aims will be accomplished by generating patient-specific CAR NKTs to treat neuroblastoma patients at four dose levels with toxicities monitored according to NCI guidelines.

“The results of this study will inform clinical development of NKT-cell based immunotherapy of neuroblastoma and will have a broad applicability for other types of cancer,” Heczey said.

This clinical trial builds on previous research by Drs. Heczey and Metelitsa using genetically engineered natural killer T-cells to target neuroblastoma.

Heczey and Metelitsa also are both members of the Dan L Duncan Comprehensive Cancer Center at Baylor.