Skip to main content

ACS Research Highlights

Update on New Treatment for Children with Neuroblastoma

This photo from ACS research grantee Andras Heczey, MD, shows a clear “win” from his phase 1 clinical trial of a new treatment for children who've been heavily treated for neuroblastoma. 

This photo shows a clear “win” from a phase 1 clinical trial of a new treatment for children who have already been heavily treated for neuroblastoma. The photo was published in a study in Nature Medicine with co-authors and American Cancer Society (ACS) research grantees Andras Heczey, MD, and Leonid Metelitsa, MD, PhD.

Neuroblastoma Tumors Before and
After Experimental Treatment

Pre and post-CAR NKT scan from childhood cancer research highlight by Heczey

These gray and blue images are from two types of scans. Images on the left show circled neuroblastoma tumors. Images on the right show empty circles—the absence of tumors after the patient received 2 doses of the experimental CAR-NKT therapy. 

We’ve followed these studies at Baylor College of Medicine since Heczey received his first ACS research grant in 2016. That’s when he was genetically adapting CAR T-Cell therapy to treat neuroblastoma. Later, on a TheoryLab podcast, Heczey talked about his work with neuroblastoma (and with pediatric liver cancer). And, we reported on his progress when he initiated his experimental treatment’s first clinical trial.

These images are from updated interim phase 1 trial results published in Nature Medicine. At this phase, anti-tumor activity wasn’t the first goal, so it’s much too early to deem this new treatment a success. What researchers did conclude was:

  • The CAR-NKT was safe.
  • Engineered CAR-NKTs multiplied after infusion as they needed to and went to places where the cancer had spread.
  • Anti-tumor activity was demonstrated in some patients.
  • The CAR-NKT therapy can be enhanced by disrupting a gene that promotes neuroblastoma growth, improving antitumor responses in preclinical trials.