New Hope for Kids with Neuroblastoma

toddler with head scarf

Andras Heczey, MD, was a general pediatrics resident at Children’s Hospital Los Angeles when he read an article that crystallized his career path in childhood cancer. The 2006 paper in Science found that two patients with metastatic melanoma – the most lethal form of skin cancer – went into remission after receiving a dose of gene-modified T-cells. In this emerging treatment known as cell therapy, a patient’s own T-cells (a type of white blood cell) are removed, genetically altered to attack the cancer, and reinfused back into the body.

“I just thought that was the coolest thing and decided I had to do something similar in pediatrics,” says Heczey, who is now a cell therapy researcher and assistant professor at Baylor College of Medicine in Houston.

To further his work in cell therapy for childhood cancer, the American Cancer Society selected Heczey for a $583,000 Mentored Research Scholar Grant, which is funded by TODAY show viewers through the “Shine a Light” campaign and subsidized by the Society. The grant will enable Heczey, under the mentorship of leading scientists, to establish a clinical trial for neuroblastoma. This type of pediatric cancer is most often seen in infants and young children, and usually starts in the abdomen (specifically in the adrenal glands, which are located on top of each kidney).

But Heczey, who is a member of the solid tumor team at Texas Children’s Hospital, notes that his recent award is not just about curing neuroblastoma. “While the Society is certainly supporting me during this project, it is also supporting something bigger,” he says. “It’s supporting the training of a future scientist who can become a leader in the field and dedicate his efforts to pediatric cancer research.”

Cell Therapy, a Form of Immunotherapy

Cell therapy is a kind of cancer immunotherapy, which uses parts of our immune system to attack the disease. (Learn more about how it works here.)

“Cancer immunotherapy harnesses the incredible specificity of the immune response. Cellular immunotherapy” – a.k.a. cell therapy – “further adds to this specificity. It’s a fantastic one-two punch against cancer,” says Susanna Greer, director of clinical cancer research, nutrition and immunology at the American Cancer Society.

A type of cell therapy called chimeric antigen receptor (CAR) T-cell therapy has major promise in childhood leukemia. A study published in October 2014 in the New England Journal of Medicine found it helped children with chemotherapy-resistant acute lymphoblastic leukemia achieve remission.

Results haven’t been as promising in solid tumors. “Solid tumors are more challenging because they have a distinct tumor microenvironment to overcome,” Heczey says. “In my opinion, solid tumors are posing a great challenge for cell therapy and a huge opportunity at the same time, because we can learn how to overcome the tumor microenvironment.”

Heczey’s project, which begins January 1, 2016, will focus on a subset of T-cells called natural killer T-cells (NKTs). This is a novel approach to cell therapy that may have even greater therapeutic potential for solid tumors in children. Heczey was lead author of a June 2015 paper in the journal Blood that established the potential of NKT cells as an effective platform for cancer immunotherapy.

He hopes to enroll 18 children with neuroblastoma in a clinical trial, but he expects the entire project – which will also involve preclinical research involving another method of cell therapy – to have a broad impact. “In addition to testing our research hypotheses, I’m [learning] how to design and conduct a clinical trial, as well as regulatory affairs specific to cell therapy,” Heczey says. “These things apply not just to neuroblastoma but to any pediatric cancer that we’ll try to cure with cell therapy in the future.”

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