Seattle Children’s Begins Recruiting Patients for Immunotherapy Research Trial for Neuroblastoma, One of the Deadliest Forms of Childhood Cancer

Trial will be one of the first of its kind; based on previous success with immunotherapy for patients with recurring leukemia


Seattle Children’s today announced the opening of patient enrollment for its new cellular immunotherapy clinical research trial designed to induce remission in children suffering from neuroblastoma, one of the deadliest forms of childhood cancer.

The phase 1 trial, which was recently authorized by the U.S. Food and Drug Administration, is based on the same science that has shown tremendous progress in the treatment of acute lymphoblastic leukemia in patients who have relapsed one or more times. Seattle Children’s is one of the first institutions to treat a pediatric solid tumor cancer using cellular immunotherapy, a technology that uses a patient’s own immune system to identify and destroy cancer cells.

“Solid tumor cancers are especially challenging to treat because the tumors create their own environment that prevents the immune system from responding,” said Dr. Julie Park, an oncologist at Seattle Children’s and lead investigator for the new trial, known as Engineered Neuroblastoma Cellular Immunotherapy (ENCIT)-01. “But we have encouraging research suggesting cellular immunotherapy can be used to safely treat neuroblastoma.”

Under the leadership of Dr. Mike Jensen, director of Ben Towne Center for Childhood Cancer Research at Seattle Children’s Research Institute, Seattle Children’s has been a leader in the development of cellular immunotherapy for treatment of pediatric cancers. Jensen began one of the studies using immunotherapy to treat children and young adults with recurring acute lymphoblastic leukemia in 2012. At least six of the patients from those research studies are now cancer free.

“We are very excited to begin studying this new immunotherapy for treatment of patients with neuroblastoma,” said Park, who is also an investigator in Seattle Children’s Research Institute’s Center for Clinical and Translational Research. “There are many children in need of innovative therapies like this. Seattle Children’s is dedicated to developing this therapy to its safest and most effective iteration.”

Participants in the ENCIT-01 trial will be children or adolescents with neuroblastoma who responded poorly to standard treatments of chemotherapy and/or radiation therapy. Clinicians will collect blood from each trial participant and deliver it to a Ben Towne Center where immune system T cells will be isolated and reprogrammed to target neuroblastoma cancer cells. The engineered cells will then be infused back into the patient, where researchers expect they will find and destroy cancer cells.

Patients may be referred to this trial from treatment centers around the world; all who are accepted for the trial will be treated at Seattle Children’s Hospital.

“These children typically have less than a 10 percent chance of surviving,” Park said. “We want to offer them and their families hope.”

About Seattle Children’s

Seattle Children’s mission is to provide hope, care and cures to help every child live the healthiest and most fulfilling life possible. Together, Seattle Children’s Hospital, Research Institute and Foundation deliver superior patient care, identify new discoveries and treatments through pediatric research, and raise funds to create better futures for patients.

Ranked as one of the top children’s hospitals in the country by U.S. News & World Report, Seattle Children’s serves as the pediatric and adolescent academic medical center for Washington, Alaska, Montana and Idaho – the largest region of any children’s hospital in the country. As one of the nation's top five pediatric research centers, Seattle Children’s Research Institute is internationally recognized for its work in neurosciences, immunology, cancer, infectious disease, injury prevention and much more. Seattle Children’s Hospital and Research Foundation works with the Seattle Children’s Guild Association, the largest all-volunteer fundraising network for any hospital in the country, to gather community support and raise funds for uncompensated care and research.

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