Neuroblastoma Advanced Therapies Center
Neuroblastoma Advanced Therapies | Research

Neuroblastoma Research & Treatment Innovations

The Neuroblastoma Advanced Therapies Center at Cincinnati Children’s is a leading center for the development and clinical testing of new approaches to neuroblastoma. Our physicians and researchers work closely together to quickly move research breakthroughs from the lab to the patient. Research is led by Dr. Brian Weiss and Dr. Natalie Wu. Current clinical research initiatives in neuroblastoma focus on the development of new treatments using:

  • Novel targeted therapies
  • Novel imaging techniques
  • New approaches to high-dose chemotherapy
  • Targeted radiopharmaceutical therapies

Cincinnati Children’s researchers are investigating novel agents or combinations of agents to treat the most difficult forms of relapsed or refractory neuroblastoma. A number of Cincinnati Children’s pilot projects have subsequently been developed into national trials by the Children’s Oncology Group and other national clinical research consortia.

131I-MIBG Therapy

Cincinnati Children’s is leading development of 131I-MIBG targeted radiopharmaceutical therapy for newly diagnosed and relapsed neuroblastoma. MIBG, a substance that sticks to and is absorbed by neuroblastoma cells, is chemically joined to 131I, a radioactive form of iodine. When this combination is given to neuroblastoma patients, the radioactive molecules are absorbed by neuroblastoma cells, killing them without causing too much damage to healthy cells nearby. To ensure the safety of patients, families and staff during 131I-MIBG therapy, Cincinnati Children’s has built two specialized therapy suites shielded against radiation. Our MIBG suites allow for parents to safely stay in the room with their child while the child is receiving 131I-MIBG therapy.

CLR 131

The team also participates in a Phase 1 study of CLR 131 for children, adolescents and young adults with neuroblastoma. This is a targeted form of radiotherapy which limits the negative effects of radiation to surrounding tissue and organs. In addition, this type of radiation has a longer half-life than 131I-MIBG and can potentially be given as an outpatient therapy.


For high risk neuroblastoma, our team was one of the first to offer the CU67 trial. This is a new compound like 131I-MIBG in that it is specifically directed at neuroblastoma cells; however, this compound can be given as an outpatient treatment.

Proton Therapy

The Cincinnati Children’s / UC Health Proton Therapy Center provides cutting-edge treatment for pediatric cancers. Our Proton Therapy Center has begun the world’s first clinical trial of FLASH radiation therapy for cancer. FLASH uses radiation delivered at ultra-high speeds up to 1,000 times faster than what is used clinically today.

> Learn more about our world-class research on cancer in the Division of Oncology.

National Referral Center

Cincinnati Children’s is a major referral center for the National Cancer Institute's two new pediatric research initiatives for children with high-risk neuroblastoma: the Pediatric Phase I Consortium and the New Approaches to Neuroblastoma Therapy Consortium. Many of our physicians serve in leadership positions for these and other pediatric cancer organizations, further strengthening our link to the latest clinical research opportunities.

Clinical Research Studies for Patients with Neuroblastoma

Physicians and researchers from the Neuroblastoma Advanced Therapies Center at Cincinnati Children’s offer clinical research studies for patients with neuroblastoma.

View a list of current clinical trials:

Pediatric Research Studies

Young Adult Research Studies

Research News

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