Neuroblastoma: Clinical Trials & Research

Memorial Sloan Kettering has been at the forefront of developing innovative drugs and treatments that prolong survival for people with neuroblastoma but don’t cause debilitating long-term side effects.

One of our approaches has been to take advantage of new drugs already in clinical trials for adult patients. Some of these drugs can enhance the effectiveness of chemotherapy without added side effects, especially in patients with disease that has become resistant to standard treatment. Other therapies are made to block the ability of neuroblastoma cells to spread. 

We are in a better position than ever before to advance our understanding of neuroblastoma.

-Nai-Kong Cheung, Pediatric Oncologist

We’re also exploring cancer vaccines to train the body’s immune system to fight cancer. These vaccines instruct the immune cells to recognize and destroy existing cancer cells and any that may subsequently develop. Other approaches we’ve worked on include different variations of immunotherapies, newer versions of antibody therapies, and the use of immune cell–based therapies such as natural killer (NK) cells. Treatments for neuroblastoma that has spread to the brain have also been a focus.  

Our investigators are translating their work in the lab into new options for our young patients, and our experiences with patients are incorporated back into our work in the lab. This allows us to offer the most promising new treatments to children through clinical trials.

Through clinical trials, our medical experts evaluate the safety and effectiveness of new therapies and diagnostic tools for neuroblastoma. Clinical trials can also help our doctors compare the effectiveness of different aspects of your child’s care or determine the causes of neuroblastoma.

Children eligible to participate in a clinical trial may have access to new therapies that are not yet available elsewhere. Our clinical research team is highly experienced in selecting children who are most likely to benefit from a particular investigational therapy. They can guide you through the process of choosing the most appropriate clinical trial for your child’s needs.

To learn more about our current clinical trials for neuroblastoma, choose from the list below.

Showing 16 trials
Title Investigatorsort icon
A Study Assessing Lower-Dose Radiation Therapy for High-Risk Neuroblastoma
Newly Diagnosed
[Protocol 14-186]
Wolden, Suzanne, MD
A Phase II Study of Cyclophosphamide, Topotecan, and Bevacizumab in Patients with Relapsed or Refractory Ewing's Sarcoma and Neuroblastoma
Newly Diagnosed & Relapsed/Refractory
[Protocol 11-183]
Trippett, Tanya, MD
Evaluating the Causes of Cancer in Young Patients
[Protocol 09-068]
Stadler, Zsofia, MD
A Phase I Study of Humanized 3F8 plus Interleukin-2 in Patients with High-Risk Neuroblastoma and GD2-Positive Tumors
Relapsed/Refractory
[Protocol 12-116]
Roberts, Stephen, MD
A Phase I Study of Difluoromethylornithine and Celecoxib with Cyclophosphamide and Topotecan in Patients with Recurrent or Persistent Neuroblastoma
Relapsed/Refractory
[Protocol 14-187]
Roberts, Stephen, MD
A Phase I Study of Anti-GD2 3F8 Antibody and Allogeneic Natural Killer Cells for High-Risk Neuroblastoma
[Protocol 09-011]
Modak, Shakeel, MD
A Phase I Study of LDK378 in Children with Cancers Containing an ALK Mutation
Relapsed/Refractory
[Protocol 13-235]
Modak, Shakeel, MD
A Pilot Study of PET Imaging of Solid Tumors Using Radioactive Iodine-Labeled Hu3F8
Newly Diagnosed & Relapsed/Refractory
[Protocol 13-147]
Modak, Shakeel, MD
A Phase I Study of the Hu3F8 Antibody plus GM-CSF in Patients with Relapsed/Refractory Neuroblastoma
Relapsed/Refractory
[Protocol 12-230]
Kushner, Brian, MD
A Study Offering Treatment with 3F8 and GM-CSF in Patients with High-Risk Neuroblastoma
Relapsed/Refractory
[Protocol 13-260]
Kushner, Brian, MD
A Phase I Trial of a Trivalent Vaccine with Escalating Doses of the Immunological Adjuvant OPT-821 Plus Oral ß-Glucan for High-Risk Neuroblastoma
[Protocol 05-075]
Kushner, Brian, MD
A Phase II Study of Intrathecal I131-3F8 in Patients with GD2-Expressing Central Nervous System and Leptomeningeal Neoplasms
[Protocol 05-122]
Kramer, Kim, MD
A Phase I Study of Intrathecal Radioimmunotherapy Using 131I-8H9 for Central Nervous System/Leptomeningeal Cancers
[Protocol 03-133]
Kramer, Kim, MD
Molecular Characterization of Neuroblastic Tumors and Clinical Outcome
[Protocol 00-109]
Cheung, Nai-Kong, MD, PhD
A Phase I Study of Humanized 3F8 Monoclonal Antibody in Patients with High-Risk Neuroblastoma and GD2-Positive Tumors
[Protocol 11-009]
Basu, Ellen, MD, PhD
A Pilot Phase II Study of Targeted Radiotherapy with 131I-MIBG and Arsenic Trioxide in Patients with Resistant Neuroblastoma or Malignant Chromaffin Cell Tumors
[Protocol 04-148]
Basu, Ellen, MD, PhD