Novel Treatment Pioneered by MSK Kids Researchers Receives FDA Approval for Patients with High-Risk Neuroblastoma

January 05, 2021 I The US Food and Drug Administration (FDA) has approved the drug naxitamab (Danyelza) for the treatment of patients with high-risk neuroblastoma. The drug, also known as humanized 3F8, was developed by researchers at MSK Kids, the pediatric cancer program at Memorial Sloan Kettering. Clinical testing of naxitamab was led by pediatric oncologist Brian Kushner and the MSK Kids neuroblastoma team. This treatment will now be more accessible to patients in the United States and worldwide. 

Neuroblastoma is a rare cancer of the nerve tissue that usually arises in the abdomen. It mainly affects young children. Although the disease used to be fatal in nearly all high-risk cases, the outlook has improved considerably in the last two decades, due in part to the techniques and therapies pioneered by MSK Kids researchers.

Naxitamab is a humanized form of the mouse monoclonal antibody 3F8, which was conceived and developed by MSK physician-scientist Nai-Kong Cheung. The drug targets tumor cells that have survived chemotherapy or radiation therapy by attaching to the cell surface sugar GD2. The antibody helps stimulate a patient’s own immune system to destroy the residual neuroblastoma cells.

“This achievement represents the collective hard work of decades of research by the neuroblastoma service here at MSK,” said Andrew Kung, Chair of the Department of Pediatrics at MSK. “We are grateful that this approval will benefit children suffering from neuroblastoma around the world. This approval is a validation of the importance of translational science in developing practice-changing therapies.”

A Major Research Milestone Achieved

Beginning in the late 1980s, MSK investigators used a mouse antibody 3F8 in combination with surgery and chemotherapy to treat neuroblastoma. This method significantly improved cure rates for children with high-risk disease. In 2007, Dr. Cheung and colleagues at MSK began developing a humanized form of 3F8 called Hu3F8. Humanized monoclonal antibodies are less likely to be rejected by the immune system and are substantially more potent than mouse antibodies. The nonprofit organization Band of Parents was instrumental in supporting the transition of the Hu3F8 blueprint from test tube to pharmacy.

Testing of Hu3F8 began in a phase I clinical trial in 2011 to determine the best and safest dose to give. In 2015, MSK licensed Hu3F8 to Y-mAbs Therapeutics for commercial development. Based on the results from early clinical studies testing this drug in children who had not responded to other treatments, the FDA granted the drug Breakthrough Therapy Designation in 2018. Now with the recent FDA approval, Hu3F8 has come full circle.

“It is gratifying for me to see this approval from the FDA,” said Dr. Cheung. “This approval is what MSK is all about at its core: research, education, and advancing patient care. Moving forward, my sincere hope is that this drug can help more kids with high-risk neuroblastoma. I am so thankful for and humbled by all of the patients and families who have supported and participated in this scientific research throughout the years. None of this progress would be possible without them.”

SOURCE: Memorial Sloan Kettering

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