Naxitamab

Naxitamab is a humanized monoclonal antibody designed for the treatment of neuroblastoma, a type of cancer that most commonly affects children and is derived from neuroblasts located in the adrenal glands. Naxitamab targets the GD2 ganglioside, which is overexpressed on neuroblastoma cells, and works by binding to these cells, making them visible to the patient's immune system, which can then attack and destroy the cancer cells.

Mechanism of Action

Naxitamab operates by targeting and binding to the GD2 ganglioside, a molecule found on the surface of neuroblastoma cells and some other types of cancer cells. This binding promotes antibody-dependent cellular cytotoxicity (ADCC) and complement-dependent cytotoxicity (CDC), leading to the destruction of the cancer cells. The GD2 ganglioside is an attractive target for cancer therapy due to its limited expression on normal cells and high expression on certain cancer cells, including neuroblastoma.

Clinical Uses

Naxitamab has been investigated and used in the treatment of high-risk neuroblastoma, particularly in patients who have not responded to or have relapsed after standard therapies. It is often administered in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) to enhance the immune response against neuroblastoma cells.

Approval and Regulation

The U.S. Food and Drug Administration (FDA) granted approval for naxitamab for the treatment of high-risk neuroblastoma in patients who are refractory to or have relapsed after previous treatments. This approval was based on clinical trials demonstrating the efficacy and safety of naxitamab in this patient population.

Side Effects

The use of naxitamab can be associated with several side effects, some of which can be severe. Common side effects include pain, including abdominal and bone pain, rash, fever, and hypertension. Due to the risk of severe infusion reactions, naxitamab is administered with premedication to mitigate these risks.

Future Directions

Research is ongoing to explore the potential of naxitamab in treating other GD2-positive cancers beyond neuroblastoma, such as certain types of sarcomas and melanomas. Clinical trials are also investigating the use of naxitamab in combination with other therapies to enhance its efficacy and reduce side effects.

See Also

• Monoclonal antibodies
• Neuroblastoma
• Immunotherapy
• Antibody-dependent cellular cytotoxicity
• Complement-dependent cytotoxicity