Nai-Kong V. Cheung, MD, PhD

My colleagues and I have developed therapies that combine multiple approaches, including surgery, chemotherapy, radiation therapy, isotretinoin (a chemical cousin of vitamin A), and more importantly, targeted therapy with a form of immune treatment called monoclonal antibodies. These strategies have dramatically extended the lives of our patients with metastatic neuroblastoma. Today, more than 50 percent of these patients treated at MSK Kids survive the disease, compared with fewer than 5 percent in the 1980s. In fact, immunotherapy has now become the standard of care for patients with metastatic neuroblastoma. Moreover, localized neuroblastoma is now considered a curable cancer, and many patients with this stage of the disease are cured with surgery alone.

Our team is also actively involved in early-phase clinical trials of promising therapies, including biologic agents and vaccines, natural killer cells, and combinations of these novel approaches with conventional treatments. We hope that effective new strategies for neuroblastoma will serve as a model for the treatment of other metastatic solid tumors in children and adolescents.

In the laboratory, we are evaluating markers of “minimal residual disease” (low levels of cancer remaining after treatment), which may predict the risk of early metastasis and recurrence, and discovering novel treatment targets in tumors. We are also engineering the next generation of antibodies to make them more potent and “human-like,” as well as arming these antibodies with either T lymphocytes (a type of white blood cell) or radioactive substances to diagnose and treat neuroblastoma. We believe many of these innovative antibodies may be useful for treating other solid tumors in adolescents and even those in adults.

We are assessing new drugs that may overcome the resistance that neuroblastoma often develops after prolonged chemotherapy. We are also trying to unearth the genetic and biochemical features of neuroblastoma to determine if a tumor’s profile at the time of diagnosis, metastasis, or relapse can tell us how aggressive the tumor may be, and what kind of therapy may be most effective. Our major thrust continues to be the translation of novel therapies from the laboratory to the clinic, where they may help our patients.

The team of experts at MSK Kids treats more patients with neuroblastoma than any other institution in the world. Using treatment approaches based on the findings of rigorous medical research, we are able to achieve better outcomes for our patients. Families who come to us benefit from the dedication and compassion of individuals from various disciplines who share the vision and the desire to eliminate this devastating disease.