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New Approaches to Neuroblastoma Therapy

What is neuroblastoma? Neuroblastoma is the second most common solid tumor and the most common extracranial cancer in children. This tumor has a unique biology. Whereas, in some children the tumors can spontaneously regress, in other children with high-risk disease, the cancer is usually metastatic and can become resistant to the best available standard therapy. In approximately one-third of children affected by aggressive neuroblastoma, gene analysis reveals the presence of malfunctioning genes (MYCN, ALK); however, genes that are frequently abnormal have not been identified in the other two-thirds of these children. For this latter group, the tumor cells enlist normal cells in their environment to help them grow, spread, and resist treatments.

What is NANT? The NANT consortium offers hope and resources to patients and their families with relapsed/refractory neuroblastoma when front-line, standard therapy fails. Established in 2000, NANT is the only consortium in the world solely dedicated to developing novel treatments and biomarkers through early clinical trials (Phase I/II) for children whose disease has become resistant to front-line therapies. NANT addresses the most difficult but most relevant question for children with high-risk neuroblastoma: "What is the best strategy to attack this disease when it has escaped all standard front-line therapies?" NANT believes that a comprehensive approach that targets the cancer cells but also their surrounding environment by a team of researchers with converging expertise will make a difference.

Our vision is to develop and test new therapies that target both neuroblastoma cells and normal cells around the tumor and in the immune system to improve the outcome for children with high-risk neuroblastoma with fewer side effects.

NANT studies include our Precision Study, which will test the feasibility of using a set of specific genetic and cellular biomarkers that could in the future guide the assignment of children with relapsed/refractory neuroblastoma to NANT trials that better fit the biology of their tumor. NANT is also conducting studies that harness the immune system to attack neuroblastoma cells, and new small molecule drugs that target specific malfunctioning genes like ALK.

How does NANT work? NANT brings together a multidisciplinary team of laboratory and clinical scientists (more than 75) from 14 pediatric hospitals and institutions in the US and Canada (see NANT membership) with complementary expertise in genetics, biology, immunology, chemistry, pathology, biostatistics, clinical investigations, and imaging all with a single focus on finding better treatments for children with high-risk neuroblastoma.

NANT expertise includes detailed pharmacokinetics, biologic correlates, and administration of complex therapies to establish feasibility and safety. NANT investigators have a proven record of moving innovative approaches into standard frontline therapy for high-risk neuroblastoma. Our NANT Biology study established a valuable repository for relapsed and refractory tumor tissue, BM tumor cells, blood, and radiologic images. This unique resource is available to the larger NB research community.

NANT closely interacts and collaborates with the Children's Oncology Group (COG). NANT pilot studies establish safety and preliminary tumor response data that are critical to support COG conduct of larger Phase II/III randomized trials. NANT has a strong track record of transferring knowledge to COG both for therapeutic and biomarker studies.

A NANT Parent Advisory Committee (NANT-PAC) provides parent input during the development of NANT clinical trials. The Committee includes parent coordinators for education, public website, fundraising, and coordination of local parent representatives.

NANT has created a "Guest Membership" designation to encourage collaboration with non-NANT investigators with innovative ideas for neuroblastoma therapy and with special expertise. Guest members may participate as individual investigators, or, if their institution has Phase I capabilities, also enter patients in that selected trial.

How is NANT supported? NANT's support comes from federal grants, the pharmaceutical industry, philanthropic foundations, and individual philanthropy. All of these sources are critical for NANT funding.