Founded in 2000 and headquartered at Children's Hospital Los Angeles, the New Approaches to Neuroblastoma Therapy (NANT) consortium is a group of 15 children's cancer centers across North America and three European sites in the Netherlands, Great Britain, and France with established research and treatment programs for neuroblastoma. NANT is the only consortium in the world focused on developing novel therapies and biomarkers for high-risk neuroblastoma. NANT clinical trials include pharmacokinetic, pharmacodynamic, biologic and immunologic evaluations, and provision for patient specimen banking. Safety and response data from NANT trials coupled with NANT leadership and infrastructure foster academic, federal and industry collaborations to accelerate transition of novel approaches developed by NANT Investigators to randomized international trials.
It is rare that a clinical trial is changed while in progress. But when results are striking enough, it can occur. Such striking results have been reported in a study led by NANT.
For patients with relapsed/refractory high-risk neuroblastoma with alterations in the ALK gene, lorlatinib was shown to be a safe, effective targeted therapy. This finding—published in Nature Medicine—was so significant that the Children's Oncology Group (COG) modified its phase 3 protocol to replace another ALK inhibitor (crizotinib) with lorlatinib to treat patients with newly diagnosed high-risk neuroblastoma with an ALK alteration. The NANT lorlatinib trial was based on compelling data from the lab of NANT investigator Dr. Yael Mosse at Children’s Hospital of Philadelphia.
Araz Marachelian, MD, Medical Director of NANT's Operations Center was the NANT study's sponsor and co-senior author with Dr. Mosse of the Nature Medicine publication. NANT's role included early collaboration on evaluating laboratory data, trial design, FDA approval, industry sponsorship, study oversight, safety monitoring, creating the study database, and analyzing and disseminating results. “We are thrilled to see many years of NANT's planning and operation come to fruition with the results of this important study,” says Julie Park, MD, Chair of the NANT consortium. “Families are urgently waiting for new treatment possibilities like this one.” Dr. Park is an established neuroblastoma expert and chair of the Department of Oncology at St. Jude Children's Research Hospital.
The mission of the NANT Parent Advisory Committee (NANT-PAC) is to enhance the work of the NANT consortium by providing parent and family input during the development and conduct of NANT clinical trials, to assist with effective communication to the neuroblastoma family community and related foundations, to develop educational tools for families, to assist with fundraising, and to provide support to families participating in NANT trials. The NANT-PAC is composed of neuroblastoma parents, the NANT Medical Liaison (pediatric oncologist), and the NANT Administrative Director.
All donations made to NANT are 100% applied to NANT projects.
(No part of donations is used for administrative costs)
Donations may be used for:
Specific NANT Trials:
Funding may be used to support the development or conduct of a specific therapeutic trial, or its associated laboratory research.
NANT Operations Center:
The Operations Center provides the infrastructure that is essential for the development, conduct, and analysis of data results for all NANT trials. The Operations Center is also responsible for meeting patient safety and other regulatory requirements. All study data is maintained centrally at the Operations Center. Funding to support the Operations Center is essential to make all NANT trials possible.
Correlative Biology Studies:
Each NANT therapy trial includes obtaining patient blood, bone marrow, and or tumor samples that are provided to collaborating NANT lab investigators for studies to confirm the proposed mechanism of a new treatment, understand possible reasons for treatment failures, and guide development of successor trials. Funding is used for the collection, processing, and shipping of these patient specimens. Some examples of correlative studies include levels of drugs in the blood, detection of minimal amounts of tumor in blood, genetic testing, and effects of drugs on the patient’s immune system and the tumor microenvironment.
Patient enrollment at NANT member sites:
Each NANT site has a lead physician (Principal Investigator) that is a leader in childhood cancer research and is recognized for their expertise in neuroblastoma. They have overall responsibility for the conduct of NANT studies at their institutions and also participate in developing the overall strategies of the NANT consortium. NANT trials also require a clinical research team at each site that is critical to assist the Principal Investigator to conduct and closely monitor each
If you have a question about a specific NANT trial, please contact the NANT Operations Center.
If you wish to have a consult regarding therapy recommendations for yourself/your child, please contact one of our NANT sites directly, or the NANT Operations Center.
If you have other questions, please contact the NANT Operations Center, located at Children’s Hospital Los Angeles. The Operations Center is responsible for the coordination and management of all NANT clinical trials and is our central communications center.
