New Approaches to Neuroblastoma Therapy (NANT)

New Therapy, New Hope

Established in 2000, NANT is the only international clinical trials consortium focused solely on developing new treatments and biomarkers for relapsed/refractory high-risk neuroblastoma through early phase (1/2) clinical trials.

High-risk neuroblastoma remains a challenge, despite improvements seen with intensive chemotherapy, isotretinoin, and immunotherapy. Further advances require increased understanding of the fundamental biology and genetics of neuroblastoma, and more individualized precision therapies.

NANT brings together a diverse team of investigators from experienced pediatric oncology centers to identify promising treatment candidates, perform pre-clinical testing, and then  design and carry out early-phase testing of these novel treatments. Because neuroblastoma is a rare disease with only 700-800 new cases in the U.S. each year, no single site could enroll enough patients to produce meaningful data. NANT sites enroll patients and collect consistent data that can then provide rationale for larger Children’s Oncology Group clinical trials and offer patients access to novel therapies across a broad geographic area in North America and Europe. NANT integrates the input of families fighting neuroblastoma via our Parent Advisory Committee.

Our vision is to cure all children with high-risk neuroblastoma using novel precision therapies developed by our expert network of laboratory and clinical investigators.


April 2023:  NANT investigators demonstrate that lorlatinib has potent anti-tumor effect in patients with neuroblastoma linked to a specific genetic alteration.

It is rare that a clinical trial is changed mid-investigation. But when results are striking enough, it can occur. Such striking results have been reported in a study led by the New Approaches to Neuroblastoma (NANT) pediatric cancer consortium, which is headquartered at Children’s Hospital Los Angeles (CHLA).

For patients with relapsed/refractory neuroblastoma with alterations in the ALK gene, lorlatinib has been shown to be a safe, effective targeted therapy. This finding—published in Nature Medicine—is so significant that the Children’s Oncology Group (COG) modified its phase 3 protocol to replace another ALK inhibitor (crizotinib) with lorlatinib to treat newly diagnosed neuroblastoma patients with an ALK alteration.

Araz Marachelian, MD, Medical Director of NANT’s Operations Center and CHLA’s Neuroblastoma MIBG Program, is the study’s sponsor and co-senior author on the Nature Medicine publication. “At NANT, we are pleased to have had a leadership role in this important discovery,” says Dr. Marachelian. 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.

First author on the paper is Kelly C. Goldsmith of Children’s Healthcare of Atlanta and co-senior author is Yael P. Mosse of Children’s Hospital of Philadelphia. The trial was conducted at national and international sites including: Vanderbilt University Medical Center, Children’s Hospital Los Angeles (USC Keck School of Medicine), Children’s Hospital of Philadelphia, University of Iowa Hospital and Clinics, Children’s Hospital of Philadelphia, Stanford University School of Medicine, Perelman School of Medicine at the University of Pennsylvania, The Royal Marsden Hospital (London, UK), Cook Children’s Medical Center, University of Chicago, University of Michigan, Hospital for Sick Kids (Toronto, ON), Harvard Medical School, Children’s Hospital Colorado, Seattle Children’s Hospital, Institut Curie (Paris, France) and UCSF Benioff Children’s Hospital. The study was funded by National Cancer Institute grant P01CA217959, Pfizer Inc. and other grants and foundations.

NANT Celebrated its first 20 years of accomplishments in 2020

You can hear NANT investigators presenting NANT’s accomplishments in its first 20 years, current NANT trials, and successor trials in the Children’s Oncology Group based on NANT trial data.  This lecture was presented via the Coalition Against Childhood Cancer (CAC2) in 2020.