The Children’s Tumor Foundation’s latest partnership is out of this world! CTF is euphoric to be part of the #YearofOpenScience, a collaboration spearheaded by the Center for Open Science, with support from NASA. Together with 15 additional organizations, these groups will convene for a series of working sessions to align collective action, culminating with a Year of Open Science conference in 2024 to showcase outputs, coalition-building, and ongoing work from these joint efforts.

Through our partnership with Sage Bionetworks, data is available and ready to use in the NF Data Portal. This centralized data repository is managed by specialists who collect, analyze, and release integrated data, accelerating the understanding of all types of neurofibromatosis and schwannomatosis, and the identification of “druggable” targets.

In 2022, the Children’s Tumor Foundation announced a groundbreaking three-year study, which it will fund for nearly $2 million, to determine if a DNA-based blood test can offer better understanding and ultimately earlier diagnosis of malignant peripheral nerve sheath tumor (MPNST) predisposition in NF1 patients. CTF has committed its support to fund the study in its entirety. The project format is inspired by CTF’s highly effective Synodos model, which brings top researchers together to solve complex problems in an open and collaborative environment.

The Children’s Tumor Foundation and the Children’s Hospital of Philadelphia (CHOP) have renewed their five-year study of optic pathway glioma (OPG) in children with NF1. The study involves 25 NF clinics, and aims to provide clinicians with clear criteria that will help them decide when a patient should be treated, and when treatment (such as chemotherapy) should be avoided. OPG develops in 15–20% of children with NF1, and can cause significant health issues. In 2022, the study was renewed, and renamed to memorialize late CTF ambassador Jeffrey Owen Hanson, who suffered from OPG.

CTF sought out the concerns of NF1 patients when establishing Synodos for NF1, a team-science initiative that began its work in 2016 and concluded in 2020. Patient concerns for better treatment options for NF1, as well as answers for low-grade glioma patients, prompted CTF to initiate three separate consortia, bringing together 24 investigators from eight leading institutions and two companies.

The NF Preclinical Initiative (NFPI) began in 2008 as the NF Preclinical Consortium (NFPC), a five-year, $7 million program that concluded in 2013. It expanded as the NF Therapeutic Consortium (NFTC), which continued the work of the NFPC, building on its infrastructure and discoveries.

Traditionally, it takes more than 15 years and costs hundreds of millions of dollars to translate a new discovery into one clinical treatment. The impact of the NFPI was clear: these teams completed 116 preclinical trials in 8 years, at a total cost of $11 million. The preclinical studies led to multiple clinical trials, many of which are currently underway. One of those clinical trials included the MEK inhibitor selumetinib registration trial.