Dissatisfied with the traditional model of scientific research, and encouraged by the success of CTF's Neurofibromatosis Preclinical Consortium (NFPC) and Neurofibromatosis Therapeutic Consortium (NFTC), the Children’s Tumor Foundation designed a prototype that emphasizes collaboration and cooperation among researchers, rather than competition and data hoarding. The Synodos approach is modeled on the acclaimed ‘Stand Up To Cancer’ effort, in which interdisciplinary ‘dream teams’ of scientists, clinicians, technicians, and other experts work in concert with the aim of finding solutions to the unique problems that affect patients.
This model, named Synodos (inspired by the ancient Greek term for "on the same path") is CTF’s unique research collaborative, where unpublished data is shared, resulting in a faster and more efficient translation of top findings for clinical benefit. Key to this partnership is Sage Bionetworks, a research institute that will 1) ensure data and knowledge are quickly disseminated throughout the consortia membership as it is produced, and 2) create an online, publicly-accessible record of the research performed by the consortia, allowing others to freely use the generated date for new purposes. Together, the groups design strategies that will accelerate the drug discovery process for NF1, NF2, and schwannomatosis.
Synodos for NF1 is a $5 million, multi-year initiative that has assembled a team of leading researchers. The aim of Synodos for NF1 is to find solutions to the unique problems that affect NF1 patients in the fastest, most efficient manner by sharing their real-time data and results.
There are three separate consortia that make up Synodos for NF1: one will study low-grade gliomas and two will focus on preclinical acceleration. With this direction, the goal of Synodos for NF1 is to both better understand the biology of an NF1-specific childhood brain tumor and to accelerate drug screening with new cell and animal models. This, in turn, will open new treatment avenues for patients with NF1.
Project 1: Low-Grade Glioma—Low-grade gliomas (LGGs) are childhood brain tumors affecting children with NF1. Despite their prevalence, the current absence of detailed information on the underlying mechanisms of LGGs results in a lack of effective treatment options for patients and their families. The goal is to transform the understanding of the cellular and molecular underpinnings of low-grade gliomas, and identify new targets that will lead to specific and selective treatment options. The LGG group includes labs from Washington University in St. Louis, the German Cancer Research Center in Heidelberg, University of California, San Francisco, and Children’s Hospital of Philadelphia.
Project 2: Preclinical Acceleration—Researchers will develop and perfect animal models that will most accurately replicate human reaction to drugs in preclinical testing. The two preclinical acceleration teams include: the University of Minnesota in collaboration with Cincinnati Children’s Hospital, the National Cancer Institute, and Recombinetics Inc. and the University of Iowa, Sanford School of Medicine, and Exemplar Genetics.
Synodos for NF2 is a $3 million, multi-year initiative that has assembled a team of researchers from twelve world-class labs and medical centers. This comprehensive, collaborative model brought together experts in basic, translational, and clinical research, who have joined forces to share information, free of bureaucratic obstacles and institutional competition.
We identified the most common medical issues by NF2 patients, requested proposals from the entire research community, and designed strategies that have a higher probability of finding solutions. The brightest minds in the NF field are now working on these solutions using a real-time data sharing platform developed through our partnership with Sage Bionetworks.
Neurofibromatosis Type 2 (NF2) is characterized by tumors that develop throughout the central and peripheral nervous system. Though NF2 tumors are benign, these patients experience significant morbidity and mortality because of the location of the tumors, their compressive effects, and the effects of current treatments.
The goal of Synodos for NF2 is to discover novel therapeutic agents for clinical treatment, that either successfully shrink NF2 tumor types, or halt tumor growth without long-term toxicity, or with a manageable long-term toxicity profile.
Synodos for NF2 aims to deliver multiple new and advanced cell and animal models to accelerate drug screening , new target pathways, and an increased understanding of response and resistance to treatment. Eventually this work will result in new clinical trials for NF2, and effective combination therapies.
Synodos for Schwannomatosis is a $1.0 million, two-year initiative that will bring together basic, translational, and clinical researchers towards the end goal of developing effective treatments for schwannomatosis, the rarest and least-studied form of neurofibromatosis. This collaboration aims to find solutions to the unique problems that affect schwannomatosis patients in the fastest, most efficient manner by sharing their real-time data and results.
Led by Dr. Gelareh Zadeh from University of Toronto and Dr. Laura Papi from University of Florence, Italy, the project aims to perform an extensive molecular analysis of schwannomatosis tumor samples to identify new therapeutic targets, and advance the understanding and management of the disease, with special focus on pain. Neurology, neurosurgery, genetics, and neurofibromatosis experts from institutions spread across four different countries are working together to 1) develop a clinically annotated schwannomatosis biobank; 2) develop a comprehensive pain registry for schwannomatosis patients; 3) establish the genomic and epigenomic landscape of schwannomatosis to identify disease-driving alterations; and 4) correlate the molecular findings with key clinical parameters, including pain.