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Children’s Tumor Foundation and Global Coalition for Adaptive Research Announce Strategic Alliance to Implement NF Platform Clinical Trial

Collaboration Will Accelerate Development of Treatments for Neurofibromatosis and Schwannomatosis Patients

A close-up of logos.NEW YORK – The Children’s Tumor Foundation (NEW YORK, NY and BRUSSELS, BELGIUM) and the Global Coalition for Adaptive Research (LARKSPUR, CA) – The Children’s Tumor Foundation (CTF) and Global Coalition for Adaptive Research (GCAR), today announced their alliance to accelerate the development of treatments for patients with NF (neurofibromatosis or schwannomatosis) – a group of rare and often debilitating diseases with high unmet medical need. CTF and GCAR are partnering to operationalize a first-of-its-kind clinical trial for patients with NF that was initially designed through the EU-PEARL initiative.

The announcement of this strategic alliance follows a 3-year, 26-million-euro investment from the Innovative Health Initiative (IHI), formerly called the Innovative Medicines Initiative (IMI).  EU-PEARL was a public-private partnership dedicated to transforming clinical trials by making them more efficient and patient-centered. Through this partnership, leading organizations representing patients, clinicians, researchers, health authorities and industry collaborated to design the first-ever Phase 1 / Phase 2 global platform basket trial for patients with neurofibromatosis type 1 (NF1) or schwannomatosis (SWN), including NF2-related schwannomatosis (NF2-SWN).

CTF is the global leader in neurofibromatosis and schwannomatosis research, dedicated to funding and driving innovative research that will result in effective treatments for the 4 million people worldwide living with NF, a group of genetic disorders that causes tumors to grow on nerves throughout the body. One in every 2,000 people is born with neurofibromatosis or schwannomatosis, which may lead to blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, or cancer.

“Central to CTF’s mission is advancing care and treatments for the NF community. The establishment of the NF platform trials will expedite the development of effective treatments for patients,” said Dr. Annette Bakker, President of the Children’s Tumor Foundation. “We are delighted to partner with GCAR, a leading expert in developing and operationalizing master protocols and adaptive platform trials to actualize this innovative trial.”

GCAR was formed with the mission to speed the discovery and development of treatments for patients with rare and deadly diseases by serving as the sponsor of innovative trials. Its first initiative was to implement the clinical trial GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment) into a self-sustaining engine of therapeutic and biomarker development for patients with glioblastoma. Since the launch of GBM AGILE, GCAR also initiated U.S. sponsorship for innovative global trials in COVID-19 and is advancing platform trials for other rare and deadly diseases such as high-grade ovarian cancer.

“We are excited to work with the Children’s Tumor Foundation to build a patient-centric clinical trial that will rapidly and efficiently evaluate multiple investigational therapies for the treatment of NF,” said Dr. Meredith Buxton, CEO & President of the Global Coalition for Adaptive Research. “GCAR is committed to leading the way in clinical trial innovation by uniting stakeholders to advance new therapies for patients. Our alliance with CTF exemplifies a collaboration that places patients and the community at the forefront of clinical research.”

The NF platform trials will be built to complement CTF’s NF Preclinical Hub, a comprehensive infrastructure to accelerate the availability of clinical trial-ready treatments, including gene therapy. The preclinical hub will provide access to a pipeline from concept to clinic, pre-negotiated master service agreements, protocols for compound testing, as well as access to testing models, data tools, libraries, and biomarkers. The plan is to launch an NF1 platform trial for four different manifestations including plexiform neurofibroma, cutaneous neurofibroma, optic pathway glioma, and low-grade glioma, with future plans to build a platform trial to support SWN patients. Additionally, the Foundation’s NF Patient Registry provides dependable access to NF patients available to participate in NF clinical trials and research studies.

With the establishment of the NF platform trials (an NF1 trial followed by an SWN trial), pharma companies are invited to explore testing of their therapies, as well as investors interested in the opportunity to fuel the progress to approved treatments.


About the Children’s Tumor Foundation (CTF)
The Children’s Tumor Foundation (CTF) is the world’s leading nonprofit dedicated to funding and driving innovative research resulting in effective treatments for the millions of people worldwide living with NF. Guided by a bold and innovative strategic plan, CTF is laser-focused on delivering therapies (right treatments at the right time) to patients who are accurately diagnosed. In addition to driving bench to bedside research, CTF manages global public-private multi-disciplinary consortia, and has invested in a gene therapy and small molecule company. The Foundation is now actively working with ten pharma/ biotech companies to advance therapies for NF, and is inviting other companies to join. CTF recognizes the unique niche role of disease foundations in the R&D ecosystem, involving the patient voice in every initiative. While being committed to building an enterprise that will benefit patients with NF, CTF hopes that the model can act as a blueprint to accelerate drug discovery and development in the larger rare disease community. CTF acknowledges Goodwin for its continuous pro bono support. For more information, please visit:

About Global Coalition for Adaptive Research (GCAR)
The Global Coalition for Adaptive Research (GCAR) unites physicians, clinical researchers, advocacy and philanthropic organizations, biotech/pharma, health authorities, and other key stakeholders in healthcare to expedite the discovery and development of treatments for patients with rare and deadly diseases. As Sponsor of innovative trials, including master protocols and adaptive platform trials, GCAR is dedicated to the advancement of science by modernizing clinical trials that support more efficient, less costly drug development. Adaptive platform trials can accelerate the time from discovery in the lab to implementation in the clinic resulting in better treatments and lives saved. To learn more about GCAR and its initiatives, visit


Click here to read more about the strategic alliance and NF platform trials.


Children’s Tumor Foundation
Simon Vukelj

Global Coalition for Adaptive Research
Rachel Rosenstein-Sisson