The Children's Tumor Foundation
12/16/2025 | Press release | Distributed by Public on 12/16/2025 15:06
No One is Delivering More Breakthroughs for NF2 than CTFEmpty heading
The impact is unmistakable - we're driving the strongest period of NF2 progress the world has ever seen.Empty heading
Brigatinib is already shrinking NF2 tumors by up to 30% and restoring hearing in clinical trials - a milestone of real, measurable progress for patients.
It was CTF's investment that uncovered brigatinib's potential - no one knew it would work for NF2 until CTF proved it.
That breakthrough is a powerful example of our rapidly accelerating NF2 engine. We are:
Co-funding multi-drug NF2 trials with Takeda - a first for NF2 and a model that gives more 'shots on goal' than ever before
Driving 24 active NF2-SWN investigations across 17 labs in 8 countries, spanning biomarkers, tumor models, hearing research, and precision medicine (read about them below)
Pushing gene therapy forward through lipid nanoparticles, opening entirely new therapeutic possibilities
Powering 4 treatments in clinical trials TODAY, not someday - today!
This is a unified NF2 portfolio - science, trials, global labs, the NF Conference and NF Summit, and the NF community - all aligned and moving together with speed and purpose.
No one has invested more, delivered more, or built a richer, faster-growing NF2 pipeline than CTF.
Every major step forward in NF2 research can be traced back to the Children's Tumor Foundation.
This is what CTF does: we build the movement, we drive the science, and we push to deliver for patients living with NF2.
And because our NF2 engine is active and accelerating, the next breakthrough is already at the starting line.
* * * Breaking News: A New NF2-SWN Trial is Ready to Launch * * *Empty heading
We are preparing to fuel a new NF2 drug trial - a powerful combination therapy designed to give NF2 patients and families another urgently needed option.
The trial is ready to begin.
The drug has been donated by a pharma partner.
And CTF has already committed $1 million to move it forward.
But we urgently need the final half million to move this drug into trial!
This is all that stands between our NF2-SWN patients and another important treatment option.
This is the world's best hope for your loved one - and the window is open right now.
Donate directly for NF2Empty heading
"No one is doing more for NF2 than the Children's Tumor Foundation."
RB Harrison, parent to a child with NF2-SWN
The Scale of NF2 Progress We're Driving Right NowEmpty heading
$4.6 million invested in 24 active NF2-SWN initiatives
This is a coordinated, global NF2 portfolio - new drugs, new models, new science - all pushing toward more treatment options for NF2 patients.
Here's a clear look at CTF's leadership across the NF2 landscape.
NF2-SWN Clinical Trials & Quality of Life
Getting treatments to patients now - and improving how trials measure what matters.
Phase 1/2 clinical trial of PRG-N-01, Bum-Joon Park, PRG S&TECH INC - $150,000
INTUITT-NF2 platform trial, Scott Plotkin, Mass General - $417,040
Repurposing anti-retroviral drugs for NF2 tumors, Oliver Hanemann, University of Plymouth - $445,141
Improving quality-of-life measures for NF2 trials, Vanessa Merker, Mass General - $149,972
Statistical support for INTUITT-NF2, Lorenzo Trippa, Dana-Farber - $44,000
NF2-SWN Gene Therapy
Advancing next-generation treatments and opening new therapeutic possibilities.
Gene therapy for vestibular schwannomas, Alexandra O'Donohue, University of Sydney - $163,373
LNP-mediated mRNA therapy in NF2-SWN, Helen Morrison, Leibniz Institute on Aging - $397,947
Vestibular Schwannoma & Hearing Loss
Protecting and restoring hearing - a top priority for NF2 families.
Targeting HIF-2 for NF2 vestibular schwannoma, Lei Xu, Mass General - $199,966
Blood biomarkers for hearing & tumor changes, Konstantina Stankovic, Stanford - $150,000
Proteome analysis in NF2 hearing loss, Isam Naber, UCLA - $155,224
NF2-SWN Drug Discovery
Identifying new therapies and overcoming drug resistance.
Multidrug resistance in meningioma & schwannoma, Sylwia Ammoun, University of Plymouth - $175,700
Apelin-mediated angiogenesis in NF2 tumors, Srirupa Bhattacharyya, Mass General - $146,862
Cell-death pathways in NF2-SWN, Anna Nagel, University of Central Florida - $151,272
Targeting YAP/TEAD in NF2, Dominique Lallemand, Inserm - $85,000
OMV-based immunotherapy for NF2 tumors, Sherif Ahmed, Mass General - $200,000
ALDH1A3 in NF2 meningioma, Libby Williams, University of Plymouth - $122,016
CCL2-CCR2 axis in tumor growth & hearing loss, Simeng Lu, Mass General - $169,898
Using multi-omics and artificial intelligence to develop precision medicine approaches for adolescents and young adults with NF2 and associated meningiomas, Leo Yefet, Mayo Clinic Rochester -$175,538
Cross-Condition Projects
hared science that advances NF2, schwannomatosis, and NF1, together.
CTF-NF Registry + Mayo electronic health records, Mayowa Osundiji, Mayo Clinic Arizona - $114,348
ClinGen Variant Curation Expert Panel for NF1, NF2 & SWN, Elisabeth Castellanos-Perez, Germans Trias i Pujol Research Institute - $149,886
Deep-learning MRI tumor-burden quantification, Gordon Harris, Mass General - $149,866
NF2-SWN Preclinical Models
Building the models that make drug testing faster and more accurate.
NF2-SWN xenograft mouse model, Helen Morrison, Leibniz Institute on Aging - $175,945
NF1/NF2 mouse model validation (CTF-Taconic-Cyagen), D. Wade Clapp, Indiana University - $152,266
Humanized meningioma mouse model, Liyam Laraba, University of Plymouth - $373,497
The Children's Tumor Foundation published this content on December 16, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on December 16, 2025 at 21:06 UTC. If you believe the information included in the content is inaccurate or outdated and requires editing or removal, please contact us at [email protected]