The Children's Tumor Foundation
06/22/2026 | Press release | Distributed by Public on 06/22/2026 09:56
CTF-Funded Research Advances Understanding of MPNST–and Identifies a Promising Drug Combination
Research funded by the Children's Tumor Foundation (CTF) is helping to answer one of the most complex questions in NF: how benign tumors transform into aggressive cancers, and how to stop that process.
A man speaks at a podium labeled "Children's Tumor Foundation" with a microphone, while others stand in the background against a blue backdrop.Eduard Serra, PhD
A study led by Eduard Serra, PhD, and his team at the Germans Trias i Pujol Research Institute (IGTP), in collaboration with multiple research groups, including the NIH's National Center for Advancing Translational Sciences (NCATS), has been published in Nature Communications. Supported through CTF's Drug Discovery Initiative (DDI), this work marks an important step forward in malignant peripheral nerve sheath tumor (MPNST) research.
Modeling How MPNST Develops
MPNST is a rare but life-threatening cancer that can develop in people with NF1. One of the biggest challenges in treating MPNST has been a limited understanding of how these tumors form and evolve.
In this study, researchers developed an advanced laboratory model using induced pluripotent stem cells (iPSCs) to recreate the progression from benign to malignant tumor states.
By systematically introducing key genetic changes known to occur in NF1-associated tumors-loss of NF1, CDKN2A, and PRC2-the team was able to model the transition from:
neurofibroma-like tumors
to early-stage MPNST
The findings show that loss of PRC2 plays a critical role in this transformation. It disrupts how cells regulate their genes, pushing them away from their normal state and toward one associated with tumor growth.
Importantly, these changes closely mirror what is observed in human tumor samples, strengthening the relevance of the model.
A Platform for Drug Discovery
Using these 3D tumor models, the researchers conducted a high-throughput screen of compounds to identify potential therapeutic strategies.
They found that PARP inhibitors-a class of drugs already used in other cancers-show selective activity in tumors with PRC2 loss, a common feature of NF1-associated MPNST.
Building on this, the team tested several combinations with the PARP inhibitor olaparib and concluded that the combination of olaparib (a PARP inhibitor) and selumetinib (a MEK inhibitor approved for NF1-related tumors) was well tolerated and significantly reduced tumor growth in this patient-derived MPNST model.
Why This Matters
This research delivers progress on multiple fronts:
Mechanism: It clarifies how genetic changes drive the transition from benign to malignant tumors in NF1.
Modeling: It introduces a new biologically relevant system for studying MPNST and testing therapies.
Therapeutic insight: It identifies a targeted drug combination with demonstrated activity in preclinical models.
Together, these advances create a clearer path toward developing more effective treatments for MPNST.
This work reflects the core purpose of CTF's Drug Discovery Initiative: to fund research that moves beyond understanding disease biology and toward actionable therapeutic strategies.
By supporting the development of better models and enabling large-scale drug screening, CTF helps accelerate the identification of treatments that can move into clinical testing.
This study is an example of that approach in action-connecting fundamental biology to potential new options for patients
The Children's Tumor Foundation published this content on June 22, 2026, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on June 22, 2026 at 15:56 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]