Better Treatment Option for some Low-Grade Glioma Patients Becomes a Reality

March 16 marked a historic day for the pediatric brain cancer community with the passing of the first-ever drugs approved by the FDA to treat children with certain types of brain cancer. The two approved drugs, Tafinlar (dabrafenib) and Mekinist (trametinib), created and manufactured by pharmaceutical company, Novartis, were authorized to be used in combination in first-line treatment of low-grade glioma (LGG) with a BRAF V600E mutation in kids one year and up. This came after the TADPOLE trial showed an overall response rate of 47% and median progression-free survival of 20.1 months for the Tafinlar + Mekinist combination compared to 11% overall response rate and 7.4 months for standard of care, according to Novartis. 


It is important to note that until last week, there had never been any drugs approved specifically for use in children battling pediatric brain cancer. The additional approval of the two drugs in liquid form is another win, as it provides an accessible option to patients as young as one year old. Dr. Brian Rood, Director, Clinical Neuro-Oncology, Medical Director, Brain Tumor Institute, and LBF Scientific advisor, said in a statement, “Over the past several years, the promise of molecularly targeted therapy has begun to be realized in the treatment of BRAF-altered pediatric low-grade glioma. The FDA approval of Dabrafenib and Trametinib for a subset of LGG represents a milestone in the advancement of treatment for these tumors, one that we hope will be just the first for these types of drugs for pediatric brain tumor therapy.” 


While this milestone is historic and shows the progress being made for our smallest warriors battling the deadliest disease in children, there is still more work to do. The Lilabean Foundation for Pediatric Brain Cancer Research (LBF) is proud to support collaborative research efforts for all types of childhood brain cancer. While LBF is hopeful that this is the beginning of many breakthroughs in the pediatric brain cancer space, it is important to note that there are many different types of brain cancer that affect children and over 100 subtypes. Certain brain cancers, such as Diffuse Intrinsic Pontine Glioma (DIPG), Glioblastoma Multiforme (GBM), and others, still have no cure and are fatal to 99% of children diagnosed. We celebrate this milestone but understand there is still more work to do. 


The approval of these drugs offers hope for a future where there are treatment options and personalized medicine for ALL children diagnosed with pediatric brain cancer. Founder and Executive Director Nicole Giroux said in a statement, “The recent approval by the FDA of Tafinlar + Mekinist shows that we, as a pediatric brain cancer community, are making strides towards our common goal of safer and more effective treatment options. For 12 years, LBF has been dedicated to evaluating research and clinical trials for childhood brain cancer. This FDA approval gets us one step closer to not just saving lives but to giving our children a chance to both survive AND thrive.”

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LBF's Transformative Partnership with CBTN Drives Groundbreaking Advances Brain Cancer Research

The Lilabean Foundation’s Transformative Partnership with CBTN Drives Groundbreaking Advances in Pediatric Brain Cancer Research

March 12, 2025
The Lilabean Foundation (LBF) has played a vital role in advancing pediatric brain cancer research, providing significant support to the Children’s Brain Tumor Network (CBTN) based at the Children’s Hospital of Philadelphia. From its origins as a small family foundation, LBF has grown into one of the most influential contributors to CBTN, driving innovative research and achieving new milestones in philanthropy. One of the most notable impacts of LBF’s support is its investment in Project Accelerate, an initiative designed to expedite research and enhance the Pediatric Brain Tumor Atlas. Through LBF’s backing, CBTN has been able to recruit essential data engineers and bioinformaticians, improving research methodologies and strengthening data models. This investment has helped break down silos and create more robust predictive models, optimizing diagnostic accuracy and advancing AI technology. This initiative is positioning CBTN to achieve transformative breakthroughs in precision care and treatment for pediatric brain tumors “The impact of The Lilabean Foundation's generosity cannot be overstated,” said Dr. Adam Resnick, Scientific Director of the Children’s Brain Tumor Network.” “With this partnership, we are paving the way for the next era of pediatric brain cancer treatment and care. Thanks to this continued support, we are one step closer to ensuring that children with brain tumors have the best possible outcomes.” Maximizing Research Potential and Driving AI Innovation: LBF’s contributions have also been instrumental in maximizing the impact of the National Institutes of Health’s (NIH) data generation initiative. With LBF’s support, CBTN was able to extract and ship over 7,000 specimens, enabling the standardization of critical molecular data. This data has formed the foundation for AI-driven technologies, putting CBTN at the forefront of AI research and allowing for discoveries that were once unimaginable. By integrating clinical, molecular, and imaging data into a single multimodal dataset, this collaboration is enhancing the ability to create more individualized and effective treatment plans for children with brain tumors. “The integration of this multimodal data is a game-changer,” said Nicole Giroux, Executive Director of The Lilabean Foundation. “It has the potential to dramatically improve how we understand and treat pediatric brain tumors.” Preparing for Future Breakthroughs: With LBF's continued support, CBTN is now poised for its next big leap in research. The foundation’s investment in Project Accelerate has led to the creation of an extensive multimodal dataset, which was instrumental in securing a first-of-its-kind award from the Advanced Research Projects Agency for Health (ARPA-H). This prestigious award, focused on pediatrics, combined with a new partnership with Amazon Web Services, positions CBTN for major advancements in AI capabilities and further opportunities for breakthrough research. A Legacy of Impact: LBF’s sustained commitment to CBTN is a testament to the power of philanthropy and the profound impact that a dedicated donor community can have on children’s healthcare. Their investments have not only laid the groundwork for groundbreaking research but are also shaping the future of pediatric brain tumor treatment and care, from bench to bedside. “We are incredibly grateful for the support of The Lilabean Foundation,” said Jena Lilly, executive director of CBTN. “Their partnership has been invaluable in driving forward our shared mission to improve the lives of children battling brain tumors. Together, we are creating a future where cures and better treatments are within reach.”
Bethesda magazine features The Lilabean Foundation hero in January/February issue

Bethesda Magazine Features LBF in January/February Issue

February 11, 2025
We are thrilled to be featured in the Bethesda Magazine January/February 2025 edition! It’s a privilege to share the work we’re doing at the Lilabean Foundation to support families and children and the heartfelt origin story of why the foundation exists. Grateful for the opportunity to amplify our mission and continue making a meaningful impact in the community. Thank you Amy Halpern and Bethesda Magazine for capturing and creating such a heartfelt feature. Click here to read the article and the January/February edition of Bethesda Magazine.
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