Targeted treatments for pediatric and young-adult gliomas

Targeted Therapies for Glioma

NIH-funded research Brigham and Women's Hospital · NIH-11178561

Targeted drug treatments aimed at the genetic drivers of gliomas in children and young adults.

Quick facts

Grant typeNIH-funded research
Study typeNIH-funded research
Funding institutionBrigham and Women's Hospital NIH-funded
Lab location1 site (Boston, United States)
Project IDNIH-11178561 on NIH RePORTER

What this research studies

Doctors and scientists at Boston hospitals are working together to develop drugs that target the specific genetic changes driving gliomas in children and young adults. For lower-grade tumors driven by BRAF changes, the team is improving RAF inhibitors such as tovorafenib and creating non-invasive, child-friendly tests to predict who will respond. For diffuse midline gliomas, researchers are exploiting a DNA repair weakness called alt-EJ and testing brain-penetrant drugs that attack that vulnerability. The program aims to move promising laboratory discoveries into treatments offered through major Boston hospitals.

Who could benefit from this research

Good fit: Children and young adults with gliomas—especially those whose tumors have BRAF alterations or who have diffuse midline gliomas—are the most likely candidates for participation.

Not a fit: Patients whose tumors do not have the targeted genetic changes or adults with unrelated brain cancers may not receive direct benefit from these projects.

Why it matters

Potential benefit: If successful, this work could produce more effective, less toxic targeted therapies and tests to identify which children will benefit, improving outcomes and quality of life.

How similar studies have performed: RAF inhibitors like tovorafenib have already shown promise for BRAF-driven pediatric low-grade gliomas, while targeted approaches for diffuse midline gliomas remain largely experimental.

Where this research is happening

Boston, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.