Why some IDH-mutant brain tumors respond to IDH-targeted medicines

Dissecting the Determinants of IDH-mutant Gliomas Response to Mutant IDH Inhibitors

NIH-funded research Massachusetts General Hospital · NIH-11164628

This project will find out why some people with IDH-mutant gliomas get tumor shrinkage from drugs that block mutant IDH while others do not.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionMassachusetts General Hospital NIH-funded
Lab location1 site (Boston, United States)
Project IDNIH-11164628 on NIH RePORTER

What this research studies

If you take part, researchers will compare tumor samples from people whose IDH-mutant gliomas responded to IDH inhibitors with samples from people whose tumors did not respond. They will use single-cell multi-omics to profile genes, epigenetic marks, and cell states in individual tumor cells before and after IDH inhibitor exposure. The team will look for signs that the drugs push immature tumor cells to become more mature glial cells and will study tumor-intrinsic factors (like ATRX, 19q, or p53 changes) and the tumor environment that might affect response. Findings aim to explain who benefits from IDH inhibitors and suggest ways to improve treatment for others.

Who could benefit from this research

Good fit: Ideal candidates are people with confirmed IDH-mutant gliomas who can provide tumor tissue and clinical treatment information, including cases treated with IDH inhibitors.

Not a fit: Patients without an IDH mutation or those who cannot provide tumor samples or treatment data would not be eligible and are unlikely to benefit directly from this project.

Why it matters

Potential benefit: If successful, this work could help doctors match people with IDH-mutant gliomas to IDH inhibitor therapy or find strategies to make those drugs work better.

How similar studies have performed: Prior clinical reports show limited benefit of IDH inhibitors in high-grade gliomas but some objective responses in subsets of low-grade tumors, so this builds on early, mixed clinical signals.

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.