New drug approaches for Balamuthia brain infection

Novel Structure-Based Therapeutic Discovery for Balamuthia mandrillaris

['FUNDING_R21'] · NORTHEASTERN UNIVERSITY · NIH-11231669

This project is creating and improving small-molecule drugs aimed at treating deadly Balamuthia mandrillaris brain infections.

Quick facts

Phase['FUNDING_R21']
Study typeNih_funding
SexAll
SponsorNORTHEASTERN UNIVERSITY (nih funded)
Locations1 site (BOSTON, UNITED STATES)
Trial IDNIH-11231669 on ClinicalTrials.gov

What this research studies

From a patient's perspective, scientists are using computer-based models of the amoeba’s proteins to design and prioritize drug-like compounds, then making those compounds in the lab and testing them against the amoeba. The team is building on earlier findings that two PI3K-targeting compounds worked better in the lab than some currently recommended treatments. Medicinal chemists will re-optimize promising molecules and laboratory biologists will measure how well they kill or stop the amoeba. This is preclinical work intended to identify candidates that could move into future clinical testing.

Who could benefit from this research

Good fit: People with confirmed or suspected Balamuthia mandrillaris infection (granulomatous amoebic encephalitis) would be the eventual candidates for any drugs developed from this program.

Not a fit: Patients with unrelated illnesses or those needing immediate lifesaving care are unlikely to benefit directly from this early preclinical research.

Why it matters

Potential benefit: If successful, this work could produce safer, more effective medicines specifically targeting Balamuthia infections and offer doctors better treatment options.

How similar studies have performed: The team previously found two PI3K inhibitor compounds with superior lab activity compared with some current CDC-recommended drugs, but translating that to proven clinical treatments remains unproven.

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.

View on NIH RePORTER →

Last reviewed 2026-05-15 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.