Using a drug for Gaucher Disease to enhance cancer treatment in triple-negative breast cancer.

Repurposing Glucosylceramide Synthase to Promote Mitochondrial Lethality and Potentiate an Anti-Tumor Immune Response in Triple-Negative Breast Cancer

NIH-funded research University of Tx Md Anderson Can Ctr · NIH-11070404

This study is looking at whether a drug called eliglustat, which is already used for Gaucher Disease, can help treat triple-negative breast cancer by making cancer cells die and boosting the immune system's ability to fight the tumor.

Quick facts

Grant typeR37 grant
Study typeNIH-funded research
Funding institutionUniversity of Tx Md Anderson Can Ctr NIH-funded
Lab location1 site (Houston, United States)
Project IDNIH-11070404 on NIH RePORTER

What this research studies

This research investigates the potential of repurposing eliglustat, a drug approved for Gaucher Disease, to treat triple-negative breast cancer (TNBC). The approach focuses on targeting a specific lipid metabolism feature in TNBC cells, which may lead to increased cancer cell death and stimulate an immune response against the tumor. By promoting the accumulation of certain toxic lipids, the research aims to shift cancer cell behavior from survival to death, while also enhancing the infiltration of immune cells into the tumor. The study will evaluate the effectiveness of eliglustat in preclinical models to establish its role as a new treatment option for TNBC patients.

Who could benefit from this research

Good fit: Ideal candidates for this research are patients diagnosed with triple-negative breast cancer who may benefit from new therapeutic strategies.

Not a fit: Patients with other types of breast cancer or those who are not eligible for immunotherapy may not receive benefits from this research.

Why it matters

Potential benefit: If successful, this research could provide a novel treatment option that not only targets cancer cells but also boosts the immune system's ability to fight tumors.

How similar studies have performed: Previous research has shown promise in repurposing existing drugs for cancer treatment, indicating potential success for this novel approach.

Where this research is happening

Houston, 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.
Conditions anti-canceranti-cancer immunotherapy
Last reviewed 2026-06-10 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.