Using a drug for Gaucher Disease to enhance cancer treatment in triple-negative breast cancer.
Official title: Repurposing Glucosylceramide Synthase to Promote Mitochondrial Lethality and Potentiate an Anti-Tumor Immune Response in Triple-Negative Breast Cancer
Funded at University of Tx Md Anderson Can Ctr · NIH-11070404 · Data updated 2026-11-12
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 type | R37 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Tx Md Anderson Can Ctr NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project start | May 31, 2023 |
| Project end | April 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $480,171 |
| NIH project number | 5R37CA269611-03 |
| Project ID | NIH-11070404 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients diagnosed with triple-negative breast cancer who may benefit from new therapeutic strategies.
✕Less likely to help
Patients with other types of breast cancer or those who are not eligible for immunotherapy may not receive benefits from this research.
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.
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 gone: Previous research has shown promise in repurposing existing drugs for cancer treatment, indicating potential success for this novel approach.
Where this research is happening
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University of Tx Md Anderson Can Ctr
Houston, United States Active
Researchers
- Principal investigator: Fahrmann, Johannes F — University of Tx Md Anderson Can Ctr
- Study coordinator: Fahrmann, Johannes F
Related research
Other NIH-funded projects on the same topics.
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Genomic study to understand how genetics and epigenetics shape triple-negative breast cancer in African ancestry
This study looks at how inherited genes and gene activity affect triple-negative breast cancer in people of African ancestry to better understand why outcomes can be worse.
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Co-Targeting IL-6 and CDK4/6 to Prevent TNBC in Women Veterans
A study aims to block two cancer-promoting pathways (IL-6 and CDK4/6) to prevent or reduce progression of triple-negative breast cancer in women veterans.
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Aimed at understanding how heavy metals like cadmium might trigger a dangerous breast cancer type (TNBC) in people, especially those with BRCA1 risk, to identify possible future treatment strategies.
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Dormancy in Triple-Negative Breast Cancer: How Liver and Skin Metastases Go Quiet or Reactivate
Researchers will study how dormant cancer cells in liver and skin metastases of triple-negative breast cancer become quiescent or wake up, using organ-on-chip models and single-cell analysis to map chromatin states.
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Rac-enhanced CAR-Macrophages against TNBC
A new cell therapy approach uses engineered macrophages with a CAR and Rac2 activation to target and kill triple-negative breast cancer cells expressing CSPG4.
-
A new peptide therapy to help treat metastatic triple-negative breast cancer
A new peptide therapy called PEG-FUD is being tested to help treat metastatic triple-negative breast cancer by normalizing the tumor environment to improve treatment delivery.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.