Using triacetin to treat propionic acidemia by balancing metabolism
Official title: Triacetin Treatment for Propionic Acidemia by Rebalancing the Acetyl-CoA/Propionyl-CoA Metabolism
Funded at Duke University · NIH-10938373 · Data updated 2026-11-12
This study is looking at whether a safe food additive called triacetin can help people with propionic acidemia by balancing certain chemicals in their bodies to improve energy and reduce health issues related to the condition.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Duke University NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project start | August 15, 2024 |
| Project end | July 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $184,617 |
| NIH project number | 1R21TR005163-01 |
| Project ID | NIH-10938373 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with propionic acidemia, particularly those experiencing complications despite dietary management.
✕Less likely to help
Patients with propionic acidemia who are not able to tolerate dietary interventions or have severe comorbidities may not benefit from this research.
What this research studies
This research investigates the use of triacetin, an FDA-approved food additive, as a treatment for propionic acidemia, a rare metabolic disorder caused by genetic mutations. The study aims to restore the balance between acetyl-CoA and propionyl-CoA levels in the body, which are disrupted in patients with this condition. By supplementing with triacetin, the researchers hope to improve energy metabolism and reduce harmful metabolic effects associated with propionic acidemia. The approach is based on preliminary data showing that acetate supplementation can enhance survival in animal models of the disease.
Why it matters
Potential benefit: If successful, this research could provide a novel and effective treatment option for patients with propionic acidemia, potentially improving their metabolic health and quality of life.
How similar studies have gone: Other research has shown promise in using metabolic interventions for similar conditions, but the specific use of triacetin for propionic acidemia is a novel approach.
Where this research is happening
-
Duke University
Durham, United States Active
Researchers
- Principal investigator: Zhang, Guofang — Duke University
- Study coordinator: Zhang, Guofang
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.