New treatment for Cushing Disease using a combination therapy
Official title: Fimepinostat, Combination HDAC and Pi3-kinase Inhibitor Tumor-Directed Therapy for Cushing Disease (IND 168501 - 07/31/2023)
Funded at University of California Los Angeles · NIH-10973945 · Data updated 2026-11-12
This study is testing a new medication called Fimepinostat to see if it can help people with Cushing Disease, which is caused by a tumor in the pituitary gland, by looking at how safe and effective it is over four weeks of treatment.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Los Angeles NIH-funded |
| Lab location | 1 site (Los Angeles, United States) |
| Project start | September 5, 2024 |
| Project end | June 30, 2028 |
| Funding year | 2024 |
| Award (this year) | $424,824 |
| NIH project number | 1R01FD007548-01A2 |
| Project ID | NIH-10973945 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with de novo, persistent, or recurrent Cushing Disease.
✕Less likely to help
Patients with other types of pituitary disorders or those who do not have Cushing Disease may not benefit from this research.
What this research studies
This research investigates the use of Fimepinostat, a dual inhibitor targeting specific pathways involved in Cushing Disease, which is caused by a tumor in the pituitary gland. The study will involve a short-term treatment period of four weeks, where patients will receive either a higher or lower dose of the medication to evaluate its safety and effectiveness. Patients will be closely monitored for changes in cortisol levels and overall health during the trial. The goal is to provide a new therapeutic option for individuals suffering from this rare and serious condition.
Why it matters
Potential benefit: If successful, this research could lead to a new effective treatment for patients with Cushing Disease, potentially improving their quality of life and reducing healthcare costs.
How similar studies have gone: While this approach is novel in the context of Cushing Disease, similar combination therapies have shown promise in other tumor types, suggesting potential for success.
Where this research is happening
-
University of California Los Angeles
Los Angeles, United States Active
Researchers
- Principal investigator: Heaney, Anthony P — University of California Los Angeles
- Study coordinator: Heaney, Anthony P
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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.