Blocking ACLY to slow cancer and metabolic disease
Metabolic regulation and inhibition of ATP-citrate lyase
Researchers are testing drugs that block the ACLY enzyme to try to slow cancer growth and help with metabolic and cardiovascular conditions.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11302626 on NIH RePORTER |
What this research studies
As a patient, this project looks at how ACLY — an enzyme that helps make acetyl-CoA — supports tumor growth and metabolic problems. The team will determine ACLY's structure and study how chemical inhibitors, including compounds related to bempedoic acid, change the enzyme's activity in cells and lab models. They will also examine ACLY's modifications inside cells (like phosphorylation and acetylation) and how those changes relate to cancer behavior. Findings will be used to prioritize which ACLY-blocking compounds might move toward clinical testing.
Who could benefit from this research
Good fit: People with hepatocellular carcinoma, other cancers with high ACLY activity, or metabolic disorders linked to elevated ACLY would be the most likely candidates for related future trials.
Not a fit: Patients whose disease is not driven by ACLY activity or those seeking immediate treatment are unlikely to receive direct benefit from this laboratory-focused work.
Why it matters
Potential benefit: If successful, this work could lead to new ACLY-blocking drugs that slow tumor growth or improve treatment options for some metabolic and cardiovascular conditions.
How similar studies have performed: Several ACLY inhibitors have been developed and bempedoic acid is FDA-approved for lowering cholesterol, but using ACLY blockers to treat cancer remains largely experimental.
Where this research is happening
Philadelphia, United States
- University of Pennsylvania — Philadelphia, United States (Active)
Researchers
- Principal investigator: Marmorstein, Ronen — University of Pennsylvania
- Study coordinator: Marmorstein, Ronen
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 below.