Targeting harmful brain-leptomeningeal cancer environment by reducing branched-chain keto acids
Official title: Targeting branched-chain keto acid accumulation in melanoma leptomeningeal disease
Funded at H. Lee Moffitt Cancer Ctr & Res Inst · NIH-11429312 · Data updated 2026-11-12
Aims to see if lowering branched-chain keto acids in the brain’s fluid can slow melanoma spread to the brain covering and help immune cells fight the cancer.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | H. Lee Moffitt Cancer Ctr & Res Inst NIH-funded |
| Lab location | 1 site (Tampa, United States) |
| Project start | August 1, 2026 |
| Project end | July 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $694,309 |
| NIH project number | 1R01CA315693-01 |
| Project ID | NIH-11429312 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with advanced melanoma who have leptomeningeal disease or are at risk for central nervous system involvement.
✕Less likely to help
Patients without leptomeningeal disease or those not eligible for central nervous system participation may not directly benefit.
What this research studies
This project focuses on leptomeningeal disease in late-stage melanoma, a tough-to-treat condition where cancer sits in the brain’s surrounding fluids. Researchers will study how branched-chain keto acids build up in cerebrospinal fluid and how they affect tumor growth and immune cell function. They plan experiments to understand whether reducing these acids can improve treatment effectiveness and T cell activity at the disease site. The work combines lab studies with analyses of patient samples to identify potential therapy targets specific to this brain-adjacent environment.
Why it matters
Potential benefit: If successful, the approach could lead to new treatments that slow tumor growth and boost immune responses in melanoma with leptomeningeal involvement.
How similar studies have gone: Early findings suggest this mechanism may influence tumor growth and immune function, but human-targeted therapies in this niche are still exploratory.
Where this research is happening
-
H. Lee Moffitt Cancer Ctr & Res Inst
Tampa, United States Active
Researchers
- Principal investigator: Smalley, Inna — H. Lee Moffitt Cancer Ctr & Res Inst
- Study coordinator: Smalley, Inna
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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.