Understanding FTLD/FTD and ALS biology to find treatments
Official title: Expanding insights into FTD disease mechanisms
Funded at University of Miami School of Medicine · NIH-11518852 · Data updated 2026-11-12
A research grant from NIH aims to uncover how frontotemporal lobar degeneration relates to ALS and to identify new biomarkers and therapeutic targets that could help people with these conditions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Miami School of Medicine NIH-funded |
| Lab location | 1 site (Coral Gables, United States) |
| Project start | December 15, 2024 |
| Project end | March 31, 2033 |
| Funding year | 2026 |
| Award (this year) | $1,055,613 |
| NIH project number | 7R35NS137447-03 |
| Project ID | NIH-11518852 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with FTLD/FTD or ALS, or individuals carrying FTLD-related genetic mutations, may benefit from future biomarker and therapy developments.
✕Less likely to help
Immediate clinical benefit is not guaranteed; findings may inform future trials rather than provide a direct treatment now.
What this research studies
This project studies the biology of frontotemporal lobar degeneration (FTLD), which underlies frontotemporal dementia and is linked to ALS. Researchers examine key molecules such as C9orf72, TDP-43, progranulin, tau, and TMEM106B to understand how disease processes start and progress. The work emphasizes translating discoveries into biomarkers and potential treatment targets that could benefit patients now and in the future. The team uses advanced approaches to tackle genetic and pathological heterogeneity and to accelerate the path toward disease-modifying therapies.
Why it matters
Potential benefit: If successful, the work could lead to new blood or brain biomarkers and novel targets for therapies that slow or halt disease progression.
How similar studies have gone: Previous FTLD/ALS research has yielded important mechanistic insights and biomarker advances, with ongoing translation in multiple projects.
Where this research is happening
-
University of Miami School of Medicine
Coral Gables, United States Active
Researchers
- Principal investigator: Petrucelli, Leonard — University of Miami School of Medicine
- Study coordinator: Petrucelli, Leonard
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.