Developing small molecules to target tau pathology in Alzheimer's disease
Official title: Targeting MSUT2 with small molecules to ameliorate pathological tau
Funded at Seattle Inst for Biomedical/clinical Res · NIH-10735826 · Data updated 2026-11-12
This study is looking for new medicines that can help fight Alzheimer's disease by targeting a specific protein called MSUT2, which is linked to the harmful buildup of tau in the brain, and it’s for anyone interested in finding better treatments for this condition.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Seattle Inst for Biomedical/clinical Res NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project start | September 1, 2023 |
| Project end | August 31, 2026 |
| Funding year | 2023 |
| Award (this year) | $2,181,250 |
| NIH project number | 1RF1AG078335-01A1 |
| Project ID | NIH-10735826 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Alzheimer's disease or related tauopathies who are experiencing cognitive decline.
✕Less likely to help
Patients with non-tau related forms of dementia or those without a diagnosis of Alzheimer's disease may not benefit from this research.
What this research studies
This research focuses on developing small molecules that target MSUT2, a protein involved in tau pathology, which is a key factor in Alzheimer's disease and related dementias. The approach includes high-throughput screening of over 100,000 compounds to identify those that can inhibit MSUT2's interaction with poly(A) RNA, potentially slowing or reversing tau-related neurodegeneration. By understanding how MSUT2 modulates tau toxicity, the research aims to create effective therapeutics that directly address the underlying causes of Alzheimer's disease rather than just alleviating symptoms.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that modify the progression of Alzheimer's disease by targeting tau pathology.
How similar studies have gone: While there has been limited success in targeting tau pathology directly, this approach is innovative and aims to fill a significant gap in current Alzheimer's treatment strategies.
Where this research is happening
-
Seattle Inst for Biomedical/clinical Res
Seattle, United States Active
Researchers
- Principal investigator: Kraemer, Brian C. — Seattle Inst for Biomedical/clinical Res
- Study coordinator: Kraemer, Brian C.
Related research
Other NIH-funded projects on the same topics.
-
Military Service–Linked ALS Risk in U.S. Veterans
This NIH grant investigates why U.S. veterans get ALS more often, by studying military exposures and health records to identify potential risk factors.
-
Targeting MSUT2 with small molecules to slow tau-related dementia
Aims to test whether small molecules that block MSUT2’s interaction with RNA can slow or reverse tau-related brain damage in people affected by Alzheimer’s disease and related dementias.
-
SPOP's role in tau-related brain disease
Researchers are studying how a protein called SPOP affects tau-related brain damage in Alzheimer's-type conditions, using a small worm model to mirror human disease and look for ways to protect brain cells.
-
Comparing medicines and weight-loss surgery for liver disease linked to metabolic problems
This program looks at how two drugs and bariatric surgery work for adults with metabolic dysfunction–associated steatohepatitis (MASH) and liver fibrosis.
-
Dementia with Lewy Bodies Consortium
A national research effort to better understand and diagnose Lewy body dementia by studying biomarkers and patient data over time.
-
Post-TBI Sleep Problems and Alzheimer’s/CTE Brain Changes
This grant studies whether sleep disruption after a traumatic brain injury increases the risk of Alzheimer’s disease and related brain changes later on, with a focus on people who’ve had mTBI.
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.