Targeting FICD to Help Spinocerebellar Ataxia Type 3 (SCA3)
Official title: Targeting the ER-resident AMPylase FICD to Ameliorate Spinocerebellar Ataxia Type 3
Funded at University of Michigan at Ann Arbor · NIH-11437540 · Data updated 2026-11-12
A brain-focused project tests whether blocking FICD, a protein in cells’ ER quality system, can slow or prevent damage in people (via animal and cell studies) who have spinocerebellar ataxia type 3.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Michigan at Ann Arbor NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project start | September 11, 2026 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $418,440 |
| NIH project number | 1R21NS151155-01 |
| Project ID | NIH-11437540 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be individuals with spinocerebellar ataxia type 3 who could participate in future trials or donate samples; current work is preclinical (animal models).
✕Less likely to help
Patients without SCA3 or those seeking non-neurological therapies would not directly benefit from this preclinical project.
What this research studies
This grant explores how inhibiting the ER-resident enzyme FICD might protect brain cells and support myelin-forming cells in a mouse model of SCA3. Researchers will use histology and biochemistry to understand how FICD inhibition preserves neurons and oligodendrocytes. They will also test a brain-penetrant FICD inhibitor to see if it can stop or slow motor decline in the model. The work aims to translate these findings toward a disease-modifying therapy for SCA3.
Why it matters
Potential benefit: If successful, the approach could slow neuron loss and motor problems in SCA3 by improving ER proteostasis and myelination.
How similar studies have gone: Prior studies suggest that targeting ER proteostasis and FICD may modulate polyQ protein toxicity, supporting a potential path to therapy.
Where this research is happening
-
University of Michigan at Ann Arbor
Ann Arbor, United States Active
Researchers
- Principal investigator: Truttmann, Matthias Christof — University of Michigan at Ann Arbor
- Study coordinator: Truttmann, Matthias Christof
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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.