New treatment for a serious brain disease affecting children
Official title: Development of a novel, targeted small molecule inhibitor of the nucleoside salvage pathway through IND enabling studies and translational mouse models for acute disseminated encephalomyelitis (ADEM)
Funded at Trethera Corporation · NIH-10999341 · Data updated 2026-11-12
This study is testing a new drug called TRE-515 to see if it can help children with a serious condition called acute disseminated encephalomyelitis (ADEM) by reducing symptoms and helping them recover better than current treatments, which can have tough side effects.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Trethera Corporation NIH-funded |
| Lab location | 1 site (Sherman Oaks, United States) |
| Project start | July 2, 2024 |
| Project end | June 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $1,050,000 |
| NIH project number | 1R44AI186534-01 |
| Project ID | NIH-10999341 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children and adolescents diagnosed with acute disseminated encephalomyelitis.
✕Less likely to help
Patients with other neurological conditions or those who do not have ADEM may not benefit from this research.
What this research studies
This research focuses on developing a new small molecule drug, TRE-515, aimed at treating acute disseminated encephalomyelitis (ADEM), a severe autoimmune disease primarily affecting children. The approach involves inhibiting a specific enzyme, deoxycytidine kinase, which plays a crucial role in the proliferation of immune cells that attack the nervous system. By using mouse models, the research aims to demonstrate that TRE-515 can effectively reduce symptoms and improve recovery outcomes for patients with ADEM. The study seeks to provide a safer and more effective alternative to current corticosteroid treatments, which often have significant side effects.
Why it matters
Potential benefit: If successful, this research could lead to a new, safer treatment option for children suffering from ADEM, potentially improving recovery rates and reducing long-term complications.
How similar studies have gone: While ADEM is a rare condition, similar approaches targeting immune cell proliferation have shown promise in other autoimmune diseases, suggesting potential for success in this novel treatment.
Where this research is happening
-
Trethera Corporation
Sherman Oaks, United States Active
Researchers
- Principal investigator: Schultz, Kenneth — Trethera Corporation
- Study coordinator: Schultz, Kenneth
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.