Improving treatment for Ataxia Telangiectasia using non-genotoxic methods
Official title: Non-genotoxic HSCT for ATM
Funded at Emory University · NIH-10951008 · Data updated 2026-11-12
This study is exploring a new, safer way to treat children with Ataxia Telangiectasia (A-T) by using a special type of stem cell transplant that aims to boost their immune system without the harsh side effects of traditional treatments.
Quick facts
| Grant type | R03 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Emory University NIH-funded |
| Lab location | 1 site (Atlanta, United States) |
| Project start | September 1, 2024 |
| Project end | August 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $117,375 |
| NIH project number | 1R03HL174883-01 |
| Project ID | NIH-10951008 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children under 11 years old diagnosed with Ataxia Telangiectasia.
✕Less likely to help
Patients with other genetic disorders or those over the age of 11 may not benefit from this research.
What this research studies
This research investigates a new approach to treat Ataxia Telangiectasia (A-T), a genetic disorder that affects children and leads to severe immune deficiencies and neurodegeneration. The study focuses on using allogeneic hematopoietic stem cell transplantation (HSCT) with non-genotoxic conditioning, which aims to reduce the risk of lymphoma and improve immune function without the harmful effects of traditional chemotherapy. By utilizing immunotoxins in a murine model, the researchers hope to correct immune deficiencies and lessen inflammation associated with A-T. This innovative method could potentially lead to safer and more effective treatments for young patients suffering from this condition.
Why it matters
Potential benefit: If successful, this research could provide a safer treatment option that improves immune function and reduces cancer risk for children with Ataxia Telangiectasia.
How similar studies have gone: While traditional HSCT approaches have been explored, this non-genotoxic method is novel and has not been extensively tested in A-T patients.
Where this research is happening
-
Emory University
Atlanta, United States Active
Researchers
- Principal investigator: Chandrakasan, Shanmuganathan — Emory University
- Study coordinator: Chandrakasan, Shanmuganathan
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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.