Improving treatment for Ataxia Telangiectasia using non-genotoxic methods
Non-genotoxic HSCT for ATM
['FUNDING_R03'] · EMORY UNIVERSITY · NIH-10951008
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
| Phase | ['FUNDING_R03'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | EMORY UNIVERSITY (nih funded) |
| Locations | 1 site (ATLANTA, UNITED STATES) |
| Trial ID | NIH-10951008 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are children under 11 years old diagnosed with Ataxia Telangiectasia.
Not a fit: Patients with other genetic disorders or those over the age of 11 may not benefit from this research.
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 performed: 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
ATLANTA, UNITED STATES
- EMORY UNIVERSITY — ATLANTA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: CHANDRAKASAN, SHANMUGANATHAN — EMORY UNIVERSITY
- Study coordinator: CHANDRAKASAN, SHANMUGANATHAN
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 below.