Gentler bone marrow transplant approach for Ataxia‑telangiectasia
Non-genotoxic HSCT for ATM
['FUNDING_R03'] · EMORY UNIVERSITY · NIH-11171641
Testing whether a non‑genotoxic (chemo‑free) bone marrow transplant approach can fix immune problems and lower lymphoma risk for people with Ataxia‑telangiectasia.
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-11171641 on ClinicalTrials.gov |
What this research studies
I have Ataxia‑telangiectasia (A‑T), a genetic condition that causes immune problems, inflammation, and a high risk of lymphoma. Researchers are using A‑T mouse models to try a different way to prepare for allogeneic bone marrow transplant that avoids DNA‑damaging chemotherapy. They will use antibody‑linked toxins (like CD117 and CD45 saporin conjugates) to remove recipient blood stem cells and then give donor stem cells, and they will watch for changes in infections, inflammation, and lymphoma development. This is preclinical work done at Emory University aiming to make transplants safer for children with A‑T in the future.
Who could benefit from this research
Good fit: Children and people with Ataxia‑telangiectasia who have significant immune deficiency or are at high risk for lymphoma would be the likely candidates if the approach moves into clinical trials.
Not a fit: People without A‑T or those whose main problems are advanced neurodegeneration rather than immune failure are unlikely to benefit from this transplant approach.
Why it matters
Potential benefit: If successful, this could lead to a safer, less DNA‑damaging transplant option that corrects immune defects and lowers lymphoma risk in people with A‑T.
How similar studies have performed: Traditional allogeneic transplants with reduced‑intensity chemotherapy have shown mixed results, and non‑genotoxic immunotoxin conditioning is a newer strategy that so far is mainly supported by preclinical data.
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.