Targeted bone marrow radiation to improve transplants for sickle cell disease
A Novel Bone Marrow Transplantation Approach for Sickle Cell Disease Using Targeted Marrow Irradiation
This work uses focused bone marrow radiation to try to make donor transplants for people with sickle cell disease safer and more successful.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Beckman Research Institute/city of Hope NIH-funded |
| Lab location | 1 site (Duarte, United States) |
| Project ID | NIH-11117141 on NIH RePORTER |
What this research studies
The team is developing an image-guided approach that delivers radiation mainly to bone marrow while sparing vital organs to prepare for donor bone marrow transplants. They are testing this in laboratory models, including normal mice and humanized sickle cell (BERK) mice, to study dose effects and how focused marrow radiation improves donor cell engraftment. The group reports an initial success in a single patient and now aims to understand the biological reasons for better donor chimerism after targeted marrow irradiation. Findings will guide whether this approach can move toward clinical trials with the goal of lowering transplant-related organ damage.
Who could benefit from this research
Good fit: People with sickle cell disease who are considering or eligible for allogeneic bone marrow transplant would be the kinds of patients targeted for future trials of this approach.
Not a fit: People without sickle cell disease or SCD patients who are not transplant candidates are unlikely to benefit from this work in the near term.
Why it matters
Potential benefit: If successful, this approach could lower organ toxicities and increase the chance of a full, curative donor transplant for people with sickle cell disease.
How similar studies have performed: There is a reported single-patient success using image-guided total marrow irradiation and other marrow-irradiation work exists, but targeted marrow irradiation for SCD is an emerging approach being tested now in preclinical models.
Where this research is happening
Duarte, United States
- Beckman Research Institute/city of Hope — Duarte, United States (Active)
Researchers
- Principal investigator: Hui, Susanta K — Beckman Research Institute/city of Hope
- Study coordinator: Hui, Susanta K
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.