Clearing excess alpha-globin to help people with beta-thalassemia
Official title: ULK-mediated autophagy of α-globin in ß-thalassemia
Funded at St. Jude Children's Research Hospital · NIH-11125822 · Data updated 2026-11-12
This research looks at whether boosting a cell 'self-cleaning' pathway (ULK1-driven autophagy) can remove toxic excess alpha-globin and help people with beta-thalassemia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project start | July 1, 2022 |
| Project end | June 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $652,585 |
| NIH project number | 5R01HL165798-04 |
| Project ID | NIH-11125822 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with beta-thalassemia caused by HBB mutations—including those who are transfusion-dependent or non-transfusion-dependent—would be the most relevant candidates for related future trials or sample donation.
✕Less likely to help
Patients whose anemia is due to other causes (for example sickle cell disease or non-hemoglobinopathy disorders) or those with irreversible organ damage from long-standing iron overload may not benefit from this approach.
What this research studies
Researchers are exploring how cells normally clear excess alpha-globin and whether enhancing that cleanup can reduce the damage that causes anemia in beta-thalassemia. In the lab they use mouse models that mimic human beta-thalassemia and cellular experiments to test the role of the ULK1 autophagy pathway and its regulation by mTORC1 and AMPK. They have tested drugs (for example rapamycin) that turn on this clearance pathway to see if it lowers immature red blood cell death, hemolysis, and anemia in preclinical models. If these approaches work in the lab, the team hopes to move toward treatments that could reduce transfusion needs or improve blood production in patients.
Why it matters
Potential benefit: If successful, this approach could reduce toxic alpha-globin, improve red blood cell production, and lower anemia and transfusion needs for people with beta-thalassemia.
How similar studies have gone: Preclinical work in validated mouse models has shown that inhibiting mTORC1 (for example with rapamycin) can stimulate ULK1-dependent autophagy of alpha-globin and reduce ineffective erythropoiesis, but human clinical use of this exact strategy has not been established.
Where this research is happening
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St. Jude Children's Research Hospital
Memphis, United States Active
Researchers
- Principal investigator: Weiss, Mitchell J — St. Jude Children's Research Hospital
- Study coordinator: Weiss, Mitchell J
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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.