Developing new treatments to increase fetal hemoglobin for patients with blood disorders
Chemical tools for modulating the fetal hemoglobin inducer BCL11A
['FUNDING_R01'] · STANFORD UNIVERSITY · NIH-11127536
This study is working on new ways to help people with sickle cell disease and beta-thalassemia by finding methods to lower the activity of a protein called BCL11A, which could help increase the production of healthy fetal hemoglobin in their bodies.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | STANFORD UNIVERSITY (nih funded) |
| Locations | 1 site (STANFORD, UNITED STATES) |
| Trial ID | NIH-11127536 on ClinicalTrials.gov |
What this research studies
This research focuses on creating innovative chemical tools that can modulate the BCL11A protein, which plays a crucial role in regulating fetal hemoglobin levels. By using advanced techniques like proteolysis targeting chimeras (PROTACs) and molecular glues, the project aims to develop therapies that can effectively target and reduce the function of BCL11A. This could lead to increased production of fetal hemoglobin, providing a potential treatment option for patients suffering from sickle cell disease and beta-thalassemia. The research is particularly significant as it addresses the challenges of targeting proteins that are difficult to drug, thereby opening new avenues for treatment.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with sickle cell disease or beta-thalassemia, particularly those who are 21 years or older.
Not a fit: Patients with other types of anemia or blood disorders unrelated to fetal hemoglobin regulation may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that significantly improve the quality of life for patients with sickle cell disease and beta-thalassemia.
How similar studies have performed: Previous research has shown promise in using similar approaches to target difficult proteins, indicating a potential for success in this novel application.
Where this research is happening
STANFORD, UNITED STATES
- STANFORD UNIVERSITY — STANFORD, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: DASSAMA, LAURA — STANFORD UNIVERSITY
- Study coordinator: DASSAMA, LAURA
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.