CAR T therapy that targets U5 snRNP200 on AML cells
Development of chimeric antigen receptor T cells targeting cell surface U5 snRNP for the treatment of acute myeloid leukemia
['FUNDING_R01'] · SLOAN-KETTERING INST CAN RESEARCH · NIH-11311808
This work will create engineered CAR T immune cells that seek out and kill acute myeloid leukemia cells that display a protein called U5 snRNP200, while aiming to spare most normal blood cells.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | SLOAN-KETTERING INST CAN RESEARCH (nih funded) |
| Locations | 1 site (NEW YORK, UNITED STATES) |
| Trial ID | NIH-11311808 on ClinicalTrials.gov |
What this research studies
Researchers discovered that a protein called U5 snRNP200 appears on the surface of some AML cells but is absent from most normal blood stem cells. They plan to build chimeric antigen receptor (CAR) T cells that recognize U5 snRNP complex members and test those cells in laboratory and animal models. The team will also study how U5 snRNP200 ends up on the cell surface and whether another receptor (CD32A) controls that process. Because U5 snRNP200 is also found on normal B cells, the project will consider potential B‑cell side effects while working toward a therapy that preferentially attacks leukemia cells.
Who could benefit from this research
Good fit: Ideal candidates would be people with relapsed or refractory AML whose leukemia cells express surface U5 snRNP200 and who are eligible for cellular immunotherapy.
Not a fit: Patients whose leukemia cells do not display U5 snRNP200, or who cannot receive cellular therapies, are unlikely to benefit from this specific approach.
Why it matters
Potential benefit: If successful, this approach could become a new targeted immunotherapy option for people with AML that kills leukemia cells while limiting damage to most normal blood precursors.
How similar studies have performed: CAR T cell therapies have cured some B‑cell leukemias by targeting surface antigens, but CAR T approaches for AML are earlier-stage and targeting U5 snRNP200 is a new, untested strategy in humans.
Where this research is happening
NEW YORK, UNITED STATES
- SLOAN-KETTERING INST CAN RESEARCH — NEW YORK, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: DANIYAN, ANTHONY FOLAWUYI — SLOAN-KETTERING INST CAN RESEARCH
- Study coordinator: DANIYAN, ANTHONY FOLAWUYI
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.