New treatment for acute myeloid leukemia using ONC201
Ph1/2 Study of the Imipridone ONC201 for Treatment of AML IND125,203 (12/23/2014)
This study is testing a new treatment called ONC201 for people with acute myeloid leukemia (AML), especially those with a specific gene mutation, to see if it can safely help kill leukemia cells while protecting healthy ones.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Tx Md Anderson Can Ctr NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project ID | NIH-10663157 on NIH RePORTER |
What this research studies
This research investigates the use of ONC201, a novel imipridone compound, to treat acute myeloid leukemia (AML) by targeting a specific cellular survival mechanism known as the mitochondrial unfolded protein response (mtUPR). The study aims to conduct a Phase 1/2 clinical trial to evaluate the safety and effectiveness of ONC201, particularly in patients with p53-mutated AML, which is known to be resistant to conventional chemotherapy. By inducing apoptosis selectively in leukemia cells while sparing normal cells, this treatment could potentially improve cure rates for AML patients. The research builds on promising preclinical findings and early clinical responses observed in previous trials.
Who could benefit from this research
Good fit: Ideal candidates for this research are patients diagnosed with acute myeloid leukemia, particularly those with p53 mutations.
Not a fit: Patients with other types of leukemia or those who do not have acute myeloid leukemia may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could significantly improve survival rates and treatment outcomes for patients with acute myeloid leukemia.
How similar studies have performed: Previous studies have shown promising results with ONC201 in treating AML, indicating potential for success in this clinical trial.
Where this research is happening
Houston, United States
- University of Tx Md Anderson Can Ctr — Houston, United States (Active)
Researchers
- Principal investigator: Andreeff, Michael — University of Tx Md Anderson Can Ctr
- Study coordinator: Andreeff, Michael
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.