Mikolaj Slabicki, PhD
Principal Investigator
Massachusetts General Hospital Cancer Center
Research project
Leveraging the ubiquitin-proteasome system to treat myelodysplastic syndromes and acute myeloid leukemia
Summary
Myelodysplastic syndromes (MDS) are a group of blood cancers that prevent the bone marrow from producing healthy blood cells, and can evolve into a more aggressive disease called acute myeloid leukemia (AML). Current treatment options for MDS and AML are limited and often ineffective, especially when the disease progresses. Our research explores a new class of drugs known as “molecular glues.” These are small molecules that work like a magnet to bring harmful proteins into contact with the cell’s waste disposal machinery—the ubiquitin-proteasome system—so they can be broken down and removed. We are developing new molecular glues that can specifically target a group of proteins called zinc finger (ZF) transcription factors, which help control how genes are turned on and off. Some of these ZF proteins are known to drive the development and progression of MDS and AML. By discovering glues that remove these proteins, we aim to stop or slow down disease progression. Our work could also reveal new proteins that are especially important in these cancers, offering fresh targets for future treatments. Ultimately, this project may lead to more effective, less toxic therapies for patients facing these life-threatening blood cancers.
Leukemia Research Foundation grant
$150K awarded in 2025
Disease focus
Myelodysplastic syndromes (MDS) and Acute myeloid leukemia (AML)
Research focus
Treatment