Using collaborative laboratory and clinical research investigation to find the cure for Myelofibrosis.
Learn about the approach at MPN-RC
What Is
Myeloproliferative neoplasm is a rare chronic blood cancer that affects the bone marrow and the production of blood cells. The bone marrow is the material inside the bone where blood cells are made.
Bone marrow is where blood cells are made. As scar tissue builds up, the bone marrow can’t make enough blood cells. The spleen, which is an organ near the stomach under the left ribs, partially takes over making blood cells. This may make the spleen get bigger, a condition called splenomegaly (splee-nuh-MEG-uh-lee).
Cells in Healthy Bone Marrow
Cells in Bone Marrow with MF
A healthy bone marrow should produce a balanced amount of red and white blood cells and platelets.
In the case when the development of myelofibrosis is present, faulty red blood cells are produced, which means less red blood cells and too many white blood cells.
Cells in Healthy Bone Marrow
A healthy bone marrow should produce a balanced amount of red and white blood cells and platelets.
Cells in Bone Marrow with MF
In the case when the development of myelofibrosis is present, faulty red blood cells are produced, which means less red blood cells and too many white blood cells.
Treatment
Nutlins, RG7388, HDM201, Kartos 232
TGF B Antagonists
Bromodomain inhibitors
CXCR 1/2 antagonists (IL-8)
Novel JAK2 inhibitors (when available)
IDH 2 inhibitor
EZH2 inhibitors
The goal of this bench to bedside approach is to efficiently translate laboratory findings into safe and effective therapies for myelofibrosis patients.
Take a look at our projects with more detail examples of our process.
Our Projects