Myeloproliferative Neoplasms
Research Consortium

Using collaborative laboratory and clinical research investigation to find the cure for Myelofibrosis.

Learn about the approach at MPN-RC

What Is

Myeloproliferative Neoplasms Research Consortium or MPN?

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).

What happens when you develop MPN?

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.

Symptoms usually develop slowly but people
with MF typically

  • Feel tired, weak or short of breath, usually because of anemia
  • Bruise easily
  • Bleed easily
  • Have excessive sweating during sleep (night sweats)
  • Dull pain or fullness below your ribs on the left side, due to enlarged spleen
  • Have fevers
  • Have bone pain

Treatment

We’re working on various candidate drugs with potential to act on these pathways

Pre-clinical studies

Investigators test drugs in the lab setting to determine which ones hold the most potential for humans

Nutlins, RG7388,
HDM201, Kartos 232

TGF B Antagonists

Bromodomain
inhibitors

E2H2 inhibitors

CXCR 1/2
antagonists (IL-8)

Novel JAK2 inhibitors (when available)

IDH 2 inhibitor

EZH2 inhibitors

Design and implement clinical trials, using these drugs, within the MPN-RC’s 11 participating sites

Evaluate and analyze data and specimen collected from to determine effectiveness of treatments

Clinical-Trials CLINICAL TRIAL #4 CLINICAL TRIAL #3 CLINICAL TRIAL #2 CLINICAL TRIAL #1
line

The goal of this bench to bedside approach is to efficiently translate laboratory findings into safe and effective therapies for myelofibrosis patients.

Want to learn more about our studies?

Take a look at our projects with more detail examples of our process.

Our Projects