Dr. Naveen Pemmaraju Discusses Myelofibrosis, MPNs, and Therapeutic Advancements


This year, thanks to COVID-19, many conferences have been held online. These play a crucial role in spreading data, facilitating conversations, and determining potential treatment options for patients with rare conditions. One such platform was the Texas MPN Workshop to discuss therapeutic advancements in the world of myelofibrosis and myeloproliferative neoplasms (MPN). In an interview with Targeted Oncology, Dr. Naveen Pemmaraju discussed hematology, treatments for patients with MPNs, and how doctors can navigate the current online landscape. Want to join in the discussion on MPN research? Check out the #MPNsm tag on social media.

JAK Inhibitors

One therapeutic option that Dr. Pemmaraju discussed during the interview was JAK inhibitors. This class of drugs, which includes ruxolitinib and fedratinib, calms overactive JAK proteins. Dr. Pemmaraju notes that those particular therapies are effective for patients even without JAK2 gene mutations. Currently, there are other JAK inhibitors in development, such as momelotinib and pacritinib. The former is being tested in the randomized Phase 3 MOMENTUM clinical trial for the treatment of myelofibrosis and anemia. The latter is being tested in a Phase 3 PACIFICA clinical trial for patients with myelofibrosis and thrombocytopenia. However, researchers need to better understand efficacy and toxicity, particularly in novel treatments.

Next, Dr. Pemmaraju notes that he is excited about promising future treatments for patients with myelofibrosis and MPNs. The first is combining a JAK inhibitor with a secondary drug agent. These can include azacitidine and decitabine. Then, another potential treatment option is the creation of new investigational therapies such as BET or Bcl-xL inhibitors.

Finally, he acknowledges that novel drugs likes SMAC mimetics or IEP antagonists offer avenues to treat patients with treatment averse myelofibrosis. As we move into the future, considering these unmet patient needs will optimize treatment offerings.


According to the Cleveland Clinic, myelofibrosis is:

a rare type of blood cancer in which the bone marrow is replaced by fibrous scar tissue. It is considered a form of chronic leukemia.

Generally, myelofibrosis occurs after age 50. Researchers are not sure exactly what causes myelofibrosis. However, it has been linked to thrombocytopenia, chemical exposure, and JAK2, MPL, and CALR gene mutations. These cause proteins called janus-associated kinases (JAKs) to become overactive. As a result, the JAKs stop regulating blood cell production, either producing excess or too few cells.

Instead of forming into healthy red and white blood cells or platelets, patients with myelofibrosis develop abnormal cells which crowd out healthy cells. For example, they may develop too many megakaryocytes, causing inflammation and fibrous scar tissue. In around 12% of all cases, myelofibrosis eventually progresses into acute myeloid leukemia (AML).

Symptoms include:

  • Anemia
  • Fatigue
  • Muscle pain and weakness
  • Shortness of breath
  • Unintended weight loss
  • Pale skin
  • Bone and joint pain
  • Blood clots
  • Enlarged spleen and liver
  • High blood pressure
  • Fever
  • Itching
  • Abnormal bleeding and bruising
  • Recurrent infections

Learn more about myelofibrosis here.

Myeloproliferative Neoplasms (MPNs)

The Cancer Support Community describes MPNs as:

blood cancers that occur when the body makes too many white or red blood cells, or platelets. This overproduction of blood cells in the bone marrow can create problems for blood flow and lead to various symptoms.

Three types of MPNs are essential thrombocytopenia, myelofibrosis, and polycythemia vera (PV). All three have been linked to JAK2 gene mutations. Learn more about MPNs here.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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