Treating Anemia in Myelofibrosis with Nelfinavir

Principal Investigator: Dr. Richard Watt​

Disease: Myelofibrosis

Research Description:

Patients with myelofibrosis, an incurable blood cancer, often have chronic inflammation, which results in high levels of the hormone hepcidin. Hepcidin lowers iron levels in the body, and decreased iron can negatively impact the body’s production of new red blood cells. This in turn can lead to anemia, which is a chronic condition in myelofibrosis patients that leads to severe fatigue, shortness of breath and the need for frequent blood transfusions. Nelfinavir has been used for 25 years to treat HIV by lowering hepcidin in HIV-positive patients and has a substantial safety profile with very mild side effects. This Phase IIA pilot trial will treat 15 myelofibrosis patients with oral nelfinavir to block the activation of hepcidin under inflamed conditions, thus potentially restoring iron delivery to the bone marrow, stimulating red blood cell production, and alleviating anemia. This trial has the potential to improve the quality of life of myelofibrosis patients and reduce the healthcare costs associated with frequent blood transfusions.

Funding Partners: Goldman Philanthropic Partnerships and others

CWR Funding Role: Primary funder