The MPNRF Interferon (IFN) Initiative is a multi-center project that has brought together internationally recognized experts in both blood and solid tumors to determine how cytokine-driven pathways affect the trajectory of the MPNs, a closely-related group of progressive blood cancers. The collaboration among this group of scientists speaks to their drive to answer these questions:

  • How and why does targeting the IFN pathway reduce the burden of mutated stem cells in MPN patients?
  • Why is targeting this pathway effective for some MPN patients but not others?
  • Why are the positive effects of targeting this pathway not permanent?

It is MPNRF’s hope and determination that this multi-year project will bring discoveries to light for MPN patients, and will provide an essential weapon in the arsenal of treatments available to them and to patients with other cancers. There is now a formulation of interferon being developed specifically for use in the treatment of MPNs called Ropeginterferon, but we don’t know if or when it will be approved by the FDA.

This initiative is funded by MPN patients and supporters, a collaborative funding partnership with Cancer Research & Treatment Fund and MPN Alliance Australia, and by generous contributions from PharmaEssentia.


Interferon (IFN) is a drug that has proven effective in viral infections, such as hepatitis C, but has largely been replaced by new generation drugs for those diseases. However, it remains the only treatment that provides disease-altering effects for MPN patients, and understanding its mechanism of action for MPNs can lead to better interferon compounds and potentially other drugs in the IFN pathway that can change the prognosis for MPN and other cancer patients.

From the NCCN Guidelines for MPN Patients:  “Interferons naturally exist in your body as part of your disease-fighting (immune) system. They can also be made in the lab and be used to treat MPN. When used as a treatment, interferon is given in much higher amounts than what the body makes. How interferon works to treat MPN isn’t fully known. It is a type of immunotherapy. Thus, it enhances the activity of immune cells. A high level of interferon also suppresses the making of blood cells…… Interferons for MPN are interferon alfa-2b, peginterferon alfa-2a, and peginterferon alfa-2b. They are received as an injection just under the skin. Most people inject themselves (p.37).”


“We have long felt that a study of Interferon would be a unique opportunity to open new avenues of treatment for MPN patients. We held an Interferon Brainstorming Session in 2016 at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University, and became convinced we should spearhead this effort.” – Barbara Van Husen, Board Chair of the MPNRF

The results of this research initiative have the potential to transform the landscape of MPN research. Interferon is the only treatment that is shown to reduce both symptom burden and the occurrence of the JAK2 mutation, and better understanding how the drug works will inevitably lead to a better understanding of the disease itself. This collaborative work could change our understanding of the biology of MPNs and explain why people develop these blood cancers. This research could also lead to new and improved treatment options that could potentially be curative, and finding out why interferon works at all could help lead to FDA approval of the drug for MPN patients, which would improve accessibility to the drug (interferon is currently prescribed off-label).

Click HERE to learn more from MPNRF’s Executive Director, Michelle Woehrle, as she discusses why it is important Interferon is researched further.


The Interferon Initiative has brought together experts from around the world in Interferon, MPNs, and solid tumors to bring both broad and deep understanding of this issue. The team of investigators includes:

  • Leonidas Platanias, MD, PhD: Lurie Cancer Center
  • Ann Mullally, MD: Brigham and Woman’s Hospital
  • Jean-Luc Villeval, PhD: INSERM/Institut Gustave Roussy, Paris
  • Joseph Scandura, MD, PhD: Weill Cornell School of Medicine
  • Steven Lane, PhD: QIMR Berghofer Medical Institute
  • Michael Milson, PhD: German Cancer Research Center

The project is guided by an Advisory Group of recognized experts in Interferon and MPNs that routinely meet with investigators to ensure that the collaboration is optimal and productive. Members of the Advisory Group include:  Andrew Schafer, MD (chair); John Crispino, PhD; Josef Prchal, MD; Robert Cohen, MD; Ronald Hoffman, MD; Richard Silver, MD; Jean-Jacques Kiladjian, MD, PhD; William Vainchenker, MD, PhD; Radek Skoda, MD and Hans Hasselbalch, MD.

 “Working together and sharing knowledge is key,” says Leonidas Platanias, MD, PhD, Director of the Lurie Cancer Center, Jesse, Sara, Andrew, Abigail, Benjamin and Elizabeth Lurie Professor of Oncology and lead investigator of the multicenter effort. “As our understanding of complex cytokine pathways and signaling networks continues to evolve, we expect to maximize the impact of these discoveries by developing new therapies for patients with MPN.”

The MPN Research Foundation provides funding to this team of investigators to pay for their ongoing research into interferon. The Foundation convenes meetings among the investigators annually so they have an opportunity to collaborate, sharing their ideas and progress with one another. The next meeting is scheduled for late fall 2019.

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