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Doctors Square Off in Important Battle Over Unapproved Blood Cancer Drug

ASHEVILLE, N.C., March 28, 2014 /PRNewswire/ -- It's hard to imagine heavyweight hematologists publicly squaring off in support of an off-label blood cancer drug.  And yet, today, that's exactly what happened in an "Open Letter to the MPN Community of the United States,"  (  published in the April issue of MPNforum Magazine. (

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The battle is over interferon.  With few good alternative therapies for an estimated 300,000 patients in the United States suffering from myeloproliferative neoplasm (MPN), the outcome of the struggle is a life and death issue.

Dr. Hans Carl Hasselbalch, professor of hematology at the University of Copenhagen, Roskilde Hospital, and Dr. Richard Silver, professor of medicine and director of the Leukemia and Myleoproliferative Center at New York Presbyterian-Weill Cornell Medical Center, publicly deplored the efforts of hematologist Dr. Ayalew Tefferi, Professor of Medicine at the Mayo Clinic (Rochester) to minimize the significance of interferon in the treatment of MPNs, a rare chronic leukemia.

"… I am worried," said Hasselbalch, "by the reported claims by Dr. Ayalew Tefferi that interferon is no better than (hydroxyurea) and busulphan in the MPNs. If Tefferi has said so, it is problematic that a colleague, who may be aware of his great impact on the 'scientific community' and MPN-patients' care, stands up and not tells the truth…I cannot dissect or understand the reasons for his anti-IFN attitude."

"I agree with everything [Hasselbalch] says," wrote Silver. "Recombinant interferon is the only drug used in the myeloproliferative diseases that has a physiological basis for its use… instead of the non-specific killing activity of hydroxyurea and other similar compounds."

The Open Letter was triggered by Tefferi's remarks on interferon in a teleconference with an MPN patient group February 23 ( during which he reported on his upcoming clinical trial of a new MPN drug, Geron's Imetelstat.  (Imetelstat was put on clinical hold by the FDA last week, reportedly due to problems in liver toxicity.) Tefferi, a principal investigator in many MPN drug trials, has publicly stated his lack of support for interferon as well as his opposition to an FDA approved myelofibrosis drug, Jakafi. 

Citing long clinical experience and publication of many peer-reviewed scientific studies, Hasselbalch and Silver make the case for early use of interferon in MPN treatment.

Recognized as a frontline drug for MPNs in Europe, interferon is only available to US MPN patients as an off-label drug. Interferon is FDA-approved for treatment of other diseases, primarily Hepatitis-C.

MPNforum and its companion MPN Quarterly Journal are open source publications entirely managed and staffed by patients and caregivers with the volunteer participation of scientists and healthcare providers. MPNforum is the largest international on-line monthly magazine devoted to myeloproliferative neoplasm patients and caregivers.

Contact: Zhenya Senyak, Editor, MPNforum

Read more news from MPNforum.  


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