New Myelofibrosis Drug Holds Promise

In a phase I clinical trial, physicians at The Mount Sinai Medical Center have identified the first drug that appears to stop the progression of myelofibrosis, a life-threatening blood cancer. The investigators found that, at low-doses, panobinostat (LBH589) successfully halted and reversed damage to the blood and bone marrow in several of the forty patients enrolled in the trial. Panobinostat, manufactured by Novartis, is a histone deacetylase inhibitor that affects the chromatin structure of malignant cells.

The study, led by Ronald Hoffman, MD, Albert A. and Vera G. List Professor of Medicine, and Director of the Myeloproliferative Disorders Research Program, and John O. Mascarenhas, MD, Assistant Professor of Medicine (Hematology and Medical Oncology), was published online in the January 21, 2013, issue of the British Journal of Haematology.

Myelofibrosis is commonly diagnosed in people between the ages of 60 and 70, and affects approximately one-to-two out of every 100,000 people in the United States annually. The disorder is characterized by anemia, fatigue, bone or joint pain, and an enlarged liver and spleen. Survival usually is between five and seven years, but some patients develop acute leukemia, which can limit survival to three-to-five months.

According to Drs. Hoffman and Mascarenhas, who designed and ran the trial, persistence and ingenuity paved the way for their findings. Signs of significant improvement appeared a year after patients were started on an oral regimen of low-dose panobinostat. “We have patients who are doing well after two-to-three years, and whose survival was predicted to be on the order of months,” says Dr. Mascarenhas.

The key to success was maintaining a low dosage. Interestingly, too high a dosage of panobinostat would have produced toxicity forcing patients to drop out of the trial before positive results could be seen, says Dr. Mascarenhas.

“Persistence led us to finally see signals of activity,” Dr. Mascarenhas recalls. “One of our patients came in for a checkup and her blood smear looked normal. I gave it to Dr. Hoffman to look at and asked him what he thought was wrong with the patient. He said, ‘Nothing. It’s normal.’ Then I told him it was one of our patients in the trial.”

To date, the only potentially curative treatment approved for people with myelofibrosis is a bone marrow transplant. But elderly patients or those with comorbidities are not eligible for transplants, and a suitable bone marrow donor is not always available.

Drs. Hoffman and Mascarenhas have now initiated a new phase I clinical trial that will combine lower doses of panobinostat with ruxolitinib, a drug manufactured by Incyte Pharmaceuticals that has been approved by the U.S. Food and Drug Administration to treat the symptoms of myelofibrosis.

This article was first published in Inside Mount Sinai.

12 thoughts on “New Myelofibrosis Drug Holds Promise

  1. Permalink  ⋅ Reply


    March 28, 2013 at 11:29pm

    Very promising! Thanks to these very dedicated and talented doctors!

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    Donna Hennessy

    March 30, 2013 at 4:54pm


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    April 1, 2013 at 9:11pm

    This is great news and the MPD program deserves a lot of credit!

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    April 4, 2013 at 1:16am

    I would like to know how to enroll my mother into this clinical trial.

    • Permalink  ⋅ Reply


      April 4, 2013 at 6:54pm

      Thank you for reaching out Sherri. For more information about the MPD clinical trial, please email

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    Mark Breaux

    April 4, 2013 at 3:00am

    Thank God for these doctors!

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      April 5, 2013 at 1:45am

      Thank you! I will send the email today! Thank you for responding and keep up the hope!

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    April 17, 2013 at 1:40am

    Amazing. I was just diagnosed today and came across this tidbit of information.
    Thank you, doctors, and the entire medical community.

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    July 1, 2013 at 4:37am

    This is great news! Clinical trials are so important when it comes to advancing medicine. I have noticed that Mt. Sinai seems to be at the forefront of cancer research. It would be great to see you tackle pain syndromes like reflex sympathetic dystrophy (RSD – what is rsd has great info).


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    Albert McGinn

    September 10, 2013 at 8:43pm

    Gives me some hope.
    Go for it.

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    Ann Hirsch

    October 24, 2013 at 7:08pm

    So glad your persistence is paying off. Too late for my husband but very good news for those struggling with this devastating disease.

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