JAK2 Inhibitors: A Breakthrough Treatment for Myelofibrosis

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Topics include: Treatments

The class of JAK2 medications is significantly improving the lives of people myeloproliferative conditions, especially myelofibrosis. While they are not a cure for myelofibrosis, in the opinion of a leading researcher, Dr. Srdan Verstovsek from MD Anderson Cancer Center, there are significant benefits. Watch now as Dr. Verstoveek, explains how JAK2 inhibitors help and why they're so important for the future of myelofibrosis treatment.

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Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Andrew Schorr:

Hello.  I'm Andrew Schorr for Patient Power. 

When myelofibrosis becomes advanced, the symptoms can be very debilitating.  How has the use of the first approved JAK2 inhibitor, ruxolitinib (Jakafi and Jakavi), made a difference in, not only improving life and alleviating symptoms, but also extending survival?  To help us understand that is a lead investigator for that drug.  That's Dr. Srdan Verstovsek from MD Anderson Cancer Center in Houston.  He joins us now from a medical conference in Madrid. 

Dr. Verstovsek, thank you for being with us.  Tell us now, what evidence is there that this drug is actually extending survival? 

Dr. Verstovsek:

The evidence that is accumulating in the use of ruxolitinib (Jakafi and Jakavi), as a therapy for patients with advanced myelofibrosis, is very positive, and I think that we can have some confidence that by controlling the signs and symptoms of myelofibrosis, in patients with advanced disease, we are extending the life of these patients.  This is related to our knowledge about what actually leads to an untimely death in patients with myelofibrosis.  It is the disease itself that causes people to die.  Although, the most common single cause for death is transformation to acute myeloid leukemia, we are talking only about 20 percent of patients transforming.  That means about 80 percent of the patients die from myelofibrosis itself or from complications related to enlargement in the spleen, enlargement in the liver, body wasting or cachexia, multiple symptoms.  And as you know ruxolitinib (Jakafi and Jakavi) is very effective in a majority of the patients in reducing enlarged spleen and reducing the liver, improving body conditions.  People gain weight, walk more.  So controlling the signs and symptoms leads to a prolongation of life. 

Andrew Schorr:

Dr. Verstovsek, beyond extending survival, and that's certainly important, do we have any evidence that the drug is having an effect on the underlying disease, in other words, stopping or reversing the scarring in the bone marrow? 

Dr. Verstovsek:

This is the area of intense research, particularly in relationship to controlling of signs and symptoms with the JAK1 or JAK2 inhibitors, because one would assume that what we see by eye, we see enlargement of the spleen going way, we see people gaining weight and walking more, we see that these clinically relevant features are improving, we would then think the biology of the disease is affected in some way.  For now, we know that the cytokines, the proteins in blood that lead to inflammation, are significantly suppressed in patients with myelofibrosis on the JAK inhibitors, and that is probably a reason why people feel better and walk more, and we expect that the long-term benefit of JAK2 and JAK2 inhibitors, in patients with myelofibrosis, may, in fact, lead to some improvements or at least a cessation in worsening of myelofibrosis in the bone marrow. 

Andrew Schorr:

Dr. Srdan Verstovsek from MD Anderson Cancer Center, thanks so much for being with us. 

Dr. Verstovsek:

It was a pleasure.  Thank you for the opportunity. 

Andrew Schorr:

We'll have much more with Dr. Verstovsek, including a discussion on the use of ruxolitinib (Jakafi and Jakavi), the first approved JAK2 inhibitor, if a patient is anemic or becomes anemic or has low platelets.  It's all coming your way in our MPN Center.  Be sure to be signed up for alerts so we can let you know as new programs are posted. 

I'm Andrew Schorr.  Remember, knowledge can be the best medicine of all. 

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

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Page last updated on January 26, 2016