How Are Genetics Transforming MPN Treatment?

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Dr. Srdan Verstovsek, an MPN expert from MD Anderson Cancer Center, explains how genetics is changing the landscape of MPN treatment.  Identifying the genes and the mutations of the disease is guiding the development of targeted therapies and medications to treat MPNs and the symptoms of MPNs.


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Even the JAK inhibitors that are specific for the JAK/STAT pathway, this is the inside?the?cell connection of different proteins that tell the cells what to do.  We call it JAK and STAT.  These are two proteins of many that are connected, and this is super active in patients with myeloproliferative neoplasms because of different mutations that make some of these proteins act abnormal. 

With the JAK inhibitors, we are targeting this pathway.  We are not eliminating disease.  We are calming down the growth.  We are calming down the inflammation.  We are only here at the beginning of our ability to develop targeted therapies.  We are inhibiting this super active JACK/STAT pathway that drives the disease process in terms of growth and inflammation.  With the control of that pathway, we are able to benefit patients greatly, including quality of life.  The weight goes up.  The ability to walk, the spleen goes down.  People enjoy life even for longer than one would think. 

However, we need the medications that would improve the blood cell count.  In a majority of patients, anemia is a major problem.  Medications that would eliminate the fibrosis and ultimately medication that would eliminate the disease itself—a way to go, but we are on the right path.  We are very optimistic about it.  The clinical research is just gearing up with new views, new combinations. Imagine, seven years ago we didn't even have anything to offer the patients, not even the studies.  With the discovery of the JAK2 mutation, we got in the mood to pay attention.  It's a new discovery.  Biology has been discovered. We have something to develop based on that, and now from JAK2 mutations we're aware of more than 30 mutations have been discovered so far.  It's very complicated, but we are getting there.  

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Page last updated on June 5, 2014