Understanding the Evolving MPN Treatment Landscape | Transcript | Myeloproliferative Neoplasms | Patient Power


Understanding the Evolving MPN Treatment Landscape

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Similarly, my colleague Animesh Pardanani is presenting on the long?term experience with momelotinib, a drug now in Phase III studies that shows also some improvement in anemia as well as other aspects of the disease, and that's very positive. 

Likewise, we're seeing long?term benefits with pacritinib.  That's being presented by my colleague Dr. Komrokji, which I'm involved with that study, benefits with the other JAK inhibitor, LY 2784544. 

Also seeing that long?term we're seeing with ruxolitinib with the pooled analysis of both the COMFORT?1 and COMFORT?2 studies, it's pretty clear that ruxolitinib is making a difference in terms of survival, and we can look at that experience in a variety of ways. 

Also I'd say something newsworthy, probably not a positive note but an important note, were developments with fedratinib, or SAR302503, very unfortunately we've seen that there are some long?term toxicities with that drug that ended up having the FDA put that drug and all who were on clinical trials for that drug on a hold.  That had the potential to cause some neurologic damage that was significant, significant enough that the companies made the decision not to develop the drug any further.  I think they've handled it very professionally and really tried to be as patient?centered with that as possible as well as trying to learn from that experience. 

I can say on a reassuring note, one, we've not really seen that as a negative risk with the other drugs and, two, of those patients that have been on that drug, still the likelihood of someone having developed that is really quite unlikely.  The number of patients through is really quite small, so the likelihood of anyone who  

I'd say the next group of things that are of great interest for patients is we're learning more about biology, so there was particularly important developments in our molecular understanding.  There is an additional new mutation that has been found to be present in many of the individuals that lack the JAK2 V617F mutation, a mutation in a new gene called calreticulum.  I'd say it's realistically a little early for us to know what that means but probably is another important clue.

In terms of other therapies, I'd say the other sets of news really fall into two—into two additional buckets.  One, combination studies, we're hearing more about those and some early benefits from combinations of ruxolitinib with other drugs like panobinostat or other drugs in development.  We're hearing some exciting news, but early news, to be very transparent, regarding new drugs such as imetelstat, a drug that has a bit of a different way it works than the JAK inhibitors, where my colleagues in Mayo Rochester have seen some benefits. 

So it's—it's an exciting time, a lot of things going on.  I'd say many of these things although exciting remain early.  I wouldn't say that we've learned anything from this meeting that means that people have to go out as soon as they hear this video and change a treatment or run out and do something drastic, but I do think that it means that be very hopeful that there is going to probably be multiple additional new options in the near future, and we're learning a lot more about MPNs. 

 

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 fo

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Page last updated on September 4, 2014