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Ongoing Research May Lead to ‘Exciting’ Advancements for Myelofibrosis Years Down the Line


Research efforts will improve the lives of patients with myelofibrosis, though those advancements may take years to come, an expert said.

Ongoing trials are laying the groundwork for “exciting” advancements in the treatment of myelofibrosis, but those new treatments will take some time, explained Dr. Joseph Scandura, an associate professor of medicine at Weill Cornell Medicine in New York City.

For example, Scandura is conducting research showing that certain biomarker changes — changes in different proteins found on myelofibrosis cells —may indicate a clinical benefit for patients taking pelabresib.

This research (and many other trials) is ongoing and may lead to better treatment options down the line, according to Scandura.


Some of the agents being used in myelofibrosis— and it's not restricted to myelofibrosis, but it's one of the most prominent — are showing changes that we've not seen with agents with drugs in development in this field.

And that's exciting, and there's a lot. The fact that there are so many drugs being tested, just provides an opportunity for some of these things to be good. And so, I think it's a very exciting time for a lot of investigators who have been stuck with inadequate therapies for a long time, because it allows us to potentially offer something to our patients that can hopefully fundamentally change their outcomes.

That, to me, is what drives this whole boat, if we're changing outcomes, we're doing something beneficial. And, the frustration is, it can take time to learn that and so although all of these studies are ongoing, and we have so many drugs in development, now, it still is going to take time for to learn enough about the long-term events survival, time to treatment, failure, event-free survival, you know. Those are what really matter for people and, fortunately or unfortunately, it takes time to learn about those things because they can be many years.

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