How and why do MPNs progress? MPN specialist Dr. Joseph Scandura shares an update on research being done to better understand–and possibly prevent–disease progression.
Dr. Joseph Scandura is an Associate Professor of Medicine and Scientific Director of the Silver MPN Center at Weill Cornell Medicine. Learn more about Dr. Scandura.
Is there research being done on MPN progression to understand how it happens or even prevent or slow progression?
Yeah. There’s a lot. I think there is a – from both the sort of basic laboratory using animal models to try to understand what are the kind of systems that are involved in how these diseases change. What genes are involved? How do they talk to each other? You know, these are not cells that live in a vacuum, right? They live in a special microenvironment. What are the signals that crosstalk between the MPN cells, the MPN stem cells, and their microenvironment?
And so, there’s a lot of research on that and the basic side of things. In humans, there’s a lot that has been done over the years in terms of trying to understand what are some of the genetic features of progression. And I think we’re beginning to get a little bit of a better understand of what are the non-genetic things that are associated with progression.
I was part of an effort from the MPN Research Foundation and still am.
They have what they call the Progression Network, where they tried to put together a number of investigators from really across the world to share ideas about the nature of progression and how we might look at studying this and understanding ways to prevent progression.
I think we do have some drugs now that show some promise in terms of being able to prevent progression. I think interferons have shown this in polycythemia vera in terms of a promise for improved long-term outcomes and delayed risk progression. I think that the gold standard randomized trials are maturing and are sort of bearing out some of the same findings that have been observed retrospectively, so sort of kind of looking back in time.
But the difficulty is that it can take a long time for patients to progress. And you say, “Oh, that’s great.” And that is great. But, from a research – from a statistical side, it means things are really slow. If you have to wait 15 years to assess whether or not people progressed less in one treatment versus another, it’s really slow going. And so, we have to do a compromise of what’s – you know, what do animal studies say? What does retrospective analysis, when we might have people who started treatment 30 years ago, and now we’re just seeing how did it all work out? It’s not a perfect study, because biases can creep in, but it’s what we have now. And so, there’s a lot. And I think, increasingly, progression is being recognized as a goal of therapy, to prevent progression.
Personally, it is one of my major goals, because I think we do a pretty good job at preventing clots with available treatments. But I don’t think we do a very good job at preventing progression, mostly, because we don’t exactly understand what’s driving that. And so, I think until we develop that deeper understanding and really invest the time and effort in terms of learning which approaches can help prevent progression, we’re going to continue to have these questions.