VIDEO: Targeted strategies, other future research in myeloproliferative neoplasms

Editor’s note: This is an automatically generated transcript. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription.

Yeah, so picking up from my from my point, developing strategies that are curative is an unmet need, making transplants better safer, as well as opening transplant options to more and more patients, for example, making donor options available for everyone. Now, with haploidentical donors and post-transplant side, I have never had to say no to transplant because of lack of donor availability, and I think that's something that needs to be considered and practiced more and more but the unmet need in the context of transplant remains relapses after transplant and making transplants safer. And that's work that we need to continue to do. We also need to continue to think about targeted strategy. What I am looking forward to is how these mutant CALR targeting strategies with monoclonal antibodies and BiTEs, how they fare in terms of their efficacy, obviously, as well as toxicity, as well efficacy in terms of decreasing the clonal burden and hence, how do they impact the overall natural history of disease, because so far, we have not had strategies or treatments that alter the natural history of neurofibrosis.

What is exciting right now is that there are combination therapies that seem to be benefiting some people up front and some people in a later stage of treatment. And what would be important for us to know, as the future of this continues to develop, is who are the patients who would benefit from combination therapies up front and who are probably more likely to have a good response to ruxolitinib or an upfront JAK inhibitor by itself? And that's important when you think about any combination, you do add more efficacy, but you also tend to add more toxicity, including financial toxicity, right? So those are important metrics to keep in mind as we think about combinations, so as to not expose everyone to those toxicities, but be able to select patients who will benefit most from that. The other thing that comes to mind is patients who develop accelerated and blast phase disease. I think the strategies or the treatment options that we have available for that subset of patients, patients with imminently progressive disease are not very successful. To be, honest, we have, we usually resort to hypermethylating agents in combination with either JAK inhibitors or BCL-2 inhibitor VENCLEXTA, but the responses remain modest and I do think that's an area that we need to develop further, in terms of treatment options.