Targeted Approaches in Myelofibrosis Treatment

Raajit K. Rampal, MD, PhD, hematologic oncologist at Memorial Sloan Kettering Cancer Center, discusses some of the promising emerging therapies for myelofibrosis that target different pathways at the Fifth Annual Miami Cancer Institute Global Summit on Immunotherapies for Hematologic Malignancies, hosted by Dr. Guenther Koehne and Miami Cancer Institute.
In the myelofibrosis treatment landscape, ruxolitinib (Jakafi) is an established JAK inhibitor, along with newer JAK inhibitors like fedratinib (Inrebic) and momelotinib (Ojjaara). Research continues to evaluate new options, including navitoclax.

He then moves into an overview of some of the significant and ongoing clinical trials that are currently investigating new treatment approaches for patients with myelofibrosis. Specifically, Rampal highlights important phase 3 trials that have had their 6-month readouts, highlighting the need for further follow-up to look at progression-free survival, overall survival, duration of response, and more.

Transcription:

0:09 | We are in a situation where there are a lot of novel and interesting therapies. Right now, phase 3 data has just [been] reported out on pelabresib [CPI-0610], which is a BET inhibitor, as navitoclax, which is the BCL-xL inhibitor, [and] both look really promising. There are things in earlier phases as well like PIM kinase inhibitors, we also have things like MDM2 inhibitors, and now we are at the advent of potentially having immunotherapies with antibodies in the market called calreticulin, which is really exciting.

0:40 | I think there are a number of [significant clinical trials]. As I mentioned, there are phase 3 trials that have had their 6 month readout. We have got to see what happens with longer-term follow-up, does it change progression-free survival, overall survival, the depth of response, to the duration of response? All of those things remain to be determined. Of course, there are a number of earlier phase trials that are ongoing.