Novel Therapies and Clinical Trials for Myelofibrosis | Updates and Innovations
What’s the latest in higher risk myelofibrosis novel therapies and clinical trials? Expert Dr. Michael Grunwald from Levine Cancer Institute discusses JAK inhibitors and other research updates along with proactive patient advice for clinical trials.
[ACT]IVATION TIP
“…it is okay and, in fact, encouraged for patients to ask about clinical trials, especially if patients have access to a clinical trial center where they might be able to be treated on a clinical trial.”
Download Guide | Descargar Guía
See More From [ACT]IVATED Myelofibrosis
Related Resources:
|
Barriers to Accessing Specialized Myelofibrosis Care in Rural Areas | Challenges and Solutions |
Helping Rural Doctors Recognize Myelofibrosis | How Patients and Providers Can Work Together |
Transcript:
Lisa Hatfield:
Dr. Grunwald, can you discuss novel therapies and current clinical trials for lower risk and higher risk myelofibrosis?
Dr. Michael Grunwald:
We’re in an exciting time in myelofibrosis because we’ve already had some new therapies introduced into the clinic in recent years and there are a number of ongoing trials that are very exciting. Some of these trials look at agents in combination with JAK inhibitors or four currently approved JAK inhibitors for myelofibrosis, ruxolitinib (Jakafi) being the oldest one. And many of these trials that are ongoing will combine a novel agent.
So there’s pelabresib (CPI-0610), which is from a class of medicines called BET inhibitors, which has shown very good efficacy in reducing spleen size when it’s combined with ruxolitinib in the treatment of newly diagnosed patients with myelofibrosis. We also have navitoclax, which is an apoptosis inhibitor or a cell death inhibitor that’s been used in combination with ruxolitinib (Jakafi) and has had promising results presented in terms of spleen reduction. There’s selinexor (Xpovio), which is a drug approved for another blood disease, multiple myeloma, and that’s being combined in trials with ruxolitinib.
And then navtemadlin as well, which is from a group of drugs called MDM2 inhibitors. Then we have drugs being looked at as a single agent. So there’s an agent called imetelstat (Rytelo) that was recently approved for a cousin of myelofibrosis called myelodysplastic syndrome or MDS, and now it’s being evaluated in myelofibrosis.
We have ropeginterferon alfa-2b (Besremi), which is approved for another MPN polycythemia vera and it’s being looked at in myelofibrosis as well. Something really exciting to me is the CALR mutant inhibitors. So many patients with myelofibrosis will have CALR mutations. Probably around 30 percent of myelofibrosis patients have that mutation. And there are some strategies being developed to try to target that mutation and kill myelofibrosis cells by targeting it. There’s a naked antibody that’s in clinical trials. There is something called a bispecific antibody that is targeting the mutation, but also trying to bring immune cells or T cells close to the tumor cells so that there’s good tumor killing by the immune system.
And finally there’s a vaccine in development to try to target this mutation. There’s also a medicine called bomedemstat (MK-3543) that’s being tested in multiple myeloproliferative neoplasms and it’s been looked at as a single agent, and I believe it’s going to be looked at as a combination with a JAK inhibitor as well. Most of those therapies are targeting intermediate and high risk MF patients. That’s where a lot of the clinical trial action is. The ropeginterferon alfa-2b study is looking at lower risk patients. And then, there are some strategies to try to improve anemia in myelofibrosis, and those strategies can also include some patients toward the lower end of the risk spectrum.
For example, there’s a drug that’s been approved for myelodysplastic syndrome to help anemia since 2019, I think it’s been, 2019 or 2020. And that’s luspatercept (Reblozyl) it’s being tested for anemia in myelofibrosis. And I think that might be a drug that would be appropriate for some patients with lower risk disease who happen to have some anemia. My [ACT]IVATION tip for this question is that it is okay and, in fact, encouraged for patients to ask about clinical trials, especially if patients have access to a clinical trial center where they might be able to be treated on a clinical trial.
