Tag Archive for: BET inhibitor

Advances in Myelofibrosis Research

Advances in Myelofibrosis Research from Patient Empowerment Network on Vimeo.

What are the recent developments in the study and advancement of myelofibrosis treatment? MPN researcher Dr. Gabriela Hobbs discusses ongoing clinical trials for new JAK inhibitors, BET inhibitors, and anemia therapies, among others.

Dr. Gabriela Hobbs is a hematology-oncology physician specializing in the care of patients with myeloproliferative neoplasms (MPN), chronic myeloid leukemia, and leukemia. Dr. Hobbs serves as clinical director of the adult leukemia service at Massachusetts General Hospital. Learn more about Dr. Hobbs.

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What about myelofibrosis, Dr. Hobbs? What advances are being made in the care of patients with this more advanced MPN? 

Dr. Hobbs:

Yeah. So, in myelofibrosis, I would say it is almost difficult to keep track of how many clinical trials are currently open. So, in 2011, we had ruxolitinib approved, or Jakafi. That was the first JAK inhibitor. Since then, we’ve had two more JAK inhibitors approved, fedratinib (Inrebic) and most recently pacritinib (Vonjo). And we’re currently awaiting the fourth JAK inhibitor to be approved, and that’s called momelotinib.   

And in addition to the JAK inhibitors, there are lots of other clinical trials underway right now that are either alone – a drug by itself or a drug in combination with ruxolitinib.  

So, there are several Phase III studies. And the reason why that’s important is that after Phase III we usually see a drug approval. So, we can expect, hopefully in the next couple of years, to see many more drugs available on the market to treat patients with myelofibrosis. Some of those include agents that block different pathways within a cell. And that includes a drug called parsaclisib. There’s a drug called pelabresib (CPI-0610), which is a BET inhibitor.  

There’s another drug called navitoclax (ABT-263), which is a cousin of venetoclax (Venclexta), which is a drug that we’ve been using a lot in leukemia. So, there’s lots of different drugs that are being used in combination with ruxolitinib. There’s also a drug called luspatercept (Reblozyl) that’s also been approved for myelodysplastic syndromes. And I suspect that that’ll be approved as well to help patients with anemia. So, really, there’s lots of drugs that are being studied right now. And I think the question that we’re all asking is, well, how are we going to use all of these different drugs? So, I look forward to seeing the results of those studies.  


Mm-hmm. Will some drugs work better for some patients and others not? 

Dr. Hobbs:

That is such a good question. And so, what I’m hoping to see is exactly that. I’m hoping to see that for patients, for example, with anemia, perhaps we’re going to be using luspatercept and momelotinib. Perhaps we’re going to see that patients with certain mutations may respond better to certain medications like the BET inhibitors or navitoclax or the PI3 kinase inhibitor, parsaclisib. But as of now, we don’t have enough information.  

We haven’t seen enough results of these studies to start to be able to know, you know, what is the patient that’s going to do better with two drugs versus one drug? And so, I think that over the next couple of years we’re going to start to have answers to those questions.  

The Latest in MPN Research: Updates from ASH 2021

The Latest in MPN Research: Updates from ASH 2021 from Patient Empowerment Network on Vimeo.

MPN specialist, Dr. Andrew Kuykendall, shares the latest news from the 2021 American Society of Hematology (ASH) annual meeting. Dr. Kuykendall discusses the latest findings in MPN research, including an update on JAK inhibitors, advances in BET inhibitors, as well as a new therapy in development aimed at reducing phlebotomy in patients with polycythemia vera (PV).

Dr. Andrew Kuykendall is an Assistant Member at Moffitt Cancer Center in the Department of Malignant Hematology. Dr. Kuykendall’s clinical and research efforts focus on myeloproliferative neoplasms (MPNs), MDS/MPN overlap syndromes and systemic mastocytosis (SM). Learn more about Dr. Kuykendall, here.

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You’re joining us following the American Society of Hematology Meeting where cancer researchers came together to share their findings. Are there highlights from the meeting that patients should know about?

Dr. Kuykendall:

Yeah, absolutely. So, the meeting we just came from, the so-called ASH meeting, is really an annual meeting. Happens every December.

It’s really a chance for researchers to share their most exciting findings and really what they’ve been working on for the past few years, and certainly in the past year.

As a clinical researcher, I think I have always a keen interest in clinical trials that are going to give us some new data so we can see how things are working, but I think this is also a big meeting for pre-clinical studies for basic scientists who get to share what’s exciting in their labs. A lot of times that’ll give a preview of what’s to come maybe four, five years down the road what we’ll see on the clinical side. From the clinical side, which is more in my realm, there is certainly a few specific things to get excited about. Within the field of myeloproliferative neoplasms, we have polycythemia vera, ET – essential thrombocythemia, myelofibrosis.

And on the myelofibrosis side of things, I think we continue to get excited about just really the proliferation of drugs that are in late-stage clinical trials. This meeting was no different from that.

We started to get a little bit more clarity as far as this agent, pelabresib, which is a BET inhibitor which is being looked at really in a variety of different settings as a single agent in combination with ruxolitinib (Jakafi) and as an add-on to ruxolitinib as well.

This was another exciting need to get an update on where the data looks to be with pelabresib. Certainly, there’s an ongoing Phase III study in the up-front setting with that agent. We’re anxiously awaiting results too. Additionally, we’ve got more information regarding other JAK inhibitors that may be coming down the pipeline in the coming months to years with momelotinib and pacritinib.

Certainly, that’s always exciting to see the data come from there, especially when we get kind of further along in their trials, we start to get very isolated assessments of their data. Looking specifically at transfusion rates and the efficacy within the subpopulations that have unmet need. And so, I think that that’s always exciting.

I think polycythemia vera – this is a really big meeting for polycythemia vera. We obviously know that ropeginterferon (Besremi) just got FDA-approved in November.

We also started to see the updated data with rusfertide, or PTG-300, which is a hepcidin memetic that aims to reduce phlebotomy rates in patients that are requiring a ton of phlebotomies which, as we know, can be very impactful on quality of life having to get recurrent phlebotomies.

I think that those were the really big highlights, and the take-aways from this is really we are starting to see these agents move into the late-stage clinical trials.

Promising ET, PV & Myelofibrosis Therapies in Development

Promising ET, PV & Myelofibrosis Therapies in Development from Patient Empowerment Network on Vimeo

MPN specialist, Dr. Srdan Verstovsek discusses the latest research and progress for the treatment of patients with essential thrombocythemia (ET), polycythemia vera (PV) and myelofibrosis (MF).

Dr. Srdan Verstovsek is Chief of the Section for Myeloproliferative Neoplasms in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Verstovsek, here.

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Dr. Srdan Verstovsek

When we talk about the new therapies in development, there are many in myelofibrosis in particular, and a few are in essential thrombocythemia and polycythemia vera. Let’s start with ET and PV. Here we are expecting either studies, or possibly even approval, of a long-acting interferon called Ropeginterferon that was approved a year ago in Europe for PV patients.

We gonna have, hopefully here in the United States, that drug for our patients in a year or perhaps studies in PV, or perhaps most definitely, I would say, studies in ET with this drug. That would be enhancement of what we done off-label using interferons that are approved for some other conditions. We know that interferons are biological agents active in these conditions to control the bone marrow, and perhaps even decrease the number of malignant cells in the bone marrow of patients with ET and PV, which may be beneficial down the road.

In myelofibrosis, the picture is completely different. In this setting, the life expectancy, unfortunately, is affected as we discussed, and we need therapy that would be perhaps improving that life longevity. As we know, the ruxolitinib JAK inhibitor that has been around for nine years can extend the life a few years, but not cure people.

So, helping JAK inhibitors by combinations with other active agents that would be biologically modifying that bone marrow, decrease the tumor burden, improving the quality of life or anemia, are at forefront of what is happening right now. So, combinations with Navitoclax which is Bcl-xL cell inhibitor, CPI-0610, which is BET inhibitor, Luspatercept which is anemia drug.

These are phase three studies that are planned to start soon for possible approval for combinations over JAK inhibitor alone for different problems that people face.

Or, later on in the course of the disease, JAK inhibitor may fail. What do you do then? So, we have studies announced that will be done in what we call a second line, after-JAK inhibitor. And the MDM2 inhibitor was announced. Imetelstat inhibitor in the second line. Momelotinib JAK inhibitor in the second line. Fedratinib is being studied, another JAK inhibitor. Pacritinib for patients with low platelets

These are all phase three studies. That’s means for approval of this drug, so that will be three and four, seven different phase three for myelofibrosis patients with different clinical scenarios, different clinical problems are being done, or about to be done, in very near future. So, my prospect is here. My view on that is that we will have, hopefully, at least some of these seven studies leading to approval of some new drugs for our patients with myelofibrosis.