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The Myeloproliferative Neoplasms (MPN) condition center is a comprehensive resource for cancer news & oncologist insights on MPN. Read more at CURE.

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CURE’s myeloproliferative neoplasms (MPN) page is an extensive resource of cancer information featuring the latest MPN news, expert oncologist insights on the latest cancer treatments, oncology research, advocacy efforts, and medical data on myeloproliferative neoplasms.

The year 2024 is now underway. After a busy 2023, we’re now looking back at some of the oncology headlines from the new year.

Notably, we have a clinical trial hold for a lung cancer drug, a trial that showed that a CAR-T cell therapy is cost-effective — though far from cheap — for lymphoma treatment, and a novel drug that’s showing promise for the treatment of myelofibrosis.

FDA Places Hold on Trial Evaluating TIL Therapy in NSCLC

The Food and Drug Administration placed a clinical hold on the ongoing IOV-LUN-202 trial, evaluating LN-145 TIL for patients with non-small cell lung cancer. With the clinical hold, new patients will not be able to enroll on the trial, and those who were previously treated will continue to be monitored.

The FDA placed the hold on the trial after the death of a patient. According to the FDA, the agency has the power to request that a trial be stopped, and then the sponsor — that’s the organization or company running the study — can reply via a response letter addressing the concerns. The FDA then has 30 days to respond, and potentially allow for the trial to resume.

Breyanzi Cost Effective As Second-Line R/R DLBCL Treatment

Research published in the journal, Blood Advances, found that the CAR-T cell therapy, Breyanzi, was found to be a promising and cost-effective treatment option for patients with relapsed or refractory diffuse large B-cell lymphoma — also known as DLBCL.

The study found that the incremental cost-effective ratio of the drug was just over $99,000 per quality-adjusted life-year from a health care perspective, and just over $68,000 from a societal perspective. Both these numbers are lower than the assumed social willingness to pay up to $100,000 per quality-adjusted life-year gained, as established by the American Society of Hematology.

While Breyanzi was deemed to be cost effective, CAR-T cell therapies are still far from inexpensive. In fact, the drug cost rose by 9% since its initial approval for DLBCL in 2022. In a CURE® Speaking Out® video series, Lee Greenberger, the chief scientific officer of the Leukemia & Lymphoma Society, expressed concern about the price of these drugs.

“Some of that the government is going to have to pay, some insurance is going to have to pay, but some of it the patients are going to have to pay as well. And the price tags are going to be significant. How are we going to manage that?

Phase 2 Trial Shows Fibrosis Reduction in Some With Myelofibrosis

Also in the blood cancer space, findings from the phase 2a trial found that a novel drug, GB2064, reduced fibrosis in the bone marrow, thereby slowing cancer progression in patients with myelofibrosis who were previously treated with Jakafi.

Myelofibrosis is a disease that affects the body’s production of blood cells in the bone marrow. The disease causes scar tissue to grow there — a process called fibrosis. By decreasing the amount of fibrosis that occurs, GB2064 has the potential to improve outcomes for this patient population.

However, we should note that this trial was completed in only a small number of patients — 10, to be exact. Six patients had a decrease of fibrosis after receiving GB2064 for six months.

For more news on cancer updates, research and education, don’t forget to 

The year 2024 is now underway. After a busy 2023, we’re now looking back at some of the oncology headlines from the new year. 

FDA Stops Lung Cancer Trial, CAR-T Cell Therapy Is Cost Effective, Novel Drug Slows Fibrosis

LeVar Burton knows the importance of storytelling quite well, as an actor, director and advocate for children’s literacy. In an interview with CURE®, he explained how he’s using storytelling to connect with the MPN community and inspire others to share their stories.

On this episode of the “Cancer Horizons” podcast, LeVar Burton sits down with CURE® to talk about the importance of MPN awareness and the power of storytelling in advocacy.

LeVar Burton Helping the MPN Community Through Advocacy and Storytelling

In this special edition, CURE spoke with Dr. Laura Michaelis, a specialist in hematology and oncology at Froedtert Hospital and Medical College of Wisconsin in Milwaukee and also a recipient of the 2019 MPN Heroes Award.

Michaelis has a mission statement to live up to when she’s treating patients: “deliver superb care in a compassionate, individualized manner.” As an MPN Hero, Michaelis has gone above and beyond to offer outstanding healthcare in and outside of the clinic, regularly speaking to MPN patient support groups to provide important information and answers in a more personal setting.

She spoke with CURE about how far we’ve come in the MPN space, how we can address current challenges moving forward, and what being an MPN Hero means to her.

CURE® is now accepting nominations for its ninth annual MPN Heroes Awards! CURE and Inctye are proud to honor heroes who have made a significant impact in the lives of those affected by MPNs. For more information or to nominate your hero today, visit, 

In this episode of the “CURE Talks Cancer” podcast, we spoke with Dr. Laura Michaelis, a 2019 CURE MPN Hero, about how far we’ve come in the myeloproliferative neoplasm space.

Recognizing Advancements and the Individuals Who Made Them in Myeloproliferative Neoplasms

In this episode of the “CURE® Talks Cancer” podcast, we spoke with Dr. Naveen Pemmaraju, associate professor of Leukemia at The University of Texas MD Anderson Cancer Center, who recently conducted research on disease awareness in patients with myeloproliferative neoplasms (MPNs).

Many patients with MPNs reported going to the internet to seek out information about their disease; however, many still did not know about some of the common markers of their disease. While the internet can be a great source of information, there is plenty of misinformation available, too, so it is always important that patients take what they find online and talk to their providers about it.

https://www.curetoday.com/view/how-to-find-reliable-cancer-information-online

Social media and the internet as a whole can offer a wealth of information and connections for patients with cancer, especially those with rarer diseases. However, patients should always bring the online advice to their health care team, says Dr. Naveen Pemmaraju.

Social Media and Rare Cancers: Stay Informed, But Tread With Caution

Myeloproliferative neoplasms, or MPNs, are a type of blood cancer that is often not heard about, which is why it is important for patients to speak with their doctors to learn more.

MPNS — essential thrombocythemia, myelofibrosis and polycythemia vera – begin with an abnormal change, or mutation, in a stem cell in the bone marrow, which leads to an overproduction of any combination of white cells, red cells and platelets.

 Talks Cancer” podcast, we teamed up with our sister publication “

” to speak with a patient-doctor duo on the disease.

Learn more from Dr. Ruben A. Mesa, director of the Mays Cancer Center at UT Health San Antonio MD Anderson, and Antje Hjerpe, a patient diagnosed with essential thrombocythemia in 1992. The pair discuss myeloproliferative neoplasms — what they are, how they’re treated and how patients can talk to their doctors to be their own best advocates.

In this special edition of the “CURE Talks Cancer” podcast, we teamed up with our sister publication “OncLive on Air” to speak with a patient-doctor duo on myeloproliferative neoplasms.
 

Patient-Doctor Duo: The Basics of Myeloproliferative Neoplasms

When a 20-something med student started to experience overwhelming fatigue and organ failure out of nowhere, he eventually took matters into his own hands.

Dr. David C. Fajgenbaum, who is now an assistant professor of medicine at the University of Pennsylvania in the Center for Study & Treatment of Castleman & inflammatory Lymphadenopathies, was diagnosed with Castleman disease — a deadly illness that presents like a mix of cancer and an autoimmune disorder.

After relapsing four times after his initial diagnosis and realizing after chemotherapy that the available treatments were not going to be enough, Fajgenbaum eventually evaluated his own samples to find a cure for his rare disease. With his radical approach to conducting original medical research, he identified and prescribed himself his own treatment that has brought about a lasting yet tentative remission.

Fajgenbaum went on to co-found and become the executive director of the Castleman Disease Collaborative Network, which uses a network of physicians, patients and other stakeholders to crowdsource ideas about what should be studied in clinical trials and who would be best suited to conduct the research. He also recently authored a memoir, titled “Chasing My Cure.”

More recently, he served as the special guest speaker at 

, held to honor those individuals who excel in their efforts to raise awareness and education around myeloproliferative neoplasms.

As a 20-something med student facing a rare disease, Dr. David C. Fajgenbaum took matters into his own hands to find his own treatment.

The Blueprint to Facing a Rare Disease

When he retired in 2010 after 30 years as a referee for the National Hockey League, Kerry Fraser looked forward to hanging up his skates and spending some well-earned time with his family. But seven years later, he was body checked by something he didn’t expect: a diagnosis of essential thrombocythemia, a rare blood cancer.

 Talks Cancer, we talk with Kerry about his diagnosis, his advocacy work, and how he uses the lessons he learned on the ice and works with his winning team to conquer each day.

In this episode of CURE Talks Cancer, we talk with retired NHL referee Kerry Fraser about his MPN diagnosis, his advocacy work, and how he uses the lessons he learned on the ice and works with his winning team to conquer each day.

Kerry Fraser: Facing Off Against Blood Cancer

Information about myeloproliferative neoplasms — or MPNs – is hard to come by. Even a simple internet search about this rare group of blood cancers turns up little. But the MPN Research Foundation’s myMPN Patient Registry aims to change that.

This week on CURE Talks Cancer, we spoke with Robyn M. Scherber, Assistant Professor of Medicine in the Department of Hematology and Oncology at the UT Health San Antonio MD Anderson Cancer Center; Lindsey Whyte, myMPN Project Manager of the MPN Research Foundation; and Michelle Woehrle, Executive Director of the MPN Research Foundation about their work with the registry, and about a recent patient data analysis that is helping to shed new light on this family of diseases.

Information about myeloproliferative neoplasms – or MPNs – is hard to come by. But the MPN Research Foundation’s myMPN Patient Registry aims to change that. Learn more about it in this week's episode of the CURE Talks Cancer podcast. 

Finding a Way Forward in Blood Cancer

For this week's episode, hear from some of 

. These Heroes are patients, survivors, caregivers, advocates or health care professionals with a passion for improving the lives and outcomes of patients with myeloproliferative neoplasms. 

You'll hear from Angela Fleischman, M.D., Ph.D., Lori Jemison, RN, Marcy Worthington, Richard French, Jaden Presaud, Celia Miltz, Jean Diesch and Rebecca Shapiro and Molly Guy. 

In this week's episode of the CURE Talks Cancer podcast, hear from some of our recent MPN Heroes.

MPN Heroes Make a Difference in the Rare Blood Cancer Space

Receiving a cancer diagnosis can be terrifying—Olympic gold medalist Scott Hamilton knows that firsthand. However, after the shock wears off, it’s possible to use cancer as a motivation to do better in the world. That’s what Scott Hamilton did by starting the Scott Hamilton CARES foundation, which is dedicated to improving the future of cancer care.

In this week’s episode, we spoke with Hamilton about his personal bouts with testicular cancer and a brain tumor, as well as losing his mother to breast cancer. The former professional ice skater also touches upon why he wanted to be involved in CURE’s 2018 MPN Heroes event.

For this episode of the CURE Talks Cancer podcast, we spoke with Scott Hamilton, an Olympic gold medalist, cancer survivor and founder of the Scott Hamilton CARES Foundation

Scott Hamilton: When Fear Flips

Among patients with polycythemia vera, the treatment pairing of Jakafi and Pegasys shows benefits with acceptable toxicity, research showed.

Jakafi, Pegasys Combination Beneficial in Newly Diagnosed Polycythemia Vera

Second-line treatment with Inrebic has been shown to be beneficial for patients with myelofibrosis previously treated with Jakafi.

Inrebic May Reduce Spleen Volume in Myelofibrosis

An expert explained the importance of education and involvement in care for patients with myeloproliferative neoplasms. 

Education on MPN Symptoms, Treatments Leads to Greater Involvement in Care

Although Jakafi has been approved for patients with higher-risk myelofibrosis, those with lower-risk disease may also benefit from this treatment.

Patients With Lower-Risk Myelofibrosis May Respond to Jakafi 

Vonjo was also shown to improve overall survival in patients with myelofibrosis treated with Vonjo in the real-world setting.

Vonjo Improves Thrombocytopenia, Anemia in Patients With Myelofibrosis

Dr. Prithviraj Bose discusses patient characteristics to potentially optimize selection of the available JAK inhibitors for patients with myelofibrosis.

Dr. Prithviraj Bose Discusses Patient Characteristics for Treatment Selection

Several patient characteristics — spleen size, symptom burden and blood counts, among others — are taken into consideration when deciding upon the best JAK inhibitor to treat myelofibrosis, a type of myeloproliferative neoplasm (MPN).

Dr. Prithviraj Bose, professor in the department of leukemia at The University of Texas MD Anderson Cancer Center in Houston, spoke with CURE onsite at the Society of Hematologic Oncology 2024 Annual Meeting, to discuss these factors to potentially optimize selection of the available JAK inhibitors for patients with myelofibrosis.

There is a lot of different patient characteristics one looks at. So, one looks at the size of the spleen, one looks at the burden of symptoms, and one looks at the blood counts. So, this is a unique cancer where treatment is not by … stage of the cancer, or, it's not very algorithmic in terms of what is front line, what is second line, what is third line. So it's really based on the need of the patient.

Now, again, generally, I will say of the four [Food and Drug Administration-]approved JAK inhibitors — again, JAK inhibitors are all we have approved in myelofibrosis — I think ruxolitinib (Jakafi), 

, can all be used in frontline [setting], but I think what really happens is that it's either ruxolitinib or momelotinib, with momelotinib being favored if the patient is anemic, and otherwise ruxolitinib. I will say the majority [of patients] get ruxolitinib because it is so good for spleen and symptoms, and it has a survival benefit that's been shown [in clinical data].

In the second line, I think it goes back more to what I was saying about the clinical presentation. If you have good counts, large spleen, by all means, go with fedratinib. If you have, anemia, predominant presentation where that's what you're trying to improve the most, momelotinib, I think, is your best choice. If the platelets are super low, which is rare to happen in [the] front line [setting], much more common in second and later lines, if it less than 50 [platelets per microliter of blood], absolutely pacritinib. That has the best data, and it has good efficacy there. So I think … you cannot set exact rules, but these are just some pointers.

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Spleen size, blood counts and symptom burden are some of the factors cancer teams take into account when selecting which of the four JAK inhibitors to treat patient with myelofibrosis.

Patient Characteristics Guide Decision Making for JAK Inhibitors in Myelofibrosis

Patients with myeloproliferative neoplasms should keep their care team up to date on any symptoms they’re experiencing, an expert said. 

MPN

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