June Blood Cancer Highlights: Top 5 Updates Patients Should Know

The ending of June calls for a time of reflection on the most recent treatment updates and FDA approvals provided during the month, as well as survivorship news. The updates that came throughout June were plentiful, following the 2025 ASCO Annual Meeting at the beginning of the month.

This meeting provided updates on many treatment options that hold much promise for patients with polycythemia vera, MDS, and even CLL. Moreover, many cancer survivors and thrivers shared their insights with CURE this month, where they highlighted the difficulties that come along with the invisibility of a blood cancer.

These updates continue to impact patients and their families, as well as caregivers, so be sure to read on for more information on these updates below.

FDA Grants Fast Track Designation to New Treatment in Myelofibrosis

The therapeutic agent nuvisertib (TP-3654) was granted fast track designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with intermediate or high-risk myelofibrosis (MF), and was supported by updated preliminary phase 1/2 data presented at the European Hematology Association 2025 Congress in Milan, Italy.

Clinical activity, such as symptom and spleen responses that correlated with cytokine modulation, were reported with the use of nuvisertib. Of the evaluable patients, 22.2% demonstrated a minimum of a 25% reduction in spleen volume, and 44.4% had at least a 50% reduction in total symptom score. Moreover, improvements in bone marrow fibrosis were reported in 42.9% of patients, 24% had hemoglobin improvements, and 26.7% had platelet count improvements.

According to the FDA website, fast track designation is given to investigational therapies for serious or life-threatening conditions that have the potential to address unmet medical needs.

What Patients With Polycythemia Vera Should Know After ASCO 2025

Following the 2025 ASCO Annual Meeting earlier this month, experts Dr. Joshua K. Sabari and Dr. Raajit K. Rampal sat down to discuss the key updates in blood cancer which were shared at the meeting, breaking down what patients should know, specifically those with polycythemia vera.

The pair discussed the investigation of Besremi (ropeginterferon alfa-2b-njft) — which is FDA-approved for polycythemia vera — in essential thrombocythemia, as well as the investigation of Scemblix (asciminib) — which is FDA-approved for frontline therapy or after failing two different TKIs — in chronic myeloid leukemia.

“Yes, a couple of key things to highlight. As you just said, it's important to point out that we actually had a hematologic malignancy presentation as part of the plenary session. That data, presented on rusfertide, a hepcidin mimetic being tested for polycythemia vera, aims to potentially liberate people from therapeutic phlebotomies. The high-level data showed that patients were, in fact, able to be rendered free of therapeutic phlebotomy and also experienced symptomatic improvement. So, if that drug gains approval, I think it may have a really useful path in the treatment of polycythemia vera,” Rampal highlighted.

Rampal is the director of the Center for Hematologic Malignancies, as well as the director of the Myeloproliferative Neoplasms Program at Memorial Sloan Kettering Cancer Center, located in New York. Sabari is the editor in chief of CURE. He also serves as an assistant professor in the Department of Medicine at NYU Grossman School of Medicine and director of High Reliability Organization Initiatives at Perlmutter Cancer Center, also located in New York.

The Invisibility of Blood Cancers to Others

Unlike many other cancers, blood cancers — like chronic lymphocytic leukemia (CLL) and myelodysplastic syndrome (MDS) — often have few visible signs to others, according to Jane Biehl. There are (typically) no surgical scare, hair loss or outward physical changes that others would be quick to notice or associate with an illness. However, this does not stop her oftentimes crippling side effects from the disease and its treatment.

“As a person with MDS, I look the same as before I was diagnosed, except I am paler and there are bags under my eyes. I feel lucky as I observe others with ports and scars and missing breasts that are more obvious,” Biehl explained in her article. “The huge side effect that is life-changing for me is a crippling fatigue. All cancers have the side effect of fatigue, whether it be from treatment or the cancer itself. However, with CLL, MDS and other blood cancers, the energy never returns. We are always anemic with low blood counts even when in remission. I am forced to take constant breaks, have an inability to stand for extended periods of time, and going up and down a flight of stairs takes forever. I cannot rush from one task to another like I used to. I plan my day to prevent overworking. In summary, the fatigue is persistent, intense, and challenging.”

New FDA Breakthroughs for Waldenstrom Macroglobulinemia

On June 5th, treatment with iopofosine-131, a potential first-in-class cancer-targeting agent, was approved by the FDA for the treatment of patients with relapsed or refractory Waldenstrom macroglobulinemia. This approval was supported by data from the phase 2 CLOVER WaM study, which demonstrated an overall response rate of 83.6% and a major response rate of 58.2% for treated patients; these outcomes exceeded the study’s primary endpoint of a 20% major response rate.

“The FDA’s breakthrough therapy designation underscores the potential of iopofosine I-131 as it may offer substantial improvement on at least one clinically significant endpoint over available therapies to address the substantial unmet medical need in this life-threatening cancer,” James Caruso, president and chief executive officer of Cellectar, said of the approval. “With robust clinical data, a favorable safety profile, expedited review designations in the United States and Europe and a compelling commercial market potential, we believe iopofosine I-131 represents an attractive candidate for potential collaborations or partners seeking impactful innovation and accelerated development pathways.”

Rusfertide Reduces Need of Phlebotomy and Improves Quality of Life in Polycythemia Vera

At the beginning of the month, Dr. Aaron Gerds sat down for an interview with CURE where he broke down the results of the phase 3 VERIFY trial, which showed that treatment with rusfertide has the potential to be a new treatment option for patients with polycythemia vera. On the trial, which was also presented at the 2025 ASCO Meeting, the agent both reduced and/or eliminated the need for phlebotomies — a procedure which removes extra blood cells from the body by using a needle to take blood from the vein — improving the patients quality of life and improving symptom control.

Gerds is an assistant professor in the Department of Medicine and member of the Developmental Therapeutics Program at Case Comprehensive Cancer Center School of Medicine, as well as a physician in the Department of Hematology and Medical Oncology at the Cleveland Clinic, all located in Ohio.

“[Rusfertide] is a new therapy that is alleviating the need for phlebotomies and improving patients' quality of life. This is incredibly important for patients. Having another tool in our arsenal to care for these patients is truly exciting,” Gerds emphasized.

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