26 Jun 2025

Research Blog: Science in Action

Some Leukaemia UK research highlights from 2025 (So Far!)

At Leukaemia UK, we know that research doesn’t just happen behind the scenes — each finding is a stepping stone to changing lives. The publication of findings is a vital step forward, providing evidence, insight, and innovation that help transform how we diagnose, understand and treat leukaemia and related blood cancers. Even the negative results tell us something we probably didn’t know yet.

Scientific publications are more than just academic milestones — they’re the way researchers share their findings with the world. A submitted paper is rigorously reviewed by experts, helping to ensure the quality and credibility of the science. Once published, these findings become part of a global effort to tackle cancer from every angle.

As we reach the halfway point of 2025, we’re proud to spotlight just a few of the many groundbreaking studies from researchers supported by Leukaemia UK. These represent a selection of highlights — not an exhaustive list — but they show the range, depth and real-world promise of our community’s work.

Targeting a New Marker in AML

Dr Konstantinos Tzelepis, University of Cambridge, JGF FUF  — “Treatment of acute myeloid leukemia models by targeting a cell surface RNA-binding protein”

In a breakthrough that could transform immunotherapy for Acute Myeloid Leukaemia (AML), Dr Tzelepis and his team discovered that a protein called Nucleophosmin (NPM1), typically found inside cells, also appears on the surface of AML cells — and, crucially, on leukaemic stem cells, which are the root of relapse in AML.

This cell-surface version, csNPM1, was found across a, making it a promising universal target. Current immunotherapies often rely on a handful of targets, and when those fail, options are limited. By identifying csNPM1, Dr Tzelepis’ team not only deepened our understanding of leukaemia biology, they have also engineered an experimental targeted therapy — one that selectively killed leukaemia cells while sparing healthy ones in preclinical models.

What this could mean:

• A potential new class of targeted therapies for AML
• Hope for relapse prevention by targeting leukaemic stem cells
• A shift in how we understand and exploit cancer cell markers

Uncovering the Immune Fault Line in Myeloproliferative Neoplasms

Dr Eman Khatib-Massalha, University of Cambridge, JGF — “Defective neutrophil clearance in JAK2V617F myeloproliferative neoplasms drives myelofibrosis via immune checkpoint CD24”

This vital study shines light on the immune system’s role in driving myelofibrosis — which is a rare, chronic blood cancer where scar tissue (fibrosis) develops in the bone marrow and is a serious complication of myeloproliferative neoplasms (MPNs). Dr Eman Khatib-Massalha and colleagues discovered that aged neutrophils in patients with the JAK2V617F mutation aren’t properly cleared from the bone marrow. Why? They overproduce a “don’t-eat-me” signal called CD24.

As a result, these rogue cells evade removal, accumulate in the marrow, and spark inflammation that leads to scarring (fibrosis). This scarring disrupts the normal structure of the bone marrow, making it harder for it to produce healthy blood cells. As fibrosis worsens, it can lead to serious problems like anaemia, fatigue, increased risk of infection, and bleeding issues.

But here’s the potential game changer: blocking CD24 in mouse models reversed this process.

What this could mean:

• CD24 may be a new target for treating or preventing myelofibrosis
• Offers hope for reducing symptoms and slowing disease progression in MPNs
• Strengthens the link between immune signalling and cancer-related inflammation

A Kinder Combination for Childhood Leukaemia

Dr Simon Richardson & Prof. Brian Huntly, Cambridge Stem Cell Institute, JGF & Project Grant — “CREBBP inactivation sensitizes B cell acute lymphoblastic leukaemia to ferroptotic cell death upon BCL2 inhibition”

B-cell Acute Lymphoblastic Leukaemia (B-ALL) makes up around 40% of all childhood leukaemias in the UK. Now, two Leukaemia UK-funded scientists — Dr Simon Richardson (John Goldman Fellow) and Prof. Brian Huntly (Project Grant)— may have found a safer way to treat it.

Their recent study combined two oral drugs, venetoclax (used in AML) and inobrodib, to create a dual-action therapy. The result? Enhanced cancer cell death through a mechanism called ferroptosis — with fewer harmful effects on healthy cells. This represents an entirely new, potentially less toxic and orally available approach to cancer treatment, opening doors to more accessible therapies.

What this could mean:

• Opens the door for clinical trials of a safer, targeted combination therapy
• Potential for outpatient, oral treatment of adults, teenager and young adults (TYAs) with B-ALL initially
• Could reduce long-term side effects often seen with current intensive treatments

These are just a few examples of how Leukaemia UK-funded researchers are changing what’s possible in blood cancer. From uncovering the roots of relapse to designing safer, kinder therapies.

With more funding, these discoveries can go further — into clinical trials, into hospitals, and into the hands of doctors treating patients. Every breakthrough moves us closer to our vision to stop leukaemia devastating lives

Together, we can help transform how these diseases are understood, diagnosed and treated — so that more people not only survive, but thrive.

Discover our research blogs.