28 Apr 2025

Research blog: Accelerating progress in AML

For the last 50 years, treatments for acute myeloid leukaemia (AML) have remained largely unchanged — intensive chemotherapy and, for some, a bone marrow transplant. Survival rates stagnated, and many patients, particularly older adults, had limited options. But in recent years, research breakthroughs have ushered in a new era of hope. Since 2017, the FDA has approved 12 new treatments for AML, with more on the horizon. Targeted therapies like venetoclax, FLT3 inhibitors, and IDH1/2 inhibitors have transformed the outlook for many patients. In addition to these a wave of new agents — including menin inhibitors and immunotherapies — are showing promise in clinical trials.

Yet despite these advances, AML is still extremely challenging to treat.  It is one of the most aggressive forms of blood cancer and the most common acute leukaemia in adults. It strikes suddenly, often without warning, and progresses rapidly. Nearly 3,100 people are diagnosed with AML every year in the UK and on average, almost 80% won’t survive beyond five years. AML affects the myeloid cells — vital components of our immune system — and disrupts their ability to function normally. What makes AML so dangerous is its speed and complexity. Driven by a tangled web of spontaneous random genetic mutations and abnormalities, the disease presents differently in nearly every patient, making treatment uniquely challenging. Left untreated, AML can be fatal in a matter of months.

The WHO currently recognises over 20 distinct subtypes of AML, a number that continues to grow as research uncovers new genetic mutations and disease pathways. This expanding complexity underscores the need for diverse treatment strategies and sustained research efforts. However, as highlighted in a recent review by Forsberg and Konopleva (2024), having many new treatments presents a new challenge: how do we best combine, sequence, and personalise these options? With so many variables — from genetic mutations to patient age and fitness — there is no one-size-fits-all approach. Clinical trials are now more important than ever, not just to test new drugs, but to figure out how to use them in smarter, more effective ways.

John’s story illustrates how he found strength in participating in research during his AML journey, and, in turn, from benefiting others. A retired technical trainer, John had just settled into a new seaside life in Anglesey with his wife Cathy when a blood test following a minor illness changed everything. Diagnosed with AML in early 2021, John initially responded well to chemotherapy. But when standard treatment began to fail, he was told that no further options were available, and it felt like the end. That was until he was offered a place on a clinical trial at The Christie Hospital in Manchester.

The trial — combining chemotherapy with a new investigational drug — gave John 15 more months of quality life. He continued to enjoy DIY, spent precious time with his family, and even beat COVID twice. The drug eventually stopped working, but Cathy (his wife) remembers how determined John was that his participation would help others, especially children. “He used to say, I am an old fella with this disease, but kids get it as well,” she said. “Ultimately, he was lending his body to hopefully benefit other people.”

Although the last five or six years have brought encouraging progress and new treatment options for AML, some specific genetic mutations—like the TP53 mutation—remain especially challenging. For these high-risk cases, doctors are actively encouraging participation in clinical trials.

Clinical trials are carefully designed research studies that test new treatments, drugs, or approaches to care. They help scientists and doctors find out what works best, and for whom. Participating in a clinical trial could mean gaining access to cutting-edge therapies that aren’t yet widely available, while also contributing to the advancement of medical knowledge that could benefit others in the future. For conditions like TP53-mutated AML, where standard treatments are often less effective, clinical trials offer hope and the potential for better outcomes.

As we mark World AML Day, stories like John’s remind us why continued investment in research and clinical trials is vital. We’ve made huge strides in just a few years and through funding researchers like Dr Samanta Mariani, our most recent John Goldman Fellowship Follow-Up fund recipient, we are making progress to find new ways to tackle AML in all its complexity.  But we’re not there yet. For every patient who doesn’t respond to treatment — for every family still waiting for better options — we will keep pushing for progress.

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References:

(i) – Forsberg M, Konopleva M. AML treatment: conventional chemotherapy and emerging novel agents. Trends Pharmacol Sci. 2024 May;45(5):430-448. doi: 10.1016/j.tips.2024.03.005. Epub 2024 Apr 20. PMID: 38643058.

(ii) – Wysota M, Konopleva M, Mitchell S. Novel Therapeutic Targets in Acute Myeloid Leukemia (AML). Curr Oncol Rep. 2024 Apr;26(4):409-420. doi: 10.1007/s11912-024-01503-y. Epub 2024 Mar 19. PMID: 38502417; PMCID: PMC11021231.