Acute myeloid leukaemia, or AML, is one of the most dangerous blood cancers. It spreads quickly and is often hard to treat, especially in older patients or those who are too weak for intensive chemotherapy.
But now, researchers from the Yong Loo Lin School of Medicine at the National University of Singapore (NUS Medicine) have published a major review about a new type of drug that could change how AML is treated.
These new drugs are called BH3 mimetics. They are designed to help the body’s own natural system for killing cancer cells—something that AML cells normally block.
In healthy cells, there’s a built-in program called apoptosis that allows damaged or unwanted cells to die.
AML cells avoid this death by using certain proteins, especially from the BCL-2 family, to stay alive. BH3 mimetics stop these pro-survival proteins from working, which helps cancer cells die off naturally.
One of the most successful BH3 mimetics so far is a drug called venetoclax. It targets the BCL-2 protein directly.
Clinical trials have shown that when venetoclax is combined with standard treatments like low-dose chemotherapy, it greatly improves patient survival. In fact, the U.S. Food and Drug Administration (FDA) has already approved this combination for older patients or those not fit for intense chemotherapy.
Assistant Professor Alan Prem Kumar, who co-led the study with Assistant Professor Courtney DiNardo from the University of Texas MD Anderson Cancer Center, explained that venetoclax has completely changed the way AML is treated. It gives patients who once had very few options a new chance at remission.
The team reviewed over 1,000 scientific articles and closely examined 236 high-quality studies. Their findings, published in the journal Nature Reviews Clinical Oncology, explain how BH3 mimetics work, how they can be used more effectively, and how resistance to these drugs may develop.
One of the exciting things about BH3 mimetics is their ability to kill even the most stubborn AML cells, including those that are not actively dividing. However, cancer cells can still adapt. Some switch from using BCL-2 to other survival proteins like MCL-1 or BCL-xL, or develop gene mutations that help them escape treatment.
That’s why researchers believe that future treatments will need to target multiple survival pathways at once. Combining different drugs or using new delivery methods may help prevent resistance and make treatments last longer.
New tools like BH3 profiling and mitochondrial profiling are also helping doctors better understand each patient’s cancer biology. This makes it easier to choose the right drug for each person, rather than using a one-size-fits-all method.
Donavan Jia Jie Tan, a medical student and co-author of the study, explained that by blocking several pathways at once, doctors can reduce the cancer’s ability to fight back.
Dr. Lam Hiu Yan, another co-author, added that these tools now help doctors predict which patients are more likely to benefit from venetoclax, and which may need other drugs targeting different proteins.
The future of AML treatment is moving toward personalised care. Assistant Professor Kumar said that with this approach, patients receive only the treatments that match their cancer’s biology, which can improve results and reduce side effects.
Professor Chng Wee Joo, a leading cancer expert not involved in the study, said that venetoclax has already helped many older AML patients who previously had few options.
The improvements in survival seen today would have been unimaginable just ten years ago. With ongoing research, there is hope that AML could become a more manageable, long-term condition.
New clinical trials are already testing venetoclax with other drugs, such as FLT3 inhibitors and CD47-targeting therapies, to see if more patients can benefit. Other BH3 mimetics targeting different proteins are also in development, made possible by advances in drug technology.
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The study is published in Nature Reviews Clinical Oncology.
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