Nucleotide Prodrugs as Treatment for Acute Myeloid Leukaemia (AML)

Acute myeloid leukaemia (AML) is a complex and aggressive blood cancer caused by the uncontrolled growth of abnormal blood-forming cells. In the UK, around 3,100 people are diagnosed with AML each year, most commonly in older adults (>60 years old). Despite available treatments, AML remains a deadly disease, responsible for approximately 2,700 deaths annually in the UK. Among the reasons for such high mortality is the lack of response to treatment, especially the drug Azacitidine, in many AML patients.

The lack of response to Azacitidine is a major issue because this drug has been transformative for some AML patients who cannot undergo intensive therapies like stem cell transplants or strong chemotherapy. It is also an issue for elderly patients, because Azacitidine has fewer side effects than many other cancer drugs so offers a safer treatment option. Given that about half of AML patients eventually stop responding to Azacitidine, these patients are left with limited treatment options, which contribute to poor survival.

To address this, our research focuses on developing new forms of Azacitidine that are designed to remain effective even in patients whose cancer no longer responds to the original drug. These newly developed drugs will aim to provide better and more durable treatment responses for vulnerable AML patients and help to improve survival rates.