AML is one the deadliest forms of blood cancer. An aggressive cancer that can develop and progress quickly, research is vital to improving outcomes and ultimately find a cure.

Dr Hassan is leading a Cancer Institute NSW Fellowship at the University of Sydney to investigate a promising anticancer drug to improve treatment – and survival - for AML.

While the five-year survival rate for leukemia in Australia has improved by more than 20 per cent since 1989 due to new and novel therapies, AML prognosis remains stagnant.

There are currently no effective ways to target leukemic stem cells in AML as they are highly resistant to treatment. This results in high relapse rates and poor survival.

Dr Hassan’s pioneering research zeroes in on a particular gene found in AML's leukemic stem cells that is critical to their survival. By targeting and inhibiting this gene (PAR1), Dr Hassan and her team have discovered a potential pathway to effectively treat AML.

“PAR1 is selectively in leukemic stem cells, so it is a really good target. We found a way to inhibit it and by doing so, we can essentially eradicate the leukemic stem cells and achieve a positive response in patients,” Dr Hassan said.

This PAR1 inhibitor is already an FDA-approved drug used to treat cardiovascular diseases. Dr Hassan and her team are specifically repurposing it for AML. If successful, this could accelerate the process of finding a much-needed treatment strategy and translating it into the clinic.

The potential to transform treatment of AML

Dr Hassan and her team are conducting a comprehensive preclinical evaluation of the PAR1 inhibitor, starting with in vitro work to testing it in vivo models.

They’ve reached a turning point in the project as the research progresses, finding the drug is having a positive effect and increasing overall survival.

“We can potentially rapidly translate this into the clinic because the drug is already FDA-approved,” Dr Hassan said.

“That is definitely a big step and is crucial in terms of finding different treatment strategies for patients who have aggressive blood cancers and limited-to-no options.”

Success of the study, the ability to eradicate the root cause of AML treatment resistance, would mean a breakthrough and provide hope for people with AML and their families.

“It would honestly represent a transformative system to how we treat blood cancer. It would be moving beyond management to durable remission or even a cure,” Dr Hassan said.

“For the research community, it’s also a step towards stem cell targeted therapy.”

For Dr Hassan, achieving this goal is a deeply personal commitment.

“Knowing your work could help transform a terminal diagnosis into a something survivable, being part of that journey to survival care and giving families more time is profoundly meaningful,” Dr Hassan said.

“A family member whose sister had AML once came to me and said how researchers, like me, bring about a lot of hope and not to give up because being in the lab is not easy. When you find something that is worthwhile, it helps us to keep going.”

Dr Hassan published an article about her research in haematology journal Blood in 2024.

Support of the Institute to break the glass ceiling

Funding from the Career Support Grant, worth $593,886, is not only supporting advancements in AML research but also in the way Dr Hassan is forging a successful career in the field. She describes the Fellowship as a game changer.

“It is instrumental, of course, in accelerating this research but also in providing me the resources and more independence to lead a project. It allows me to break that glass ceiling, explore and go beyond,” Dr Hassan said.

“This includes further funding, skill development and the confidence to mentor the next generation of Australian scientists – positioning me for future leadership and discovery as well. It’s not just propelling me in NSW, but also the research field across the globe.”

Dr Hassan has taken her work to international collaborators and audiences, with the Fellowship supporting her to build capacity and establish these vital connections. 

“It takes a village to find a cure. Through this Fellowship, we are finding a treatment strategy for AML, and it helps to forge strategic collaborations with researchers, labs and industry partners around the world.”

Dr Nunki Hassan is a Postdoctoral Research Associate specialising in Leukemia Stem Cell Biology and Cancer at the University of Sydney.

Support for Dr Hassan's work

• 2023 Early Career Fellowship - PAR1 inhibition – developing a novel targeted therapy to eradicate leukaemic stem cells