Review presents a new perspective on treatment response in CML

In line with World CML Day today, SAHMRI’s Blood Cancer Program has published a new review, revealing how lipid metabolism inside leukaemia cells may play a role in treatment response for chronic myeloid leukaemia (CML).

Published in Cancers, the review was led by Dr Ilaria Pagani and PhD student Molly Tolland, in collaboration with Professor Tim Hughes, Professor Deb White and Professor David Ross. 

Researchers examined pre-clinical and clinical studies through a unifying lens of lipid metabolism, reinterpreting existing data and emerging therapeutic strategies, including statins, pioglitazone and hydroxychloroquine, to better understand why some patients respond differently to tyrosine kinase inhibitors (TKIs), the standard treatment for CML.

While the results were mixed, some showed promising improvements in deeper treatment responses, highlighting the need for further research and improved disease modelling.

This year marks the 25th anniversary since the introduction of TKIs, which have transformed CML from being a death sentence, into a manageable chronic illness. Despite this outstanding progress, some patients still fail to respond, or eventually develop resistance, leaving them with limited treatment options.

Dr Pagani and Ms Tolland say the review offers a fresh perspective on where the next breakthroughs may come from.

“By understanding and disrupting this process, we may be able to improve responses to existing drugs and expand the options available for patients who currently face a poor prognosis.” Dr Pagani said.

Many of the drugs mentioned in the review, such as pioglitazone, a diabetes medication, and hydroxychloroquine, originally an antimalarial, are already widely used for other conditions and have well-established safety profiles, opening up possibilities for repurposing.

Further studies will seek to identify potential biomarkers and therapeutic targets that can predict who will benefit most from lipid-targeted approaches and to test these strategies alongside newer generations of TKIs.

Dr Pagani says she wants all people living with CML to have access to the care and treatments they need.

“Equal access to diagnostics, treatment, and specialist care is essential. Our goal is to continue advancing research, repurposing therapies, and supporting patients so that no one is left behind,” Dr Pagani said.