Glasgow woman who had cancer four times welcomes development
A drug commonly used to treat parasitic worm infections could offer a new way to tackle one of the most difficult childhood blood cancers to treat, research funded by Leukaemia UK has identified.
Dr Noelia Che and her colleagues at UCL GOS Institute of Child Health, investigated whether the drug mebendazole, could be repurposed and used in combination therapies to treat childhood acute myeloid leukaemia (AML).
While survival rates for childhood leukaemia have improved dramatically in recent decades, AML remains one of the most challenging forms of the disease.
Established treatments such as chemotherapy and stem-cell transplants, while effective, are intensive and can often lead to relapse and serious long-term side effects for patients.
This was the case for Nichola Smith, whose diagnosis of AML at aged 12 led to decades of difficult treatment, relapse and serious life-long consequences. “I was constantly tired and run down,” recalled Nichola, now 44, from Glasgow.
“Then one day after playfighting with my cousins I noticed an enormous hand-shaped bruise where one of them had grabbed me. Then the next day I collapsed and my mum took me to Yorkhill Children’s Hospital where, to my shock, I was diagnosed with leukaemia”.
Nichola underwent intensive chemotherapy. After relapsing a year later months later, she also required a stem cell transplant and more chemotherapy.
While her treatment was successful, the impact on Nichola has been life-long. In 2006, a routine mammogram revealed pre-cancerous cells in both her breasts, a recognised long-term effect of the leukaemia treatment she had received as a child. She underwent a double mastectomy and several major operations.
Then, in 2022, Nichola was diagnosed with myelodysplastic syndrome (MDS), a blood cancer that can develop into AML.
“I was devastated,” she said. “I kept saying to myself, ‘Here we go again’ and ‘Why me?’ I couldn’t believe I was going to have to fight another blood cancer after everything I’d already been through.”
Her condition later progressed and she once again required chemotherapy, radiotherapy and a stem cell transplant.
Experiences like Nichola’s are driving researchers to find more targeted and less toxic ways of treating AML. Reducing the devastating long-term effects.
Dr Che, a recipient of the Leukaemia UK John Goldman Fellowship, is focusing her research on a protein called MYB, which AML cells rely on to survive and grow.
She and her colleagues found that mebendazole can reduce levels of MYB within leukaemia cells, depriving them of a key survival mechanism and causing them to die.
She hopes the approach could form part of a new generation of targeted therapies. Unlike chemotherapy, which attacks all rapidly dividing cells, targeted therapies attack cancer cells more precisely while reducing damage to healthy tissue.
Dr Che and her colleagues are now exploring further how mebendazole can be used with newer targeted therapies rather than chemotherapy, identifying a promising treatment strategy for children with AML. Potentially avoiding the increased toxicity associated with more intensive chemotherapy.
Dr Noelia Che said: “Our research is exploring whether mebendazole, a safe and well-established drug, could be used in a completely different way to target vulnerabilities within AML cells.
“We hope this work will contribute to gentler, more effective treatments, so that children survive without life altering complications.”
Simon Ridley, Director of Research and Advocacy, Leukaemia UK, said: “We’re proud to support innovative researchers such as Dr Che who are exploring bold new approaches to treatment.
“It is important to note that this research is still in early stages, but it shows one of the many routes that researchers are working to fight leukaemia through new strategies, repurposed medications and innovative combinations.
“Which, in the future, could reduce the long-term impact of treatment on patients and their families.”
