Repurposing mebendazole and albendazole as new therapies in paediatric acute myeloid leukaemia

Project title: Repurposing mebendazole and albendazole as new therapies in paediatric acute myeloid leukaemia

Funded by The Little Princess Trust and administered by CCLG
Lead investigator: Prof Ken Mills, Queens University Belfast
Award: £86,458.00
Awarded January 2017

Leukaemia is the most common childhood cancer. The two main types of childhood leukaemia are called acute lymphoblastic leukaemia (ALL) and acute myeloid leukaemia (AML). While ALL is more common than AML, it generally has a favourable outcome. However, the survival rates for childhood AML are much poorer, with only 6 out of 10 children diagnosed expected to survive more than 5 years. This is made more difficult by the severe treatment side effects that children often experience including vomiting, loss of appetite and hair loss. Drug repurposing is the process of using drugs already approved for a certain disease in new diseases. It is a good option because the time from drug discovery to patient is much quicker than traditional drug discovery. Furthermore, as the drugs are already approved, the cost and risk in drug development are also significantly less.

Professor Ken Mills and his team at Queens University Belfast have tested the effect of 760 drugs in leukaemia cells. Of the 760 drugs tested, they found 38 drugs had killed over half of the leukaemia cells. The researchers identified two drugs called mebendazole (MBZ) and albendazole (ABZ) as part of these 14 candidate hits. MBZ and ABZ are used to treat intestinal worm infections, particularly in children. They are used routinely in GP practices all across the UK. From published articles and speaking to GPs directly, it is clear that both MBZ and ABZ are safe drugs with very few side effects, even for patients who have had to take the drugs for up to 10 years. Since this, the team have found in several experiments that MBZ and ABZ are effective against different types of childhood leukaemia, but have little or no effect in normal, healthy cells.

In this project, Professor Ken Mills’ three aims are:

  1. Understand how MBZ and ABZ are working to kill childhood AML cells.
  2. Test the effect of MBZ and ABZ in childhood AML patient cells.
  3. Investigate the effect of MBZ and ABZ to treat model systems with childhood AML