Using existing drugs in a new way to treat high-risk acute lymphoblastic leukaemia

Project title: Novel drug treatments through repurposing of FDA-approved drugs for improved treatment of high-risk acute lymphoblastic leukaemia 

Funded by The Little Princess Trust and administered by CCLG
Lead investigator: Professor Christine Harrison, Newcastle University 
Award: £188,962.00 
Awarded March 2022 

Chromosomes contain part or all of a person’s DNA and problems with them, such as extra copies or issues with their structure, can cause a wide range of problems. In childhood cancer, chromosome 21 can often have problems and could therefore make a good target for new drugs to treat a wide range of cancers. The research team at the Newcastle University Centre for Cancer, led by Dr Christine Harrison, have already found a high-risk subgroup of children with acute lymphoblastic leukaemia (ALL) where there are lots of problems with chromosome 21. This group doesn’t respond well to normal chemotherapy. In a previous project, they showed that increasing the levels of treatment made a big improvement in these children’s survival. However, this comes with a cost of serious long-term side effects, showing that we need safer and more effective treatments.  

Dr Christine Harrison’s team have shown that certain genes on chromosome 21 are expressed too much in all patients in the high-risk subgroup, and that these genes feature in other types of leukaemia where there are too many copies of chromosome 21. Therefore, this group would be useful to help find new treatment strategies that could treat a wide range of cancer patients. The researchers have sequenced the genome of lots of patients from the high-risk subgroup and also patients with other types of leukaemia. Using these data, they have found which genes are responsible for driving this type of high-risk ALL. Next, they will try to find drugs that can target these genes.  

In order to increase the probability of finding a drug that is already licenced for use, the researchers will also screen FDA-approved drugs. This would mean that the new treatment could help patients sooner. The researchers are working with a team who have a way of quickly testing drugs to see which could kill leukaemia cells. Dr Christine Harrison hopes to find drugs that are already licensed that can quickly start to be used against at least one type of high-risk childhood leukaemia. The team will also be looking for any promising new drugs that could be developed in the future.