Scientists in the North East have made an exciting breakthrough testing a drug that could be used to treat children with life-threatening leukaemia.
Nearly 40% of youngsters with the illness whose disease returns after treatment could benefit from medication designed to treat colon, skin and lung cancer.
Clinical trials are now planned after experts at Newcastle University successfully used selumetinib in mice to treat leukaemia carrying a common set of genetic faults.
Acute lymphoblastic leukaemia (ALL), the most common childhood cancer, is caused by out-of-control growth of abnormal white blood cells, leading to a breakdown of the immune system.
The Newcastle team collaborated with scientists in Glasgow and Berlin, screening DNA from 206 children diagnosed with leukaemia between 2001 and 2012 whose disease had relapsed after chemotherapy.
The researchers found that the presence of faulty genes linked to ‘ras proteins’ in leukaemia cells was a potential cause of early relapse, resistance to chemotherapy and spread of leukaemia to the central nervous system.
Scientists turned to cancer drug selumetinib, which has been designed for other cancers to inhibit a key protein in the ras pathway.
After successful targeted killing of leukaemia cells in cells isolated in the laboratory, the drug was tested on mice with leukaemia that contained the faulty ras pathway.
Selumetinib produced dramatic reductions in the number of cancer cells in the mice, with limited side-effects.
Dr Julie Irving, who led the research at Newcastle University, said: “It is very exciting to have a set of data that is prompting clinical trials as the next obvious step forward.
“The results provided are very powerful in showing that this drug might be used for a large proportion of children with relapsed acute lymphoblastic leukaemia.
“New drugs are desperately needed for children with ALL who relapse after their chemotherapy. Our findings show that selumetinib could be very effective at treating a substantial proportion of these children.
“The drug actually targets faults specific to the leukaemia cells, limiting damage to healthy cells and debilitating side effects.”
Around nine in 10 children with ALL will now survive their disease long-term, but the outlook for children whose cancer comes back after initial treatment is much poorer.
The findings of the Newcastle study are published in the journal Blood and it is hoped that clinical trials will begin soon, with experts in the region leading the study.
Dr Matt Kaiser, head of research at Leukaemia & Lymphoma Research, said: “This hugely promising research is a great example of the potential of personalised medicine in treating childhood leukaemia.
“Using the latest technologies to screen children who have specific genetic faults driving a relapse, we can start to identify the most effective treatments available.
“And by understanding the biological drivers, we can look beyond traditional disease boundaries for potential cures.” In partnership with North of England Children’s Cancer Research and the region’s Great North Children’s Hospital, Newcastle University has recently launched a campaign to ensure that research like this continues to thrive.
The Future Fund aims to raise £5.5m to create the Newcastle University Centre for Childhood Cancer, a state-of-the-art facility where the city’s academics and clinicians can advance and accelerate the children’s cancer research and treatment.
To give a donation to the Future Fund go to www.futurefund.co.uk , call 0191 208 7250 or text NCFF01 and the amount of your donation to 70070. You can also get involved at https://www.facebook.com/futurefundnewcastle, on Twitter @FutureFundNCL and use the hashtag #NCLFF