Aussie researchers' ‘magic bullet’ gives new hope to children who relapse from blood cancer

Cancer scientists at the prestigious Children’s Cancer Institute Australia (CCIA) have discovered that an existing drug being trialled for adult cancer treatment also has the potential to treat children who relapse from acute lymphoblastic leukaemia (ALL), one of the most common types of paediatric blood cancers.

The current survival rate for children diagnosed with the ALL is 80 per cent, however if a child relapses this survival rate decreases to just 20 per cent.

“What is so exciting about this drug is it has the potential to not only improve the survival rate of children who have relapsed but, since it acts as a ‘magic bullet’, it only targets the cancer cells, leaving the healthy cells untouched!” explains Professor Lock, Head of the Leukaemia Biology Program at CCIA.

Dr David Ziegler, paediatric oncologist with the Sydney Children’s Hospital, said “Our patients and their parents can’t afford to wait years to have new treatments developed. We plan to start a clinical trial of this new therapy for children with leukaemia by the end of this year.”

Researchers are calling the therapy a ‘magic bullet’ because it works by targeting a protein which is present on ALL cells but not on the healthy cells which help to replenish blood cells. It therefore delivers the treatment directly to the cancer, minimising the harmful side-effects of treatment.

The drug, called SAR3419, has already been in clinical trials for the treatment of adult cancer. However, in experiments using a laboratory model of paediatric ALL, researchers found this same drug, when it is combined with a three-drug treatment of standard therapies, has the potential to prevent children from relapsing from their cancer.

Professor Lock, said, “The combination we used specifically targeted the leukaemia cells when tested on aggressive or drug-resistant acute lymphoblastic leukaemia. It significantly delayed disease dissemination and progression into the major organs and prevented leukaemia relapse in those organs.

The Sydney Children’s Hospital at Randwick is currently working with CCIA to fast-track a clinical trial for this new drug, ensuring this discovery can be safely made available to the children with ALL who are in need more effective treatments.

Please click here to visit the CCIA press release, for more information.