Targeting the NAD pathway as a new therapeutic strategy for high-risk leukaemia in children

Targeting the NAD pathway as a new therapeutic strategy for high-risk leukaemia in children

Recipient: Dr Michelle Henderson
Institute: University of NSW
Funding: $138,179 July 2019 to June 2022

Rapidly dividing cancer cells can be dependent on a cellular biochemical called nicotinamide adenine dinucleotide (NAD) as an energy source.

While survival rates for paediatric acute lymphoblastic leukaemia (ALL) have improved dramatically over recent decades, the outlook for certain subgroups remains dismal. These include children where treatment fails, those who relapse during treatment, or whose leukaemia harbours genetic abnormalities – something that commonly occurs in infant ALL and is referred to as MLL-R leukaemia.


Behind the science: Dr Michelle Henderson


New, more specific therapeutic agents, tailored to patient needs are urgently needed.
 
Together with colleagues in the United States, the Henderson lab discovered and developed OT-82, a new drug that blocks the production of NAD and has remarkably strong anti-leukaemia activity, even against the most aggressive subtypes of leukaemia.
 
Over three years, this research project aims to further develop this drug so it then can be tested as a viable therapeutic strategy for aggressive leukaemia in children, to ultimately improve their survival and minimise the side effects of treatment.

This project recieved further support through Cancer Australia's Priority-driven Collaborative Cancer Research Scheme.

Children’s Cancer Institute Australia is affiliated with UNSW Sydney and The Sydney Children’s Hospitals Network.