Dr Charles Mullighan talks about his translational research project into acute lymphoblastic leukaemia

Dr Charles Mullighan is an Australian who found his way to the USA to pursue his passion for research into leukaemia. Charles is leading the way in finding a cure to paediatric leukaemia so we feel the LLS-SF-LF grant he has received is a wonderful investment for blood cancer. To read about his project, his greatest discovery so far and how important your donation is to finding a cure please read the interview below.

Q. Your area of research speciality is paediatric leukaemia specifically acute lymphoblastic leukaemia (ALL) – what led you down this specific path?
A. My clinical training as an adult haematologist in Australia opened my eyes to the dismal outcomes of acute leukaemia. I pursued postdoctoral research at St Jude to gain experience in genetic analysis and experimental modelling of acute leukaemia. Initially, this focus was on both AML and ALL. With the technology available at the time, we made exciting discoveries in ALL and over the last decade have focused my efforts largely on ALL. St Jude has provided a remarkable and unique environment to perform large scale analyses of thousands of leukaemia samples in great detail coupled with state of the art experimental and cutting edge technical approaches. This work includes studying the disease both in children and adults, and this project is relevant to ALL across the age spectrum.

Q. If you had to summarise what your LLS-SF-LF research project is about in 1 sentence what would it be?
A. Developing a new therapeutic approach – protein degradation – against a difficult but important target in high risk leukaemia

Q. What will be the impact of this research project on leukaemia patients?
A. An entirely new treatment approach to treat high risk ALL, if our promising current data prove ultimately successful. Such patients currently lack effective treatment approaches, even in the era of immunotherapy.

Q. What has been your most exciting discovery?
A. Discovering a number of unsuspected genetic changes that drive leukaemia, that transformed our approach to diagnosing, modelling, and treating the disease.

Q. What has been your most frustrating project?
A. Developing genetically engineered mouse models to study a common target of mutation that has been technically challenging – but ultimately overcome. Perseverance!

Q. How important are independent grants funded by Philanthropic donations compared to NHMRC or Pharmaceutical grants
A. Vital. A large proportion of my research funding is from Foundations, which often have an exclusive focus on pediatric cancer research. Such foundations often fund more high risk, innovative research in a timely way.