Professor Melissa Little
Murdoch Children’s Research Institute
29 March 2022

Professor Melissa Little leads the Kidney Regeneration Laboratory at the Murdoch Children’s Research Institute where she holds an NHMRC Senior Principal Research Fellowship and CEO of the Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW). She received the 2021 Marshall and Warren Ideas Grant Award for her work generating kidney organoids from human pluripotent stem cells and developing novel engineering approaches to integrate transplanted tissue to the host kidney. 

Research Council chair Professor Steve Wesselingh and Professor Melissa Little on the evening of the NHMRC Research Excellence Awards held in March 2022.

Being recognized with this Marshall and Warren award is a clear signal of the innovation of our research. In the spirit of Marshall and Warren, we are attempting what has been regarded as not possible because the impact will be so significant. End stage kidney disease is increasing in prevalence at 6 per cent per annum, costs in excess of $US25 billion per annum in the US alone and has been identified as one of the major chronic diseases of today. Despite this, only 1 in 4 patients will receive a transplant, mortality on dialysis is 60 per cent across a five-year period and no other treatment options are available. 

As pioneers in the field of creating human kidney tissue from pluripotent stem cells, we have very rapidly made the impossible possible and begun to apply kidney organoids to better understanding of human development and the modelling of genetic kidney disease.  We have also begun the process of showing that we can scale up tissue production using cellular bioprinting or bioreactor culture. The step from this to engineering a functionally useful and transplantable tissue is a really enormous one, but we have identified the many challenges and how they may be addressed. The specific aim of this application is connectivity. If we succeed in showing we can induce connectivity of a transplant to the underlying patient organ, the path to clinical trial is open to us. 

I have worked on the kidney for my entire career, from renal physiology, renal cancer, renal development and renal genetics. It is on the more than 30 years of basic understanding of the cellular and molecular basis of kidney development that these major advances in stem cell-derived kidney engineering are based. A key driver throughout my career has been to ask what my research may be able to do for the patient population. To this end, I have always applied whatever technology is available and let the question guide what research needs to be performed. Ensuring that fundamental research is supported, but also that it does enable medical advancement, is a critical role for the NHMRC and I am incredibly grateful for their support.

Read more about Professor Little's work funded through the Human Frontier Science Program.