Stem cell derived organoids could one day help treat conditions like kidney disease

A scientist who questioned the value of her work to the human race has been recognised in this year’s Australia Day Honours’ list.

Professor Melissa Little was appointed as Companion of the Order of Australia in recognition of her service to medical research through pioneering contributions to stem cell medicine regenerative therapies for kidney disease, and to stem cell medicine.

One day, she believes, scientists will be able to grow tissues from stem cells and transplant them into patients with conditions like kidney disease to restore their function and health.

“I think stem cells as a field  has the ability to transform medicine,” Little, who heads up the Kidney Regeneration laboratory at the Murdoch Children’s Research Institute, told Cosmos.

“Stem cells were not something that were discussed when I was growing up. Even when I was at university,” says Little.

Little completed a Bachelor of Science in Physiology at the University of Queensland in 1984 and worked as a research assistant in kidney cancer in the very early days of molecular biology. Her subsequent post-doctoral research in the United Kingdom involved working on a gene mutated in children with Wilms’ tumour – a type of kidney cancer.

It wasn’t until about 1998, when the first human pluripotent stem cell was derived from human embryos, that she started to ask the question: “What is my research on kidney development ever going to do that’s of value to anybody?”

Read more: Australian research brings scientists closer to making blood stem cells in the lab.

This is when she started to look at the potential of stem cells, in recent years at pluripotent stem cells which can give rise to any tissue in the body.

Little’s personal research is in rebuilding the human kidney using pluripotent stem cells.  Her team are pioneers in the field having produced the world’s first kidney organoid in 2015 – a kidney in a dish, essentially.

Little explains that in the space of about three weeks they were able to induce pluripotent stem cells to form into a micro tissue with nephrons – the filtering units in the kidney – with the appropriate surrounding cell types and blood vessels.

Professor melissa little, stem cell expert
Professor Melissa Little. Credit: MCRI

“This is obviously still a model of human foetal tissue, but it’s quite remarkable,” Little says.

“It’s remarkable that we can mimic that self-organising process that happens in one tiny area of the embryo in a laboratory dish and use this to model human disease.”

Having relocated her work to the Murdoch Childrens Research Institute in 2015, this has placed her on the campus of the Royal Children’s Hospital in Melbourne, where her team has since then been able to use their kidney organoids to model inherited forms of kidney disease.

“One in 15,000 kids born has an inherited form of kidney disease,” explains Little.

“And we are looking at how we do more than just create an accurate model of those diseases, but also move to the point of screening drugs to find treatments, which don’t currently exist.”

“These children may die or go into renal failure, which requires either transplantation or dialysis. The treatment options are the same as in an adult. There are no other options for renal failure patients, nor has there been for more than 70 years,” she says.

Read more: Kidney disease affects women more than men.

With organoids, instead of testing the efficacy of drugs on completely irrelevant cell lines or on animal models that don’t have the same condition, drugs could be tested on a human model of kidney disease using organoids.

Little is an internationally recognised expert in stem cells and has played a large role nationally in promoting stem cells as a research field. She was Chief Scientific Officer of the Australian Stem Cell Centre, Program Leader of Stem Cells Australia, President of the Australasian Society for Stem Cell Research, and most recently, the President of the International Society for Stem Cell Research.

“I’m very passionate about not just doing the science, which I love, but making a connection between a great idea and a product at the end.”

“We need to actually provide an environment where scientists can translate their research into outcomes,” says Little.

Little is currently the Chief Executive Officer of the Novo Nordisk Foundation Centre for Stem Cell Medicine – reNEW – at the University of Copenhagen. reNEW is an international centre with researchers in Copenhagen, Denmark, in Leiden in the Netherlands, and at the MCRI in Melbourne, Australia. reNEW aims to develop new therapies based upon stem cell research excellence. Little has an active kidney research laboratory at MCRI but is establishing more kidney research in Denmark.

Little is also a passionate advocate for a greater role and recognition for women in science. She’s acutely aware that fewer women than men reach the most senior levels of their profession.

“I am a scientist and a Mum. My husband and Iraised two kidsand we also have a granddaughter,” she says.

“I think science is a fantastic career for women. I’m not saying there aren’t barriers, and there’s barriers for women in almost every career, particularly if they choose to have children, but science gives you enormous flexibility in terms of time management.

“I’ve loved being a scientist and a mother and I would never choose one or the other.”

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