Grants secured to advance research into childhood conditions

Ten Murdoch Children's Research Institute (MCRI) scientists have received federal funding to advance research into childhood conditions including leukemia, food allergy, kidney disease, epilepsy, obesity, reproductive disorders, sex development disorders and COVID-19.  

Professors Andrew Elefanty, Andrew Sinclair, Melissa Little and Mimi Tang, and Doctors Elizabeth Ng, Katie Ayers, Nicole Messina, Peter Houweling, Sara Howden, Simranpreet Kaur received National Health and Medical Research Council (NHMRC) Ideas Grants, announced by Federal Health Minister Greg Hunt.  

Professor Sinclair's Disorders of Sex Development (DSD) project aims to identify enhancers of genes that control testis or ovary development. "We will determine whether disruption of these enhancers leads to a baby being born with a DSD to improve diagnosis and clinical management of these patients," he said.

Dr Ayers has helped create a human embryonic testis petri dish model from stem cells, which she will use to study genetic and environmental causes of reproductive disorders. She said her findings would empower families and doctors to choose the right clinical care for their children, improving their health and wellbeing.

Dr Kaur's project aims to accelerate treatment for epilepsy in children with a specific genetic mutation that causes patients to die within the first five years of life. "We will use innovative models such as stem cells and algorithms to identify new therapies for these life-threatening epilepsies," she said. 

Dr Houweling is investigating the loss of a key gene in modern evolution that improves muscle function and metabolism, and how it might negatively affect people today. Dr Houweling said he hoped his research would help better treat weight gain and improve outcomes for obese adults and children.

Dr Howden has developed a method to recreate human kidney tissue from stem cells to better understand a type of kidney disease that causes kidney and liver failure in babies and children. "We will use patient blood samples to make a model of their disease in the lab, which will hopefully lead to new, personalised treatments for patients with this severe kidney disease," she said.

Professor Little's project aims to engineer replacement kidney tissue with the use of stem cells to treat chronic kidney disease. Professor Little said this technology had the potential to revolutionise kidney disease treatment and would bring us a step closer to creating stem-cell derived replacement kidneys.

Dr Ng and Professor Elefanty's projects aim to make blood stem cells in a petri dish to improve therapies for patients with inherited blood diseases including leukemia and bone marrow failure. 

"Recreating a human bone marrow organ from stem cells in the lab would improve our ability to make blood stem cells for patients with leukaemia or bone marrow failure who cannot find a matched donor," Dr Ng said. Professor Elefanty said success in his project, which was also aimed at patients who lacked a donor, would accelerate the translation of these human stem cell derived blood therapies to clinical settings.

Professor Mimi Tang's project aims to find a long-term treatment for food allergies to help improve quality of life and prevent death. "By better understanding the underlying immune changes that lead to remission we hope to develop effective long-term treatments for food allergy, possibly offering a cure," she said.

Dr Messina's project aims to harness the beneficial effects of a tuberculosis vaccine to boost protection against COVID-19. She said her project would investigate how BCG boosts immune responses to COVID-19 and if it improved protection and boosted existing COVID vaccines. 

Dr Messina's study will use samples from the MCRI BRACE trial, the world's largest trial on the off-target effects of BCG vaccine, which includes almost 7000 healthcare workers across Australia, Brazil, Spain, the Netherlands and the United Kingdom. 

The NHMRC Ideas Grant scheme supports innovative research projects addressing specific health and medical questions and provides particular opportunities for early and mid-career researchers.

Each Ideas Grant team will receive funding for up to five years.