Doctor looking in child's throat

Murdoch Children’s Research Institute researchers have secured prestigious United States National Institutes of Health (NIH) grants worth a combined $11.8 million to advance research into how children’s immune systems respond to Strep A infection and to trial treatment for rheumatic heart disease.

Strep A is a common bacteria that causes sore throat and school sores (a highly infectious skin condition), predominantly in school-aged children. Left untreated, it can develop into life-threatening infections and rheumatic heart disease which together cause over 500,000 deaths globally each year.

Despite more than 100 years of efforts to develop a vaccine against Strep A, there is still no vaccine available, and only a handful of vaccine candidates have completed a Phase 1 human trial (which tests the safety, side effects, dosing and timing of a new treatment) in recent times.

Professor Andrew Steer, Doctors Hannah Frost, Joshua Osowicki, Melanie Neeland, Nicole Messina and Associate Professors Catherine Satzke and Daniel Pellicci received a $2 million National Institute of Allergy and Infectious Disease grant (1R01AI173567-01to establish tests that could help better understand how the immune system responds to Strep A and support development of a vaccine.  

Led by Murdoch Children’s in collaboration with Karolinska Institute, the project aims to use samples from the world’s only Strep A human challenge model to develop tests that can assess the effectiveness of vaccine candidates on the immune system.

“The World Health Organization (WHO) has highlighted difficulties in Strep A vaccine development, including the lack of standardised tests that measure immune protection,” Dr Osowicki said. “Our goal is to overcome this by using cutting-edge immunology approaches to establish tests that will help us understand how the human immune system recognises and responds to the bacteria.”

Professor Steer and Doctors Daniel Engelman and Anneke Groebler also received a $9.8 million grant from the NIH National Heart, Lung and Blood Institute (1R61HL166441-01to investigate new treatments for rheumatic heart disease, which can result from untreated strep A sore throat and cause irreversible heart valve damage.

Rheumatic heart disease affects around 40 million people worldwide and is common in low-and-middle-income countries, and in Indigenous Australians. Most children with latent rheumatic heart disease (which occurs when patients have no symptoms but have early signs of heart damage typical of the disease) present years too late, missing the opportunity to benefit from antibiotic treatment. Tragically, many die prematurely as a result.

Led by Cincinnati Children’s Hospital and Medical Center in collaboration with Murdoch Children’s and the Uganda Heart Institute, the project aims to recruit over 1,000 school-aged children in Uganda to test the effectiveness of two different penicillin therapies for latent rheumatic heart disease.

Building on the group’s previous work which found that penicillin administered by intramuscular injection significantly reduces disease progression in children, the trial will compare intramuscular penicillin to oral penicillin, which is less painful and less costly to the health system.

Professor Steer said, “The results of our study will inform international policy on the standard of care for children with latent rheumatic heart disease, which could help better treat the disease, saving many thousands of lives.”

The National Institutes of Health are a part of the U.S. Department of Health and Human Services and is the largest biomedical research agency in the world.


This research is funded by a United States National Institutes of Health National Institute of Allergy and Infectious Disease grant and a National Institutes of Health National Heart, Lung and Blood Institute (grant numbers listed above).