Innovations - Biotech

A new drug screening approach identifies children at risk of developing heart problems during chemotherapy

What's the child health challenge?

Children undergoing life-saving cancer treatments are at risk of developing heart problems due to side effects from chemotherapy. Cardiotoxic chemotherapy is the leading cause of premature death and health complications in children, adolescents and adults who have had cancer.

Anthracycline-based chemotherapies treat over 70 per cent of childhood cancers and have significantly improved cancer survival rates. However, anthracyclines can cause heart damage and dysfunction. This risk can carry on into adulthood, impacting those who had childhood cancer well after it has been treated.

With the lack of child-specific treatments available and clinicians’ dependence on anthracycline-based chemotherapies, there is a clear need to develop drugs that minimise the effects of this treatment. The ideal compound would not influence treatment efficacy, but instead, act to protect the heart from lasting damage. Without this, children with cancer will continue to be at risk of heart problems until a solution is found.

What's the discovery or innovation?

Researchers at MCRI used heart muscle cells produced from stem cells in a dish to screen a library of small molecules. This helped them identify compounds that can help to protect young hearts from anthracycline-induced damage, thereby safeguarding children against the long-term side effects of cancer treatment.

These findings will enable MCRI's researchers to develop a portfolio of compounds that can be further developed into clinically valuable drugs. The major outcome of this research will be a strong dataset that includes important information on several lead compounds. This will give us a secure foundation for the development of new therapeutic products to prevent heart disease in children undergoing chemotherapy.

How did the MCRI Innovation team contribute?

Our Innovation team is supporting MCRI's ongoing commitment to precision child health by ensuring children benefit from faster diagnosis and treatment plans tailored to their unique healthcare needs.

Working alongside MCRI Associate Professor David Elliot and Professor Enzo Porrello, the Innovation team helped to ensure the drug screening process and relevant research were possible. We also assisted with the development of commercialisation plans and protection of MCRI's Intellectual Property (IP) through patents.

How is it changing children’s lives?

The research undertaken at MCRI will be instrumental in helping clinicians identify the children who are most at risk of anthracycline-based chemotherapy heart damage.

This will give children the best chance of living a healthy lifestyle post their completion of cancer treatment. It will also minimise the likelihood of adverse effects from this vital treatment for the years to come.

Innovative immunotherapy may help children with allergies eat peanuts freely

What's the child health challenge?

Avoiding allergens can be difficult for children and can result in accidental allergic reactions. Although allergic reactions can be mild for some children, life-threatening or fatal anaphylaxis may occur in others.

The unpredictability of reactions and the caution required to avoid an allergen can cause psychological distress and severely reduce the quality of life for children and their families.

Worldwide, peanut allergy is one of the leading causes of death in people with food allergy. Food allergies are one of the most common chronic conditions in children from Western countries.

Nearly 3 per cent of Australian children under the age of one have a peanut allergy and this number is increasing at the fastest rate of any other food allergy. There is an urgent need to help children consume peanuts safely.

What's the discovery or innovation?

MCRI has developed a new oral immunotherapy approach that induces remission of peanut allergy. This will allow children to incorporate peanut and peanut products into their regular diet and may lead to a substantially improved quality of life for many children.

ProtaTherapeutics, a biotech startup company, has partnered with MCRI to develop and commercialise this innovative immunotherapy.

In the future, this approach may also help to treat other common food allergies affecting children, including allergies to other nuts, shellfish, egg and milk.

How did the MCRI Innovation team contribute?

Our Innovation team assisted MCRI Professor Mimi Tang and the Allergy Immunology group to identify the value of Professor Tang’s discovery and protect her invention. The Innovation team filed international patents in order to protect MCRI's intellectual property and newly developed technology.

We also supported Professor Tang in the development and progression of the therapy. By working together to study the effects of the therapy, MCRI aims to positively impact the lives of children across the globe. 

How is it changing children’s lives?

For decades, MCRI has been helping lessen the burden for children and families living with food allergies. Prota’s therapy will enable children with peanut allergy to consume peanuts freely without fear or anxiety. Most importantly, this will allow them to enjoy a greatly improved quality of life.

Initial studies have shown that around half to three-quarters of children who have participated in our oral immunotherapy trials have achieved remission after 18 months of treatment. Of these initial treatment responders, 60 to 70 per cent remain in remission three to four years after treatment.

The aim is that children who receive Prota’s treatment will no longer need to avoid peanuts. This will reduce their risk of anaphylaxis and give them the opportunity to live a happier, allergy-free life.

RV3-BB vaccine helps save the lives of children across the globe

What's the child health challenge?

Currently, 77 million, or 41 per cent, of children globally are unable to access the life-saving rotavirus vaccine.

Rotavirus is a global pathogen that causes children to experience diarrhoea, tummy aches, fever and/or vomiting. Some children with severe rotavirus infections may also experience dangerous levels of dehydration and shock and in some instances, it can be fatal. 

Mostly infecting infants between the ages of six months and two years old, rotavirus is responsible for around 215,000 child deaths per year. Despite being vaccine-preventable, rotavirus is one of the leading causes of child illness and death in the developing world. 

What's the discovery or innovation?

MCRI has led the global effort to introduce rotavirus vaccines to children all over the world by improving access in countries that need it most.

Until the discovery of rotavirus in 1973 by Professor Ruth Bishop and her team, more than 800,000 children globally were dying from this acute form of diarrhoea.

In 1999, research conducted at MCRI inspired the development of four oral rotavirus vaccines that have since helped saved the lives of countless children in more than 114 countries. The WHO Global Vaccine Action Plan shows that from 2011-2020, up to 900,000 deaths were prevented through the development of the rotavirus vaccine. 

Our leading researchers have since developed a new oral vaccine, RV3-BB, that has several advantages for developing countries. The new vaccine has been trialled in Africa and has shown that a reduced dose can also produce a strong immune response in newborns. 

This significant scientific achievement is the culmination of four decades of work and has been created to help ensure all children across the globe can live healthy and fulfilled lives.  

The RV3-BB vaccine is now being trialled in a birth dose strategy which is hoped to improve protection in babies and children from severe rotavirus disease.  

How did the MCRI Innovation team contribute?

Supporting Professor Julie Bines, the MCRI Innovation team helped researchers secure manufacturing partnerships in India and China, where the greatest impact would be achieved.

These partnerships have improved access to the vaccine in some of the world’s largest and most vulnerable populations, where the death rate linked to rotavirus is still high. The Innovation team has taken the vaccine profile and picked the best development strategy for India and China. 

We are now seeking additional manufacturers from across the globe to develop and commercialise the RV3-BB vaccine. These efforts will help make the vaccine more widely available and ensure more children can benefit from its life-changing effects.

How is it changing children’s lives?

The development of oral rotavirus vaccines is a great breakthrough in Australian science, one that has had a profound and far-reaching impact on the lives of children everywhere.

By 2013, the number of deaths had halved while in Australia, hospital admissions dropped by over 70 per cent, down from approximately 10,000 admissions of children under five every year.  

Today, rotavirus vaccines are helping to save the lives of children in 114 countries. 

Whilst still under development, researchers at MCRI hope the new RV3-BB vaccine will be licensed for use in the coming years, joining existing vaccines already preventing rotavirus in children.

It is our aim to ensure that all children can access this life-saving vaccine.