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CHILDREN'S MEDICAL RESEARCH INSTITUTE

We are incredibly excited to announce the creation of a new postdoctoral researcher position, made possible by the Team Simon Foundation, to work on gene therapy for Cystic Fibrosis. The Team Simon Foundation for Cystic Fibrosis alongside CMRI are committed in helping change the lives of children like our son Simon and future generations.

It all starts with BELIEF!

FAMILY FOUNDATION FUND NEW RESEARCH

Children’s Medical Research Institute (CMRI) is excited to announce the founding of a new Postdoctoral Researcher position, focused on Cystic Fibrosis, thanks to the generosity of the Team Simon Foundation which has a very personal connection to the cause.

The Team Simon Foundation is the brain child of Teresa and Harry Bazouni who want to see treatments and cures developed for kids with CF, a condition affecting their son Simon and many thousands of children worldwide.

In May 2014, Simon was born seemingly healthy before things rapidly changed. Over the following months, Teresa and Harry watched him struggle to breathe before he was finally diagnosed with CF, and the whole family’s life changed forever.

“It was a big shock – a devastating day”, Teresa said. “It was overwhelming.” The daily routine for a child with CF involves physiotherapy, using an inhaler to clear out their chest, and 25 pills a day. “A lot of people don’t know what a genetic disease is,” Harry said. “Simon’s condition is invisible. From the outside he looks all nice and healthy, but everything is happening on the inside.”

His family was determined to change the future for Simon and other children with CF, so the founded the Team Simon Foundation. Connecting wih CMRI, through Jeans for Genes™, the family learnt about the gene therapy revolution. Instead of daily treatments for genetic diseases, gene therapy aims to treat, or even cure, a patient through one single therapeutic administration.

Head of CMRI’s Translational Vectorology Unit, Associate Professor Leszek Lisowski and his team have been researching ways to use gene therapy to treat CF. His team creates microscopic delivery vehicles, called vectors, which make gene therapies possible. The vectors deliver gene therapeutics into the patient’s cells affected by the disease. The therapy adds a working copy of the faulty gene or corrects the faulty gene with a functional version.

“Cystic Fibrosis impacts the lungs, so in order for us to deliver a gene therapy, we have to go directly into the lungs. This is especially difficult int the case of CF, where the airways are covered with a thick mucus that is impenetrable to therapeutic vectors.” Associate Professor Lisowski said.

“We are thus working to develop novel bioengineered viral vectors that would enable safe and effective delivery of gene therapeutics to the affected lung cells via the blood stream. If successful, this new technology could be life changing for patients with Cystic Fibrosis but also other lung disorders. As an independent medical research institute, we aren’t guaranteed government funding so we are extremely grateful to the Team Simon Foundation for investing in our work, which we hope will benefit current patients as well as future generation of Children.”

The Team Simon Foundation’s pledge of $464,000 will fully fund the postdoctoral position over three years. Teresa and Harry said they wanted to create a legacy for others in their situation. “We have toured the labs, we have met the researchers and we have great faith in the potential for them to make a real difference in the lives of children living with CF”

JOINING FORCES FOR CF

Gene therapy researchers at Children’s Medical Research Institute (CMRI) are developing a new way to target the lungs more effectively, with the aim of improving treatment of cystic fibrosis.

Associate Professor Leszek Lisowski and researchers Dr Andrea Perez-Iturralde, Maddy Knight, and Kim Dilworth were excited to show their progress during a recent lab tour with the Cure4 Cystic Fibrosis Foundation and the Team Simon Foundation, who have generously supported this research.

It’s rare for us to have our researchers in the same room as patients, parents, clinicians and the foundations funding our work. But on this special occasion we were joined by Olivia, an ambassador for Cure4CF Foundation who is living with CF, as well as Harry and Teresa Bazouni, co-founders of the Team Simon Foundation whose son Simon has CF, as well as Professor Hiran Selvadurai from the Children’s Hospital at Westmead who is collaborating with A/Prof Lisowski on this project. All were excited to be in attendance to hear about CMRI’s progress toward a gene therapy for cystic fibrosis patients.

“I had a fantastic time connecting with our research partners at CMRI,” said Cure4CF CEO, Suzy Dimaline. “A/Prof Lisowski and his team are making significant strides in addressing cystic fibrosis at its core. Touring their labs allowed me to witness their important work first hand.”

Andrea explained that one of their key aims is “the development of a vector that is able to reach the lungs, because there is no specific vector that goes to the lung.” Vectors are microscopic delivery vehicles that can carry a healthy copy of a gene to replace a malfunctioning gene, an approach already successfully used to cure other genetic diseases. 

The team explained the complex process of designing new vectors and were excited to share they had narrowed the search down from an initial 64 million vector options, to a small subset that appears to successfully target the lungs.

Maddy explained: “I found these peptides,” which act as the address label on the vectors, “that had been previously published. I inserted them into these three promising vectors, making a total of 18 new variations. Of those 18, there was one peptide that was more successful at reaching the lung by about 7-20%. But more interesting is that this vector, which tends to go towards the liver, was significantly de-targeted from the liver which is very exciting.”

“This is very early, and we need to do many other experiments, but it is something that is already very exciting. Now we try to build on this success to make the vectors even better.”

Everyone had the opportunity to witness Maddy’s discovery in the lab, seeing tissue culture cells that were targeted with one of the new vectors, which turned them green with fluorescent reporter protein!

CMRI received the major Holckner Family CF Impact Grant from Cure4 Cystic Fibrosis Foundation in 2023. It is the second major grant to CMRI researchers from Cure4 Cystic Fibrosis Foundation, thanks to fundraising efforts of the Team Simon Foundation who have a long history with CMRI.

“It was an absolute pleasure meeting the team at CMRI alongside our partners from CURE4CF,” said Teresa Bazouni. “This opportunity wouldn’t have been possible without the support from our generous sponsors and donors. We’re grateful to be a part of life-changing moments.”

“It’s inspiring to realise that this is where transformative advancements take place,” said Suzy. “I feel honoured to be in the company of such talented individuals dedicated to creating a future free from cystic fibrosis.”

See the article here: https://www.cmrijeansforgenes.org.au/news/families-and-experts-join-forces-to-fight-cystic-fibrosis

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Team Simon Foundation for Cystic Fibrosis Ltd

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