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Giving cystic fibrosis patients longer, better lives

March 11, 2022

Dr Gerard Kaiko has developed a new molecule that could help cystic fibrosis patients live longer and with a better quality of life. 

With the generous support of donors, the Foundation wants to help fund Gerard’s research and bring this life-changing treatment to clinical trials.

A baby is born with cystic fibrosis every four days on average in Australia. The inherited genetic disease causes severe damage to the lungs, digestive system and other organs.

When cystic fibrosis was first recognised 80 years ago, most babies with the condition died before their first birthday.

Life expectancy is about 38 years – less than half that of the general population – and Gerard wants to extend it further.

“Existing drugs don’t restore enough of the function of the defective gene to give patients the same life expectancy as the general population,” Gerard says.

“There’s no reason why, with the advent of targeted therapies, like those we’re working on, that we can’t make greater strides.”

Gerard and his colleagues at Hunter Medical Research Institute and the University of Newcastle were selected for the Foundation’s 2021 Matched Funding Program for their project using personalised medicine for the treatment of cystic fibrosis.

Under the Program, the Foundation will match, dollar-for-dollar, donations made by other supporters to provide a total of $100,000.

A generous donation from Whiteley Corporation, an Australian manufacturing company specialising in cleaning and infection control and prevention products, has brought the funds raised to $70,000.

We’re looking for donors to cover another $15,000, which, matched by the Foundation, will meet the funding target.

The project uses stem cells in two ways. The drug molecule is made using living stem cell ‘biofactories’; it can’t be made synthetically through chemical reactions in a traditional manufacturing lab.

“Stem cells allow us to make the large quantities we will need for treatments in the clinic.”

The project also uses stem cell-derived tissues from individual patients to test how different people might respond to the drug.

Gerard says that personalised treatment is particularly important in cystic fibrosis. Although only one gene is involved, there are about 2000 different mutations. Not everyone has the same prognosis or the same symptoms.

A biopsy from a patient’s airways or colon provides cells that are grown into ‘mini-lung’ or ‘mini-gut’ tissues that allow Gerard’s team to test the stem cell ‘biofactory’ drug candidate in living cells with the patient’s unique genetics.

“With this disease, you see some really sick kids. You can also see patients who don’t go downhill until later in life. Being able to study how treatments work in a wide range of patient-derived tissues from the actual patients in the clinic with different mutations will mean we can get to clinical trials much faster.”

If you want to make a donation to support Gerard’s research, contact Graeme Mehegan via [email protected].

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