Mater helps bring new hope to patients with cystic fibrosis

Friday 19 October 2018

A new triple-combination drug trialled at Mater in Brisbane could increase the life expectancy of patients with cystic fibrosis (CF).

Dr Lucy Burr, Mater’s Director of Cystic Fibrosis and Mater Research Institute-The University of Queensland (MRI-UQ) researcher, said Mater had seven patients enrolled in Phase 2 of the clinical trial, which aims to treat the underlying cause of CF in approximately 90 per cent of CF patients.

“The new drug known as VX-445, in combination with two existing treatments, has demonstrated lung function improvement of around 10 per cent–which is really significant for cystic fibrosis patients,” said Dr Burr.

“Patients in our trial had significant increases in their lung function that they hadn’t been able to achieve previously.”

As co-author of the paper and site principal investigator, Dr Burr said the early phase study showed some really exciting progress. 

“In CF, most patients will die from lung disease, so improving or maintaining their lung function is the mainstay of treatment.”

In Australia, a baby is born every four days with CF. There is currently no cure for the genetic disorder which primarily affects the lungs and digestive system, reducing life expectancy to37.

Dr Burr explained that in CF, a protein called CFTR doesn’t do its job to transport salt and water in and out of cells.

“This affects the pancreas, liver, lungs, sinuses… everything. A lot of our therapies are directed at treating the consequences of CFTR dysfunction: decreased lung function, bowel function, pancreatic problems and really thick, sticky, dehydrated mucus,” said Dr Burr.

“There are two drugs on the market in Australia which actually target the CTFR protein to make it work better in patients with specific gene mutations–giving access to treatment for about 50 per cent of the CF population. This triple combination has the potential to treat 90 per cent of CF patients.”

Dr Burr said this trial used a triple-combination therapy, to target the CFTR protein in patients with a ‘F508del’ gene mutation.

“Two of the drugs help the protein move through the cell, and one of the drugs helps the protein channel open. Adding the extra drug seemed be the key to making the dual combination work much better,” said Dr Burr.

 “What we found in the trial was that the drug improved the protein function, which we measured through sweat chloride in patients, it also significantly improved their lung function and quality of life – and that was only over a four week period.”

Dr Burr said that the development of drugs targeting the CFTR is an exciting step forward for CF care.IMG_E5022.JPG

“When you’re targeting the actual problem there’s great potential for these medications to change, and lengthen, the lives of patients with CF.”

As a large centre for Adult CF patients, Mater collaborated in the international study across 38 sites in the United States, the Netherlands, Belgium, and Australia.

Dr Burr said research is embedded into clinical practice at Mater.

“By participating in pharma trials, we provide patients with access to new, front line drugs to combat cystic fibrosis.”

Mater is currently participating in Phase 3 of the clinical trial.

Dr Lucy Burr’s paper is published in the New England Journal of Medicine

Dr Lucy Burr is a Betty McGrath Fellow, and part of the Respiratory and Sleep Medicine unit, with a specialty in cystic fibrosis.