Friday , October 18 2019
Home / australia / Cystic fibrosis drug trialled in Queensland could add decades to the lives of sufferers

Cystic fibrosis drug trialled in Queensland could add decades to the lives of sufferers



Updated

September 29, 2019 07:46:43

Cystic fibrosis sufferer Darren Bullock has never been able to breathe easy.

Key Points:

  • Prince Charles hospital in Brisbane trialling new cystic fibrosis drug
  • Drug costs $ 250,000 per patient each year, but there is hope it will be part of the PBS
  • It makes the faulty cystic fibrosis gene work effectively, reducing mucous on the lungs

His lungs run at 40 per cent capacity, he cesses incessantly, battles constant lung infections and has been hospitalized twice for collapsed lungs.

The 44-year-old is pinning his hopes of survival on a breakthrough drug being trialled by Queensland scientists.

The pills, taken three times each day, are the first to treat the faulty gene that causes cystic fibrosis.

International trials have shown so far that it tricks the "druggable" gene, and therefore the body's exocrine system, into working effectively and reducing excessive mucous that gathers on sufferers' lungs.

It could add decades to Mr Bullock's life.

"It would be life changing, give me a second chance at life," Mr Bullock said.

His dream is to see his 11-year-old twins Alex and Abigail grow up.

He cries when he realizes this could now be a possibility.

"To see my daughter get married, to see my son have his first girlfriend, go on a date, graduate from university," he said.

"To be there for my wife in old age and enjoy those later years. That would be everything really."

Brisbane's Prince Charles Hospital is trialling the drug, which was first developed in the United States.

It comes on the back of three international trials, with a blueprint for ongoing care for patients being unveiled at the European Respiratory Society Congress in Spain today.

The Prince Charles experiment has only been underway for weeks, but Because the drug works within days, wards that were once overflowing now have free beds.

The hospital's Professor Scott Bell, a world-renowned Brisbane cystic fibrosis researcher, is also part of a global team that has been trialling the drug.

He said the trial had shown that the cystic fibrosis transmembrane conductance regulator (CFTR) gene is druggable for the first time.

It does not correct the mutated gene at the DNA level, but manipulates it into working better in what Professor Bell describes as the most significant breakthrough since the condition was discovered in the 1930s.

"Patients report major improvements in quality of life, a massive increase in lung function and fewer infections," he said.

"Much better than any of the system based treatments we've had so far.

"They are really having a major impact on the small number of patients being able to access the drugs."

Treatment costs $ 250,000 per year

Professor Bell said the biggest challenge now was making therapy affordable with one patient for more than $ 250,000 a year.

Patients would need to take the tablets for the rest of their lives.

"They are very expensive to develop," Professor Bell said.

"It takes about 10 to 30 years to get a drug from discovery to a patient in the clinic."

The therapy is currently being evaluated by the US Food and Drug Administration.

Professor Bell hoped the drug would then be approved by Australia's Therapeutic Goods Administration and funded by the Pharmaceutical Benefits Scheme within two years.

Associate Professor David Reid, who runs the Prince Charles Hospital Adult Cystic Fibrosis Center as well as QIMR's Lung Infection and Infection Laboratory, said it was an exciting time for the world's 90,000 sufferers.

About 3,400 Australians have been born with the disease.

In the 1960s the life expectancy of a child with cystic fibrosis was just 10, now people can survive into their 50s.

That is expected to improve further if, or when, the drug goes to market.

"When I was a medical student, my only contact with cystic fibrosis was to see teenage people coming into the hospital to die," he said.

"When I retire we will be, I think, the last generation of physicians to see classical cystic fibrosis.

"[These therapy drugs] could dramatically alter the natural history of this disease.

"One would hope if you maintain lung function in your later adult life that things like transplantation will no longer be needed."

Mr Bullock is not part of the Queensland trial, but he has given up hope that the drug could soon be available to him.

"I'm hanging out for that one day I'll get the call and the doctor will say 'Darren guess what's coming in? We've got it for you'," he said.

"It would be one of the best days of my life.

"I've been blessed to live this long, have a beautiful family, and I just want to continue blessing."

Topics:

health,

health-policy,

health-administration,

medical-research,

medical-ethics,

medical-specialization,

respiratory-diseases,

diseases and disorders,

government-and-politics,

australia,

qld,

brisbane-4000

First posted

September 29, 2019 05:48:06


Source link