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Cystic fibrosis drug tried in Queensland can bring decades to life in the sick


September 29, 2019 5:48:06 PM

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

Key points:

  • Prince Charles Hospital in Brisbane tries new cystic fibrosis drug
  • Drug costs $ 250,000 per patient each year, but it is hoped it will be part of PBS
  • It gets it defective cystic fibrosis gene to function effectively, reduces mucus on the lungs

His lungs have a capacity of 40 percent, he coughs incessantly, fights constant lung infections and has been hospitalized twice for collapsed lungs.

The 44-year-old is hoping to survive on a breakthrough drug being sought by Queensland researchers.

The pills, three taken daily, are the first to treat the defective gene that causes cystic fibrosis.

International studies have so far shown that it tricks the "druggable" gene, and therefore the body's exocrine system, to function effectively and reduces excessive mucosa that accumulates on the sufferers' lungs.

can add decades to Mr Bullock's life.

"It would be life changing, give me another chance at life," Bullock said.

His dream is to watch the 11 year old twins Alex and Abigail grow up.

He cries when he realizes that this may now be a possibility.

"To see my daughter marry me, see my son have his first boyfriend, go on a date, graduate from university," he said.

"To be there for my wife of old age and enjoy the later years. That would be all real."

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

It comes on the back of three international studies, 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 running for several weeks, but because the drug works over days, departments that were once crowded now have free beds.

The hospital's Professor Scott Bell, a world-renowned scientist in cystic fibrosis from Brisbane, is also part of the global team that has tested the drug.

He said that the trial had shown that the genetic cystic fibrosis transmembrane conductance regulator (CFTR) could be pulled for the first time.

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

"Patients report significantly improved quality of life, a tremendous increase in lung function and minor infections," he said. [19659008] "Much better than some of the system-based treatments we have had so far.

" They really have a big impact on the small number of patients who have access to the medicines. "

Treatment to cost $ 250,000 per year

Professor Bell said that the biggest challenge now was to make therapy affordable with treatment for a patient for a year that cost more than $ 250,000.

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

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

"It takes about 10 to 30 years to get a drug from findings to a patient at 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 benefit scheme within two years.

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

About 3,400 Australians are born with the disease.

In the 1960s, the life expectancy of a child with cystic fibrosis was only 10, now humans can survive to the 50s.

It is expected to be further improved if, or when, the substance comes on the market.

"When I was a medical student, my only contact with cystic fibrosis was to see teenagers coming into hospitals to die," he said.

"When I retire, we will be the last generation of doctors who see classic cystic fibrosis.

" [These therapy drugs] can dramatically change the natural history of this disease.

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

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

"I hang on the one day I get the call and the doctor will say & # 39; Darren guess what has come in? We have it for you & # 39 ;, he said.

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

"I have been blessed to live so long, have a beautiful family, and I just want that blessing to continue."



health policy,

health management,

medical research,

medical ethics,

medical specialization,

respiratory diseases,

diseases and disorders,

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