Local cystic fibrosis treatment called a miracle - San Diego, California News Station - KFMB Channel 8 - cbs8.com

Local cystic fibrosis treatment called a miracle

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SAN DIEGO (CBS 8) - It's the closest scientists have come to a cure for cystic fibrosis and it was discovered in San Diego.

CF affects 30,000 people in the United States and the average life expectancy is 37-years-old. But a new medication could mean much longer lives, for more than 15,000 CF patients.

There is groundbreaking research being done in a San Diego lab, where scientists have developed two drugs that could change the lives of people living with cystic fibrosis.

Ashton and Lola Ferguson appear to be healthy kids, but jumping on their backyard trampoline is part of their therapy.

"It rattles their lungs around, it shakes them up," said their mom, Jennifer, who plays an active part in the treatment.

Both 12-year-old Ashton and seven-year-old Lola have cystic fibrosis.

Each day, they spend at least an hour connected to a machine that helps loosen the mucus in their chest.

Their gene defect clogs up many vital organs.

"Their lungs get dry and mucous gets really thick and that's where the bacteria breeds," explained Jennifer.

Advancements in medicine mean people with CF are living far longer than they used to. In the 1960's, most kids died by the age of 10. However, both Ashton and Lola know they may not live past their 30's.

"My daughter the other day was talking about having children, and she goes well, ‘if I live long enough,'" Jennifer said.

But Jennifer is more hopeful than ever before that her children will outlive her because of a medical breakthrough discovered at a San Diego lab.

"We're excited every day to come to work, to try and find these medicines for these patients," said Paul Negulescu.

Paul Negulescu is Vice President of research for Boston-based Vertex Pharmaceuticals. He manages the San Diego lab, where scientists have developed a tiny pill that could be the huge miracle drug, which extends the lives of CF patients by decades.

"It's currently in human trials, it's in the final stage of human trials which is what's so exciting," noted Negulescu.

Used in conjunction with a drug already approved by the FDA, the new drug is designed to give the majority of CF patients dramatic relief

Nearly 60 years in the making, the Cystic Fibrosis Foundation has been raising money in search of a cure since 1955.

"There's little to no government funding for cystic fibrosis research so we are involved in every single drug that comes out," explained Laurent Quenaud with the CF Foundation of San Diego & Imperial Counties. "I think that we can get a cure for this."

The new drug would technically be a fix, but families say this miracle of science is the closest they've come to a cure.

"It means less pills, better digestion. It means bigger, healthier lungs. It means longer lifespan. It means being able to have kids, it means a lot of things," continued Quenaud.

If this last phase of clinical trials goes as planned, this life changing drug could be in the hands of patients in the next two years.

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