A new treatment for cystic fibrosis, or CF, is being hailed as a groundbreaking therapy that can potentially extend the lives of people with the condition.
Nearly 400 people in Maine have the genetic disease that affects the lungs and other organs. And while many in the cystic fibrosis community are celebrating the news, there is concern about the medication's cost — more than $300,000 per year.
One of the worst things about having cystic fibrosis, says 11-year old Adriana Pettengill of Belgrade, is that some of her peers treat her differently.
"Like, some people avoid me because they think I'm going to get them sick." Then, she says, there are the people who say they feel bad for her. "I'm like, 'I don't have a hard life.' I mean, I do have a disease, but there are people out there who have it way worse than me."
Adriana and her 13-year old brother Blake, who also has cystic fibrosis, do follow daily routines that are different from most kids’. The disease causes mucus to build up in organs, mainly the pancreas and the lungs, which can interfere with nutrition and the ability to breathe. The mucus can also trap bacteria and cause infections. To get the excess mucus out of their lungs, Adriana and Blake need to slip on a special vest every morning and night.
Their dad Travis Pettengill helps Blake hookup tubes from his vest to a machine about the size of a computer printer. "So this machine is a compressor that inflates and it also pulsates,” he says. “So it inflates and gets very tight on their chest. And the pulse is what shakes them around to loosen the mucus," which they eventually cough out.
Blake and Adriana spend more than an hour a day wearing the vest. They also use nebulizers and take several medications. And now another treatment is going to be added to that routine. Last month, the FDA approved a new medicine called Trikafta. It's being described as the single greatest therapeutic advancement in cystic fibrosis.
"I never thought in my lifetime that this would happen," says Dr. Ana Cairns, who has been director of the cystic fibrosis center at Maine Medical Center for more than two decades.
Cairns says Trikafta is groundbreaking for a couple of reasons. First, it targets the underlying cause of cystic fibrosis at the cellular level. Second, Trikafta is expected to help about 90 percent of patients.
"It creates tremendous hope. I mean it creates hope that they can live a long life into midlife and beyond."
Cairns stresses that it is not a cure. But the new medicine will likely reduce the cycle of inflammation and infections that cause lungs to scar and deteriorate to the point that a lung transplant is the only option to prolong life. And some patients, she says, will see improved lung function.
Eighteen-year old Melayna Benner hopes that her lungs, which function at around 65 to 70 percent, will get a boost when she starts taking Trikafta.
"I just got approved the other day," she says.
Benner is studying to be a surgical tech at Southern Maine Community College. Even though cystic fibrosis is a progressive, terminal disease, she says she has always held out hope for a long life.
“To me, it was always like, I'm going to live to 100. And now it's like, I'm really going to live to 100.”
Back at the Pettengill's house in Belgrade, 13-Year old Blake will start taking the new medication right away. Trials for younger children are underway, but Adriana will be eligible in a year, when she turns 12.
"I'm just so excited for the day to come, because I think it's awesome that people are helping us out."
Blake and Adriana's parents say their kids will now have a shot at a longer, healthier life. But Travis says there is another aspect to his kids' future that he worries about.
"Right now, sometimes it's a struggle with paying for medication,” Travis says. “You know, when they get into adulthood, when they have to get a job and have to worry about insurance and have to worry about being able to afford to pay for their nebs (nebulizers) and their meds and medical equipment. That's more the things that I'm starting to think about now."
Trikafta comes at annual price tag of more than $300,000. A spokesperson for the company that manufactures it, Vertex, says in an email that their drugs have been broadly accepted by both public and private insurance, and the price of Trikafta reflects the efficacy of the medication and the company's investment in the development of new medicines.
But Dr. Ana Cairns says she is worried that the high cost of Trikafta could restrict access for those who are eligible for the drug. And for the 10 percent of CF patients who are not eligible because of their particular form of the disease, Cairns says the search for a cure must go on.
Originally posted 4:27 p.m. Nov. 13, 2019