True grit: The story of Cailyn Fowler (Part 1 of 3)

NORWAY — For most of the students of Oxford Hills Comprehensive High School, Wednesday, February 8 is just another day of classes, interacting with peers, and looking forward to the final bell.

But for one junior, Cailyn Fowler, the day is anything but ordinary.

The 17-year-old has a hard time focusing on history, despite the fact that it is taught by her favorite teacher, Mr. Vandemark.

"Good luck," he tells her, as she walks out of the classroom. She goes on to her culinary arts class, where she spends three hours trying to concentrate on making ranch dressing and a veggie plate with her partner and friend, Deanna.

"Good luck," says Deanna.

Good luck. It is a phrase she has heard all day, from many of her peers and teachers.

At 1:52 p.m., the minute the final bell rings, most of the students are wandering to their extra-curriculars, or taking the bus home, or going to work, or making arrangements to play video games with friends.

Cailyn hurries to the parking lot and gets into her car (the license plate reads CUR4CF). At 4-foot-10 and 83 pounds, she is both shorter and thinner than her classmates. When she first got the car, an adult friend asked if she needed a booster seat, only half-joking. As it turns out, she does not.

Cailyn looks for all the world like a happy, well-adjusted teen, dressed in hip, colorful outfits and shooting a shy smile to all who acknowledge her. She chats with friends, marking the latest in teen fashions, television programs, and teen heartthrobs. (When it comes to the love triangle at the center of the Twilight saga, she favors Jacob, the Native American werewolf over Edward, the 100-year-old vampire whose cold skin, she says, would be kind of a turn-off.)

To the casual observer, there are only minor clues to Cailyn's medical condition. Her slight frame is one. Then there is her chronic cough. She has also missed school a lot for medical reasons. Other than these signs, no one would guess that, as a baby, she was diagnosed with cystic fibrosis (CF), a debilitating condition that afflicts 30,000 Americans.

No one would guess that, though she is just a teenager, she is also, by all expectations of the medical establishment, middle-aged.

Two weeks ago, there wasn't a doctor in the world who would have told her she would live past her thirties. In fact, the average age of death for those who were born with CF is in their mid-twenties.

Cystic fibrosis is uncurable, unmanageable, unforgiving. It doesn't care about the beauty of youth, the right of a human being to live and love and grow old. When it enters a person's life, it takes all that they have, and all that they're ever going to have.

But today is Wednesday, February 8.

It is, hopes Cailyn, a very special day.

Cailyn hops in the car, drives the half-mile or so to the Norway Savings Bank on Main Street, where her mother, Cindy, has worked for 30 years. 

Cailyn has grown up in the bank. When she was very young, she would run from desk to desk, asking the amused adults questions, and then running upstairs to practice gymnastics.

Photo: Matt Hongoltz-Hetling

FIRST CHANCE — Cailyn takes her first dose of ivacaftor, a new drug that should dramatically improve her battle against cystic fibrosis, while the staff of Norway Savings Bank cheers her on.

Today, there is a festive atmosphere in the bank.

The bank's employees, nearly all women, are gathered in a crowd. They cheer when they see her. Their eyes shine with tears of hope and joy. From their maternal expressions, any one of them looks like they could be her mother.

Cailynn stands by Cindy, her actual mother. Cindy helps to deflect some of the crowd's excitement, bantering and speaking while Cailyn stands, smiling quietly.

Everyone asks her how she feels, if she is excited.

She is, she tells them, she is excited. She is happy, but also seems a little unsure of what to say, of what could justify the attention being bestowed on her by the assemblage of adults, who eagerly hang on her every word.

Finally, the moment comes.

Her mother hands her a pill bottle and gets her a drink. Pills are not new to Cailyn. After all, she takes 37 pills a day.

But this little blue pill is different. This pill may be the pill to end all pills.

This pill, says her mother, is the closest thing to a miracle that she and Cailynn have ever seen.

A cure

Where did the miracle pill come from?

The short answer is that it came from a pharmacy operated by the Cystic Fibrosis Foundation.

The pharmacy is located in a five-story brick building in a commercial district of Bethesda, Maryland, a life-saving center nestled in among its more ordinary neighbors; a bank, a bicycle store, a hardware store and a bakery.

It is an unassuming location for the extraordinary work that is done there.

On Tuesday, January 31, just eight days before Cailynn arrived at the bank to cheers and applause, the Food and Drug Administration approved a drug called ivacaftor.

Based on the amazing success ivacaftor had in human trials, FDA said that pharmacies would be allowed to dispense the drug for a small percentage of those who suffer from CF.

The drug was approved at noon.

Just two hours later, by coincidence, Cailyn and Cindy Fowler were in the Cystic Fibrosis Clinic of the Maine Medical Center in Portland, talking to her nurse, Mary Ellen Corrigan.

A perfect blend of practicality and warmth, Corrigan has been working with cystic fibrosis patients for decades.

She has seen first-hand the progress that has been made in the battle against CF.

"We have about a hundred patients in our kids clinics and probably about 75 in the adult clinic," she says. Sensing that her point has not been taken, she clarifies why this is important.

"Twenty years ago, we didn't have an adult clinic," she says. "... We didn't have adults."

Corrigan has known Cailyn and Cindy since Cailyn was an infant, since those days when there was no adult clinic for CF patients. Over the years, Corrigan has developed a strong appreciation for the small girl with the deadly disease who, said Corrigan, has faced her affliction with style and courage.

Cindy remembers asking Corrigan about whether the new drug could help her daughter. She had heard about it through Facebook, from a friend who also has a child living with CF.

"The nurse looked at Cailyn and said, 'what mutation do you have?'" says Cindy. "Cailynn said 'I don't know,' so she said, 'I'm gonna check to make sure it wouldn't help. She went and checked and came back jumping up and down, saying, 'Cailyn, you're a match, you're a match!' She said, 'oh my God, you're a match!'

"Cailynn and I looked at each other and said 'what does that mean?' It's the closest thing to a cure you can get, that's what she told us."

Cailyn knows something about long odds. Even in cases when both parents do carry the recessive CF gene, there is only a one in four chance that the child will be born with CF. The chances of a baby being born with CF is just one in 3,500.

Based on those numbers, Cailyn's had more rotten luck than the rest of us can imagine.

But now, it seems, her luck may be turning around.

CF is the result of any one of 1,800 different mutations. Of all of those mutations, the drug ivacaftor fixes just one, referred to as G551D.

That's why Corrigan was jumping up and down in the CF clinic in Portland. She had gone to the computer, barely daring to hope. She was just checking, really, to make sure that it wasn't going to help, that Cailyn didn't have the G551D genetic mutation.

But, remarkably, it is the exact mutation that Cailyn has.

Once she identified the match, Corrigan went into a state of frenzied action.

"Then I had to figure out how to get it for her," said Corrigan. "Drugs like that are really expensive and you can really only get them at specialty pharmacies."

Over the course of the next few days, the situation changed a couple of times as Corrigan tried to get an order for a supply of ivacaftor, which is marketed under the name Kalydeco™ by a company called Vertex.

She put in a call almost immediately to the CF pharmacy in Bethesda.

"Of course, their phones were all abuzz with people from all over the country calling," said Corrigan.

Vertex officials reported that they were working quickly to complete packing and shipping of bottles containing 30-day supplies of ivacaftor.

At first, it seemed like it might take two weeks to get a prescription filled. Then, the estimate changed to several weeks, with a target date of sometime this spring. Then it changed again, and a pharmacist called Cindy to tell her that the order would be shipped on Wednesday, February 8.

"They said that Cailyn is the first one that they've shipped this out to," says Cindy.

The unimaginably precious blue pills were put into a white pill bottle with Cailyn's name on it, packed into a small, square box, and shipped up the coast via UPS.

Cindy says that waiting for the pills was nerve-wracking in and of itself.

"I didn't get a lot of work done," she says. "I've been pacing all day."

Now, on this special day, Cailyn holds a pill in one hand, and the sports bottle in the other.

With a nervous grin, she downs the pill. There are cheers and applause. The crowd begins to break up, with bank employees wishing her well before drifting off in groups of one and two.

Karen Hakala, the bank's senior vice president, bends down to give Cailyn a tight hug before Cailyn leaves.

"I love you," she tells the girl.

"Love you too," says Cailyn, hugging back.

Click here for Part 2