She waits a beat before saying to the audience: “Wouldn’t that be hilarious if He healed me right now?”
Since the play, when she was just starting Trikafta, Jones’ lung function has gone up 20 percent. She now has a full-time job as a bookkeeper, which she never thought would be possible. She opened a Roth IRA, which she is proud of even though it only has some paltry dollars from nannying.
As we talked, she was taking her morning dose of Trikafta. She first heard about the drug being in development about a decade ago and remembers crying and wishing she didn’t know about the possibility of the drug, because she knew it would take years and years of development.
“It still kind of blows my mind to think now, I’ll be alive in such a way, not crippled,” she said. But she also knows that she is daily dependent on the drug, and complications could develop that kick her off the drug. “I feel like I’m on vacation from CF. But one thing that I didn’t expect is … constant fear that it’s going to come back.”
She hears about someone with liver problems who has to come off Trikafta, and imagines that happening to her. Jones has been going to therapy. “It’s like, I’m home from the war, and I can deal with all the fear.”
HEALING GIVE PATIENTS mental space for the pent-up emotional and psychological problems to come rushing in. Many have dealt with near-death experiences at a young age, friends who have died from the disease, and the prospect of deteriorating health.
“You grow up staring down that barrel,” said Robert, 32, whose full name we are not using to protect sensitive medical information. He has CF and has struggled with severe depression since he was a child, as well as substance abuse from his teens into adulthood. “Death is scarier when you’re right up close to it.”
At 22, he almost died when one of his lungs collapsed, and he spent weeks in the hospital with a chest tube draining out blood and other fluids from his lung. At 25 he started smoking and returned to drugs, not great for already sensitive lungs: “I was just super unhappy. … What I really wanted to do when it was bad, was to not exist.”
Studies have shown that as CF patients live longer they deal with a burden of psychological issues and are at a greater risk for depression.
Only when Robert got on a gene modulator (Kalydeco, a precursor to Trikafta) did he begin to address his substance abuse issues and depression. He’s now in Alcoholics Anonymous, has quit smoking, and hasn’t had a depressive episode in a while. “Seeing a big jump in my quality of life made me believe I could get better,” Robert said. He recalled being able suddenly to run and not be out of breath. “That felt like a magical miracle.”
The effect has been similar for Joanie Santander. Santander began taking Trikafta in December, as her brother was recovering from his double-lung transplant. She has had side effects: high liver enzymes, and less dexterity. She and her husband had talked about having children, but haven’t conceived yet, and she wonders if the drug affects fertility.
But she has lost her cough, and her lung function is now the highest it’s been in a decade.
Taking the drug, she says, “is like giving time back to us.”
On the fast track
Though Joanie Santander wished the drug had come earlier for her brother, the breakthrough drug approval came earlier than anyone anticipated, even Vertex, the drug developer. The last few years, the FDA has been fast-tracking more drugs for life-threatening conditions, according to an analysis by The Wall Street Journal, approving a record 43 via the fast-tracking process in 2018. That fast-tracking brought Trikafta to cystic fibrosis patients, but it also means patients and insurers are paying for other potentially expensive drugs without proven records.
In announcing its fast-track approval for Trikafta on Oct. 21, the FDA’s acting commissioner said “we’re consistently looking for ways to help speed the development of new therapies for complex diseases, while maintaining our high standards of review.” —E.B.