But that year, she started coughing up blood from time to time, and she began having more flare-ups of inflammation and acute infections in her lungs. Then in February, an artery burst in her lung and nearly killed her. The surgeons patched her up, but for the rest of that year and the next, she spent more nights at the hospital than at home, coughing up blood, running fevers, struggling to breathe and to tame the infections in her lungs. Polyps in her sinuses that had been removed three times were growing again and giving her terrible migraines, but she wasn’t healthy enough to endure another surgery. Instead, she drew the blinds and packed her face in ice to cope with the pain.
“The path I was going down was terrifying,” she says now. “I knew my body was declining.”
In October 2013, Honaker joined a new clinical trial testing a pair of drugs, lumacaftor and ivacaftor, that are specifically designed to restore the function of the defective protein that causes her CF. By this past spring, she was feeling well enough that when she found out the Food and Drug Administration was holding a public hearing on whether to approve the new drug combination, called Orkambi, she signed up to testify.
Before starting the drugs, she’d rarely traveled by airplane. Flying would put stress on her weak lungs, and she didn’t want to be too far from her local CF center in case something terrible happened again. But now she was willing to risk it. In early May 2015, she flew from her native North Carolina to Washington, D.C., to tell the panel and the world what Orkambi had done for her.
“My quality of life has dramatically improved,” she said in her testimony. She sleeps through the night, instead of waking up repeatedly to cough. Her migraines have vanished. She has more energy; she rarely coughs up blood and has had only one lung infection in the last year and a half. “I ask that the FDA please approve these drugs,” she said. “They will prolong many lives.”
That day the panel voted 12-1 in favor of the combo; the official FDA approval followed in July.
Orkambi is hardly a magic bullet. It is proven to work only in people over the age of 12 who have the same pair of gene mutations (two copies of the same delta F508 mutation, also known as homozygosity for the mutation) that Honaker has: about 8,500 people in the United States, out of a total of about 30,000 people with CF. On average, people who take it get only somewhat better — Honaker, for example, still has to keep up her daily routine of treatments and drugs.
What makes patients, families, researchers, and doctors so optimistic is that Orkambi is just one of a new wave of treatments designed to restore function to a defective protein caused by a specific genetic error that reults in the disease. Only two are now on the market — Orkambi and Kalydeco (ivacaftor alone) — but there are many more in development. The trend suggests that the future of CF treatment is going to be entirely new. It also suggests that at least for some people, CF may soon become a manageable disease.
“It’s a most exciting time for CF in our lifetimes,” says Jennifer Taylor-Cousar, an associate professor of medicine and pediatrics at National Jewish Health in Colorado and a CF expert. “On the horizon is the possibility that we will be able to treat all patients at the molecular level in the next 10 to 15 years — maybe even sooner than that.”
The promise of personalized medicine, in which each patient is treated with a therapy designed specifically to target the underlying cause of his or her disease, is still a distant vision for most people and most illnesses. In CF, it might soon be a reality.