On Thursday, July 15, 2021, Emily’s Entourage (EE) held a “Patient Listening Session” with the U.S. Food and Drug Administration (FDA) to advocate on behalf of the final 10% of the cystic fibrosis (CF) community that does not benefit from existing targeted therapies. You can read a recap of the session here. Below is a testimonial from one of the six members of the CF community who shared their experiences living with the disease.
Hello, my name is Diane Shader Smith. I would like to tell you about my daughter Mallory who passed away at the age of 25, two months after receiving a double-lung transplant. When I read about what currently available modulators are doing for 90% of CF patients, my heart breaks that Mallory didn’t get to live. My hope is that patients in the 10% non-treated group still in the fight will have an easier time of it and a better outcome.
Mallory was diagnosed with CF at the age of three. She had lots of symptoms — a persistent cough, a chronic runny nose, and serious GI problems. The doctors started her on treatment the day after she was diagnosed.
As a toddler, Mallory hated doing treatments. Every day when it was time to start, she would hide … in the closet, under the bed, anywhere she thought we wouldn’t find her. Mark made up a game he called Astronaut and Pat Pat. Astronaut for the mask that delivered her inhaled medicines, and Pat Pat for the chest percussion therapy. It was unbearable for all of us.
Eventually, Mallory got used to it all. She had a very happy childhood, was always compliant … until one day at the age of nine, she refused to do treatment anymore. That’s when we introduced the idea that CF could be fatal — and explained it’s why she had to follow such strict protocols. She cried for three days but then seemed to accept that her life was different.
When Mallory was 12, she came home from camp very sick and had her first hospitalization, the result of an aggressive B. Cepacia infection. Treatments got more complicated; life got harder.
Mallory kept a journal for 10 years. In it, she wrote, “For a CF patient, time is the meanest of forces. Cystic fibrosis does a lot of taking – of dreams, of time, of travel, of friendships, of freedom, of potential, of plans, of lives. It’s like an earthquake, constantly moving the ground under our feet so we’re always struggling to regain balance, to find our footing. It’s hard to look forward when we must always be looking at the ground beneath us; we’re more lurching than walking, stumbling to stay upright.”
Having CF forces children to straddle the line between the sick and the well worlds and complicates normal childhood development and becoming independent. Doing lengthy treatments two to four times a day is disruptive academically and socially.
My daughter was a straight-A student, a three-sport varsity athlete, and beloved by family and friends. For most of her life, CF was invisible, so everyone thought she was the golden girl who had it all. Mallory would have traded everything for the chance to take a breath that didn’t hurt … to have a life that wasn’t defined by illness.
She was often worried about her disease and what life would bring. To manage her anxiety, she spent time in nature whenever possible and wrote her college thesis in parallel stories comparing the slow destruction of the Hawaiian ecosystems with her declining lung function from a superbug. The analogy helped her process what was happening to her body. It was terrifying to her and terrifying to us.
When Mallory spoke at graduation she quoted the universally respected English philosopher, Winnie the Pooh: “I used to believe in forever … but forever was too good to be true.” Mallory chose her words carefully to be relevant to her classmates but the second, private meaning, was inspired by what she refers to as, “The dark voices in my head, the brokers of hopelessness.”
Mallory died two months after receiving a double-lung transplant. In one of our last conversations, I told her I was so sorry she had CF and that I hated the disease. She wrote in response: “CF is a complex, unpredictable, irreversible, progressive, painful, suffocating, choking weed of a disease and it’s okay to hate it.”
We couldn’t be more grateful to Diane and the five other members of the CF community for courageously sharing their candid, personal experiences of living with this fatal disease. We remain in eternal awe and admiration of their commitment to advocating for the urgent unmet treatment needs of those in the final 10% of the CF community.