I was a mess the week Trikafta was approved. It felt like I could finally exhale
characteristic of this disease. In secondary school, Sammie and I argued regularly as she pushed back against my hypervigilance. The more she struggled for independence, the more tightly I held on. If she missed a respiratory treatment, I accused her of being cavalier with her life. The truth was, I was terrified.
The summer before her final year of school, Sammie was hit with a triple lung infection. She was hospitalised, given intravenous antibiotics and airway clearance therapy every three to four hours, around-the-clock. Despite weeks of intensive treatment, first at the hospital and then at home, she didn’t bounce back as we had hoped.
At 18, her routine included hardcore prescription pills with terrifying warning labels and insulin shots for Cf-related diabetes, a common development. It felt like my daughter had veered down a path with narrowing possibilities.
Another friend’s daughter succumbed in her twenties. At her funeral, I sat between two other CF mums. We were the lucky ones whose daughters were still alive. Silent, we stared ahead, clutching one another’s hands. I envisioned the three of us on a rowboat, in the middle of a stormtossed ocean, sharks circling—waiting to tear us apart.
At 22, Sammie joined a clinical trial for a triple-combination drug to target the underlying cause of CF: its mutated protein. For years, we’d heard about this new generation of drugs in the pipeline that focused on restoring that protein’s function. Less powerful versions had come to market for smaller CF populations with rare mutations, and those were gamechangers. This new drug would address the most prevalent mutation, and Phase 2 trials looked promising.
For the first few months of the Phase 3 trial, nothing changed for my daughter. We assumed she was among those taking the placebo.
Then the study entered its open-label stage when all participants received the drug.
Within hours of taking her first pills, Sammie began coughing violently as her airways were purged of mucus. Within a week, her lung function skyrocketed, and her chronic cough disappeared. Within months, her blood sugar levels normalised, and she could stop injecting herself with insulin, although she continues to take other medications.
The drug, Trikafta, received approval by the US Food and Drug Administration in November 2019. It arrived like the hero in an action film, snipping the wire on a ticking time bomb with only seconds remaining on the clock.
I was a mess the week Trikafta was approved. It felt like I’d been holding my breath for 20 years and could finally exhale. I inhaled stories about young adults with CF suddenly moved off lung transplant lists or able to have kids. My daughter got tired of hearing me talk
about it.
“I know you’re excited about the drug, and I get it, but you have to understand. I was never afraid of dying from CF,” she said.
I sat with that a moment.
“Good,” I said. “That means I did my job well.”
Parenting a child with a chronic illness is, in many ways, like parenting any child. It requires trust and a willingness to live with uncertainty. Eventually, you have to let go and hope your child has absorbed the lessons you tried to impart.
My daughter has grown into a remarkable young woman. Strong, confident, and capable. She no longer needs me to be her caretaker. I can just be her mum.
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