On the day we received the news, my five-year-old daughter, Lily, peered up at me with concern through her vibrant pink glasses. “Why are you crying, Mommy?” she asked.
“I’m worried about your medicine,” I replied. “We’re not sure how we will get it soon, and without it, you could get sick.” She stared at me silently for a moment, then, filled with trust and confidence, she said, “I’m not sick, Mommy. I won’t be sick.”
Oh, how I wish that was the truth.
The phrase “get sick” is a euphemism for a harrowing reality: she could die. How do you explain to a five-year-old that she is dealing with a relentless, fatal illness? That her condition will only worsen without the medication which is about to be discontinued in October? This essential drug is effective and safe; it just won’t be available for her or many other children like her. Someone made the decision that it’s not worth continuing, and Lily is the one who will suffer the consequences.
Lily is a spirited little girl, full of dreams and joy. She adores her pink heart-shaped sunglasses, twirls in her tutu, and often breaks into song with “Let It Go.” Her favorite show is “Pioneer Woman,” and she often wonders aloud about the day’s happenings on the ranch. She envisions a future where she gets married, becomes a doctor, and follows her older sister everywhere.
However, Lily is also battling a rapidly progressing neurodegenerative disease called Niemann-Pick Type C (NPC), sometimes referred to as Childhood Alzheimer’s due to its propensity to cause early-onset dementia. It brings on seizures, tremors, and choking. Yet, the true cruelty lies in what the disease robs from her: her laughter, her memories, her movement. It will ultimately take the most precious thing of all: her life.
The medication, VTS-270/Adrabetadex, is currently her lifeline against NPC. It has allowed her to maintain a delicate balance against this devastating illness. Tragically, in January 2021, the pharmaceutical company, Brightwood Pharmaceuticals, announced it would cease all access programs and distribution of the drug in October 2021. Time is running out—only six months remain.
Lily receives this medication bi-weekly through a lumbar puncture at a remarkable children’s hospital. She bravely endures the procedure while surrounded by a team of compassionate doctors and nurses who uplift her spirit. They wear matching pink glasses and unicorn pins, bringing a touch of magic into her life. These dedicated medical professionals recognize the significant impact of this drug and advocate for its continued availability.
Patients with Niemann-Pick Type C have safely used VTS-270 since 2010. Lily began treatment at 19 months old under a compassionate use program. Before starting, she struggled to walk, was weak, and frequently choked on food. We were uncertain if she would reach her second birthday due to the aggressive nature of her condition.
But then, a miracle happened. After just five doses of VTS-270, she began to walk and flourish. Today, she’s in pre-K, rides her tricycle, and climbs into her sister’s bunk bed. This medication has revived her spirit, and without it, her future dims.
The complexities surrounding the approval of this drug are extensive, making it one of the most challenging stories in drug development. The details of the clinical trial are intricate, but the bottom line is simple: without this medication, Lily’s life is at stake. And the same is true for countless other children battling NPC.
As a mother, I’m not concerned about assigning blame. What matters is finding a solution, urgently. This medicine works, and her doctors can attest to that. I am focused on saving my daughter’s life and the lives of others like her who depend on this treatment. It’s about preserving the joy of her childhood and the connections she cherishes.
This is a solvable issue. We can rally for a change to secure this medicine. But we need your support. We need an army of mothers to advocate for our children. Join us as we fight to ensure a future for them. They are counting on us; let’s lead them to safety rather than despair.
To follow our mission to save access to this life-saving treatment, check out our other blog posts, like this one on home insemination. For expert insights, visit Intracervical Insemination and Progyny for invaluable resources regarding pregnancy and home insemination.
Summary
A mother shares her heart-wrenching story about her daughter, Lily, who is facing a life-threatening condition known as Niemann-Pick Type C. The only medication that has been effective for Lily is set to be discontinued in six months. The mother calls for urgent support from the community to advocate for this essential treatment to save her daughter’s life and the lives of other children battling the same disease.