Lifestyle
The moment we received the news, my five-year-old daughter, Lily, gazed up at me with concern through her bright pink glasses. “Mommy, why are you crying?” she asked.
“I’m worried about your medicine,” I replied. “We don’t know how we’ll get it soon, and without it, you could get sick.” She studied my face for a moment, then with complete faith and confidence, she smiled and reassured me, “I’m not sick, Mommy. I won’t be sick.”
Oh, how I wish that were true.
“Get sick” is a euphemism, a gentle veil over a horrific reality: she could die. How does one explain to a child that they are facing a terminal illness, and that the sole medicine keeping it at bay will no longer be available in October? This drug works, it’s safe, and it exists — it simply won’t be provided to her or to many other children like her. Someone made the decision that it’s not worth it, and she will bear the consequences of that choice with her life.
Lily is a vibrant five-year-old full of dreams and aspirations. She delights in wearing pink heart-shaped sunglasses over her regular glasses, twirls in her tutu, and joyfully sings “Let It Go.” She adores watching “Pioneer Woman,” often pondering where the host’s husband is on the ranch. She dreams of marrying one day, becoming a doctor, and following her older sister around for eternity.
But Lily also suffers from Niemann-Pick Type C (NPC), a severe neurodegenerative disease often dubbed “Childhood Alzheimer’s” as it can lead to dementia in young children. NPC causes seizures, tremors, and choking, but perhaps the most cruel aspect is what it takes away: her laughter, her memories, her very essence. It will rob her of movement, dance recitals, birthday celebrations, and cozy hot chocolate nights, ultimately stealing the most precious thing of all: her life.
Her medicine, VTS-270/Adrabetadex, is her lifeline. Right now, Lily is stronger than the disease, but the situation is precarious. In January 2021, Mallinckrodt Pharmaceuticals announced the termination of all access programs and distribution of this vital drug by October 2021. The countdown has begun — we have only six months left.
Every other week, Lily receives this treatment via a lumbar puncture — a procedure she bravely endures while awake at Children’s Hospital Los Angeles. She is supported by an exceptional team of doctors and nurses, who bring joy and hope into her life. These dedicated professionals recognize the significance of this drug and advocate fiercely for its continued access.
Niemann-Pick Type C patients have been receiving VTS-270/Adrabetadex since 2010 — for over a decade. Lily began her treatment at 19 months through a compassionate use program. Before her treatment, she struggled to walk, was frail, and faced significant challenges with feeding and energy. We feared she might not see her second birthday due to the aggressive form of NPC she has.
Then, everything changed. After just five doses of VTS-270, she began to walk and flourish. Today, she’s in pre-K, power walking, riding her tricycle, and climbing into her sister’s top bunk bed. This medicine has revived her spirit, and without it, all that joy will vanish.
The complexities surrounding the approval of this medicine are extensive and convoluted, leading to delays that endanger lives. The reality is stark: without VTS-270, Lily, along with countless other children, will not survive.
As a mother, I don’t seek to assign blame. What I do want is urgent action. This drug works, her doctors see its benefits, and we need to advocate for its continuation. It’s not just about my daughter; it’s about all those battling this rare, life-threatening disease who rely on this medicine for a fighting chance. We need to ensure that children like Lily continue to experience the joys of childhood — love, friendship, and happiness.
This is a solvable issue, and we can ignite change to preserve this critical medicine. We need a united front of mothers — an army to advocate for our children and secure their futures. Please join us in this fight, not just for Lily, but for all those who depend on VTS-270 to stay alive.
To stay updated on this vital mission, connect with us on Facebook and Instagram. For more information on how you can support our cause, please visit our campaign “Save Our Medicine.”
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Summary:
Lily, a five-year-old battling Niemann-Pick Type C disease, faces the impending discontinuation of her life-saving medication, VTS-270. As her mother, Clara shares the emotional turmoil and urgency of the situation. The drug, which has been effective for years, is critical for Lily’s survival and the survival of many other children. Clara calls for collective action from mothers and advocates to ensure access to this essential medicine and preserve the lives of these children.