My Daughter's Future Relies on a Drug Set to Be Discontinued Soon

The moment we received the news, my five-year-old daughter, Lily, gazed up at me through her adorable pink glasses, her expression filled with concern. “Mommy, why are you crying?” she asked.

“I’m worried about your medicine,” I replied softly. “If we can’t get it, you might get sick.” She paused for a moment, then, with unwavering confidence, reassured me, “I’m not sick, Mommy. I won’t be sick.”

Oh, how I wish that were true.

“Get sick” is merely a gentle way of saying something far more devastating: it means death. How do you explain to a five-year-old that they are facing a relentless and fatal illness? That they have a progressive disease, and the only treatment keeping it at bay will cease to be available in just six months? This medicine is effective and safe; it simply won’t be provided to her or many other children in need. Someone decided it wasn’t worth it, and she may pay the ultimate price.

Lily is a vibrant five-year-old, bursting with enthusiasm for life. She loves donning her pink heart-shaped sunglasses over her regular glasses, twirling in her tutu while belting out “Let It Go.” Her favorite show is “Pioneer Woman,” and she often wonders aloud about the adventures of the show’s host. She dreams of growing up to be a doctor and following her older sister around for the rest of her life.

However, Lily is also battling Niemann-Pick Type C (NPC), a devastating neurodegenerative disease that is sometimes likened to Childhood Alzheimer’s due to its potential to cause dementia in young children. NPC leads to seizures, tremors, and choking. But more heartbreaking than the symptoms it brings is what it robs from her: her laughter, her memories, and ultimately, her life.

The medicine, VTS-270/Adrabetadex, is what currently stands between her and the cruel progression of NPC. It has granted her strength, but time is running out. In January 2021, Mallinckrodt Pharmaceuticals announced the discontinuation of all access programs for this drug, set for October 2021. The countdown has begun, and we have only six months left.

Every other week, Lily bravely undergoes a lumbar puncture at the remarkable Children’s Hospital Los Angeles to receive her medication. Surrounded by compassionate doctors and nurses who share her love for pink glasses and unicorns, she faces each session with courage. These dedicated healthcare professionals understand the importance of this drug for Lily and other children like her.

Patients with Niemann-Pick Type C have safely used VTS-270/Adrabetadex since 2010. Lily began her treatment through a compassionate use program in 2017, when she was only 19 months old. Before treatment, she struggled to walk, was extremely weak, and couldn’t keep down food. We were uncertain if she would even reach her second birthday due to the aggressive nature of her illness.

Then, something miraculous happened. After just five treatments, Lily began to walk and thrive. Now, she is in pre-K, riding her tricycle, and even climbing into her sister’s top bunk. This medicine has given her life, and without it, her future is bleak.

The reasons behind the drug’s delayed approval are complex and tangled, but the reality is stark: without this medicine, Lily’s life is at risk. The same goes for other children like Emma in Minnesota, Jeg in Illinois, and Abby in Texas.

As a mother, I don’t care about assigning blame; I just want action. This medicine works, and her doctors know it. My priority is to save my daughter’s life and the lives of all children battling this rare and fatal disease who depend on this treatment.

There is hope. This is a solvable issue, and we can drive change to save this drug. We need support from other parents—an army of mothers—to advocate for our children’s futures. Please join us in this fight to ensure access to this critical medicine. Our kids are counting on us; let’s guide them toward a brighter future instead of allowing them to slip away.

For more information on this vital mission, please connect with us on Facebook and Instagram. To assist in navigating the complexities of this issue, consider visiting our campaign, “Save Our Medicine,” or explore helpful resources like this one. And if you are looking to boost fertility, check out these supplements.