“I’m worried about your medicine,” I said. “We don’t know how we will get it soon, and if you can’t get your medicine you will get sick.” She watched me silently for a moment, and then full of trust and reassurance, pleased with her solution, looked in my face and said, “I’m not sick, Mommy. I won’t be sick.”
I wish more than anything that could be true.
“Get sick” here is the ultimate euphemism. It is code for a brutal and unbearable truth: you will die. How do you tell a five-year-old that they are slowly dying? That they have a relentless fatal disease, and the only medicine to hold their disease back will stop being distributed in October. That this medicine works and is safe and still exists — it just won’t be given to her and dozens of other kids just like her. That someone, somewhere, decided it’s not worth it — and she will pay the price for that decision with her life.
My daughter Marian is five and filled with a fiery desire to live and love. She loves to wear pink heart sunglasses over her pink eyeglasses, wiggle into a tutu and prance around the living room singing “Let It Go.” Hands down her favorite show is “Pioneer Woman,” and she sits watching raptly and says, “I wonder where Ladd is on the ranch today.” She dreams of her future, wants to get married, be a doctor, and follow her big sister around everywhere she goes for the rest of her life.
She also has a rapidly neurodegenerative fatal disease called Niemann-Pick Type C (NPC), sometimes referred to as Childhood Alzheimer’s because it can cause dementia in young kids. It also causes seizures, tremors, and choking. But more than what it brings, it’s what the disease takes away that is most cruel. The disease will take her laugh, her smile, her memories, her songs, her voice. It will take away movement, dance recitals, birthday party games and hot chocolate nights. In its place will be suction machines, G-tubes, immobility, and a mini hospital unit in our living room. Ultimately, it will take the most precious thing of all: her life. It will take her.
This is what her medicine, VTS-270/Adrabetadex, is fighting. Because of this medicine, Marian is stronger than the NPC right now — a scale that, at this moment, is precariously balanced. To our devastation, in January 2021, the pharmaceutical company — Mallinckrodt Pharmaceuticals — said they are discontinuing all access programs and distribution of the drug in October 2021. The clock is ticking. We have six months left.
Marian gets this medicine every other week through a lumbar puncture — that she sits up fully awake for — at the incredible Children’s Hospital Los Angeles. She is beyond brave. Every other week she is cheered on by some of the most beautiful and exceptional doctors and nurses in existence. They have matching pink glasses and unicorn pins, because they bring the magic into her life. Marian loves them. These phenomenal doctors, NPC expert physicians, and many more strongly recognize the benefit and power of this drug and critical importance to save access.
Niemann-Pick Type C patients have safely taken VTS-270/Adrabetadex since 2010. For 11 years. Marian began treatment at 19 months, in 2017, through a program called “compassionate use.” Before treatment she couldn’t walk, was extremely weak, choked on some baby foods, wasn’t gaining weight, and she was sleepy all day long. We didn’t know if she would live to be two years old due to her aggressive infantile version of NPC.
But then, it was like a miracle. After only five treatments with VTS-270, she started walking and thriving. Today she is in pre-K, power walks, rides a tricycle, and climbs into her sister’s top bunk bed. She came alive with this medicine. It gives her life, and without it, she won’t have one.
The story of why this medicine isn’t sailing through to approval is being called one of the most complex in drug development. The troubles with the clinical trial for this investigational drug are many and complex. The history is complicated, but the truth is simple: without this medicine Marian will die. And so will Emma, a two year old in Minnesota, and so will Jeg, a seven year old in Illinois, and Abby, a six year old in Texas, and on and on and on.
As a mom, I don’t know or care exactly who is at fault for this. What I know is this: it needs to be fixed. Urgently. This medicine works. Her doctors see that it works. What I care about is saving my daughter’s life and the lives of all 40-some children and adults battling an ultra rare fatal disease who need this drug to keep living. I care about saving a medicine that gives my daughter independence and childhood experiences and friendships and joy in life. There’s nothing Marian loves more than to connect with others and love them. That’s what this is for: it is to keep her here. To hold her trusting hand and guide her forward, not betray her childhood faith in the world and instead have her slip away.
This is a solvable problem. We can inspire the change to save this medicine. But our children need your help. We need all the moms, an army of moms, to stand up for our children to save them. Please help us fight for them and inspire the solution to save this medicine and save them. Please help give our children a future. They are trusting us; let’s help guide them to safety instead of the end.
To follow this life saving mission to save access, please connect on Facebook and Instagram. For more information and to help fund the expert help needed to navigate the complexities of this issue, please visit our campaign “Save Our Medicine.”