She’s 14 months old and needs a drug that costs $2.1 million to save her life

At first, Ayah Lundt was a typical, healthy baby. She was born at 8.3 pounds. At six months, she crawled and tried new foods. However, at 9 months of age, her development hit a steep decline. She stopped lifting her head, clapping, or sitting on her own. Whenever she tried to crawl or stand up, her legs buckled. Despite being able to eat oatmeal with a spoon multiple times before, she suddenly couldn’t. Then she was diagnosed with spinal muscular atrophy. And to treat it, she needs a drug that costs $2.1 million. [1]

Baby Needs a Drug That Costs $2.1 Million

Spinal muscular atrophy is a rare genetic disease that affects one in 10,000 babies all over the world. It’s caused by a defective gene that kills nerve cells, causing the children’s muscles to deteriorate, including the muscles that allow them to swallow and breathe. The infants affected often suffer from finger tremors and curved spines. They can’t stand or walk on their own. Many pass away from respiratory failure while still very young. [2] Zolgensma is the treatment for this disease, and at $2.1 million a dose, it’s considered the world’s most expensive drug. 

“I think about the cost every day, and it weighs me down,” said Ayah’s mother, Mary Mithika, from Bornholm, Denmark. “And then I look at Ayah and see her getting worse. As a parent, what would you do if you knew there’s something out there that can save your child?”

Even so, Zolgensma is not a guaranteed cure. According to her doctors, it’s unlikely that Ayah will ever walk again. But the treatment could improve her muscle movement and function. However, there’s another obstacle aside from the exorbitant price. Zolgensma is not approved under universal health care in Denmark. Ayah’s parents, Mithika and Frank Lundt, need to have her treated in the United States, where it’s approved for kids aged two and under. They’ve already contacted Boston Children’s Hospital about the treatment. However, during a year of a pandemic and financial difficulties, the family struggles to attain the funds.

“We just want what every parent wants — the best for their child,” Lundt said.”We were so excited about having another princess. She is perfect. She has always been such a happy child, even now with all this going on.“

More About Spinal Muscular Atrophy

There are four types of SMA that all appear at different ages. Therefore, the symptoms of SMA depend on the type and the severity of the case. Common symptoms are:

• Difficulty standing, walking, and sitting up
• Muscle weakness and twitching
• Changes in the shape of the chest, spine, and limbs
• Problems with swallowing and breathing

Since SMA is degenerative, the symptoms usually worsen as time goes on and muscles become weaker. It’s a genetic disease that requires both parents to be carriers for it to affect their child. Even so, there’s still a 1 in 4 chance their child will have it. [3]

Why The Drug Costs $2.1 million

Zolgensma is a type of gene therapy only given to children under two years of age. At 14 months, Ayah doesn’t have much time to receive this infusion. However, it’s her best chance at a cure.

“The potential for gene therapy products to change the lives of those patients who may have faced a terminal condition, or worse, death, provides hope for the future,” the FDA wrote in its approval letter. “Children with SMA experience difficulty performing essential functions of life. Most children with this disease do not survive past early childhood.”

According to Dave Lennon, president of Novartis Gene Therapies, which developed the drug, the exorbitant price is relative to the value it brings to the health care system. It’s also relative to the cost of long-term care of patients with SMA. As he explained, “Treating and caring for patients with SMA can cost up to… $6 million in the first 10 years of a patient’s life.”

However, Zolgensma isn’t sanctioned in Denmark as the Danish Medicines Council debates whether the price should be reimbursed by their universal health care system. Right now, Ayah’s parents are waiting for Denmark to agree to cover the high cost. The other option is to raise the $2.1 million themselves.

Ayah Waits For Treatment

Remember, Ayah has until her second birthday; then, she won’t be able to receive the treatment. The family set up a GoFundMe page to raise money for the drug that costs $2.1 million, as well as the additional travel and medical expenses. In the meantime, Ayah receives breathing treatments twice a day, a preventative therapy for her lungs. Ayah dislikes the apparatus, so her mother sings “You Are My Sunshine” to calm her as she places it over her daughter’s face. She also receives medicine that doesn’t seem to help. Her parents also help her with activities to try and strengthen her muscles.

“She’s like a four-month-old trapped in a 14-month-old’s body,” Mithika said. “She gets frustrated she cannot move. When her older sister dances around her, she tries to join her from the wheelchair, but she can’t. Every patient is different. But in just the last few weeks, she’s deteriorated so much.” [4] In addition to their GoFundMe, the family set up a social media campaign called “Friends of Little Ayah.” There, they share videos of the baby and encourage people to donate.

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