By SABRINA BERNDT

Washington Daily News

 

Nicky and Brandon Hosey knew something was wrong when their son, Wade, did not show signs of walking by 12 months, though their doctor told them that everything was normal.

At 18 months, they grew more concerned and put Wade into physical therapy, but his physical therapist found he had the leg strength of an 11-month-old and recommended genetic testing. The results proved Wade had Spinal Muscular Atrophy Type 2, or SMA.

“You have a whole range of emotions,” Nicky Hosey said. “You never expect it to happen to your kid. And then when you read about how rare this is and how serious it is, I think it makes you appreciate everything even more.”

According to the SMA Foundation, the disease is the result of “the absence of or defect in the Survival Motor Neuron 1,” which causes muscle atrophy and loss of motor function. The SMN protein is responsible for the nerves cells in the spinal cord that help muscles contract.

It affects approximately one in 10,000 infants, and one in 50 people are carriers for the disease in the SMN1 gene.

“In (your) spinal cord, you’ve got all these chromosomes, and (Wade) is missing one,” Nicky Hosey said. “He’s got an SMN1 gene and an SMN2 gene and the two should back up, but as far as well know, he does not have a one, which is why he has this disease.”

Due to the rarity of the disease, there are few treatment options. The first approved drug for treating SMA was Spinraza in 2016, a spinal tap injection that is proven to slow the disease’s progression. However, each injection costs $125,000, and injections are given every four months, making the process costly.

A new gene therapy treatment, on the other hand, was released on the market in May and is proven to be cheaper that the alternative in the long run — Zolgensma.

“It sits beside his SMN1 gene, which he’s completely missing, and feeds it protein,” Nicky Mosey said. “It’s the closest thing they have to a cure. … These kids that could never do anything, they sat there and physically could not move, they can sit up and they can move their torso.”

Zolgensma is a one-time injection that is known as a miracle drug, but it can only be administered to children younger than 2 years old and comes with a hefty price tag that the Hosey family cannot afford — $2.1 million, making it the most expensive drug on the market.

According to the Institute of Clinical and Economic Review, an organization that analyzes the monetary value of pharmaceutical drugs on the market, the value of Zolgensma is $1.2 million to $2.1 million. The estimation is based on their “per life gained” benchmark, which values drugs based on the number of years gained after administering the drug during the clinical trial.

The value is also based on the assumption that insurance companies will cover a portion of the cost, but Hosey’s situation says otherwise.

“Insurances are not willing to pay for it,” Nicky Hosey said. “It’s pretty much impossible to meet (their criteria for coverage). You’re not going to tell me that my kid’s life is not worth $2.1 million.”

To cover the outstanding cost, Nicky and Brandon Hosey created a Go Fund Me fundraiser, allowing anyone to donate to their cause. They are also hosting local fundraisers, including a fundraiser at Copper Canyon Wellness on Sept. 7-8.

“When I tell you the outpouring of support we’ve gotten in less than 24 hours, it will blow your mind,” Nicky Hosey said. “This small town has started to come together in 12 hours for somebody that they don’t know. … I just don’t want other parents who are dealing with this to be forgotten.”

For more information about Wade and SMA, along with a link to donate, visit www.walkingwithwade.com.