OLYMPIA, Wash. — A medical breakthrough has been both a blessing and a blow to an Olympia family.
The Hay family includes two sons with the same form of muscular dystrophy, but only one of the boys qualifies for a newly approved treatment.
In typical 5-year-old fashion, Kellen Hay loves running around his backyard and is looking forward to Halloween.
Having an open space like this is vital for the family, where the boys can keep moving and walking.
Kellen Hay has Duchenne muscular dystrophy. It’s the most severe form of the disease that worsens over time, weakening muscles. Most patients don’t make it to 30.
“A lot of hospital visits and a lot of living day-to-day, rather than thinking about the future,” said Tristan Hay.
It’s genetic and Kellen’s older brother Liam Hay also has it.
“You kind of have to let go of what you thought your lives were going to be like,” said Heather Hay.
But this summer brought some hope, at least for Kellan Hay.
In June, the U.S. Food and Drug Administration (FDA) approved the use of Elevidys, the first gene therapy for Duchenne muscular dystrophy.
The FDA based its ruling on research that showed the treatment slowed the progression of the disease, but its use was only approved for patients who are 4 and 5 years old.
"It's kind of funny because so many people have been focusing on our good news, but there's bad news too," said Heather Hay.
Kellen is 5, but big brother Liam is 8.
“How do you explain this to an 8-year-old. Yeah, your brother gets this and you don’t,” said Heather Hay.
On Thursday, Kellen began receiving the gene therapy at Seattle Children’s hospital. He’ll go into isolation for a month before starting kindergarten.
Meanwhile, his brother is waiting and his parents are hoping that the FDA either expands its approval or there’s another breakthrough.
“But with this disease, it’s so kind of fast-progressing, we don’t have time to wait for long time studies to be done,” said Tristan Hay.
The FDA said approvals are made based on the risk and reward to the patients. The Hay family wishes they could make that choice.
“We understand the risk and the reward, so without anything," said Heather Hay.
"Without anything we know the outcome, so. Yeah. I’ll take almost anything to help them,” finished Tristan Hay.