PHOENIX (3TV/CBS 5) -- Last September, we took a look at how a brand new drug for muscular dystrophy is helping people with spinal muscular atrophy (SMA) maintain their muscle.
Now, just a year later, an even more promising drug was just approved for SMA. It could be just in time to save the life of one Arizona baby.
Stella Lackey was born this past May. Her parents thought she was healthy, and doctors at the hospital saw no signs of trouble, sending the family home a few days after her birth. But a few weeks later, their pediatrician noticed Stella didn’t cry or kick much.
"He asked us, 'Is she always this lazy?'" said Stella’s mother, Samantha. She and Stella’s dad Jeremy thought they just had an "easy" baby because she didn’t fuss much or kick them when they changed her diaper.
Their pediatrician suspected something else.
Stella was soon diagnosed with SMA, a form of muscular dystrophy characterized by muscle weakness. That weakness usually worsens with age.
Stella was already losing strength quickly. By six weeks old, she could barely kick her leg or move her arms. Her cries were soft and muted. Her tiny belly pumped up and down as she struggled to breathe.
"No parent should have to ask their doctor how long their child has to live," said Samantha. "And Dr. Bernes told me right away, 'No, we're not going to talk about that, because there's treatment now.'"
At six weeks old, Stella made history, becoming the first baby in Arizona to receive Zolgensma.
"This is probably the biggest thing I’ve seen in my 32-year career here," said Dr. Saunder Bernes, a pediatric neurologist at Phoenix Children’s Hospital.
"This drug is basically a gene that’s missing in these children, and this gene has been put into a viral vector." said Bernes. "It takes a gene into your body and gets it into the places where it’s not working."
Stella’s parents noticed almost immediate changes. Her cries were getting louder. Her legs were kicking again and she was breathing more easily.
"The first time we saw her head move," said Stella’s father Jeremy Lacky, "that was big."
Dr. Bernes noticed changes right away too. "Stella was diagnosed quite early," he said. "She was quite weak. And the response to this has been quite dramatic in that we could see improvement literally in the first week."
In clinical trials for Zolgensma, children diagnosed within the first few months after birth were later able to feed themselves and breathe on their own. But for babies diagnosed immediately after being born and then given the drug, they were able to walk, even run a few years later.
Stella’s dad Jeremy says he hopes she will be able to live as normal of a life as possible. "It might not be walking, it might not be running or playing sports, but being able to adapt to the future and being able to live as normal a life as possible. That’s what I’m hopeful for."
Stella’s mom shared a similar hope for Stella. "That she will continue to be strong and that she will continue to change the world."
Stella and Jeremy, along with Dr. Bernes would like to see SMA added to the newborn screen in Arizona. Currently, 7 states have this in place, and it could be added in Arizona in the next year or two.
But Dr. Bernes calls it "horrific" to wait that long. He says babies born in Arizona should have the same fighting chance as babies born in other states.
For more information, check out the Cure SMA Facebook page here.
And to support Stella and her family, check out "Strong Like Stella" on Instagram.