Family of NC 5-year-old with rare disease searching for hope after drug trial ends

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CHAPEL HILL, N.C. (WNCN) — Sadie Haywood, 5, has a rare, incurable, neurological disorder.

For the last couple of years, she’s been one of six children in the world to participate in a clinical trial for an experimental drug.

Dozens of photographs document Sadie’s visits to UNC Hospital to take part in the clinical trial. She’s been there every week for the past two years.

The pictures show more than treatments. They reveal a little girl growing from a toddler to an energetic 5-year-old, with a smile that sparkles in her eyes and a love for Disney characters.

Watching Sadie’s development is pretty incredible to her parents.

“Where she is now compared to most children with Sanfilippo, she should not be where she is,” said mom, Ashley Haywood.

Sanfilippo syndrome is a rare, and terminal, genetic condition, sometimes described as childhood dementia.

Sadie’s doctor, Joseph Muenzer, a pediatric biochemical geneticist at UNC, has studied rare diseases, including Sanfilippo, for decades.

“It is a progressive neurologic disease,” he explained. “You are normal at birth, you start developing normal for a while, typically, then you start slowing down. You peak sometimes around three years of age development and then you regress.”

When doctors diagnosed Sadie, her parents realized what her future would likely hold.

“They typically don’t live past 15, 16,” said Ashley Haywood. “They start regressing and lose their ability to speak, to walk, to feed themselves.”

So far, her parents say Sadie hasn’t lost her abilities, and they believe the experimental drug she received has something to do with that. She was one of just six children in the world taking part in the clinical trial.

“It’s a miracle we got into that study,” said Ashley Haywood.

“I think it was probably helping her, but unfortunately now who knows what will happen,” said Dr. Muenzer.

Sadie recently received her last weekly treatment after the drug maker decided to stop the trial.

“We are giving this family hope and then, unfortunately, that hope disappeared,” said Muenzer. “They elected to stop before we had a lot of efficacy, effectiveness data and there was really no safety concern at that point. It was most likely some other reason, primarily probably a business decision that I have really no insight into.”

“I understand business decisions,” said Ashley Haywood. “But then it’s also like this kind of business decision affects more than just a business — it affects a child’s life.”

Sadie, her family, and her medical team celebrated her last treatment with party hats and pompoms, but their smiles hid a sense of sadness.

“Most likely, in time, she will start to regress, unfortunately,” noted Muenzer.

“It’s hard to imagine that, you know, in a couple of months she’s probably not going to be this good,” said Sadie’s mom. “It’s hard to picture that because she’s always happy and always smiling.”

The Haywoods still hope that another clinical trial may come along, and despite the way this study ended, they are glad Sadie got to take part in it.

“We’re still grateful that we got those two years,” said Ashley Haywood.

Now they’re making the most of every moment.

The family is working with the Cure Sanfilippo Foundation to try to raise money for clinical trials.

They are hoping to fund a gene therapy trial that could potentially help Sadie or other children like her. If you’d like to help visit, www.CureSFF.org/Sadie

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