Fighting for Clara and a cure

In a matter of weeks, Ryan and Jenny Bragg went from uncertainty to despair to an unexpected hope.

The Ross Bridge couple’s daughter, Clara, had started as a healthy baby, but at about 14 months, she stopped hitting the right milestones: She wasn’t walking independently, had stopped speaking and wasn’t using nonverbal communication. The Braggs enrolled Clara in therapy at the Bell Center and began the hunt for a diagnosis. After neurologists, geneticists and rehab specialists, a full genome sequencing revealed in August 2016 that Clara has GM1 gangliosidosis.

The disease, Ryan Bragg said, is similar to Tay-Sachs disease and destroys brain function through an inability to process and break down material in neural cells. 

GM1 causes trouble with speech and mobility, and later more severe problems such as blindness, deafness, seizures and inability to eat. Jenny Bragg said the diagnosis meant instead of being upset their 2-year-old daughter wasn’t progressing, now they realized they should be glad that she wasn’t moving backward.

“That was something to be celebrated,” Jenny Bragg said.

A diagnosis of GM1, however, is always fatal. In Clara’s case, her late infantile case has an expected life span of 5 to 10 years. Ryan Bragg recalled being told their family had no options except to go home and enjoy their years with Clara while they had her.

When they began researching advice on caring for a child with GM1, the Braggs were surprised to find a cure might be closer than they thought. 

At Auburn University, veterinary medicine professor Doug Martin has been studying GM1, which also occurs naturally in cats, for several years. He developed a gene therapy treatment that was successful in cats and is now ready for a National Institute of Health trial for a similar treatment in humans.

“He’s been able to save a lot of their lives; he’s been able to restore a lot of their functions,” Ryan Bragg said of Martin’s work with felines.

When they found out about Martin’s work in September 2016, Ryan Bragg said one of the major hurdles for the drug trial to start was about $1.5 million in funding. Less than a month after a diagnosis that they thought spelled the end of their daughter’s life, the Braggs jumped with both feet into fundraising for a concrete cure.

“At this point, it’s a matter of when, not if. Unfortunately for kids with this disease, they don’t have time. A few extra months can matter a lot,” Ryan Bragg said. “If there (are) delays, it’s not going to be because they can’t pay the bill.”

Since that time, the Braggs and other families with GM1 children have raised about $1.1 million through private donations and a variety of silent auctions, restaurant nights, fashion shows, cookie sales, movie nights and other fundraisers.

“Our goal is just, ‘Let’s make this trial happen,’” Jenny Bragg said.

Day-to-day parenting of Clara is difficult, her parents said. She’s developmentally a 1-year-old, despite being nearly 3, and she doesn’t use verbal or typical nonverbal communication. Jenny Bragg said her daughter expresses most things with her face and understands simple commands, but it’s hard to tell how much she’s able to comprehend of the world.

“It’s very hazy in terms of trying to figure out exactly what she understands, and maybe exactly what she chooses not to do,” Jenny Bragg said.

Clara can walk with the help of a walker, but she falls easily and can’t be left alone.

“There’s a lot of hazards for her,” Jenny Bragg said.

“It’s hypervigilance. There’s no such thing as ‘Just let her play,’” Ryan Bragg agreed.

This can take a toll not only on Ryan and Jenny Bragg, but also on their 5-year-old son Tanner. Meeting Clara’s needs means they don’t always have the free time they’d like to spend with Tanner, but Jenny Bragg said he’s always loving to his little sister and willing to help in small ways.

Ryan Bragg said they’re in a unique situation because they aren’t fundraising for a “someday” cure, but for a specific trial with a background that leaves them optimistic for success. If fundraising and other hurdles are cleared, he said the trial could begin as soon as the end of 2017. 

If Clara is accepted as a trial patient, she would receive a one-time gene-therapy treatment and be closely monitored for a few years, then remotely monitored for up to 13 years to track results.

“This, I think, is a unique opportunity [and] a remarkable opportunity to solve something forever. In three or four years, people are going to be talking about GM1 the same way you talk about polio or smallpox or tuberculosis,” Ryan Bragg said.

“That is what our sights are set on. From everything we’ve seen, this is it. This is our shot,” Jenny Bragg said.

The Braggs’ next fundraiser will be a party for Clara’s third birthday from 7-11 p.m. April 8 at the Redmont Hotel. Tickets for the adults-only cocktail are $75, and there will be drinks, food, music, a silent auction and a Kendra Scott jewelry pull.  For more information on the event, go to acureforclara.com.