CURE FOR CLARAHow trial could save a child with terminal diagnosis
Written by Lauren Dowdle
She might never take her first steps, sing a nursery rhyme or learn how to ride a bike. Those missed milestones are just reminders of the cruel progression of Clara’s disease, not that her family could forget.
But from that pain comes a determination and hope that could save this 3-year-old’s life and many others.
All that’s missing are the funds to make that dire prayer a reality. And raising awareness for that need is a daily mission for Ryan and Jenny Bragg.
The Braggs moved to Hoover for Ryan’s job at Frito-Lay, a division of PepsiCo, where they now live with their two children: Tanner, 5, and Clara, 3.
But life as they knew it changed last year when they received news no parent wants to hear: Their youngest had a terminal disease.
The couple first started noticing something wasn’t quite right when Clara was about 14 months old. They became concerned because she hadn’t started to walk on her own, instead falling when she would try to take steps, Jenny says.
“We then began noticing that her fine motor skills were not where they should be and that she had plateaued developmentally across all areas,” Jenny explains.
Soon after that, they began realizing Clara was actually regressing and losing skills she had once mastered. So, they began a year-long journey searching for a diagnosis.
It was during a research study in August 2016 with Hudson Alpha and UAB when Clara received her diagnosis: late infantile GM1 gangliosidosis. The results came after they analyzed Ryan and Jenny’s DNA, along with Clara’s DNA, and found both parents carried the recessive GM1 gene — and Clara inherited both.
GM1 gangliosidosis is a terminal, inherited disorder that progressively destroys nerve cells in the brain and spinal cord. Clara’s life expectancy is 10 years, as the disease will slowly take away her skills. Late infantile GM1 is found in 1 in 200,000 to 250,000, Jenny says.
“We pour all of our emotions into fundraising, as this cure and other promising research are our only options to potentially saving her life,” Jenny says. “We’re very action-oriented. There’s promising research, and we’re very grateful for that.”
The family has held a variety of fundraisers since receiving the diagnosis last year, raising $1.7 million for GM1 research. All of the money they raise goes straight to the nonprofit Cure GM1 Foundation and is 100-percent tax deductible. And that money could directly save lives in the very near future.
“Clara is very determined. As difficult as it is for her to move her body in the way she wants to, she is constantly trying and never gives up.”Jenny Bragg
Unlike many other terminal diseases, someone has actually found a cure for GM1. The family has focused specifically on Dr. Doug Martin’s research from Auburn University since he’s cured the disease in cats, allowing them to live normal lives.
Researchers just need the funds to move from animal testing to a human trial — something that could save Clara’s life.
“It’s the first time in history a terminal disease has been cured with gene therapy,” Jenny says. “They just need the funding to go to trial.”
Jenny says they’re hopeful the trials will begin for humans in the first or second quarter of 2018. Once trials begin, Clara would have to qualify to be a part of them. So, the sooner they start the better, since she’s gradually losing her skills and abilities, which could potentially disqualify her from participating.
While the odds may seem to be stacked against Clara, she definitely doesn’t let her diagnosis hold her back. “Clara is a very social little girl,” Jenny says. “She loves being around other kids and adults.”
Although Clara gets frustrated when she’s unable to do certain things — like walk or pick up things — she doesn’t know she’s sick or different than other children, Jenny says.
“She is a very happy child and always ready to play,” Jenny says. “She wants to move all of the time. She can crawl and bounce on her knees.”
Clara continues to impress her family with everything she does, and they’ve learned not to take any moment for granted.
“Clara is very determined. As difficult as it is for her to move her body in the way she wants to, she is constantly trying and never gives up,” Jenny says. “She is feisty and opinionated, in spite of her being nonverbal. She has a very strong personality: It’s amazing to see.”
To learn more about Clara’s story or support the GM1 trial, visit Acureforclara.com.