Meet Kid Captain Maggie Larson
Although Maggie Larson and her twin brother, Will, were born five weeks prematurely, the Urbandale, Iowa, siblings were hitting early developmental milestones.
“Will was developing his gross motor skills and Maggie was developing her fine motor skills and her speech earlier,” her father, Samuel, says.
“She talked and everything,” her mother, Heidi, adds. “She spoke in full sentences.”
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But Maggie struggled when it came to walking. Heidi says Maggie’s legs would just crumble out from under her. The Larsons initially suspected Maggie’s difficulties in walking were related to cerebral palsy or a stroke, but an MRI came back as normal.
Eventually, the family got an answer, albeit a difficult one to hear. Maggie was diagnosed with a rare, terminal disease—metachromatic leukodystrophy—in February 2020, when she was almost 3 years old, at a children’s hospital in Minnesota.
Losing hope, gaining knowledge
Metachromatic leukodystrophy causes fatty substances to build up in cells, particularly in the brain, spinal cord, and peripheral nerves, due to a deficiency of an enzyme that helps break down lipids. Maggie’s parents were told she had a short life expectancy, and nothing could be done.
“We had just been told that not only did our daughter have this debilitating disease that would rob her of her ability to play and move and speak and more, but also that she had a life expectancy of two to five years, and we should just do our best to keep her comfortable,” Heidi says. “We were not given much in the way of hope or promise that Maggie would have anything resembling a normal childhood or life. It was soul-crushing.”
Holding her daughter and sobbing, Heidi searched for information about the disease online and came across a clinical trial at University of Iowa Stead Family Children’s Hospital that might help slow its progression.
“We met an amazing group of individuals who were so caring and loved her the moment they met her,” Heidi says. “They just wanted her to be OK.”
“They were deeply invested in giving her the best care,” Samuel adds.
Slowing disease progression
John Bernat, MD, PhD, a clinical geneticist at UI Stead Family Children’s Hospital and one of the only people in Iowa who studies the disease, told Maggie’s parents that even if she didn’t enter the clinical trial, he wanted to continue following her because of the rarity of the condition. It’s estimated between one in 40,000 to one in 160,000 people have the disease.
Under the clinical trial, Maggie receives weekly infusions of the enzyme her body lacks into the fluid surrounding her brain and spinal cord, which her parents believe have helped improve her symptoms and quality of life.
“The goal of the study is to slow down the progression,” Samuel says. “And we think we’ve seen that.”
“I think our expectations have become more realistic,” Heidi adds. “We’ve seen more movement out of her. We’ve seen more communication. She smiles. She thinks it’s hilarious when her brothers get in trouble.”
And just this year, Maggie began giving the “thumbs-up” sign at appropriate times. “That’s huge,” Heidi says.
“We have found a support system that has given us hope and that has helped Maggie thrive. We cannot say enough about the care she and we have received.”
Now 6 years old, Maggie is in first grade and absolutely loves school and being around other students and staff. “She has taught us to love with reckless abandon, because we know that our time is limited,” says her mom, Heidi, “and she’s taught a lot of other people that, too.” (Photo courtesy of Heidi Larson.)
A small yet global community
Worldwide, just 42 children were expected to enter the clinical trial. The families form a very small community, connecting with one another from all corners of the world through Facebook groups.
Since her diagnosis, all of Maggie’s care has been transferred to UI Stead Family Children’s Hospital, including Botox injections meant to relax her muscles to lessen cramping.
“The clinical research team has become like family to us,” Heidi says. “They want her to live life fully and do all the things that kids should get to do. They have gone above and beyond for our family and for her care. And it hasn’t stopped with research. This has been our experience throughout the entire hospital. The different specialties work so well together and come together to make a plan that works well for Maggie and keeps our goals for her at the forefront.”
Samuel remembered a special day at the hospital.
“When Maggie could still speak and you’d ask her what her favorite color was, she’d say ‘rainbow,’” he says. “They all dressed up as a rainbow for her birthday by wearing colors of the rainbow, with rainbow-colored balloons in the room.”
University of Iowa Stead Family Children’s Hospital is home to the only comprehensive medical genetics and genomics services in the state of Iowa. Our experienced teams contribute to the diagnosis and care of children and adults with rare genetic disorders. We strive to provide the best, evidence-based and comprehensive health care services to Iowans.
Feisty, sassy girl
Now 6 years old, Maggie is in first grade and absolutely loves school. Maggie loves being around other students and staff. She also likes “girlie” things, including Barbies and cheerleaders, and has her own pom-poms.
“She is feisty. She is sassy,” Heidi says. “Maggie communicates with her eyes. If she doesn’t like what you’re saying, she’ll repeat ‘no’ to you over and over (by closing her eyes) and finally look away. She’s hilarious.”
Her parents attribute Maggie’s quality of life primarily to the care she has received at UI Stead Family Children’s Hospital.
“We have found a support system that has given us hope and that has helped Maggie thrive,” Heidi says. “We cannot say enough about the care she and we have received, and, in spite of how hard it is to have a terminally ill child and know that their life is limited, we are so grateful that they are doing everything to make sure that her life is full and that she has every opportunity to be a kid.”
“She has taught us to love with reckless abandon, because we know that our time is limited,” Heidi adds, “and she’s taught a lot of other people that, too.”
