Ask 6-year-old Wesley Cash Benham to show off his muscles, and a smile will spread across his face before he proudly holds up his arm, bending it so his bicep pops up on his small frame. He's strong in more ways than one, carrying the weight of his Duchenne muscular dystrophy (DMD) diagnosis.
“It is not curable at this time,” his mom, Tiffany Benham, said.
But Wesley's life is changing thanks to innovative research and treatment at Arkansas Children's Hospital (ACH). He is the first pediatric patient in Arkansas and one of only about 25 children in the United States to receive the FDA-approved gene therapy known as Elevidys, meant to extend the life of patients with DMD. The drug was approved in June to treat children 4 to 5 with a confirmed DMD gene mutation.
Aravindhan Veerapandiyan, M.D., known as “Dr. Panda,” a pediatric neurologist at Arkansas Children’s Hospital (ACH) and an associate professor of pediatrics at the University of Arkansas for Medical Sciences (UAMS), was the principal investigator on the Elevidys trial.
He is director of the comprehensive neuromuscular program and the Parent Project Muscular Dystrophy Certified Duchenne Care Center, as well as co-director of the Pediatric Muscular Dystrophy Association Care Center, all at ACH.
“Our hope is that this treatment is going to slow down the disease progression, and we can expect him to live longer,” Panda said.
The youngest of seven children, Wesley is their “angel baby,” said parents Tony and Tiffany Benham, who live in Manila, about 30 miles from Jonesboro.
"He knows that he's the baby and expects to get what he wants. He's a great kid. He's very loving," his mother said, with his father adding, "He cares a lot about everybody and everything. He worries for other people."
Wesley was 9 months old when he was diagnosed with DMD, a highly progressive, rare muscular dystrophy impacting boys. Females can be carriers of the DMD mutation.
"They usually present to us around the age of 4 to 5 years with muscle weakness. They're not able to run, they're not able to walk, they're not able to climb stairs. So that's how they typically present," Panda said. "It is a muscle disease. It affects your skeletal muscles and also affects your heart and the breathing muscles. So as time goes on, these kids tend to progress, deteriorate."
A genetic change in the DMD gene usually causes DMD. Boys with the disease have muscle weakness because they lack a "dystrophin protein."
As a baby, Wesley began having febrile seizures. While other hospitals could not figure out what was wrong, their family was finally given answers when they came to ACH in July 2018.
"His CK levels skyrocketed," his father Tony said. Tiffany added, "That's creatine kinase levels, and that's how much protein is built in a body. For a normal person, it should be under 50. For Wesley, it's at 31,000 as of last year."
Thomas Burrow, M.D., a pediatric geneticist working with genetic and metabolic disorders at ACH and associate professor in the department of pediatrics in the division of genetics and metabolism and a clinical and medical biochemical geneticist at UAMS, suspected two possible conditions that could be causing Wesley’s seizures, ultimately confirming DMD with additional testing.
"We came in, I think it was like a month later, and that's when he broke the news to me," Tiffany said. Tony, a truck driver, was traveling at the time. "He was on the road. ... He can't stop what he's doing because if he stops what he's doing, then our family stops moving."
The family learned DMD is an extremely rare disease in children. According to the nonprofit CureDuchenne, about 15,000 boys in the U.S. live with DMD and about 300,000 worldwide.
"It breaks down every single muscle in the body," and children typically end up in a wheelchair, his mother said. "The death causes are normally like they suffocate, or they die from heart failure."
Though there is no cure yet, Panda said the disease has typically been treated with steroids like prednisone or deflazacort, which slow down DMD's progression, allowing boys to walk longer and delay cardiomyopathy. This disease makes it harder for the heart to pump blood throughout the body. Before using steroids to treat DMD, boys would stop walking around age 9 or 10 and die by 18.
“Now we have boys living up to age 20 and 30 years old with the current treatment that we have,” Panda said.
On Aug. 11, Wesley received the gene transfer treatment Elevidys, a one-time intravenous infusion, at ACH.
"It's actually giving them a miniature version or a small version of this dystrophin protein that's already missing in these boys. So the research has been going on for the last five years or even more than that," Panda said. "We know that the boys who were treated in the research have actually shown improvements in the dystrophin protein itself, and also functional improvements, the way that they are walking and jumping and things like that."
Tiffany explained her faith in Panda made all the difference in the family’s decision to move forward with Elevidys.
"I said, 'What do you think?' He was like, 'Well, I think it's going to be great.' I was like, 'No, you don't tell me as a doctor, I want you to tell me as a parent, what do you think about this medicine?' He said, 'Well, I'm the doctor that worked on this medicine, and I approve of it, 100%,'" calling Wesley the "best prospect" for this medicine, Tiffany said.
Payment for the treatment had to be approved by Medicaid in just two weeks before Wesley turned 6, as Elevidys is set aside for 4- and 5-year-olds.
"Arkansas Children's Hospital worked pretty hard getting all of this approved. I'm thankful for it," Tony said.
Panda said they consider many factors before selecting a boy for this treatment, including his age, the specific genetic mutation required by the FDA to receive the treatment and the cooperation of the child and his family.
"There's a lot of safety associated with this drug, and they need more close follow-up and monitoring. I know they're a reliable family; they're very consistent that I can have them in a follow-up with me as much as I wanted to make sure that he's doing OK and he's safe after receiving the therapy," Panda said.
Since August, the family has traveled the roughly five-hour round trip once a week to ACH for bloodwork to check Wesley's organ function and side effects from the drug. Panda said Wesley has had no side effects or adverse reactions from the treatment.
"As far as Wesley's future care, I think we will continue to follow him in our multidisciplinary clinic with (a) team of providers as any other boy with Duchenne would get," Panda said. "It's kind of too soon to say how he's going to respond. But we would expect that this treatment would delay the disease progression and improve his lifespan and his quality of life."
For the Benham family, the long drive is a small price to pay for the care received.
Tiffany said ACH team members have done everything to make Wesley as comfortable as possible each time he visits, from playing games with him to bringing in Play-Doh, anything “to help him feel relaxed.”
“As a mother, there’s no better feeling in the world than to have a place to go that you feel safe and you can trust the people around you to help you with your child,” she said. “... We could have been in a situation not knowing that he had this and fight with his regular pediatrician and him slowly dying, and we don’t know it just because someone in our area didn’t know any better.”
Panda said the Benham family has been easy to work with, allowing him to check on Wesley as much as needed.
"He's a cute little boy. I love working with him. He's very responsive. He's very cooperative, and his family is always a pleasure to work with," Panda said.
The family's loyalty to ACH extends beyond Wesley's care. Tony explained ACH saved his daughter Elizabeth, who suffered seizures every 30 seconds as a newborn. She was transported by Angel One to the ACH Neonatal Intensive Care Unit and stayed six weeks.
"There's nothing like (Arkansas) Children's Hospital. He's a child. He's not a grown adult. So therefore, our surgeons down there wouldn't know what to do with him. So, this is our first option. Like I said, it's not the first child I've had that was sick," Tony said. "And I was a little nervous coming here the first time because I thought maybe the one in Memphis would be better for us; it was closer. But when we got down here, the doctors proved me wrong with Elizabeth. They really did. They had her on two different medicines within a week, saved her life. … I don't care about the drive. This is why we're here. I would walk it to come here."