Hope for the Haslams: UNM offered $6 million gene therapy treatment to two young Rio Rancho brothers

RIO RANCHO — The Haslam family knew early on that their 10-year-old and 12-year-old were suffering from muscular dystrophy, but what they didn’t know is that one day, the University of New Mexico Hospital would administer the two boys a $6 million gene therapy treatment and would be the first in the state to receive the treatment.

For years, the boys’ mother, Danielle Haslam, and father, Aaron Haslam, followed news on gene therapy — a medical treatment that can prevent or treat diseases by altering a person’s genetic makeup — after learning both their sons were suffering from Duchenne muscular dystrophy (DMD).

“It causes weakness over a long period of time in childhood, and ultimately leads to heart failure, or potentially lung failure,” said Meeta Cardon, a child neurologist and pediatric neuromuscular specialist at UNM Children’s Hospital. “Although we have had some treatments come out over the last several decades, we don’t yet have a cure for this condition.”

The disease is most common in boys, who make up nearly all cases. The disease affects approximately 1 in 3,500 to 6,000 live male births and 1 in 50,000,000 live female births, according to the National Library for Medicine.

The diagnosis isn’t always in childhood, and many people live with it for years before being diagnosed. The family realized early on when the oldest of the two brothers struggled with walking that he might have Duchenne’s. After testing, the family learned that if one sibling has the disorder, another might have it. They tested the younger Haslam brother and discovered he also had Duchenne’s.

“We didn’t really know anything about this when our sons were diagnosed,” said Danielle. “They were 18 months and 2 years old, not walking and having a hard time getting off the floor, different things like that. We caught it at a young age. Most of the time, kids don’t get diagnosed until they’re 4 or 5 years old.” Currently, the life expectancy of people diagnosed with muscular dystrophy is around 30 years old.

After learning the Haslam brothers would require medical attention and regular check-ups to keep an eye on the disease, the boys began meeting with Cardon. When Cardon learned that the gene therapy treatment — Elevidys — was approved for all ages, the Haslam family worked closely with her to begin the process of treatment.

Elevidys can increase a patient’s life expectancy drastically, can extend the length of time the brothers will be able to walk and is the only treatment available for Duchenne muscular dystrophy. Though doctors cannot guarantee a specific result, the medication slows the progress of the disease based on how the patient’s health is currently.

Although the Rio Rancho family was feeling hopeful, there was one problem. Elevidys is a one-dose treatment patients receive once in their lives, and each dose costs $3 million. UNM Hospital decided to cover the bill for the family to make sure they received treatment as soon as possible without having to travel to another state.

“It was definitely a huge relief,” said Aaron, who is a clinical psychologist for Veteran Affairs. “UNM was willing to go out on a limb and front the money. There’s no way we could have done that.”

The treatmentOn Sept. 18, the Haslam brothers received their Elevidys treatment, being the first in the state to do so. The two arrived bright and early at the hospital and received the treatment through an IV infusion that took roughly one hour to administer. UNM provided the 10- and 12-year-olds with Legos and video games to pass the time and made sure to monitor them through the afternoon. The family was able to return home later in the evening.

“The company that produces the medicine, Sarepta, worked really closely with the medical team to make sure they knew what they needed to do, what procedures (will) follow,” Danielle said. “Even on (the) day of the treatment, they had a guy come out and was there the whole time making sure they were doing it correctly. They’re definitely a top-notch medical team.”

Following the treatment, Cardon texted and called the family regularly to check on the boys and see how they were holding up. Because the medicine was recently approved, UNM wanted to give the family the opportunity to try the treatment and see how it affected the Haslam brothers. UNM had also worked with the brothers for several years, and both the Haslam family and UNM staff felt comfortable because they had extensive knowledge of the history of the boys.

“Gene replacement therapy patients might feel kind of like flu-like symptoms within the first week or two,” Cardon said. “They might feel a little bit sore, pretty fatigued, low energy, they might feel a little bit nauseous, a little sick to their stomach and they might throw up. Over the period of time that we’re monitoring post-infusion, there are different things that can come up at different time periods.”

While the oldest of the two Haslams recovered following his flu symptoms, the other required additional hospital visits to receive steroid infusions following liver complications, a common side effect of Elevidys. He will continue his remaining steroid treatments but has recovered gradually since. The boys’ last appointment was Jan. 9, and since then, they both said that though the effects are minimal so far, they have begun to feel better and have more energy.

“It’s a huge relief,” Aaron said. “It’s really rare for someone with this disease at their ages to even be walking and almost impossible to go up the stairs. They’re still able to walk, and to be able to preserve that is a modern miracle.”

While the future of the Haslam brothers isn’t clear, as modern medicine develops and the boys feel the effects of the gene therapy, the Haslam family feels hopeful.

“I just think they’ll have more opportunities,” Danielle said. “Most boys get to maybe 30 years old, but they’ll have the chance to go to college and hopefully have more options for different careers and just have more of a future that maybe they wouldn’t have had before.”