By Kate Marino

While playing outside a few months before his ninth birthday in 2021, Damarius White fell down.

When his calf became enlarged, his parents took him to the nearest emergency room. Damarius then went to an orthopaedist for concerns of compartment syndrome, a painful condition that occurs when pressure builds in and around muscles. When that was ruled out, he was referred to Amy Harper, M.D., who is part of the Department of Neurology and Neurosurgery at Children’s Hospital of Richmond at VCU.

Within weeks, Damarius was diagnosed with Duchenne muscular dystrophy (DMD), a genetic disorder that affects the muscles, causing them to become weaker and deteriorate. This happens because the body isn’t able to produce enough dystrophin, a protein muscles need to work properly. Over time, DMD can impact walking, breathing and even how the heart functions. It occurs in males almost exclusively.

“To an extent his muscular dystrophy slows him down, but he’s not a quitter,” said Damarius’ mom, Michaella White.

While running and climbing stairs can be hard at times, his positive attitude and zest for adventure keep him going strong. His winning demeanor also put him on the path to work with researchers to discover the next generation of gene modifying therapy for other kids, by being the first patient admitted for a research study in the Children’s Tower. 

A hub for cutting edge pediatric inpatient care and research

As part of the study, Damarius is testing a new gene modifying therapy to skip exons that code for dystrophin. By skipping faulty areas of the code, partially functioning dystrophin protein can be produced.

The overall research focuses on the next generation of these gene modifying therapies. Newer methods allow for better and more efficient skipping to occur, and thus more proteins are made.

“The medication we’re trialing is designed to go into the cells and stay in the cells longer. The longer it stays, the more skipping it can do,” said Harper, who specializes in caring for kids with muscular dystrophies. “Not all similar treatments have the same skipping efficiency. These next generation skippers have the potential to further improve health and mobility in our boys with DMD.”

The medication in this trial is given by infusion in the hospital setting, so study specific labs and procedures can be done at precise intervals around the clock. With safety at the center, the entire CHoR clinical team also monitors for reactions to the new study medicine.

The timing worked out perfectly, with the opening of the Children’s Tower and Damarius’ eligibility for the trial. Overnight stays for pediatric research did not take place previously. Longer visits were limited by the outpatient pediatric unit hours.

“Before the Children’s Tower and the addition of the pediatric research unit nurse, we couldn’t offer studies that required inpatient pediatric admissions. This meant certain protocols were not a good match for us,” Harper said. “Children’s Hospital Foundation recognized the need for pediatric research everywhere across CHoR and funded a pediatric research nurse coordinator position that goes to the patient. The combination of the new environment and this position – this is BIG.”

As it turned out, a familiar face was the perfect one for the job. Megan Beatley, BSN, RN has been with VCU Health and CHoR for more than a decade, first as a pediatric hematology and oncology nurse and clinical coordinator, then in the bone marrow unit. She’s experienced in helping kids and families through complex health situations with leadership and grace.

In her new role, Beatley provides clinical care to pediatric research participants, oversees the operations of the pediatric research unit and educates nurses in the acute and intensive care units on the intricacies involved in caring for patients as part of research studies.

“I have a vision to grow pediatric research by helping study teams across all pediatric specialties at CHoR, both inpatient and outpatient, and facilitating collaboration between various research and clinical teams,” Beatley said. “I'm honored to be a part of the important work that's being done here and motivated by the growth I've seen already and that which is surely to come. Our efforts are expanding opportunities for pediatric patients."

11-year-old Damarius White loves baseball and cars. He was diagnosed with Duchenne muscular dystrophy in 2021 and receives care through the Children’s Hospital of Richmond at VCU. (Contributed photo)

Tracking pediatric trial progress, with plenty of playtime

Damarius stayed in the hospital for two nights, but the research isn’t done.

Harper and her team will continue to follow Damarius closely to track his progress. There will be medical checks, more infusions and motor assessments performed by our DMD-certified physical therapists. Damarius has also been seeing our nephrology team over the years for elevated kidney levels, benefiting from coordinated care from CHoR’s nationally ranked teams.

Damarius, who just finished fifth grade, enjoyed his time in the hospital, a testament to the attention put into making it kid friendly.

“The food is good, I like playing video games in the teen lounge and I get to miss school,” he said with a laugh. 

And the clinical trial isn’t stopping this baseball and car fanatic from enjoying summer break. Damarius is now busy playing outside, riding bikes and keeping up with his brother, sister, cousins and neighbors.

Harper’s goals for pediatric muscular dystrophy care align with this strength.

“With the new Children’s Tower, along with our patients, community and team members, the best is yet to come!”

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