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Damarius’ story: Children’s Tower supports next generation gene modifying therapy research for Duchenne muscular dystrophy
June 29, 2023
CHoR patient Damarius flexing with his muscular dystrophy research team

    Children’s Tower allows for research into the future of care for kids

    CHoR patient Damarius with his baseball trophyDamarius White helped us mark an exciting milestone as the first patient admitted for a research study in the Children’s Tower. The 11-year-old baseball and car fanatic and his CHoR team are working together to discover the next generation of gene modifying therapy for Duchenne muscular dystrophy, or DMD.

    From DMD diagnosis to enrolling in an innovative investigation

    Damarius fell while playing outside a few months before his ninth birthday. When his calf became enlarged, his parents took him to the nearest emergency room. He 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 Dr. Amy Harper on our neurology team.

    “Damarius first came to me in February 2021. Within about two weeks, I was able to diagnose his Duchenne muscular dystrophy and refer him to our outpatient interdisciplinary pediatric neuromuscular clinic,” said Dr. Harper.

    DMD is 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.

    Dr. Harper specializes in caring for kids with muscular dystrophies – and in shaping the future of muscular dystrophy care in children through research and participation in clinical trials. This study with 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. Damarius’ study focuses on the next generation of these gene modifying therapies. Newer methods allow for better and more efficient skipping to occur, and thus more protein 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 Dr. Harper. “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.”

    Stars aligned – clinical trial eligibility, a pediatric research nurse and a new home for care

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

    The timing worked out perfectly, with the opening of the Children’s Tower, the addition of a dedicated pediatric research nurse to the CHoR team and Damarius’ eligibility for the trial. He had to modify his current clinical treatment to meet the requirements. This preparation took several weeks, enough time for the research and inpatient teams to plan for the Children’s Tower opening and Damarius’ participation in the trial.

    A hub for cutting edge pediatric inpatient care AND research

    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,” explained Dr. Harper. “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 Megan’s new role, she 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.

    “My previous roles in nursing leadership helped me to be successful in the infancy of this new position,” said Megan. “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. 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, which is really exciting!"

    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.

    He’s also a fan of Dr. Harper, Megan, his nurses and research coordinators Mehreen Qureshi and Kathy O’Hara who work alongside Dr. Harper in child neurology.

    Tracking progress and success as the muscular dystrophy trial continues

    Damarius stayed in the hospital for two nights, but the research isn’t done. Dr. Harper and team will continue to follow him 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, benefitting from coordinated care from two of our nationally ranked teams.

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

    This summer, Damarius will be busy playing outside, riding bikes and keeping up with his brother, sister, cousins and neighbors. While running and climbing stairs can be hard, his positive attitude and zest for adventure keep him going strong.

    Dr. 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!”

    Discover how our teams are improving tomorrow’s care for kids through pediatric research.

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