In 2004, I started receiving care at Cincinnati Children’s Hospital Medical Center (CCHMC) through its neuromuscular clinic. Luckily for me, this was a time when great advances were being made treating patients with Duchenne muscular dystrophy (DMD).
One of the first neurologists I consulted with was Brenda Wong, MD. She continues to be a leading physician for DMD and now oversees a clinic at UMass Memorial near Boston. I recently spoke with Dr. Wong to learn about how she had a pioneering impact on the treatment and quality of life associated with the disease.
Two decades ago, treatment for DMD was like night and day when compared to current treatments. Wong explained the clinic at CCHMC included only 2 primary specialists – a neurologist and rehabilitation doctor for obtaining medical equipment.
“I felt like it was not adequate to just pronounce the diagnosis with nothing else to offer,” Wong said.
An inflection point occurred when contact was made with Parent Project Muscular Dystrophy (PPMD), a charity that advocates for families of DMD patients. PPMD’s involvement contributed to the use of steroids as a treatment that revolutionized how the disease progresses for the better.
“I got to see what happens outside the clinic where there are families,” Wong said.
Over the next 5 years, the groundwork was laid for a more multidisciplinary approach to treating DMD. Finding a way to effectively maintain focus on the neurological, cardiac, and pulmonary trio of muscles the disease damages most was pertinent.
This involved coordination between specialists in each discipline. A pivotal moment occurred when a single space in the hospital became dedicated to bringing these doctors together for consultations with their patients in a single visit. Later, a dietician, genetic counselor, physical therapist, and social worker were added to this rotation.
“Some patients alluded to us as a team being the quarterback for their care,” Wong said.
Through 2010, the neuromuscular clinic at CCHMC experienced a period of rapid growth in terms of the reach to patients from across the country. At one point three-fourths of patients visiting were outside of the hospital’s local region, Wong said.
The advances in care for DMD patients eventually earned recognition for its specialized treatment by the National Committee for Quality Assurance – an organization that assesses the efficacy of medical facilities around the country. It was also named a Certified Duchene Care Center by PPMD.
Through all the successes, it’s witnessing her patients have positive health outcomes and live a fulfilling life that brings pride, according to Wong.
“It’s great to see the boys grow up and do well. A former patient wrote an essay that I will never forget. He said, ‘If you can’t stand up, then you can stand out,'” Wong said. “It has been an honor and a privilege to be growing up with the families. I feel like a grandma.”
The milestones reached under the watch of doctors such as Wong have made it possible for clinical trials focusing on DMD treatments to expand. In 2006, the first US gene-therapy trial for the disease was conducted at CCHMC. Several of the largest pharmaceutical companies, such as Pfizer, are now involved in research.
At CCHMC, a variety of conditions that fall under the neuromuscular umbrella are treated. In 2018, Wong moved on to UMass Memorial, so she could realize her dream of starting a clinic that caters mostly to DMD care.
Since becoming a founding member at her new practice, Wong has served pediatric to adult populations of DMD patients and carriers of the disease. The only other condition treated is Becker Muscular Dystrophy, a disease that acts like Duchene but progresses over a longer period of time.
When asked what keeps her motivated to pursue DMD treatment and research towards a cure, Wong says initially it was just a job. But now she is motivated by a desire to see her patients have positive outcomes.
“That human aspect of seeing them do well reinforces that what we’re doing makes a difference,” Wong said.
From my perspective, the work Wong has been a part of is a fine example of how a rare disease can effectively be treated when a multidisciplinary team approach is adopted. It gives providers an opportunity to share knowledge from their points of view in order to coordinate the best possible treatment plan.
I’ve also been struck by the passion and compassion some of the doctors I’ve encountered have shown. I can still remember Wong helping me feel calm when going through difficult experiences at the hospital. And having doctors that join together for a common passion is necessary in ultimately solving what used to be seen as incurable.