Miller School of Medicine Named Wilson Disease Association Center of Excellence
The rare but treatable disease results from excess copper overwhelming the liver, brain and other organs, leading to serious and even fatal complications.
A team of 11 University of Miami Miller School of Medicine specialists under the co-direction of Jason Margolesky M.D., and Cynthia Levy, M.D., have seen their hard work pay off as the school was recently designated a Wilson Disease Association (WDA) Center of Excellence.
The team includes specialists across disciplines including hepatology, neurology, ophthalmology, physical medicine and rehabilitation, speech and language pathology, nutrition, neuropsychology and social work. With fewer than 15 Centers of Excellence worldwide, this distinction will play a major role in helping those suffering from Wilson disease find specialists, enabling the team to treat a much wider range of patients.
About Wilson Disease
Wilson disease is an inherited liver condition during which the body retains excess copper. The copper overwhelms the liver, brain and other organs, leading to neurological, psychiatric and liver complications.
Currently, one in 30,000 people are living with the disease. Symptoms usually arise during childhood, adolescence and early adulthood (though later onset occurs, as well). Diagnosis can be made through blood, urine and genetic testing, and treatment initiated prior to symptom onset by screening a patient’s siblings.
To receive the WDA Center of Excellence designation, institutions must meet criteria including:
• At least one physician dedicated to Wilson disease patients
• Physicians well-trained in diagnosis and treatment
• Clinical diagnostic laboratory studies for metal analysis and related testing
• Wilson disease education for rising physicians
• Access to clinical trials to advance medical and surgical treatments
• Patient, family, public and continuing medical education
Basic research into the underlying pathophysiology of disorders of copper metabolism is required, as well as new experimental therapies for the disorder. Centers must also serve as an educational and consultative resource to Wilson disease patients and those who treat them.
Dr. Margolesky, director of the neurology resident program and associate professor of clinical neurology at the Miller School, shared what it means to join this prestigious group.
“Being named a WDA Center of Excellence is really exciting and a long time coming. It highlights just how much of a need there is for these centers, since there are so few of them,” said Dr. Margolesky. “This is a wonderful recognition for the work we’ve been doing that allows us to expand our care for a group that needs help.”
A Crucial Service for Patients
The center team will connect patients with specialists across different disciplines. Researchers have participated in two recent studies, one of which is ongoing. The first study explored pharmaceutical-driven genetic treatment, while the other focused on a new method to measure copper levels in blood. The University of Miami hopes to continue to be a part of the research pipeline in Wilson disease, with the WDA Center of Excellence designation attracting future studies and investigators.
Dr. Levy, the Arthur Hertz Chair in Liver Diseases, director of the Schiff Center for the Liver Diseases and professor of clinical medicine at the Miller School, explained the vital role the WDA Center of Excellence will play in getting patients the care they need.
“The Center of Excellence team will serve as liaisons for people living with Wilson disease, putting them in touch with the right specialists to pave the way for earlier diagnosis, optimal treatment and improved outcomes,” said Dr. Levy.
The Miller School was previously named a WDA Center of Excellence, but when center director and hepatologist Dr. Tamir Miloh abruptly passed away, the center paused operation. For Dr. Margolesky, earning back the Center of Excellence distinction was the culmination of tremendous effort.
“It’s hard to find physicians willing to dedicate their time and interest to a rare disease. The engagement from our team made all the difference,” said Dr. Margolesky. “This is a very treatable disease, if caught early enough, when paired with a zinc supplement and the few available medications on the market. But early detection is our main goal.”
Now officially a Center of Excellence for a second time, the team aims to ensure Wilson disease is something all Miller School students learn about during their matriculation. Looking toward the future, the team hopes to grow the center by adding a psychiatrist and a dedicated geneticist, adding annual updates on the disease to course material and building a comprehensive Wilson disease patient database to springboard future research endeavors.
The partnership with the WDA, a nonprofit organization dedicated to education, support and research, will optimize student, physician, patient and caregiver engagement and education, with a goal of spreading awareness, early diagnosis and holistic intervention for affected people.
Tags: center of excellence, Department of Neurology, Dr. Cynthia Levy, Dr. Jason Margolesky, movement disorders, neurology