As a parent, it can seem like a horrible nightmare: One day, they are a healthy and active child, and months later, they cannot even walk or talk. For one Gainesville family, this scenario was exactly what they were facing when their 7-year-old son Tyler Staab was diagnosed with dystonia. What could have been despair and bleakness, however, has turned into hope for not just their son, but for millions of children with the same diagnosis. Dystonia is a neurological movement disorder affecting the central nervous system. It is characterized by involuntary and often painful spasms of the muscles. This disorder currently has no cure and progressively causes loss of the ability to control voluntary movements, even walking. As soon as Dr. Michael Okun at UF Health diagnosed Tyler with dystonia, the family established Tyler’s Hope for a Dystonia Cure in 2006 to raise money for dystonia research. They were inspired by the strong cadre of researchers and clinicians at UF Health who specialize in movement disorders. Tyler’s Hope Foundation was established to advance research for a cure, discover effective treatments and to promote awareness and education of this devastating disorder. A few years after Tyler’s diagnosis, their daughter Samantha was diagnosed with generalized dystonia. The Staabs continue to take an active role in the foundation today.
“Our ultimate goal is we are looking for a cure, and we are so close,” said Rick Staab. “We have lines of breakthrough science pathways to achieve that goal, which includes neuroscience research, newly discovered gene therapy and protein therapy. We have already come so far.”
A collaboration between Duke, UF and the University of Michigan yielded a new pathway that will either cure or affect dystonia and other neurological conditions. This pathway led to a pharmaceutical breakthrough, which targets the gene that cause these conditions. Since dystonia is not degenerative, if symptoms are relieved, then the condition is cured. Another collaboration has led to the identification of a gene that could potentially prevent the mutation of the protein that causes dystonia. Protein therapy could delete that mutation. The incredible amount of fundraising done by the Tyler’s Hope Foundation has made this kind of progress possible. Since 2006, Tyler’s Hope has raised about $4 million, elevating UF into the epicenter of dystonia research and care. In 2008, the UF Center for Movement Disorders and Neurorestoration, co-directed by Dr. Okun and Dr. Kelly Foote, became the home of the Tyler’s Hope Center of Excellence. In 2010, the center recruited the country’s most promising dystonia researcher, Dr. Yuqing Li, with funding from Tyler’s Hope. After raising $1 million, the center also constructed a state-of-the-art laboratory for dystonia research. Tyler was the youngest patient ever, at 7 years old, to receive deep brain stimulation surgery at UF Health, which greatly improved his symptoms. The Staabs attribute much of their success to support from the Greater Gainesville community.
“We could not have accomplished everything without the support shown by so many people in this community,” Staab said. “When this community gets behind a cause, they really affect change and make things happen. We not only were humbled by the amount of financial support that we have received but also the level of sweat equity from our volunteers.”
Of every dollar donated to Tyler’s Hope, 98% goes directly into research. This funding has buoyed major advances in neurological research since Tyler’s diagnosis, including widespread awareness of the condition, the creation of definitive lab tests that confirm the condition and a patient registry. This registry allows patients to verify their condition and have an inventory of patients for clinical trials. Tyler is doing much better today, although he still endures his share of struggles. Samantha, who also underwent deep brain stimulation surgery, is a straight-A nursing student at the University of Alabama despite her lingering struggles with balance. Staab said the foundation will continue to work each day until they get to their ultimate goal.
“We are going to change the world when we cure dystonia. That I am sure about,” Staab said.