Lurie Children’s leads $4 million study examining improved treatment for severe form of epilepsy

Twenty-six years ago, Dr. Tracy Dixon-Salazar’s then two-year-old little girl went from being a healthy, typical toddler to having hundreds of seizures a day virtually overnight.

Her daughter Savannah was eventually diagnosed with Lennox-Gastaut Syndrome (LGS), a serious and rare form of epilepsy that begins in infancy and early childhood.

LGS is typically accompanied by severe disabilities in gross motor, fine motor, communication, eating and other important skills needed for daily functioning. There is no known cure.

Back in 1998 when Savannah was diagnosed, Dr. Dixon-Salazar recalls that doctors were flummoxed about the root cause of her seizures, giving the family little understanding of their daughter’s illness and no effective treatment options. Needing more answers, Dr. Dixon-Salazar, then a stay-at-home-mom without a college degree, enrolled in a community college and started taking neurobiology courses.

Flash forward more than twenty years, and today Dr. Dixon-Salazar is a PhD neuroscientist and the executive director of the LGS Foundation, a nonprofit dedicated to providing information and support to families affected by LGS. And in collaboration with Lurie Children’s faculty and researchers, she is now spearheading what she calls one of the most powerful research efforts she has ever seen for examining best treatment options for the condition that devastated her daughter’s life.

In March 2021, Sandi Lam, MD, MBA, Chief of Neurosurgery at Lurie Children’s, along with Anne Berg, PhD (research professor and epidemiologist) and Marc Rosenman, MD, MPH (pediatrician, health services researcher, and medical informatics specialist) were among research team members approved for a $4 million funding award by the Patient-Centered Outcomes Research Institute (PCORI) to conduct a study, Comparative effectiveness of palliative surgery versus additional anti-seizure medications for Lennox-Gastaut Syndrome.

The results from this study will help parents and providers make more informed choices about treatment for children with LGS and will highlight areas for improvement in providing the best possible health care for this severe, lifelong disorder, Dr. Lam said.

An all-star team will work together to complete this project, with researchers at Lurie Children's and partner pediatric institutions across the United States. Another unique aspect of the study is that it will draw on the vast health data resources of PCORnet, the National Patient-Centered Clinical Research Institute, to conduct this study more efficiently. With health records for 66 million patients available for observational studies, the PCORI-funded PCORnet

provides vast scale to potentially power research on conditions affecting even small numbers of people, said Dr. Lam.

“It has long been suspected that surgery for LGS can lead to even better outcomes than the next medication, but families are reluctant to try surgery as it is a difficult and painful decision for anyone to make for their child,” said Dr. Dixon-Salazar. “And there is hardly any data to support surgery over the next medication. This study will change that and that is a really big deal!”

Savannah is now a 28-year-old “who is living a wonderful life,” said Dr. Dixon-Salazar. “It's not perfect. She suffered much brain damage from seizures. But we are out of that horrible constant crisis mode that most of the LGS families I work with are still living day-to-day. What I want most is for others to find their way out of LGS hell just as Savannah and our family did. I want families to find their treatments, their cures. And I want to help if I can. This research is one way to do that.”

Read more about the LGS Foundation and Tracy and Savannah’s story here. https://www.lgsfoundation.org/fighting-for-a-cure-for-lgs/

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