Researchers receive $250,000 to study inventive cell-transfer therapy to treat cortical dysplasia

University of Florida researchers will receive $250,000 to study an inventive cell-transfer therapy to treat cortical dysplasia, a condition where the brain develops abnormally before birth, causing severe epilepsy in children and adults.

Citizens United for Research in Epilepsy, also known as CURE, has given its 2010 Falk Medical Research Trust Award to neurosurgeon Steven Roper, M.D., to collaborate with the laboratory of McKnight Brain Institute Executive Director Dennis Steindler, Ph.D., to study the use of a versatile type of brain cell to restore brain function in an animal model of the disease.

Cortical dysplasia causes seizures due to problems with the electrochemical connections in the brain. Currently the only treatment options are anticonvulsive medications and surgery, both of which have shown limited effectiveness in people with cortical dysplasia.

Using tissue surgically removed from epilepsy patients, Steindler's lab will isolate and grow a population of cells known as adult human neural progenitors, which have the ability to develop into a variety of brain cells needed to treat neurological disease.

Roper and his team transplant the cells into a rat model of cortical dysplasia. The goal is to replenish a type of brain cell called an inhibitory neuron, which is in short supply in these animals.

"There are different theories about what causes seizures in these individuals, but many researchers believe it is because of an imbalance of excitatory and inhibitory neurons in the brain," Roper said. "Taking cells from the adult human brain and having them turn into functional neurons in a rat is a novel approach. If this can be done for individuals with epilepsy due to cortical dysplasia, it would provide an important new treatment option."

The researchers want to demonstrate the effectiveness and safety of the technique in animals first, with the goal of eventually moving the research to humans.

"This grant allows our cells to be used in Dr. Roper's lab's exquisite epilepsy models, and learn how to mobilize a patient's own population of these cells to treat their own epilepsy, truly personalized neuroregenerative medicine," Steindler said.

CURE is a volunteer-based, nonprofit organization founded by parents of children with epilepsy who were frustrated with their inability to protect their children from the devastation of seizures and the side effects of medications. Unwilling to sit back and accept the debilitating effects of epilepsy, these parents joined forces to spearhead the search for a cure.

"This project offers a new direction for treatment of intractable epilepsies using targeted cellular therapies," said Vicky Whittemore, Ph.D., CURE scientific consultant. "If this research is successful, the use of adult human donor cells will facilitate the translation of this approach to treatments for individuals with epilepsy due to cortical dysplasia."