$11.7 million epilepsy treatment grant will help determine importance of ‘getting it right the first time’

Nearly one year ago, a team of researchers led by Tracy Glauser, MD, changed the course of epilepsy treatment when they published the results of a study in the New England Journal of Medicine.

The NIH-funded study involved 446 children and 32 medical centers nationwide. It showed that of the three most commonly prescribed antiseizure medications – ethosuximide, valproic acid and lamotrigine – ethosuximide provided the best short-term results for kids with absence, or “petit mal,” epilepsy.

Ethosuximide – a medication that has been on the market since the early 1950’s – provided the best combination of seizure control and fewest attention-related side effects in children with absence epilepsy who had received no prior treatment.

More to the Story

For most researchers, that is where it would end – determining which drug is best for the short term. But Glauser and his team weren’t satisfied, and the National Institute of Neurological Disorders and Stroke is fully behind their efforts to take it further.

The new four-year study is evaluating how the same cohort of children fares over the long term. The study will explore which of the three medications does the best job of preventing the long-term problems of epilepsy and which has the least negative long-term impact on quality of life and cognitive function.

“We want to know why specific patients respond differently to different drugs, how we can improve our overall rate of success with initial therapy, and whether medications that work best in the short-term continue to be the best choice over the long term.”

Better Than 53

Percent Although the first trial established ethosuximide as the evidence-based choice for initial drug therapy, the researchers found that even this “best” treatment failed in nearly half of newly diagnosed cases. Ethosuximide was successful – meaning it controlled seizures with fewest side effects – only 53 percent of the time.

When treating a child who can suffer as many as 100 petit mal seizures a day, Glauser says, a drug that works only a little more than half the time just doesn’t seem good enough.

Same Kids, New Questions

The new trial is an observational study that will follow the same cohort of children, but ask new questions. Using statistical and mathematical models, the researchers plan to use the information obtained from the first study to further explore which of the three medications tested was the “best first” drug for each child based on their long-term outcomes.

“The current study is important because it will tell us how important it is to get it right the first time when selecting a medicine for a child with epilepsy,” Glauser says.