July 17--Houston researchers have dramatically reduced the occurrence of
seizures in a small group of children with epilepsy, using an existing cancer
drug they think may be able to treat up to 1.5 million people with intractable
forms of the disease.
In a new study, Texas Children's neurologists found that children with a rare,
severe type of epilepsy received great benefit from Everolimus, a smart drug
approved for the treatment of kidney and brain tumors. The drug targets faulty
signaling in the brain that causes uncontrolled cell growth.
"The exciting thing is that this is anti-epilepsy therapy, not just anti-seizure
therapy," said Dr. Angus Wilfong, the director of the comprehensive epilepsy
program at Texas Children's and one of the study's principal investigators. "For
the first time, we have a drug that addresses the underlying cause of seizures,
changing the brain and the way it sends and receives disease-causing signals."
The study, now online in the journal Annals of Neurology, is important even
though it only enrolled 20 children because the benefits were so "life changing"
for most of them, Wilfong said. He added that the difference in children before
and after treatment was "like night and day."
Wilfong will lead the Texas Children's arm of an international trial that will
test Everolimus on patients whose type of epilepsy is more common but still
characterized by the same signaling problems. The trial, already started in
Europe, aims to enroll 500 patients at as many as 70 sites, including some in
Asia and Latin America.
The small study, conducted at Texas Children's and Cincinnati Children's
hospitals, tested Everolimus on patients with tuberous sclerosis complex, a
genetic disorder that causes non-malignant tumors to form in different organs.
Those in the brain affect signaling in a key molecular pathway and cause
The condition is found in one in 6,000 births and accounts for between 1 and 2
percent of the nation's 3 million cases of epilepsy. But Wilfong said the same
pathway signaling problems occur in 30 percent to 50 percent of cases.
Children in the study received Everolimus for 12 weeks, during which time their
symptoms were monitored. Seventeen of the 20 participants experienced an average
72 percent reduction in their overall seizure activity -- four became completely
seizure free and the others reported seizures that were milder, shorter and less
Among the successes was Aidan Maxwell, a 9-year-old boy who, diagnosed at birth,
began suffering seizures at 23 months. By the time he was 6, his frequent,
severe seizures caused autistic-like symptoms -- developmental delay, a lack of
communication, tantrums -- that his parents assumed meant he'd never be a
About that time, Aidan began taking Everolimus. Though he's not completely
seizure-free and his development lags behind, he is, for the first time,
relatively healthy and independent.
"I thank God for that drug," says Lisa Maxwell, his mother. "I don't know who my
son would be today without it. He's now like any other kid, playing and
The drug is not without downsides. It costs several thousand dollars a month and
appears likely to need to be continued through one's life since its withdrawal
would result in a resumption of the brain's faulty signaling -- though Wilfong
thinks early use might prevent the growths that cause the condition.
Though Everolimus is a mild immunosuppressant, Wilfong said side effects were
minimal. Short-lived mouth ulcers were the most common side effect.
Janice Buelow, vice president of programs and research for the Epilepsy
Foundation, called the study "a good, promising start," noting the impressive
results occurred with very difficult-to-treat cases.
She said she looks forward to the larger trial attempting to replicate the
Wilfong, who got the idea to conduct the study after noticing the decline in
seizures among tuberous sclerosis complex patients treated with Everolimus for
tumor growth, said there's a buzz over the potential new application of the
"I haven't been to a conference recently where this therapy hasn't been the
subject of discussion," said Wilfong, also a Baylor professor of pediatrics and
neurology. "I think this is the dawn of a new era in seizure treatment."
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