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Cortical dysplasia (CD) is the most common cause of epilepsy in children, and a high proportion of these cases are refractory to drug therapy. Although surgery is being increasingly used to treat drug-resistance in CD, there is still a lot of uncertainty in terms of outcome. Researchers are therefore keen to find new types of medication that might succeed where conventional anti-epileptic drugs (AEDs) have failed.
Thishas been a difficult task, however, because it requires an animal modelthat displays all of the features of CD, and until now, this has notexisted.
Investigators at Rutgers, the State University of New Jersey, have successfully remedied this situation. They bred mice that lacked a gene called Pten, which blocks cell growth in some neurons, causing them to display all the characteristic traits of CD: enlarged cortical neurons and abnormal EEG activity with spontaneous seizures. There was also increased mTor activity in these models.
When they administered rapamycin, the team found that the severity and duration of seizures was strongly suppressed. Dr D'Arcangelo, the project leader commented, "We demonstrated that rapamycin is a novel and effective anti-epileptic agent that suppresses seizures in our mice as well as in the TS model, and this has raised some hope for the future."
The group hopes that these results will lead to human trials of rapamycin for CD, in the same way as it has for TS. If these show the same promise, rapamycin could be developed as a possible treatment for patients with epilepsy associated with CD and similar disorders.
Thishas been a difficult task, however, because it requires an animal modelthat displays all of the features of CD, and until now, this has notexisted.
Investigators at Rutgers, the State University of New Jersey, have successfully remedied this situation. They bred mice that lacked a gene called Pten, which blocks cell growth in some neurons, causing them to display all the characteristic traits of CD: enlarged cortical neurons and abnormal EEG activity with spontaneous seizures. There was also increased mTor activity in these models.
When they administered rapamycin, the team found that the severity and duration of seizures was strongly suppressed. Dr D'Arcangelo, the project leader commented, "We demonstrated that rapamycin is a novel and effective anti-epileptic agent that suppresses seizures in our mice as well as in the TS model, and this has raised some hope for the future."
The group hopes that these results will lead to human trials of rapamycin for CD, in the same way as it has for TS. If these show the same promise, rapamycin could be developed as a possible treatment for patients with epilepsy associated with CD and similar disorders.
