Epilepsy & learning deficits can be reversed in animals with Tuberous Sclerosis
Results of animal research have just been published that show the drug Sirolimus (Rapamycin) can not only prevent seizures in an animal model of TSC, but also reverses seizures in older animals. Enhanced survival and improvement in behavioural aspects in two different animal models was also demonstrated.
Click here to view a longer article.
The synopsis below was posted today (27 June 08) by the TSAlliance - our counterpart in the USA:
This week, an article published by Ehninger et al., 2008, showed that rapamycin can improve cognitive deficits in an animal model of TSC. This study utilized the Tsc2+/- animals derived in David Kwiatkowski’s laboratory, and showed that the animals improved their performance in the Morris water maze, 8-arm radial maze, and in a test of context conditioning that tests their response to fear. These publications have, of course, stirred all of us to think that a new treatment for epilepsy and for the cognitive and intellectual disabilities in TSC may be near. The TS Alliance is moving rapidly to put a group into place to develop a protocol for a clinical trial that will provide the evidence that is needed to identify if these drugs will work to improve seizures and cognition in individuals with TSC, and if the drug(s) are safe for individuals with TSC. We will keep you informed about the plans for this study as we go forward in the next few weeks. It is important that we first do the clinical trials to determine if the mTOR inhibitors will be efficacious and safe for individuals with TSC, especially for children.
References:
Ehninger D, Han S, Shilyansky C, Zhou Y, Li W, Kwiatkowski DJ, Ramesh V, Silva AJ (2008) Reversal of learning deficits in a Tsc2+/- mouse model of tuberous sclerosis. Nature Medicine June 22, 2008, Published online
Meikle L, Pollizzi K, Egnor A, Kramvis I, Lane H, Sahin M, Kwiatkowski DJ (2008) Response of a neuronal model of tuberous sclerosis to mammalian target of rapamycin (mTOR) inhibitors: effects on mTORC1 and Akt signaling lead to improved survival and function. J Neurosci 28(21):5422-32
Zeng LH, Xu L, Gutmann DH, Wong M (2008) Rapamycin prevents epilepsy in a mouse model of tuberous sclerosis complex. Ann Neurol 63(4):444-53
