A new compound targeting the protein Pumilio as an antiepileptic drug

Regular functioning of the brain relies on balancing neuronal circuitry, and while changes to this system, such as the excitation-inhibition system, can result in numerous disorders, no drug exists that can help the body regulate these important circuits.

A vital compound has been developed which can aid in this needed homeostasis.

Significant neurological disorders; epilepsy, schizophrenia and autism, are not well treated by current medications. It is accepted that novel, game-changing, approaches are required. A commonality of these diseases is that they arise from pathological alteration to the excitation-inhibition balance of the brain. Moreover, it is probable that perturbation of this balance occurs during early nervous system development, which then becomes ‘locked-in’.

The excitation-balance of the brain is regulated and maintained by neuronal homeostasis. All neurons employ homeostatic mechanisms to manage excitability (i.e. to maintain circuit stability). Given its importance, neuronal homeostasis is a potentially very attractive drug target. However, no such drugs have been identified.

Researchers at The University of Manchester have developed a compound that increases expression of a key homeostatic regulator: Pumilio. As predicted, this compound is a potent anticonvulsant in mouse models of epilepsy (through restoration of the excitation-inhibition balance). This activity demonstrates the potential for manipulation of neuronal homeostasis. It is anticipated that development of this compound, and similar drugs, will have enormous clinical potential.

For more information, please contact:

Arnaud Garçon
arnaud.garcon@uominnovationfactory.com
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