Pursuing first-in-clinic treatment for severe infant epilepsy
Researchers at the Florey, in collaboration with the University of Melbourne, will pursue a potentially life-changing precision medicine treatment for children with severe forms of epilepsy, thanks to a new multi-year grant awarded from the Medical Research Future Fund (MRFF).
Minister for Health, the Hon. Greg Hunt announced the boost in funding as part of the MRFF Genomics Health Futures Mission – Projects which support several vital genomic projects.
Professors Ingrid Scheffer, Steven Petrou and Sam Berkovic will lead the project, aside Drs Snezana Maljevic and Geza Berecki, which uses the advancement of precision medicine for life-threatening infantile epilepsies which account for an estimate 5% of all epilepsy. Their research will include the first-in-clinic investigation trialling a novel gene therapy for children with SCN2A developmental and epileptic encephalopathy (SCN2A DEE). There remains urgent clinical need to develop a treatment for this devastating disorder in which children experience uncontrolled seizures and profound developmental impairment.
By taking a precision medicine-approach the aim is to develop a therapy to control seizures and halt, or even reverse, developmental decline. A successful trial of this therapy could have significant implications in saving lives and paving the way for similar treatment approaches in other forms of genetic epilepsy and neurogenetic diseases.
The program will see the Florey, the University of Melbourne and the Murdoch Children’s Research Institute working together. The team is uniquely positioned to undertake this innovative work as world leaders in epilepsy genetics.
Article originally published on the Florey Institute of Neuroscience and Mental Health website
Professor Steve Petrou is a member of the Centre for Stem Cell Systems, conducting research captured in the Stem Cells and Disease Modelling research theme.