Sanfilippo syndrome Brain in a Dish project to continue with major new grant from the Medical Research Future Fund

15 Mar 2023

We are thrilled to announce that the Sanfilippo Brain in a Dish research team have been awarded a $730,000, two-year grant from the Australian Government's Medical Research Future Fund Stem Cell Therapies Mission. This grant allows the team to continue this important flagship project to screen and validate drugs that may help improve the symptoms of Sanfilippo syndrome.

The Brain in a Dish platform was established through a previous $2 million MRFF grant to the Sanfilippo Children's Foundation. The team will use this panel of deeply characterised cells donated by children with Sanfilippo that have been grown into neurons in the laboratory. They will continue the rapid drug screening program to identify drugs that might restore healthy function to the cells. The top candidate drugs will then be further tested in cells to determine their mechanism of action, before being validated as potential therapies in a mouse model of Sanfilippo syndrome.

The team is led by Associate Professor Cedric Bardy at the South Australian Health and Medical Research Institute (SAHMRI) and Flinders University, together with Dr Nicholas Smith (Women's and Children's Hospital, Adelaide), Professor Kim Hemsley (Flinders University, Adelaide) Professor Mark Hutchinson (The University of Adelaide), Dr Chris Bye (The Florey, Melbourne), Dr Zarina Greenberg (SAHMRI) and Dr Lisa Melton (Sanfilippo Children's Foundation). Dr Cara O'Neill from Cure Sanfilippo Foundation, USA and the Childhood Dementia Initiative will also provide significant input to the project.

Sanfilippo Children's Foundation Head of Research Dr Lisa Melton said "It is wonderful to see this important project gain ongoing government funding - it has enormous potential to help slow down disease progression and improve quality of life for children living with Sanfilippo syndrome. We are excited to continue working with these excellent researchers as well as bringing new researchers into the Sanfilippo field."