SAHMRI hosts special Sanfilippo syndrome symposium

SAHMRI’s neurobiologists recently partnered with the Sanfilippo Children's Foundation to host a unique symposium, bringing together international researchers and families from across Australia who all have children living with Sanfilippo syndrome, the most common form of childhood dementia.

The event included a special lab tour, giving parents a firsthand glimpse of the world leading research behind ongoing efforts to find a cure in SAHMRI’s Bardy Lab, led by Professor Cedric Bardy.

Neurobiologist Dr Zarina Greenberg, who helped lead the tour, says the experience was as valuable for the researchers as it was for the families.

“As researchers, we want to bring these families into the work we’re doing, because they’re the ones who’ll eventually benefit from it,” Dr Greenberg said.

"Seeing them face to face reminds us of the urgency of our work.

It really hits hard knowing we’re fighting against the clock," Dr Greenberg said.

The tour showcased state-of-the-art equipment, including high-throughput robots like the Janus liquid handler, which allows researchers to screen a far larger number of drugs in a much shorter timeframe, optimising output while ensuring consistency.

Attendees were eager to learn about the progress of the Sanfilippo ‘Brain in a Dish’ project, which is supported by the Sanfilippo Children’s Foundation and a grant from the Medical Research Future Fund (MRFF). After years of preparation, the project has now reached the drug screening phase, where readily available medications are being tested to identify any potential utility for the treatment of Sanfilippo syndrome.

“We’ve got the first batch of 60 drugs on the way and we’ll be working our way through them over the next few months,” Dr Greenberg said.

“This is the moment we've all been working for. We're very passionate about finding a solution.'"

"With each step forward, we bring these families closer to a future where effective treatments for Sanfilippo syndrome are within reach.”

For more information on the Brain in a Dish project and other research visit www.bardylab.com.