Targeting the immune systems as a treatment approach for Sanfilippo

08 Jul 2020

The Foundation has awarded funds to Associate Professor Kim Hemsley (Flinders University) and Dr. Marten Snel (South Australian Health and Medical Research Institute) for a two-year translational project that will evaluate if drugs that target the immune system could benefit children with Sanfilippo.

In this project, Assoc. Prof. Kim Hemsley and Dr. Marten Snel will test compounds that target a part of the immune system on a mouse model of Sanfilippo Type A, by intravenous injection into the bloodstream, or via direct injection into the cerebrospinal flui

The team will determine how effective the therapies are in preventing cell degeneration and cognitive decline. If a therapy is proven to be successful, this research will provide a new avenue for exploration in the treatment of Sanfilippo.

Megan Donnell, Executive Director of the Sanfilippo Children’s Foundation, said: “This project will help to develop a greater understanding of the role of the immune system in Sanfilippo and identify potential therapeutic avenues. By limiting the level of inflammation in the brain, it may be possible to slow down the cognitive decline experienced by children with Sanfilippo and improve their quality of life.”

Read more about the project here