The Brain in a Dish research project began in July 2019 with the support of a $2 million grant from the Australian Commonwealth Government’s Medical Research Future Fund and additional funds from Sanfilippo Children’s Foundation and their generous donors.
The aim of this study is to grow cells in the laboratory from children with Sanfilippo Syndrome that mimic or model what is going on in their brains.
The researchers take skin cells from patients and reverse engineer them into stem cells and then into neurons, creating a “brain in a dish” – an individualised representation of a person’s brain tissue. Skin cells from healthy volunteers will also go through the same process for comparison.
They will study the function of the cells and then test a range of different drugs that have already been approved for other conditions on the cells, to see if any of them can improve the health of the Sanfilippo brain cells.
We are excited to fill you in on the great progress that has been made so far. The project is about two thirds of the way through and all study participants have been recruited and have provided skin samples.
The research team have extracted the skin cells, known as fibroblasts, from these samples and are converting each of the sets of fibroblasts into stem cells and then into neurons.
The samples that have already been converted into brain cells are currently being analysed in detail to explore how the Sanfilippo cells’ structure and function differ from the cells from healthy individuals and develop the best methods for screening the activity of the panel of drugs.
Some examples of the stages that the cells go through as they advance through the Brain in a Dish pipeline are shown below. The researchers are pleased that the work is going well and the mature brain cell cultures are “looking beautiful”, enabling groundbreaking research into how these brain cells differ from healthy cells.
This year, the team will conduct the drug screening to hopefully identify some promising candidates for further study. These drugs would then need to go through a series of further detailed experiments to confirm that they show promise in altering the disease course or managing symptoms and improving quality of life before testing in children with Sanfilippo syndrome.
The project was originally planned to last for two years in total, however, there were some delays to the project, particularly in recruitment of patients due to the COVID pandemic. A one-year extension has been secured from the Government funding body to continue and complete the work.
You can watch the video below to learn more about the Brain in a Dish project.
We are enormously grateful to the participants in this study and their families, all the donors and supporters, and the Commonwealth Government who have made this study possible. We will update you with more news as it becomes available.