Dr. Maria Francisca Coutinho from Portugal’s National Institute of Health Dr. Ricardo Jorge (INSA) has received funds from the Sanfilippo Children’s Foundation to develop nanoparticles to deliver substrate reduction drugs to the brain.
Substrate Reduction Therapy (SRT) aims to decrease the production of heparan sulfate so that there is less to build up inside the body. The project will develop cutting-edge nanoparticles to deliver SRT drugs through the blood-brain barrier and into the brain.
“This study will help to investigate new approaches to treatment delivery, with an emphasis on drug delivery to the brain. This represents an important step in the drug development process for Sanfilippo”, said Megan Donnell, Executive Director of the Sanfilippo Children’s Foundation.
Reducing heparan sulfate accumulation inside the brain could help to reduce symptoms and slow disease progression. The team will test this approach in neurons in the lab and if successful future projects will test them in animal models.