The winners of the Australian and New Zealand Batten Disease Research Grant for 2021 have been announced by Batten Disease Support and Research Association (BDSRA) Australia, the program sponsor.
They are Anthony White, PhD, Associate Professor and Principal Investigator at QIMR Berghofer Medical Research Institute in Brisbane, Australia, and Anthony Cook, PhD, Associate Professor at the Wicking Dementia Research and Education Center at the University of Tasmania in Hobart , Australia.
The program supports local research that advances current understanding, advances treatment, or improves diagnosis, education or management of any or all of the many types of Batten disease.
White and his team received a grant worth AU $ 49,600 (approximately $ 35,000). They will focus on studying the possibility of reuse of drugs, treatments are already on the market for other indications, to treat Batten disease.
“If a drug is found to be suitable, it is much faster to use for patients with Batten disease because the drug is already past the initial stages of development and is being used by other patients,” the page states. Web ad.
Their project, “Integrate computational and in vitro approaches to achieve repositioning of Batten disease drugsâEmphasizes the use of computer algorithms that analyze how sets of genes interact in Batten disease. Then, the algorithms will try to identify the existing drugs that act on these interactions. Candidates will be tested in cellular models of Batten disease in the laboratory before possibly moving on to clinical studies in patients.
Cook’s team will use their AU $ 55,000 (approximately $ 39,000) grant to study how the blood-brain barrier is affected by changes in blood pressure. CLN3, the gene that is mutated in juvenile Batten and other forms of Batten disease. The blood-brain barrier is a membrane made up of cells that protect the brain from the outside environment (i.e. toxic substances in the blood) while allowing essential nutrients to reach the brain. Any damage to this barrier can cause starvation of brain cells, as they are unable to receive energy.
Researchers have developed a human stem cell model of Batten disease. Stem cells will be transformed into the type of cells that form the blood-brain barrier, and researchers will assess how changes in CLN3 alter normal cell function, in the hope of identifying potential therapeutic targets.
“Such models are needed to accelerate the transformation of lab results into effective and safe treatment options for people with this disease,” the webpage says.
During the project, “Dissect the effects of CLN3 variants on the blood-brain barrierThe scientists also plan to create a mock-up of the blood-brain barrier in the lab to serve as a platform to test how and if new drugs can enter the brain.
Grants are awarded for a period of one year and may be renewed based on progress made during that year.
Researchers who wish to apply for the 2022 grant are invited to submit expressions of interest at the opening of this cycle in May.