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Australia and New Zealand
Batten Disease Research Grant Program

Each year, BDSRA Australia invests in promising local research through its Batten Disease Research Grant Program. The objective of the Program is to advance research across Australia and New Zealand that has the potential to:

  1. Further our understanding of the disease mechanisms for all or any of the Neuronal Ceroid Lipofuscinoses (NCLs);
  2. Advance therapeutic strategies; or
  3. Improve current diagnosis, treatment, education and/or management practices or family support practices (including those that improve quality of life, palliative care or other).

BDSRA Australia typically awards up to AU$100,000 in total each year, across one or more projects.

The 2022 Research Grant round is now open for Expressions of Interest (EOIs) until 5 pm (AEST) Wednesday 29 June 2022. See Key Dates for 2022 below.

Before applying, please read the Expression of Interest - Information for Applicants and the full Research Program Guidelines.

To receive Research Grant Program updates please email

Key Dates for 2022

Milestone Date
Expressions of Interest open: Monday 16 May 2022
Expressions of Interest closed: 5pm (AEST) Wednesday 29 June 2022
Successful EOIs will be notified by: Early August 2022
Deadline for full Research Proposals: 5pm (AEST) Wednesday 5 October 2022
Funding announcements: Mid-November 2022

2021 BDSRA Australia Batten Disease Research Grant Recipients

Integrating computational and in vitro approaches to achieve drug re-positioning for Batten disease

Chief Investigator Associate Professor Tony White (left) and Project Co-Investigators Dr Zachary Gerring, Dr Lotta Oikari and Prof. Eske Derks (right)

Developing drugs to treat brain diseases is a very slow and costly process, often taking 10-15 years and billions of dollars. Yet, the success rate is also very low, with only 1-2% of drugs actually end up being used to treat patients. A major part of this slow and complex process is all the early safety analysis and determining if the drug can actually penetrate into the brain. To help overcome the slow delivery of drugs to patients with rarer diseases, drug-repositioning can be investigated. This is where drugs that are already in use in the clinic for other diseases are investigated for their ability to work on another disease i.e. Batten disease. If a drug is found to be suitable, it has a far quicker path to use for Batten disease patients, as the drug has already passed the initial development stages and is used by other patients.

Although the testing of clinically-approved drugs can be done randomly, there are many thousands of drugs to test and this is a costly and high risk, low outcome approach. Instead, we propose to use computer-based algorithms that analyse the gene pathways involved in Batten disease and compare this to known drug action on these pathways. In this manner, we anticipate that we can identify drugs (that are already in the clinic for other diseases) which may be able to target specific key genes/proteins in Batten disease. To confirm this, we will select several of these identified drugs and test them on cell models of Batten disease in the laboratory to determine how they affect the cells. The outcomes from this will potentially provide a rapid pathway for identifying drugs that could then be tested in advanced clinical trials on Batten disease patients.

Dissecting the effects of CLN3 variants on the blood-brain barrier

Chief Investigator Associate Professor Tony Cook (right) and Project Co-Investigator Associate Professor Brad Sutherland (left)

The blood-brain barrier (BBB) is a specialized part of our bodies that separates our blood supply from our brain. The BBB controls the delivery of oxygen and energy into the brain to keep it functioning and healthy. When the BBB breaks down, the cells of the brain are starved of energy and can no longer function normally. While we know that the BBB is involved in Batten disease, we do not fully understand how this occurs. Variation of the CLN3 gene occurs in Batten disease and our goal is to determine how the BBB is affected by variants in the CLN3 gene. To do this, we have used advanced techniques in human stem cell biology and gene editing technology to develop a new human cell-based model of Batten Disease.

Such models are needed to accelerate moving laboratory findings into effective and safe treatment options for people with this disease. We can turn these stem cells into the cell types that form the BBB, and we will study how CLN3 gene variants change the normal function of these cells. We will identify molecules that explain these changes, and which may be suitable targets for development of new therapeutics. We will also develop techniques to re-construct the BBB in the laboratory, which will allow us to study this structure in greater detail; this is important because getting drugs to cross the BBB is a significant bottleneck to finding new drugs to treat diseases affecting the brain. Thus, we will provide a platform for testing how and if new drugs are able to enter the brain, and thereby accelerate development of treatment options for people living with Batten disease.