Dr Lotta Oikari
A researcher at Brisbane's QIMR Berghofer whose dedication to solving the mysteries of rare neurodegenerative disorders is fueled by a mix of scientific rigor, motherhood and her Finnish roots.
In February 2026, BDSRA Australia’s Project Support Officer, Dr Tahmina Tabassum, dropped by the lab to meet Dr Lotta Oikari and the team behind their latest Batten research project.
Dr Lotta was a recipient of a BDSRA Australia Batten Disease Research Grant in 2023-24. She recently submitted her final report for the project "Validating therapeutic effects of repositioned drugs in Batten disease brain cell types using medium to high-throughput approaches" and is currently working on the manuscript to publish the outcome of this research work.
Lotta completed her PhD in fundamental neuroscience and did not imagine herself working in the rare disease field in her early career. It was only until she returned from maternity leave when her mentor, Prof Anthony White, presented her with a new project funded by the BDSRA Australia Grant Program. "I had never heard of Batten disease, to be honest. But when I started reading about it, I felt straight away really passionate because I also have small children. I was really excited to start working on a project knowing that it’s under-researched and underfunded. Also, I come from Finland and after starting to work on Batten disease, I discovered that some of these mutations are quite prevalent there.” Though she had previously worked on more common neurodegenerative conditions like Alzheimer’s, the unique urgency and the underserved nature of Batten disease turned this lab assignment into a passion project that occupies her thoughts even in her spare time. She found the curiosity, passion to find treatments, and willingness to participate in research, displayed by the parents at the Australian NCL Conference 2025 very encouraging. A typical day for Lotta includes conducting experiments, mentoring students and applying for the next funding opportunity to keep the research moving forward. When the pressure of grant writing and complex data becomes too much, she turns to a hidden talent: the piano. "It’s a good way to get your mind off things," she shares, noting with a laugh that her children rarely let her play in peace. Lotta is genuinely optimistic about the future of the field and believes drug repurposing may speed up getting treatments to patients and leveraging cross-disease knowledge from conditions like Alzheimer’s and ALS can accelerate progress. She emphasized that advances in technology, such as patient-derived organoids (mini-organs grown in a dish) and methods to cross the blood–brain barrier, are expanding research capabilities. Her team is also currently following up on several promising compounds that could eventually serve as supportive treatments to reduce symptoms and improve quality of life for those living with Batten disease.
Summary of the Project Outcomes:
Title: Validating therapeutic effects of repositioned drugs in Batten disease brain cell types using medium to high-throughput approaches
Developing a new drug is an expensive and time-consuming process and it can take up to 10 years to deliver a newly developed drug to patients. Drug repurposing is an approach where new uses are found for already existing drugs that have been clinically tested for other diseases. Our aim was to use computer-based analysis tools to identify new drugs for Batten disease. We then tested the effects of these drugs in our laboratory cell models to identify which drug could have therapeutic effects in Batten disease. From our drug repurposing analysis, we proceeded with 6 drugs for further investigation. We tested the safety of these drug candidates on human brain cells and found that none of the drugs were toxic for human brain cells. We then investigated the potential therapeutic effects of the candidate drugs on cells obtained from an individual with CLN3 Batten disease by analysing which genes were modulated by the drug candidates in these cells. Interestingly, we found that one of the drugs: GlcNAc, increased CLN5 gene expression as well as the expression of many other lysosomal genes that are associated with lysosomal storage disorders. We also found that GlcNAc and a compound called SMLC were able to reduce lipofuscin accumulation in CLN3 cells. These outcomes warrant the further therapeutic investigation of GlcNAc and SMLC, which are both considered as dietary supplements, in Batten disease.
A message for the Batten disease community:
"We are really passionate about the work we do. We take it very seriously and it’s on the top of our minds a lot. There are highly experienced experts behind the scenes, and through collaboration, there will be hopeful findings in the near future." 
Touring the lab: BDSRA Australia's Project Support Officer, Dr Tahmina Tabassum, visited QIMR Berghofer to meet Dr Lotta Oikari and her team behind the BDSRA Australia funded project.