mRNA Victoria has awarded funding to two groundbreaking Alzheimer's disease research projects at the Florey Institute, marking a significant step toward harnessing mRNA technology for neurological conditions. The funding, announced by Victoria's Minister for Economic Growth and Jobs Danny Pearson MP, supports innovative approaches to both prevent and treat Alzheimer's disease using mRNA-based therapeutics.
APOE-Targeted mRNA Therapeutics for Treatment
Dr. Abdel Belaidi is developing an mRNA-based therapeutic system that represents the first attempt to use mRNA technology to penetrate the blood-brain barrier for neurological treatment. His approach builds on the discovery that certain individuals with genetic variants causing inherited Alzheimer's disease remain protected from developing dementia.
"These people carry rare genetic variants that protect against inherited Alzheimer's disease. I recently uncovered that APOE and APOE receptors exert an important function in the brain by protecting neurons from death," Dr. Belaidi explained. His research focuses on developing a therapy that uses mRNA to deliver beneficial protein variants directly to neurons, potentially slowing or halting disease progression.
The therapeutic targets apolipoprotein E (APOE), a protein that Dr. Belaidi's research has shown plays a crucial protective role in brain function. "I am now working to develop a therapy that will use mRNA to deliver beneficial protein variants to protect neurons and slow progression of the disease," he said.
mRNA Vaccine for Alzheimer's Prevention
Dr. Rebecca Nisbet is pursuing a complementary approach through the development of an mRNA vaccine designed to prevent Alzheimer's disease before symptoms appear. Her research targets amyloid-beta, a toxic molecule whose accumulation in the brain is one of the primary causes of Alzheimer's disease.
"One of the causes of Alzheimer's disease is the clumping together of a toxic molecule called amyloid-beta in the brain," Dr. Nisbet said. "Targeting amyloid-beta and clearing it from the brain shows promise as a treatment."
The vaccine aims to stimulate the immune system to produce protective antibodies that clear amyloid-beta from the body and prevent its accumulation in the brain. If successful, this approach could enable long-term pre-symptomatic treatment and potentially prevent Alzheimer's disease from developing in later life.
Addressing Critical Medical Need
Both projects address a significant unmet medical need, as Dr. Belaidi noted: "Alzheimer's disease is the leading cause of dementia and a major cause of death in Australia. Despite recent breakthroughs in mRNA technology and Alzheimer's research, we still lack effective treatments."
The research leverages the rapid advancement of mRNA technology that gained prominence during the COVID-19 pandemic. Since then, researchers worldwide have been exploring ways to apply this technology to neurological conditions, making these projects particularly timely.
Broader Implications for mRNA Therapeutics
The projects have implications beyond Alzheimer's disease treatment. Dr. Belaidi emphasized that his mRNA brain-penetrating technique "could be used to treat other neurological conditions," potentially opening new avenues for treating various brain disorders.
Dr. Nisbet highlighted the platform potential of her work, noting that "the project lays the foundation for establishing a platform for developing mRNA vaccines for neurodegenerative diseases, with the potential to expand Victoria's mRNA clinical pipeline."
Both researchers emphasized the strategic importance of their work for Victoria's position in the global biotechnology landscape. "This project not only has the potential to change the treatment landscape for Alzheimer's disease but also positions Victoria as a leader in the development of mRNA-based therapies, enhancing the State's reputation as a hub for innovative biomedical research," Dr. Belaidi said.
