In a breakthrough that could reshape treatment for Parkinson’s and Alzheimer’s, scientists have discovered how to block brain cells from dying — a first step toward next-generation neuroprotective drugs.
Key Points at a Glance
- Researchers identified a molecule that blocks the cell death protein BAX
- Findings open a new path to treat neurodegenerative diseases like Parkinson’s
- Study builds on decades of research into how and why cells self-destruct
- Discovery made using advanced screening at Australia’s National Drug Discovery Centre
Every day, millions of cells in our bodies die off — a natural, necessary process known as programmed cell death. But when this mechanism goes awry, particularly in the brain, it can lead to devastating consequences. Excessive neuronal death is at the heart of neurodegenerative diseases like Parkinson’s and Alzheimer’s. Until now, medicine has struggled to slow or stop that decline. A new study by Australian scientists could change that.
Researchers at the Walter and Eliza Hall Institute (WEHI) have discovered a way to selectively block this lethal process. The study, published in Science Advances, reveals a small molecule capable of stopping BAX — a key protein that triggers cell death — from reaching and damaging the mitochondria, the cellular “power plants” essential for survival.
“For the first time we could keep BAX away from mitochondria and keep cells alive using this molecule,” said Kaiming Li, lead author and member of the Dewson Lab at WEHI. This finding may sound technical, but its implications are vast: protecting neurons from premature death could potentially transform how we treat degenerative conditions, where no current therapies can halt progression.
BAX is a member of the BCL-2 protein family and functions as a pro-death signal. Once activated, it perforates the mitochondrial membrane, initiating a cascade of events that ultimately dismantle the cell from within. In cancer, drugs that trigger cell death via BAX have revolutionized treatment. But doing the opposite — blocking this pathway to preserve neurons — has been far more difficult.
That challenge may now be surmountable. Using high-throughput screening technologies at the National Drug Discovery Centre, WEHI scientists sifted through more than 100,000 chemical compounds. They found one that acts as a molecular “bodyguard,” binding to BAX and preventing it from attacking mitochondria. While this wouldn’t help most cells — where other death pathways exist — neurons appear uniquely vulnerable to BAX, making this a promising neuroprotective strategy.
“This could be game-changing,” said Professor Grant Dewson, co-corresponding author and head of the WEHI Parkinson’s Disease Research Centre. “Currently there are no treatments that prevent neurons from dying to slow the progression of Parkinson’s. Any drugs that could be able to do this could be game changing.”
The research builds on WEHI’s rich history in cell death science. Back in 1988, their scientists discovered the first protein that prevents apoptosis (programmed cell death), laying the groundwork for today’s anti-cancer drugs. The current work flips that script — instead of killing rogue cells, it aims to save them.
Professor Guillaume Lessene, co-author of the paper, emphasized how this molecule’s specificity could be its greatest asset. “While not the case in most cells, in neurons turning off BAX alone may be sufficient to limit cell death,” he explained.
The discovery also showcases the power of multidisciplinary collaboration. Chemists, molecular biologists, and neuroscientists joined forces to uncover a mechanism that had remained elusive for decades. It’s a reminder that the path to new medicine is rarely a straight line — but with the right tools and minds, progress is possible.
For patients, this could mark the beginning of a new chapter. Parkinson’s and Alzheimer’s affect millions worldwide, slowly robbing individuals of memory, movement, and independence. Current treatments offer only symptomatic relief. The potential to intervene at the cellular level, to actually protect neurons from destruction, holds enormous promise.
The WEHI Parkinson’s Disease Research Centre is now turning its focus to translating this molecular discovery into a viable drug candidate. They’re exploring how to refine the molecule, test it in models of disease, and one day move into clinical trials. Backed by the Bodhi Education Fund and Australia’s National Health and Medical Research Council, the team is optimistic.
Cell death has long been seen as inevitable in degenerative diseases — a one-way path. But this research suggests there might be an exit, hidden deep within our cells, waiting to be unlocked.
Source: Walter and Eliza Hall Institute
