Sydney– In a significant breakthrough that could pave the way for new treatments for neurodegenerative diseases, Australian scientists have identified a small molecule capable of blocking brain cell death. The discovery could lead to promising therapies for conditions such as Parkinson’s and Alzheimer’s disease.
The research, conducted by a team at the Walter and Eliza Hall Institute of Medical Research (WEHI) in Melbourne, focused on finding chemical compounds that can inhibit the process of cell death—an underlying cause of many degenerative brain disorders.
After screening more than 100,000 chemical compounds, the team identified a small molecule that effectively neutralizes a protein called BAX, a well-known trigger of cell death. By inhibiting BAX, the molecule successfully prevented the destruction of neurons in laboratory models.
“We were thrilled to find a small molecule that targets the killer protein BAX and stops it from functioning,” said Professor Guillaume Lessene of WEHI. “In most cells, shutting down BAX alone isn’t enough, but in neurons, turning off this protein may be sufficient to prevent cell death.”
BAX promotes cell death by damaging mitochondria—the energy-producing structures in cells. Blocking its activity helps preserve the mitochondria and, by extension, the survival of the neuron.
“For the first time, we were able to prevent BAX from reaching the mitochondria and keep brain cells alive using this molecule,” said lead author Kaiming Li from the Dewson Lab. “This discovery opens the door to developing next-generation cell death inhibitors to fight degenerative diseases.”
The findings, published in the journal Science Advances, mark a major step toward developing drugs that can halt or slow the progression of neurodegenerative conditions, which currently have no cure and limited treatment options.
While drugs that promote cell death have revolutionized cancer therapy, finding compounds that can prevent cell death—especially in neurons—has been a much greater challenge. This study demonstrates the feasibility of such an approach and could offer a blueprint for future therapies.
“This research provides a proof of concept that drugs targeting the cell death pathway may lead to the development of much-needed disease-modifying treatments for conditions like Parkinson’s and Alzheimer’s,” the researchers concluded. (Source: IANS)
