SYDNEY: A team of Australian scientists has identified a small molecule that blocks cell death, an advance that could lead to new treatments for neurodegenerative conditions like Parkinson’s and Alzheimer’s.
The team from the Melbourne-based Walter and Eliza Hall Institute of Medical Research (WEHI) aimed to find new chemicals that block cell death, which could help treat degenerative diseases in the future.
The findings offer hope for treatments that could slow or stop the progression of neurodegenerative diseases.
After screening over 100,000 chemical compounds, the team found a small molecule that targets a killer protein called BAX.
By interfering with a well-understood cell death protein, the molecule effectively stopped cells from dying.
“We were thrilled to find a small molecule that targets a killer protein called BAX and stops it working,” said Professor Guillaume Lessene from WEHI.
“While not the case in most cells, in neurons turning off BAX alone may be sufficient to limit cell death," Lessene added.
While drugs that trigger cell death are transforming the treatment of certain cancers, the development of cell death blockers -- that could be similarly game-changing for neurodegenerative conditions -- has proven challenging.
The new molecule targets a killer protein called BAX, which kills cells by damaging mitochondria, the powerhouse of cells.
“For the first time we could keep BAX away from mitochondria and keep cells alive using this molecule,” said lead author and Dewson Lab researcher Kaiming Li.
“This could pave the way for next-generation cell death inhibitors to combat degenerative conditions,” Li said, in the paper published in the journal Science Advances.
While drugs that trigger cell death are transforming the treatment of certain cancers, the development of cell death blockers – that could be similarly game-changing for neurodegenerative conditions -- has proven challenging.
The study demonstrates the potential to identify drugs that block cell death and may open a new avenue to find much-needed disease-modifying drugs for Parkinson’s and Alzheimer’s, said the researchers.