Scientists from the Weizmann Institute of Science have discovered two molecules that could slow, and even reverse, the effects of the incurable and neurodegenerative Huntington’s disease.
Huntington’s disease stops parts of the brain from working properly over time. People living with the disease develop uncontrollable dance-like movements and abnormal body postures, as well as problems with behavior, emotion and thinking.
The brain is a well-guarded control center surrounded by a well-protected cell barrier, preventing substances from getting in or out of the brain, making treatment of Huntington’s almost impossible.
But a new study by Weizmann’s Prof. Rivka Dikstein identified two molecules that have managed to penetrate this barrier, potentially reducing levels of Huntington’s, as well as reversing some of its symptoms.
The treatment has already proven to reduce the mutant copy of the disease in mice, and alleviate some of the damage caused by it in the brain.
“We were delighted to find that small molecules managed to reach the brain without undergoing changes and without disintegrating along the way,” said Dikstein.
“While other experimental treatments require repeated brain or spinal surgery, these molecules, delivered orally or by means of an injection, could pave the way for an effective and safe treatment of Huntington’s disease.”
The study was recently published in EMBO Molecular Medicine.
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