A new study carried out by the Weizmann Institute of Science in Rehovot has identified two molecules capable of penetrating the blood-brain barrier and reducing the levels of a defective protein that causes Huntington’s disease, an incurable neurodegenerative disorder.
The research not only slowed the progress of the disease in mouse models but even reversed some symptoms, the Institute said.
The signs and symptoms of Huntington’s disease—slight involuntary movements, general clumsiness and increased anxiety—most commonly emerge around the age of 40. The disease develops over time and inevitably leads to death.
Past attempts to treat the disease were unsuccessful, while drugs aimed at tackling the root cause—the defective protein—found it difficult to distinguish the defective protein from the normal one.
During the new study, “We were delighted to find that small molecules managed to reach the brain without undergoing changes and without disintegrating along the way,” said Weizmann’s professor Rivka 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,” she continued.
“It’s become clear that a certain function of large regulatory proteins can be precisely targeted using tiny molecules without harming the overall functioning of these proteins. This understanding could lay the foundations for new treatments for a variety of diseases,” she said.
