A drug for Huntington's disease that will minimize its cause instead of just alleviating symptoms has been developed by scientists at the Faculty of Biology of the University of Gdańsk.
Huntington's disease is a progressive neurodegenerative disease that causes the death of neurons in the brain. Some patients describe it as a combination of Parkinson's disease, Alzheimer's disease, and motor neuron disease.
Its symptoms include emotional disorders (mood changes, irritability, depression) memory impairment and deteriorating physical coordination, usually after the patient reaches 30 years of age. The disease kills slowly, within 10 to 20 years of the onset of symptoms. Symptoms can be relieved, but their progress could not be stopped. The disease affects 7 to 10 people out of 100,000, men and women equally.
Now scientists say they have come up with a solution.
One of the scientists involved, Professor Anna Herman-Antosiewicz, said: “The essence of the invention is the new use of 2-isothiocyanatoethylbenzene, a compound naturally occurring in cruciferous plants, for the production of a drug that actively inhibits the accumulation of abnormal protein deposits, which causes Huntington's disease.
“These deposits disrupt the proper functioning of nerve cells and lead to their death, and 2-isothiocyanatoethylbenzene not only prevents their accumulation, but also accelerates their elimination.
“Importantly, this compound is relatively safe for other, normal cells. All this makes it a promising drug for this neurodegenerative disease (and probably also for other diseases where abnormal proteins accumulate in nerve cells), a drug with minimal toxicity to healthy cells.”
Monika Rogo from the University of Gdańsk press office added: “Using this compound could allow cells to survive despite having a defective copy of the gene encoding the toxic form of the huntingtin protein by inhibiting the production of this toxic protein and allowing it to be removed efficiently by the cell.
“As a result, the compound could protect nerve cells from death, a major cause of neurodegeneration in Huntington's disease, and therefore prevent or slow the development of the disease.”
The inventors of the new drug are: Professor Anna Herman-Antosiewicz, Dr. Aleksandra Hać from the Department of Biology and Medical Genetics and Joanna Brokowska from the Department of Molecular Biology.
(PAP)
author: Robert Pietrzak
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