European patent for invention to help treat Huntington's disease


prof. Grzegorz Węgrzyn

Researchers from the UG Department of Biology with a new European patent. The patent relates to a treatment method for Huntington's disease, a severe genetic disorder that runs with neurodegenerative processes and very severe symptoms. The invention offers hope for a drug that will slow down or halt the progression of the disease.

The technology developed enables the medical use of 5,7-dihydroxy-3-(4-hydroxyphenyl)-4H-1-benzopyran-4-one, a compound popularly known as genistein, to produce a drug that causes a reduction in the size and number of protein aggregates and a decrease in the total amount of the mutant form of huntingtin. Consequently, this leads to a significant slowing down or even inhibition of the progression of Huntington's disease. The invention was worked on by a research team comprising scientists from the UG Biology Department: prof. dr hab. Grzegorz Węgrzyn, dr Sylwia Barańska, dr Aleksandra Hać and dr Karolina Pierzynowska.

Huntington's disease belongs to the group of genetic neurodegenerative diseases. In developed countries, the disease occurs at a frequency of approximately 1 in 15,000 people and represents a major economic burden due to the high cost of patient care, amounting to approximately EUR 25,000 per person per year. The direct cause of the disease is a mutation in the gene encoding the huntingtin protein. The mutated form of the protein shows a tendency to form protein aggregates in cells, leading to neuronal dysfunction and progressive neurodegeneration of certain areas of the brain, with the clinical consequences being impaired motor coordination, and dementia.

Despite ongoing research, no effective treatment for Huntington's disease has emerged to date. Existing drug therapies and physiotherapies primarily serve to alleviate the symptoms of the disease. Genistein, a compound from the isoflavone group, naturally found in plants in free form (as aglycone) or, more commonly, glycosidically bound (genistein), results in the reduction of the mutant form of huntingtin in human cell cultures.

‘The patent relates to a method of treating Huntington's disease, a genetic disease with a severe course of neurodegenerative processes and very severe symptoms. To date, there is no effective treatment for this disease,’ says prof. Grzegorz Węgrzyn. ‘In our research, we have shown that the chemical compound covered by the patent causes a reduction in the level of toxic protein in cells from patients with Huntington's disease in a cellular model of the disease and in an animal model (mice). Furthermore, we obtained a definite improvement, and even normalisation, of the behaviour of mice with Huntington's disease after treatment with the proposed therapeutic preparation. This gives hope for a new, effective drug for this hitherto incurable and prematurely fatal disease.’

The essence of the invention is the medical use of 5,7-dihydroxy-3-(4-hydroxyphenyl)-4H-1-benzopyran-4-one to produce a drug that actively inhibits the size and number of protein aggregates. Studies conducted have shown the positive effect of genistein in the form of increased cell survival. The method described results in a reduction of the mutant form of huntingtin in human cell cultures.

Fot. Alan Stocki

dr hab. Karolina Pierzynowska. Fot. Alan Stocki/UG

Przypomnijmy, że zespół naukowców z UG pod kierownictwem prof. Grzegorza Węgrzyna uzyskał w roku 2021 patent na wynalazek z wykorzystaniem genisteiny pt. „Genisteina do zastosowania do leczenia choroby Alzheimera”.

- Fakt, że mamy te dwa patenty na dwie choroby, z których jedna - choroba Alzheimera dotyczy większej liczby pacjentów, daje nadzieję, że zainteresować się nimi mogą firmy farmaceutyczne - mówi dr hab. Karolina Pierzynowska.  

Julia Bereszczyńska/Press Office