Clinical trials with the first drug for progeria have been successful, US scientists said. The study’s findings were published in the journal Proceedings of the National Academy of Sciences
The farnesyltransferase inhibitor (FTI), initially used in the treatment of cancer patients, has caused children suffering from progeria to gain weight, improve bone structure, and most importantly, improve cardiovascular function.
The discovery that some of the blood vessel destruction processes in progeria can be slowed or even reversed with 2,5 years of treatment with an FTI called lonafarnib is a major breakthrough as cardiovascular disease is the leading cause of death in children with progeria, says Leslie Gordon. , the lead author of the study, director of the Progeria Research Foundation and the mother of a child with this condition.
Progeria, also known as Hutchinson-Gilford progeria syndrome (HGPS), is a very rare genetic disorder that causes the body to age very quickly. Children suffering from progeria develop diseases that are characteristic of the elderly. As a result of rapidly progressing atherosclerosis, children with progeria are at risk of heart attacks and strokes as early as 5 years of age, and their average life expectancy does not exceed 13 years.
28 children with progeria from 16 countries participated in the study of the activity of a farnesyltransferase inhibitor. At the start of the tests, they accounted for 75 percent. of all known cases of this disease in the world.
The children took FTI orally twice a day and traveled to Boston every four months to be checked at Boston Children’s Hospital.
The FTI allowed to block the attachment of the farnesyl group to progerin, an aging-related protein, which is overproduced in children with progeria. Progerin impairs the functioning of cells in the human body, and the presence of the farnesyl group is responsible for part of the harmful effect. Progerin is also produced in the body of healthy people, and its production increases later in life.
Scientists hope that FTI will help in the future in the fight against cardiovascular disease that affects millions of people who age at a normal pace (PAP).
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