- A new treatment for Progeria, a rare genetic disease that accelerates aging, has been approved in the United States.
- This drug, Zokinvy, saves two and a half years of life expectancy for people with this disease.
Progeria is a disease rare which concerns approximately one in 4 to 8 million births, according to Inserm estimates. Also known as syndrome de Hutchinson-Gilford, it is characterized by the abnormal aging of cells which occurs from the first months of existence.
Children who suffer from this pathology present symptoms similar to those observed in individuals of an advanced age: joint pain, cardiovascular disorders, alopecia… the life expectancy linked to progeria is 12-13 years on average.
In order to increase life expectancy and improve the condition of these patients, American researchers have just completed clinical trials for a drug approved by the Food and Drug Administration (FDA) on Friday, November 20.
First treatment of this kind
Led by the American Progeria Research Foundation (Peabody, Massachusetts, United States), this research is the first to present results that have developed a drug to treat this disease. The treatment in question, called Zokinvy, comes in the form of capsules.
“This is only the first. We will find other even more effective treatments ”says Dr. Leslie Gordon, medical director of the Progeria Research Foundation and pediatric disease researcher at Hasbro Children’s Hospital (Providence, Rhode Island, USA).
Although not inherited, progeria is caused by genetic mutation which causes a damaging build-up in cells of a protein called progerin. Zovinky would block the production and accumulation of progerin, which would have the effect of slowing down premature aging.
An extended life expectancy of two and a half years
A total of 62 children took the drug twice a day. Their results were compared with those of 81 untreated children worldwide, based on their age and other characteristics. Participants were followed for 11 years, and those who took the drug lived an average of two and a half years longer.
A total of four studies were conducted at Children’s Hospital in Boston, 22 children and young adults who took this drug since 2010 or before. Leslie Gordon sees in this treatment a hope to allow patients to have “a longer life, a stronger heart and the possibility of moving towards a cure ”.
The price of Zokinvy has not yet been released but is likely to be expensive, given the rarity of the disease. The Californian company responsible for its development, Eiger BioPharmaceuticals, however, promises financial assistance so that all patients can benefit from it.