The U.S. Food and Drug Administration (FDA) has licensed the use of Progeria, a treatment for a remarkably rare genetic disorder in children. Because of this disorder, children age remarkably quickly, before reaching the age of puberty. Because of this, they have a small body and a tense look even before they reach school age. Most of these children die of heart disease in their early teens. However, a new drug, Zokinvi, slows down this rapid aging process.
“Zokinv is a healing medicine and not a cure. However, we can say that it can increase life expectancy by 2.5 years,” said Leslie Gordon, a pediatrician at Brown University. He is also the Medical Director of the Progeria Research Foundation.
Gordon and his family played a pivotal role in the creation of Zokvin. Their goal was to find a cure for Gordon’s son, Sam Bernie. He was diagnosed with progeria in 1998 and he died in 2014.
As a teenager, Sam became a recognizable face of Progeria. He has given interviews to leading publications about his condition, arranged public speeches (his speech at TEDx has been viewed more than 40 million times). Sam was filmed by HBO.
Sam was one of the first children to adopt Zokinv, and Gordon said this extended the life of his son. After his death, the family continued to run the Progeria Research Foundation as usual. They first published evidence of the effectiveness of Zokin in 2012, although obtaining an FDA permit took a lot of time and effort.
The main problem was the following: There are a total of 20 people with progeria in the US, and each of them was involved in a clinical trial. There was no control placebo group to determine the efficacy of the drug. After that, the foundation began searching for volunteers around the world.
“We were looking for information on children who were not being treated for zucchini. It was a kind of external control group for us,” said Leslie Gordon.
A new member, Eiger BioPharmaceuticals, was soon added to the fund. The latter has become the maker of zokin for the volunteers involved in the study. The pharmaceutical company also manufactures medicines to treat hepatitis. It was with their help that the FDA was convinced of the effectiveness of the drug.
Currently, Sam’s family is working on a better medicine to treat progeria.