Slow the Aging Kids
Dave and I complain about getting old, but that's because together we have more than a hundred years. In children with a genetic disorder called Progeria, they're old and frail before kindergarten and most die in their early teens from heart disease or stroke.
Now, for the first time, a drug called Zokinvy can extend their lives. It blocks an enzyme that modifies cellular proteins. This enzyme adds molecules such as fats onto proteins so that they can attach to cell membranes.
In children with Progeria too much fat is added onto these proteins, causing cell nuclei to become unstable and leading to early cell death. Children with progeria appear normal at birth and then fail to grow at a normal rate. They develop prominent eyes, a thin nose, small chin, protruding ears, hair loss, loss of fat under the skin and begin to look aged. Their arteries also start to harden so that they face the risk of heart failure, heart attack and stroke; however, their intelligence and motor coordination are unaffected.
But for the eighty children in clinical trials with the drug, Zokinvy, their lives were increased. In the first three years of treatment, their lifespan grew by three months. After eleven years of the drug, the children lived an average two and a half years longer.
That's significant given the short lifespan of those with Progeria and offers hope that one day, it will be stretched even more.
More Information
FDA Approves First Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies
The U.S. Food and Drug Administration approved Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older. Zokinvy is not approved for use in patients with other progeroid syndromes or laminopathies...
FDA Approves First Drug For Rare, Rapid-Aging Genetic Disorder
The Food and Drug Administration has approved a treatment for progeria, a genetic disorder that causes children to age rapidly. Zokinvy is the first drug approved by the agency for the disorder...
Progeria Research Foundation: About Progeria
Hutchinson-Gilford Progeria Syndrome ("Progeria", or "HGPS") is a rare, fatal genetic condition characterized by an appearance of accelerated aging in children. Its name is derived from the Greek and means "prematurely old." While there are different forms of Progeria*, the classic type is Hutchinson-Gilford Progeria Syndrome, which was named after the doctors who first described it in England; in 1886 by Dr. Jonathan Hutchinson and in 1897 by Dr. Hastings Gilford...