JOIN US A drug first developed to cure cancer has shown great promise in treating children with Progeria, a rare and fatal rapid-aging disease, a new study has found.
Promising results have emerged from the first-ever clinical drug trial for the condition, including improvements in weight gain, bone structure and heart health.
A third of children placed on the drug Ionafarnib demonstrated a greater than 50 per cent increase in annual rate of weight gain or switched from weight loss to weight gain, due to increased muscle and bone mass.
On average skeletal rigidity, which was highly abnormal in patients at the time of the trial, improved to normal levels after treatment, the Daily Mail reported.
Arterial stiffness, which is linked with an increased risk of having a heart-attack, decreased by 35 per cent.
The genetic condition, which affects one in eight million people, causes the body to age eight times faster than normal.
The disorder is said to have inspired the F Scott Fitzgerald novella and Brad Pitt movie The Curious Case of Benjamin Button, in which the character is born an old man and ages backwards.
“To discover that some aspects of damage to the blood vessels in Progeria cannot only be slowed by the drug called lonafarnib, but even partially reversed within just 2.5 years of treatment is a tremendous breakthrough, because cardiovascular disease is the ultimate cause of death in children with Progeria,” study author Dr Leslie Gordon, from Boston Children’s Hospital, said.
The two-and-a-half year drug trial involved 26 children with the disease - which was three-quarters of the known Progeria cases when it started.
The study was published in the journal Proceedings of the National Academy of Sciences.