A new drug test yielded results that helped children with progeria gain weight and improve cardiovascular health. The disease, which is caused by a single letter mix-up in the human genome – manifests itself in prematurely aging children. Most suffer hair-loss and smaller faces than normal before eventually succumbing to either heart attack or stroke before they finish their teens.
It was a repurposed experimental cancer drug called lonafarnib that saw the most promising results. Five years ago researchers brought in more than two dozen children suffering from the disease and began administering the drug. Nine of the children experienced a more than 50% increase in weight gain and modest improvements to cardiovascular health.
The findings were published in Proceedings of the National Academy of Sciences and are being hailed as a promising first step in fighting this tragic and heartbreaking disorder. Though as of yet it’s unclear if the drug tests will help actually extend the life of the patients.
“By keeping these children on lonafarnib and seeing how they do 5, 6, 10 years from now, that’s a long-term project,” Dr. Leslie Gordon, a staff scientist at Children’s Hospital said. “We know we have a treatment that does something, but we don’t know if those somethings are meaningful toward rate of heart attack, stroke, and longevity. We’re excited to find out.”
(Source: Boston Globe)