Pill for rare ageing disease on the horizon

By Dr. Ananya Mandal, MDJul 1 2011

Scientists have found that a drug used in transplant patients could reverse the effects of premature ageing and extend human life by over 10 years.

A team of US scientists took skin cells from children with Hutchinson-Gilford Progeria Syndrome (HGPS), a rare genetic condition in which babies rapidly grow old and die at around 12 years, and turned them healthy again by using rapamycin, known as the “forever young drug”. The word progeria comes from the Greek word meaning “prematurely old.”

Rapamycin, used to suppress the immune system in organ transplants, has been created from a bacterium found in the soil on Easter Island. Past research has hinted that the drug may have the power to extend human lifespan by more than a decade.

The new study, published in Science Translational Medicine, may give experts clues toward a cure for Progeria patients. While the drug has only been tested in Petri dishes, scientists are excited about rapamycin's potential to treat the condition.

“Part of the problem with aging starts when debris is accumulating in the cells and it's not getting removed, and this particular drug is able to enhance the removal process,” said Dr. Dimitri Krainc, lead author of the study. “It would be too optimistic to say this could completely cure Progeria patients, but we're hoping that this drug could make these kids live longer with fewer complications.”

“Parents and patients have reason to be hopeful and excited,” said Krainc. “It's a devastating illness, and these effects are pretty dramatic. We really hope that this will help translate into clinical treatment.” Krainc said the Progeria Research Foundation is “doing everything possible” to create a clinical trial with the drug to use in Progeria patients as soon as possible.