Cancer Drug Used in Treatment of Progeria

A new drug has been found to treat progeria, a rare rapid aging disease, that affects several children across the world

Researchers from the Progeria Research Foundation (PRF) and Boston Children's Hospital have found that farnesyltransferase inhibitor (FTI) - a drug originally developed to treat cancer - could be used to treat progeria.

Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS), is a rare, fatal genetic disease characterised by an appearance of accelerated aging in children. All children with progeria die of heart disease that affects millions of older adults in their 60s or 70s, but these children may suffer heart attacks and strokes as early as age five. They die before they become 13.

The discovery was made while analysing the data of more than 25 children from six different countries.

During the study, 26 children with progeria travelled to Boston every four months to receive comprehensive medical testing through the Boston Children's Hospital's Clinical and Translational Study Unit. The doctors gave FTI twice a day over the course of the study.

Researchers then evaluated the children's rate of weight gain compared to their pre-therapy rate as the primary outcome because children with progeria experience severe weight loss.

The study found that children improved their ability to gain additional weight and increased flexibility of blood vessels or improved bone structure.

"To discover that some aspects of damage to the blood vessels in progeria can not only be slowed by the FTI 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," said Leslie Gordon, medical director at the Progeria Research Foundation.

One in three children demonstrated a greater than 50 percent increase in annual rate of weight gain or switched from weight loss to weight gain, due to increased muscle and bone mass.

Researchers also found that skeletal rigidity (which was highly abnormal at trial initiation) improved to normal levels after FTI treatment. They also found that arterial stiffness, strongly associated with atherosclerosis in the general aging population, decreased by 35 percent. Vessel wall density also improved with treatment.

"One of the main reasons we achieved breakthrough results in this first trial is because of the tremendous supporters who provided funding, and helped get us one step closer to achieving our ultimate goal - a cure for progeria," said Audrey Gordon, Executive Director at the Progeria Research Foundation.