New study of rare disease provides clues on aging

A new drug originally developed as a cancer treatment has show promise in helping children with the rare and fatal disease Progeria, which causes children to age rapidly. 

The Wall Street Journal reports that the drug, called Lonafarnib, appeared to slow, and in some cases reverse, some of the damage caused by the disease, including arterial stiffness, which also is linked to heart problems in the normal aging population.

Researchers found that arterial stiffness decreased by 35 percent, according to Science Daily, and that nearly all of the participants gained weight. 

"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," Leslie Gordon, MD, PhD, lead author of the study told Science Daily.

Gordon is also the mother of a 15-year-old son with Progeria.  Gordon told CNN that the study gives her hope that a cure is possible. 

"I think that we have just sort of just broken open the first of many possibilities here for the children," Gordon said.

"It is possible that some systems affected in Progeria may be resistant to treatment. But if a cure means getting rid of progerin and really affecting the cardiovascular system in a big way so that these children live longer lives, then trials gives us a lot of hope. I think we can do this for the children and I think it's what the children deserve."

Hutchinson-Gilford Progeria Syndrome, is extremely rare and only strikes approximately one in every 4 million to 8 million infants, reports CNN.  Only about 200 children worldwide have the disease. 

Most children born with Progeria die of heart attacks or strokes by the time they are 13 because of the accumulation of a protein called progerin. The children experience a loss of body fat, hair and are unable to gain weight. They often develop osteoporosis and their bones become brittle and weak. 

The study has been extremely promising and it's benefits may be extended to older people who suffer from general problems associated with aging. 

Francis Collins, director of the National Institutes of Health and one of the discoverers of the gene that causes Progeria, told the Wall Street Journal that it was important to question how the drug might benefit a wider group. 

"One concern is to make sure you are not inducing an untoward effect in people who are not as severe as kids with progeria," Dr. Collins said.