A Cure for Progeria, the Disease that Makes Children Die of Old Age

One in every 4 million children suffers from a genetic disorder called progeria that causes them to age prematurely — developing wrinkled skin and baldness before the age of 10, and usually dying of heart disease or stroke by puberty. The disease has always intrigued researchers interested in unlocking the genetic key to aging. Now it looks like a group of geneticists at the National Human Genome Research Institute in Maryland may have found a drug that can reverse the effects of heart disease caused by premature aging. In an article published today, the team also suggests that this could have ramifications for adults with heart disease too. The group, led by geneticist Francis Collins, used transgenic mice designed to develop symptoms of heart disease like human children with progeria do. They treated the mice with a cancer drug called tipifarnib, part of the FTI family of drugs which are being used experimentally to treat several forms of cancer. It worked incredibly well. Below, you can see a diseased blood vessel at left, and on the right a healthy one treated by the drug.


According to Collins:

This approach worked much better than we thought it would. Not only did this drug prevent these mice from developing cardiovascular disease, it reversed the damage in mice that already had disease.

Now the question is whether it will work in humans. Study co-author Elizabeth Nabel said:

If these drugs are found to have similar effects in children, this could mark a major breakthrough for treating this devastating disease. In addition, these findings shed light on the potential role of FTI drugs to treat other forms of coronary artery disease.


In addition, the more the researchers learn about reversing the effects of premature aging, the more tools they'll have to reverse the effects of normal aging in the general population. Not only could these FTI drugs prevent kids with progeria from dying young — they could potentially prevent them from dying for centuries. Top image via Carlen Altman. Anti-cancer drug prevents, reverses cardiovascular damage in mouse model of premature aging disorder [NIH News]

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