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Home » U.S. News » Health » Rapamycin A Drug Which May Treat Progeria

Rapamycin A Drug Which May Treat Progeria

Posted by: Jineta Raval    Tags:  aging disease, Drug, IBT, LA Times, Progeria, Rapamycin    Posted date:  July 12, 2011  |  No comment



Progeria, which begins in childhood, is known to be rare disease that affects an individual’s aging. The disease rapidly ages a person, giving them an average lifespan of 13 years. Having symptoms like hair loss, diminished subcutaneous fat, premature atherosclerosis and skeletal abnormalities, children in their teens typically die from heart attacks or strokes. According to International Business Times, a recent study that was published in Science Translational Medicine discovered that rapamycin, “an immunosuppressant drug, could treat Hutchinson-Gilford Progeria syndrome.”

LA Times describes how the disease is caused by a mutation in a protein called Lamin A, which maintains the cell’s nucleus.  As a result, it accumulates a toxic protein in cells, breaking down the cells and making them sick. Scientists have discovered that the rapamycin drug treatment on the affected progeria cells reverses the impairment from the toxic, removing waste and reducing the symptoms of aging. At the same time also having the ability to reduce aging symptoms in healthy cells.

IBT mentions how scientists believe that genetic programming and environmental damages are the causes of senescence, or biological aging. Accumulation of toxins damages to cells and DNA, and cross-linking of sugar to proteins are some examples of environmental damage. The reversal of these aging affects is known to slow the effects of natural aging. A study according to IBT describe how “mice treated with rapamycin live 13 percent longer for females and 9 percent longer for males. rapamycin treatment has also extended the lifespan of worms, fruit flies and yeast.”

Kan Cao of the University of Maryland in College Park, after reading the study with her colleagues treated the cells of three progeria patients and four health individuals with rapamycin in culture. LA Times describes how “the progeria cells showed a reversal in the structural alterations of the nucleus that are associated with the disease and less DNA damage… it also reduced the symptoms of aging in both the progeria and healthy cells.

Although the drug is known to be efficient, it can come with serious side effects if administered as an anti-aging drug to humans. It has the ability to suppress the immune system’s ability to fight disease and can cause “elevated levels of cholesterol and triglycerides in the blood” known as hyperlipidemia.

The drug works by interfering the pathway of mTOR(Mammalian target of rapamycin), slowing the process of aging. Calorie restriction diets are known to be associated with longevity, impeding mTOR.

Professor Randy Strong of University of Texas describes rapamycin’s effect to IBT as “tricking the cells into thinking that they’re depleted of nutrients. Rather than the animals losing weight—we haven’t noticed any weight loss—they may be just using their proteins more efficiently.”


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