New Potential Treatment for Progeria, Rapamycin

Article

A recent study focuses on a possible future Progeria treatment which will hopefully impede the effects of the childhood disease.

The Progeria Research Foundation has published an optimistic report concerning a recent study on the possible future Progeria treatment—a drug called rapamycin—which will hopefully impede the effects of the disease.

The study was performed at National Institutes of Health and Massachusetts General Hospital in Boston.

Progeria, also known as Hutchinson—Gilford Progeria Syndrome, is a disease which causes accelerated aging in children. It is an extremely rare disease—only seventy-eight children worldwide are living with it today—but it is debilitating and often kills children who suffer from it before they reach adulthood.

Rapamycin is an immunosuppressant drug, andhas been typically used to prevent organ rejection in transplant patients. In this recent study, though, the researchers found that the drug decreased the amount of protein responsible for rapid aging by 50% in progeria cultured cells and mice, causing the cells to live longer in both experiments.

Other studies have found that female mice treated with rapamycin lived 13% longer than untreated females, while male mice treated with rapamycin lived 9% longer than untreated males.

One drawback of this study, however, is that it was performed on non-Progeria mice.

Even so, the results are promising, and not just for children suffering from Progeria; finding a treatment (cure?) for aging would undoubtedly benefit mankind, as such knowledge could lead to treatments for disorders and diseases caused by aging, such as arthritis.

To further this excitement over the possible applications of this drug, rapamycin was found to decrease the symptoms of aging in healthy cells as well as the progeria cultured cells.

The possibilities of the drug, however, are reportedly undercut by its dangerous potential, as there could be some severe side effects associated with administering the drug as an anti-aging treatment to humans. The nature of rapamycin is to suppress the immune system’s ability to defend the body against diseases; also, it has been found to cause hyperlipidemia, which can lead to elevated levels of cholesterol and triglycerides in the blood.

This study was published in the journal Science Translational Medicine.

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