Progeria is a genetic disorder that affects approximately 200 children every year.
Children with progeria suffer from accelerated aging and most often succumb to complications associated with old age.
Children with progeria have a misplaced letter or base pair in their genome, resulting in an inefficient waste removal system in the cell.
Normally, as people age, more protein slowly accumulates in the cell. In children with progeria, there is an accelerated accumulation of one specific toxic protein called progerin.
Healthy people have progerin in their cells, however they also have a waste removal system, which in normal cells removes any excess and unwanted proteins.
In a recent study published in Science Translation Medicine, researchers found that a drug called Rapamycin may be the clue to finding a treatment or cure for this devastating disorder.
The drug is given to transplant patients because it suppresses the immune system, preventing organ transplant rejection.
Researchers have found that cells in patients with progeria look old and unhealthy. Once the cells are injected with the drug they look healthy again. Researchers believe that the drug may activate the cell’s innate waste removal system that removes the excess progerin.
The results of the study are promising, however there are serious complications associated with Rapamycin. The drug increases cholesterol and suppresses the immune system, making it hard to fight off infections.
Scientists may consider using a modified version of Rapamycin, called RAD001 for clinical trials because it has fewer side effects.
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