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Rapid-Aging Disease In Kids And Its Potential Treatment From A Organ Transplant Drug

Progeria
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A new research has found that a drug presently used to counteract rejection among recipients of organ transplant may be instrumental in reducing DNA cell damage in children who experience rapid ageing.

The researchers of US National Institutes of Health along with a couple of universities and hospitals have found the antibiotic rapamycin to help in cases of children who have the Hutchinson-Gilford Progeria Syndrome (or HGPS). Progeria is known to be deadly during the teenage years of those who suffer from it, and have been documented 100 times since its discovery at the start of the 20th century.

Rapamycin was discovered to increase the ability of the cells to flush out a protein called progerin, the same protein that triggers skin and joint problems in children, in addition to promoting cardiovascular diseases. In normal cells, progerin is found in minimal amounts and only progresses with age.

NIH Director Dr. Francis Collins, a researcher in the study published in the June 29 journal Science Translation Medicine, said that they find the drug’s profoundly positive effect on cell cultures to be pretty exciting. He went on to add that this is one of the best part of his time being a physician - as he is challenged to further understand the molecular basis of diseases and to develop targeted therapies thereafter.

Researchers have found Rapamycin to focus on a different path than the already discovered one class of cancer-fighting drugs known as farnesyltransferase inhibitors (FTIs). Progeria Research Foundation’s medical director and co-founder, and also a mother with a teenage son who has a Progeria disorder - Dr. Leslie Gordon said that combining the pathways used by FTIs and Rapamycin may “someday prove to be a one-two punch to progeria.”

Gordon said that they are incredibly fortunate as they have a wealth of information to draw from, and the potential to attack progeria from different angles is very plausible. She further mentioned that as the drug’s safety has been established by the US Food and Drug Administration, approving the drug to treat progeria may now be streamlined.

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