Rapamycin – an immunosuppressant drug used to prevent organ rejection in transplantation – has been found to reverse a very rare, fatal genetic disease called Hutchinson-Gilford progeria syndrome, characterized by very rapid, dramatic appearance of aging.
Remember our friend and Mprize Lifespan Achievement winner, Dr. David Z. Sharpe? He and his team have already proven that Rapamycin extends the lifespan in healthy mice and now researchers are hoping to uncover new insights into treating progeria and other diseases related to aging.
Published in the journal Science Translational Medicine, a new study finds that Rapamycin can reverse the defects from skin cells of patients with progeria (namely, decreased growth, deformities in their membranes, and early death) by enhancing the cells’ ability to degrade the protein progerin, accumulated in excessive amounts in progeria patients who suffer with issues typically linked with old age: balding, joint pain, hardened skin, hip dislocation, heart disease, and not to mention arteriosclerosis that leads to higher chances of heart attack and stroke.
Top image shows a toxic protein called progerin (green) spread evenly throughout the cells.
Bottom displays the treated cells with the concentrated protein removed much more effectively.
The findings may have relevance beyond the treatment of this rare genetic disease. Progerin accumulation in normal cells, though not nearly as concentrated as those of progeria sufferers, may still be a factor of the aging process.
Previous research has shown that cellular maintenance failure is a key component of aging. Associate professor of neurology at Harvard Medical School and one of the authors of the paper, Dimitri Krainc indicates that age-related diseases like Parkinson’s and Alzheimer’s also result in defects of the “trash-removal” system of the cells. In simpler terms: Failure of cellular maintenance is a key component of aging.
“With normal aging… you start accumulating by-products of normal cell functions,” says Krainc. Though this study only focused on the effects of Rapamycin on progerin, it may also help clean up other toxic proteins as well.
Dr. David Sinclair, Mprize competitor and director of the Paul F. Glenn Laboratories for the Biological Mechanisms of Aging at Harvard Medical School, hopes that “the study increases the search for molecules to replace Rapamycin” so as not to have the immunosuppressant side effects. Such alternatives could be a major step forward in the fight against aging.
Scaffidi P, Gordon L, Misteli T (2005) The Cell Nucleus and Aging: Tantalizing Clues and Hopeful Promises. PLoS Biol 3(11): e395. doi:10.1371/journal.pbio.0030395
Vezena, Kenrick. “Drug Reverses ‘Accelerated Aging’ in Human Cells.” Technology Review. MIT Technology Review, 29 June 2011. Web. 1 July 2011.