Premature Aging Drug, Rapamycin, Shows Promise For Progeria Patients As Well As Extending Human Life

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Main Category: Pediatrics / Children's Health
Also Included In: Genetics;  Seniors / Aging;  Cardiovascular / Cardiology
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Nicknamed the "forever young drug", Rapamycin, which was created from a substance found in the soil of Easter Island, has been found to have potential for reversing the effects of premature aging, and could even help extend our lifespans by ten years, researchers reported in Science Translational Medicine.

Rapamycin, also known as Sirolimus, is an immunosuppressant medication used to prevent rejection after organ transplantations. It is commonly used after kidney transplants. It is a macrolide antibiotic and was first discovered in Rapa Nui, Easter Island, hence its name. It is a product of the bacterium Streptomyces hygroscopicus. It was originally developed as an antifungal agent. However, as soon as its immunosuppressive and cell growth inhibiting (antiproliferative) properties were discovered, scientists focused on those two areas instead. In laboratory experiments, it has been shown to extend the lifespans of mice. Experts believe it may also be useful for treating certain cancers.

In this latest study, Rapamycin was used on children with HGPS (Hutchinson-Gilford Progeria Syndrome), a devastating genetic condition in which the child ages quickly and reaches old-age by the time they are 12 years old. The accelerated aging is caused by an accumulation in every cell in the body of a protein called Progerin.

The scientists explained that Rapamycin got rid of the progerin in the cells, leaving them healthy.

HGPS is a very rare disease. According to the NIH (National Institutes of Health), approximately 100 cases have been documented over the last 100 years.

Co-author and NIH Director Dr. Francis Collins, said:

"We found it pretty exciting that this drug has such a profoundly positive effect on cell cultures. The ability to understand the molecular basis [of diseases] and develop targeted therapies makes this a very exciting time to be a physician."

The researchers concluded in an Abstract in the journal:

"Our findings suggest an additional mechanism for the beneficial effects of rapamycin on longevity and encourage the hypothesis that rapamycin treatment could provide clinical benefit for children with HGPS."

Progeria affects children, who age much faster than they should. There are different forms of Progeria; Hutchinson-Gilford Progeria Syndrome (HGPS) is the most common. The condition was first described in an academic journal in 1886 by Dr. Jonathan Hutchinson (England), and then in 1897 by Dr. Hastings Gilford (England).

It is estimated that between 1 newborn in every 4 to 8 million has Progeria. Both sexes have the same risk. Progeria rates appear to be the same all over the world, regardless of race, geographical location, or ethnic group.

Babies with Progeria are born looking healthy. At the age of about 10 to 24 months they start showing signs of accelerated aging, which may include: Aging skinFailure to growGeneralized atherosclerosisHair lossJoint stiffnessLoss of body fatHip dislocationStrokePatients with Progeria die between 8 and 21 years of age (average 13 years). Virtually all patients die from heart disease. Patients commonly have cardiovascular problems, such as angina, stroke, hypertension (high blood pressure), enlarged heart, and heart failure - all of them are conditions associated with aging.

Experts have always said that any breakthrough in Progeria treatment would probably have results which would also benefit adults with diseases associated with aging.

Specialists say that it is unlikely that Progeria is an inherited disease. It is probably due to a rare genetic change which happens randomly. Non-twin siblings of a child with Progeria have the same risk of developing the condition as any other child outside the family. However, in approximately 1 in every 100 cases of HGPS, the syndrome may be passed down to the next generation.

"Rapamycin Reverses Cellular Phenotypes and Enhances Mutant Protein Clearance in Hutchinson-Gilford Progeria Syndrome Cells"
K. Cao, J. J. Graziotto, C. D. Blair, J. R. Mazzulli, M. R. Erdos, D. Krainc, F. S. Collins
Sci. Transl. Med. 3, 89ra58 (2011).

Written by Christian Nordqvist
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