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Drug Extends Progeria Survival


A therapy originally developed to treat cancer could help children with this rare, fatal disease to live longer.

A study published in the Journal of the American Medical Association suggests that an experimental drug therapy can extend the lives of children with progeria, a rare genetic disorder that causes premature aging and death.

The research showed that children with progeria who were treated with lonafarnib, a drug originally developed to treat cancer, were more likely to survive over the course the study compared with children with progeria who did not receive the drug. The study is preliminary, but the researchers say the results suggest a promising avenue for treating a condition for which there are currently no approved therapies.

“This study provides supporting evidence that we can begin to put the brakes on the rapid aging process for children with progeria,” says Leslie Gordon ScM’91 MD’98 PhD’98, lead author and a professor of pediatrics (research). “These results provide new promise and optimism to the progeria community.”

The study took place at Boston Children’s Hospital, Brown University, and Hasbro Children’s Hospital.  Researchers followed 27 children with progeria who had taken twice-daily doses of lonafarnib in a clinical trial at Boston Children’s. The study group was compared to a group of 27 children of similar age who had progeria but were not part of the trial and did not receive the drug. The study showed a significantly lower mortality rate in the group receiving lonafarnib treatment. After two years, mortality in the treatment group was 3.7 percent, compared to 33.3 percent in the untreated group.

Francis Collins, MD, PhD, director of the National Institutes of Health, led the lab that first identified the mutation that causes progeria.

“My lab did some of the original research on cellular and mouse models that showed potential benefit of this class of drugs for progeria,” says Collins, who was not involved in the new study. “It was encouraging to see those results translated into a clinical trial.”

Gordon is medical director of the Progeria Research Foundation, which funded the research. She and her husband, Scott Berns, MD, MPH, clinical professor of pediatrics, cofounded the nonprofit organization in 1999 after the couple’s son, Sam, was diagnosed. Sam died in 2014 at age 17.

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