For Kids with Progeria, New Therapies May Offer Revolutionary Hope for a Longer Life

Sammy Basso is one of around 400 young people in the world with progeria.

Courtesy of Basso

Sammy Basso has some profound ideas about fate. As long as he has been alive, he has known he has minimal control over his own. His parents, however, had to transition from a world of unlimited possibility to one in which their son might not live to his 20s.

"I remember very clearly that day because Sammy was three years old," his mother says of the day a genetic counselor diagnosed Sammy with progeria. "It was a devastating day for me."

But to Sammy, he has always been himself: a smart kid, interested in science, a little smaller than his classmates, with one notable kink in his DNA. In one copy of the gene that codes for the protein Lamin A, Sammy has a T where there should be a C. The incorrect code creates a toxic protein called progerin, which destabilizes Sammy's cells and makes him age much faster than a person who doesn't have the mutation. The older he gets, the more he is in danger of strokes, heart failure, or a heart attack. "I am okay with my situation," he says from his home in Tezze sul Brenta, Italy. "But I think, yes, fate has a great role in my life."

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Jacqueline Detwiler-George
Jacqueline Detwiler is the former articles editor at Popular Mechanics and former host of The Most Useful Podcast Ever. She writes about science, adventure, travel, and technology. For stories, she has embedded with high school students in Indianapolis, jumped out of a plane with a member of the Red Bull Air Force, and travelled the country searching for the cure for cancer. Most recently, she trailed the Baltimore Police Department's Crime Scene Investigation team for a book for Simon & Schuster's Masters at Work series. It will be published in April, 2021.
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The Cocoanut Grove fire in Boston in 1942 tragically claimed 490 lives, but was the catalyst for several important medical advances.

Boston Public Library

On the evening of November 28, 1942, more than 1,000 revelers from the Boston College-Holy Cross football game jammed into the Cocoanut Grove, Boston's oldest nightclub. When a spark from faulty wiring accidently ignited an artificial palm tree, the packed nightspot, which was only designed to accommodate about 500 people, was quickly engulfed in flames. In the ensuing panic, hundreds of people were trapped inside, with most exit doors locked. Bodies piled up by the only open entrance, jamming the exits, and 490 people ultimately died in the worst fire in the country in forty years.

"People couldn't get out," says Dr. Kenneth Marshall, a retired plastic surgeon in Boston and president of the Cocoanut Grove Memorial Committee. "It was a tragedy of mammoth proportions."

Within a half an hour of the start of the blaze, the Red Cross mobilized more than five hundred volunteers in what one newspaper called a "Rehearsal for Possible Blitz." The mayor of Boston imposed martial law. More than 300 victims—many of whom subsequently died--were taken to Boston City Hospital in one hour, averaging one victim every eleven seconds, while Massachusetts General Hospital admitted 114 victims in two hours. In the hospitals, 220 victims clung precariously to life, in agonizing pain from massive burns, their bodies ravaged by infection.

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Linda Marsa
Linda Marsa is a contributing editor at Discover, a former Los Angeles Times reporter and author of Fevered: Why a Hotter Planet Will Harm Our Health and How We Can Save Ourselves (Rodale, 2013), which the New York Times called “gripping to read.” Her work has been anthologized in The Best American Science Writing, and she has written for numerous publications, including Newsweek, U.S. News & World Report, Nautilus, Men’s Journal, Playboy, Pacific Standard and Aeon.

Conner Curran, now 10 years old, can walk more than two miles after gene therapy treatment for his Duchenne's muscular dystrophy.

Courtesy of the Curran family

Conner Curran was diagnosed with Duchenne's muscular dystrophy in 2015 when he was four years old. It's the most severe form of the genetic disease, with a nearly inevitable progression toward total paralysis. Many Duchenne's patients die in their teens; the average lifespan is 26.

But Conner, who is now 10, has experienced some astonishing improvements in recent years. He can now walk for more than two miles at a time – an impossible journey when he was younger.

In 2018, Conner became the very first patient to receive gene therapy specific to treating Duchenne's. In the initial clinical trial of nine children, nearly 80 percent reacted positively to the treatment). A larger-scale stage 3 clinical trial is currently underway, with initial results expected next year.

Gene therapy involves altering the genes in an individual's cells to stop or treat a disease. Such a procedure may be performed by adding new gene material to existing cells, or editing the defective genes to improve their functionality.

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Ron Shinkman
Ron Shinkman is a veteran journalist whose work has appeared in the New England Journal of Medicine publication Catalyst, California Health Report, Fierce Healthcare, and many other publications. He has been a finalist for the prestigious NIHCM Foundation print journalism award twice in the past five years. Shinkman also served as Los Angeles Bureau Chief for Modern Healthcare and as a staff reporter for the Los Angeles Business Journal. He has an M.A. in English from California State University and a B.A. in English from UCLA.