Danson, Steenburgen join effort to raise awareness about progeria

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Ted Danson and Mary Steenburgen with Sam, age 9. Sam has progeria, a genetic condition that causes rapid aging. The actors have lent their voices to public service announcements to promote public awareness of the disease and the research efforts of The Progeria Research Foundation. Courtesy of The Progeria Research Foundation

Progeria is a genetic condition that causes rapid premature aging in children. Ted Danson and Mary Steenburgen have lent their voices to public service announcements to promote awareness and research into the disease.

By Melonie Magruder / Special to the Malibu Times

Malibu residents Ted Danson and Mary Steenburgen have joined the fight against a disease that, in every known incidence, has killed every one of its victims. Hutchinson-Gilford Syndrome, also known as progeria, is an extremely rare, and always fatal, genetic condition characterized by accelerated aging in children. They literally die of old age by their mid-teens. First diagnosed in 1886 by Dr. Jonathan Hutchinson and Dr. Hastings Gilford in England, the illness develops rapidly, usually showing up in babies before their second year. Children suffering from this disease simply fail to grow. They lose body fat and hair; their skin wrinkles and ages; they develop arthritis, hip dislocation, generalized cardiovascular disease and usually die of a heart attack or a stroke before they reach the age of 13-normally the onset of puberty in most children.

Because of the extreme rarity of progeria (with an incidence of approximately one in four to eight million births), the disease has not received much attention and even less federal funding for research. That changed in 1998, when a Massachusetts couple, Dr. Leslie Gordon and Dr. Scott Berns, learned that their son, Sam, was diagnosed. When they began to research origins and treatments for the disease, they found there was practically no medical lexicon. There was no way to test for progeria, no research efforts or funding, either privately or publicly, and no one advocating for children suffering from this living death sentence. In 1999, they established The Progeria Research Foundation, with Gordon acting as PRF’s medical director for both medical and research efforts for children with progeria.

Berns’ and Gordon’s son, Sam, is one of only 14 children in America living with this disease. When Berns was working on a White House fellowship in 2003, he was introduced to Danson and Steenburgen at a presidential black-tie event. A friendship quickly developed and, when the star couple met Sam, they fell in love with the boy. After learning of the critical status of funding for research into the disease, Danson and Steenbergen decided to lend their names and efforts to PRF.

“We are honored to be a part of this tremendous effort to raise awareness about progeria,” said the couple in a press release announcing their participation in public service announcements to promote public awareness of the disease and the research efforts of the foundation.

The ads feature voice-over announcements and images of Danson and Steenburgen with children suffering from the disease, and started to run in California markets in the late summer.

Attorney Audrey Gordon, sister of Gordon and aunt to Sam, is president and executive director for the Progeria Research Foundation. Despite a vested interest in finding immediate treatment to slow or halt the effects of the disease, she is optimistic about the foundation’s progress.

“We launched this research only seven years ago, working mostly with grassroots fundraising efforts, and we managed to isolate the gene that causes progeria last year,” she said. “That progress is unheard of in medical research!”

Although the disease is caused by genetic defect, it is not passed from parent to child. The genetic mutation occurs only within rare individuals and, now that the culpable gene in which the mutation occurs has been identified, therapies can be developed to separate the defective protein that causes the cells to develop abnormally.

Now that the mutation has been identified, PRF has created a diagnostics testing program that can look for the specific genetic change in the progeria gene that leads to the disease. Gordon stressed the importance of early testing on children who do not seem to be developing normally.

“Although there is no cure yet, there is certain treatment we can recommend to parents that will positively affect the quality of life of their children,” she said. “Many of these therapies are what we would recommend for normal complaints by elderly people, such as aspirin to reduce arthritic inflammation.”

The foundation’s research focus is on developing farnesyltransferase inhibitors, or FTI, originally designed to treat cancer, and which are capable of reversing the dramatic nuclear structure abnormalities that are the hallmark of cells from children with progeria.

Gordon said that children suffering from progeria have a remarkably similar appearance, characterized by overly large round, bald heads and tiny, fragile bodies, even when coming from differing ethnic backgrounds.

“And just because they have this disease that gives them such a dramatic physical appearance, most of the kids are sharp as tacks and totally normal,” she said. “My nephew, Sam, goes to public school, plays the drums and baseball and does all the things normal kids do.”

She said there are 42 children around the world known to suffer from progeria, and the progress of their research has prepared them to start clinical trials for FTI treatment as early as next year.

“And the cost of the clinical trials is only going to be about $2 million,” she said. “It is just totally doable, with the help of friends like Ted Danson and Mary Steenburgen spreading the word about the immediate need for funding. We have very high hopes.”

Gordon noted the amazing philosophical acceptance of these children to their disease. John Tacket, who, at age 15, was the oldest child living with progeria in the U.S., summed it up: “Well, no one really knows how long they are going to live. So I don’t worry about it.”

John Tacket died of complications of progeria shortly thereafter, in 2003.

More information on progeria can be obtained at the Web site, www.progeriaresearchfoundation.org