New gene therapy being tested

Amish and Mennonite communities

The Associated Press

STRASBURG, Pa. -- Lancaster County's Amish and Mennonite people, the plain folk who mostly live, work and dress the way they did during the 19th century, are nevertheless serving as test subjects in one of the most advanced areas of medicine -- gene therapy.

Scientists hope to learn more about genetic diseases by studying members of the insular community, people who often ride in horse- drawn buggies and don't have electricity but are willing to climb into an MRI machine.

"I can see how it could be confusing if you don't understand our beliefs," said Leon Newswanger, a Mennonite whose son will be one of the first participants in human trials of a new gene therapy. "But if we don't do whatever it takes, if we know what could help -- then that is worse."

The Amish and Mennonites, known as the Plain community for their simple, dark clothes and rural way of life, have a higher rate of certain inherited diseases than other people.

They have become instrumental in genetic research because the estimated 75,000 sect members are descended from just 47 families. The sects have been providing blood samples and family histories to geneticists for years.

Similarly, scientists have studied Ashkenazi Jews, Cajuns in Louisiana and other groups with a high degree of genetic uniformity.

"Amish people know the reality of genetic diseases and so any advances are welcomed," said Dr. Holmes Morton, who opened the Clinic for Special Children in the heart of Pennsylvania's Amish country in 1990.

It is one of the most advanced gene research clinics in the country, situated in a timber-frame house surrounded by fields plowed by mules.

Newswanger's son, Marlin, and two other children from Morton's clinic will be the first subjects in a test of a process called chimeraplasty, which involves tricking the body into correcting a gene mutation. If successful, the technique could be used to treat up to 80 percent of all genetic diseases.

All three children are stricken with Crigler-Najjar, a rare liver disease that is more common among Amish and Mennonites. About 300 people worldwide have Crigler-Najjar; 16 trace their roots to Lancaster's Plain sects and are being treated by Morton.

Crigler children suffer severe jaundice and must sleep under special rehabilitating lights for up to 16 hours a day to prevent deadly brain damage.

Altogether, Morton treats hundreds of children with more than 70 rare genetic diseases, and many regularly undergo MRIs and complicated blood tests and may be hospitalized for months at a time. Some children must be fed special formulas and vitamins -- another contrast in a community where nearly all the food is homegrown.

"This is what it takes to keep him alive," said Glenda Groff, a Mennonite whose 8-year-old son has a genetic disorder called maple syrup urine disease, which is fatal without a proper diet.

The disease -- extremely rare in the general population but more common among Mennonites -- prevents the body from processing certain amino acids, which then become toxic. One of the symptoms is urine that smells like maple sugar.

With the opening of Morton's clinic, many Amish and Mennonite have shed their traditional fatalism about disease.

"There is a perception in the Plain community that some things are a fact of life," said Rebecca Smoker, an Amish worker at Morton's clinic. "For years, these children lived their short little lives and died without any medical person seeing them because everybody knew they were going to die."

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