Helping families thrive through genetics

Kensington father of 12 works to find cure for muscular dystrophy

Wednesday, June 21, 2006

Click here to enlarge this photo Naomi Brookner⁄The Gazette Eric Hoffman, head of the Center for Genetic Medicine at Children’s National Hospital Center, enjoys some rare downtime with his kids, Alanna, 4, Ryan, 3, and Caleb, 4.

On a recent afternoon, the director of Children’s National Medical Center’s pediatric genetic research lab sat at the dining room table of his Kensington home, waxing intellectual on genetic ethics and his work on muscular dystrophy. As Hoffman spoke, his 3-year-old son Ryan squirmed on his lap and squealed in his ear, his 4-year-old son Caleb sat nearby rolling a truck on the table while Caleb’s twin, Alanna, apparently made something crash in another room.

A second crash was the only thing that made Hoffman miss a beat.

‘‘What’s Alanna doing?” he asked Ryan and Caleb, who simply looked at him. ‘‘Can you go check on what Alanna’s doing for me?”

The boys bolted into the other room, their small feet pounding on the hardwood floors.

‘‘That worked,” he said laughing.

After another crash only seconds later, Hoffman decided to go in for a look. He returned after a minute or so with Ryan following close behind on a tricycle.

It’s a happily chaotic scene, one in which the 47-year-old scientist seems perfectly at ease, which is easy to understand considering he and his wife, Ruth Hoffman, have 12 children between them in their blended family. Three at a time is no sweat.

It’s this quality of life that Hoffman’s work could allow more families to enjoy, as he continues to work toward a cure for muscular dystrophy.

Hoffman and his wife moved to Kensington in 1999 when he was hired to start the research center for genetic medicine at Children’s National Medical Center in Washington, D.C. Living here has been conducive to his family’s lifestyle, he said, allowing him to bicycle commute to work and share the childcare duties with his wife, who runs a local chapter of Candlelighters, a nonprofit organization providing advocacy for children and adolescents with cancer.

To reach the door of the Hoffman residence, visitors must navigate two tricycles, a small stroller carrying a Cabbage Patch Kid and dodge the hose from Alanna, who is watering the lawn. The tricycles and stroller sat parked after a sort of working play date Hoffman spent with the kids. He walked to the C & O Canal with Ryan, Caleb and Alanna, who rode the trail while he read through a stack of research grants.

This is what amounts to a day off for Hoffman, who had just spent the last few days traveling around the country for various speaking engagements before muscular dystrophy researchers, muscle biologists and the American College of Sports Medicine.

‘‘It’s great to be able to go to the C&O Canal and review grants with the kids,” he said.

One of Hoffman’s primary research focuses is finding a cure for muscular dystrophy. After earning a doctorate in biology and genetics from Johns Hopkins University in 1986, Hoffman went to Harvard Medical School to apply his research in fruit fly molecular genetics to muscular dystrophy in humans.

His team ultimately identified the gene for Duchenne muscular dystrophy in 1987. Hoffman believes the discovery will eventually lead to a cure for the disease, but there is still a way to go, he said.

‘‘The funky thing about genetics is once you have the gene in hand....people just thought that gene therapy was going to be a magic bullet,” he said. ‘‘People said, ‘You cloned the gene, so it’s going to be cured overnight, right?’”

His research center at Children’s has teamed up with other agencies in the region, including the National Institutes of Health, to set up a 23-site clinical trial network in 11 countries. Hoffman estimates he and his colleagues have managed to leverage between $40 million and $50 million for muscular dystrophy research.

‘‘Even though it’s the most common genetic disorder among children, it gets one-tenth of the funding,” he said.

Since starting the Center for Genetic Medicine at Children’s, Hoffman has grown the program and now oversees about 90 scientists collaborating on a variety of other conditions as well.

‘‘Working with him, he’s like this genius and it’s hard to keep up,” said Marie Pichaske, Hoffman’s administrative coordinator, who is responsible for his insane schedule.

Pichaske first came to work for Hoffman when he opened the research center at Children’s, but she had heard of him years ago when he discovered the muscular dystrophy gene. Her son, Peter Renzi, now 23, was born with the disease.

‘‘Back when my son was diagnosed, it was, your son has muscular dystrophy and you take him home and he’s going to die,” Pichaske said. ‘‘That has been completely turned around to, ‘He has muscular dystrophy, now let’s see what can we do about it.’”

For his next trick, Hoffman is working with scientists from Japan to attempt for the first time to cure a dog with Duchenne muscular dystrophy.

‘‘I think we’ll do it,” Hoffman said as Ryan continued to squirm in his lap, cracking himself up with the sound of his own high-pitched voice.

But today is his day off, which means instead of doing research, he’s off to a dress rehearsal with the Master Chorale of Washington for an upcoming performance at the Kennedy Center with the National Symphony Orchestra.

‘‘I was a music major,” he said. ‘‘I just sort of picked up biology by reading newspapers.”