Chelsea and Cameron Pratt lost a statistical lottery by having two children with cystic fibrosis.
But they consider themselves blessed and the children — Ben, 4, and Lauren, 2 — consider themselves healthy, playful and a bit mischievous.
Outside the Potomac house where Cameron Pratt and his seven siblings grew up, Ben and Lauren rolled in the grass and bounced on trampolines last week while their parents talked about therapies and cures for the genetic disease, which causes a progressive decline in respiratory and digestive functions.
The Pratts are the lead organizers the Potomac Great Strides Walk for the Cystic Fibrosis Foundation, a fund-raising event at the Bullis School May 21.
“Our children are very healthy but some kids are born very sick and they’re sick from day one,” Chelsea Pratt said. “[Ben] knows he has cystic fibrosis but he doesn’t think he has any limitations because of it.”
“We want our kids to have a fairly normal lifestyle,” Cameron Pratt said.
Cystic fibrosis is the result of a genetic mutation that prevents the body from producing a cellular protein. The result is that patients accumulate a thick, sticky mucus in their lungs that impairs their ability to breath and provides a breeding ground for infections.
One in 24 white Americans carries the genetic mutation but does not have any symptoms of the disease. Only when both parents are carriers can a child be born with cystic fibrosis, and even then the chance is only one in four.
Those circumstances aligned when Ben was born with the disease. In having Lauren, the Pratts used an in-utero fertilization technique that includes screening to prevent having a symptom-bearing child. The technique is believed to be 99 percent effective but the Pratts attribute its failure to human error.
But cystic fibrosis is treatable.
“We are very, very optimistic,” Cameron Pratt said. “The trajectory of development of therapies is so steep, we kind of believe it’s always going to stay ahead of our kids. We don’t really worry about our kids dying.”
That would not have been the case 15 years ago. The median survival age for people with cystic fibrosis was 14 in 1969. Five years ago, newborns with cystic fibrosis were predicted to live to age 32. Last week researchers announced that predicted survival is now close to 40,
Chelsea Pratt’s younger sister died from cystic fibrosis 10 years ago at age 15.
“Not that long ago people didn’t live past their teenage years and not long before that they didn’t live beyond childhood,” said Brent Pratt, Cameron Pratt’s father and a Potomac resident of more than 20 years. “The life expectancy has moved up dramatically because of the Cystic Fibrosis Foundation. … This is like polio. This is solvable and we’re really on the brink of it.”
The dramatic improvements in cystic fibrosis care are largely the work of the foundation, which directs 91 cents of every dollar to research, making it one of the most efficient medical foundations anywhere.
The foundation maintains its own staff of doctors and scientists and also makes grants to large pharmaceutical companies for the development of drugs that treat the disease.
The pharmaceutical companies have little incentive to invest in cystic fibrosis therapies without the grants because the disease affects only about 30,000 Americans, a small number compared to other ailments.
“Years ago they were able to raise money but they didn’t know what to do with it — they didn’t have the science to use it. And now they’ve got so much science and there are so many different drugs that they’re working on,” said Meg Pratt, Cameron Pratt’s mother. “But it takes millions and millions of dollars to develop the drugs.”
And while drugs — antibiotic inhalers, and mucus-thinning therapies, for example — have contributed to increases in life expectancy and quality of life, even greater hope lies in genetic therapy, which could someday solve the root cause of cystic fibrosis rather than just the symptoms.
“We really feel like we are in a better place than we’ve ever been,” said Julie Puzzo, executive director of the Washington Metropolitan chapter of the Cystic Fibrosis Foundation and a Bethesda resident. “We really do believe that we will see a cure in our lifetime.”
Cameron Pratt , 32, grew up in Potomac, the oldest of eight siblings who all attended Bullis. The youngest, 18, will graduate this year.
Cameron Pratt now works for Foulger-Pratt, Brent Pratt’s real estate development company, and lives in the Lakelands community of Gaithersburg.
Last year he organized a Rockville-based Great Strides Walk that was held at Cabin John Regional Park.
That walk was successful, but he said that it lacked the level of community involvement that he had hoped for. This year, he broke the event into two separate walks — the Potomac walk at Bullis and another one in the Kentlands on the same day.
The May 21 event will feature food, music, and information about cystic fibrosis. Walkers can register in advance on the Cystic Fibrosis Foundation Web site but no advance registration, pledge-gathering, or participation fee is required.
“You don’t have to necessarily be involved to come,” Puzzo said. “It’s really just a fun day to come out with your family.”
There will be 10 walks in the Washington area among more than 1,000 nationwide that are expected to collectively raise some $35 million.
“The goal is to make cystic fibrosis a nuisance disease,” Puzzo said, “Something that you die with not from.”