She suffered from an ailment that was guaranteed to take her life. Her condition had been declining for years. And yet, when Dana Jesse Marella died in July, eleven days shy of age twenty, it came as a shock to her parents and many others whose lives she had touched. “Even though for twelve years we knew that Dana had a fatal disease, we had really come to the expectation that we were going to be able to get her what she needed to always be with us,” says her father, Phil Marella. “We thought that progress would come quickly enough to save her, even if it was in a more limited life.”
Before Niemann-Pick Type C, a neurodegenerative condition brought on by an inability to metabolize cholesterol, whittled away her physical and mental faculties, Dana was the happiest of children, with a contagious laugh and a disarming smile. “One of her friends used to say that everybody loves Dana because she never has a mean word to say about anyone, that she’s always so nice,” remembers her mother, Andrea. “I guess that was a unique quality, but she was always that way.”
For all the memories her passing evoked in family and friends, Dana’s greatest legacy may be in having served as the namesake and inspirational force behind the Greenwich-based charitable foundation, Dana’s Angels Research Trust (DART), which her parents launched to fight Niemann-Pick Type C. She would become known far and wide as her parents set off on a determined quest to raise money and help find a cure for the disease. Hundreds of mourners filed into Leo P. Gallagher and Son Funeral Home last summer to pay their respects at her wake and filled the St. Catherine of Siena Church in Riverside for her funeral. “Phil and I stood for five straight hours at the wake,” says Andrea. “It went an hour overtime and they finally had to shut the doors. That’s how many people came and waited in line.”
And it wasn’t just in Greenwich that people mourned. Her father, who has come to know researchers and others involved in the fight against Niemann-Pick from around the United States and abroad, says he was overwhelmed by the response when after the funeral he returned to attending conferences and other meetings related to the disease.
“To have people from all over the world come up to me with tears in their eyes, and hug me and say that they just can’t believe Dana’s gone was so moving,” says Phil, an attorney in the television industry. “She wasn’t the only kid in the world with Niemann-Pick, but most everybody knew who Dana was.”
A Family Battle
Back in 2002, when Phil and Andrea learned that their daughter had Niemann-Pick Type C, it was thought that Dana would live no longer than her early teens. She was eight at the time. “I cried every night,” remembers her mother.
Three years later, they received another blow: son Andrew, now fourteen and the youngest of their children, was diagnosed with the condition as well. Both Dana and Andrew would eventually be treated with experimental drugs. And though the medication came too late for Dana, Andrew has largely staved off progression of the illness and its more debilitating symptoms. The Marellas’ other children—Julia, who is twenty-two, and Philip, seventeen—apparently have been bypassed by the disease.
The years have brought their share of emotional challenges for the Marellas. Indeed, having two children with such a malady is among the heaviest of burdens to carry. Dana’s decline demanded constant care and hospitalizations. When her condition was at its worst, Andrea would describe their home as a “virtual hospital,” complete with a special bed in their daughter’s room, cabinets filled with medications, oxygen tanks and medical equipment of all sorts. “We have our own kind of normal,” Phil liked to say.
Rather than surrender to the disease, the couple chose to battle back. Just months after Dana was diagnosed, they founded DART, which has since raised more than $3 million to help scientists find a cure.
The Marellas have never taken salaries. Donations to the charity are directed toward scientific research. In the process, Phil and Andrea have become respected voices in the drive to spur scientific investigation and clinical trials for drugs that might extend and even save the lives of the afflicted. Phil has gone before the U.S. Food and Drug Administration to help in the efforts—unsuccessful so far—to win approval for one of the drugs, Zavesca, to become an official treatment for Niemann-Pick Type C. Dana’s Angels has even helped sponsor post-doctoral fellowships and medical internships to encourage greater involvement by the scientific community in taking on the disease. With only about 200 known cases of Niemann-Pick Type C around the country, it’s not a condition that researchers, or financial donors, typically hear much about.
As Dana’s passing would show, the Marellas’ work is truly a matter of life and death. “They’d do anything in the world for their children” says Kathie Lee Gifford, the television personality and Greenwich resident who with her husband, Frank, has hosted the group’s fundraising event for seven years. “And they don’t take no for an answer.”
Many a marriage has collapsed under the weight of a child’s devastating illness. Much of the time and energy of one or both parents suddenly becomes channeled toward that child, whether it’s for hands-on care or trips to doctors or extended hospital stays. Priorities change. Stresses mount. Sometimes one parent will be in denial. Another may be unable to cope with the new demands, or his or her grief.
“It can go one of two ways,” says Andrea, who met her husband when they both were working in corporate planning at ABC, Inc., the parent company of the television network, during the eighties. “The divorce rate is 50 percent as it is. Then you bring in critically ill children and it could be that much worse. Or it could go the other way and make you even stronger together and bond you. That’s what’s happened in our case. It has truly forged our relationship and made us lean on each other even more.”
Their healthy kids, Julia and Philip, have received less attention from their parents than they would have under normal circumstances. Still, Phil and Andrea have always tried to give each of their children individual time and support. Andrea’s lifelong friend Cindy Schaffer remembers one day last spring when she sat with Dana, who had been hospitalized for a serious infection, so that Andrea could be home to see son Phil off for the SATs. (The elder Phil was out of town.) “She wanted to be there to make him eggs and make sure he had pencils, that kind of thing,” says Cindy.
And last spring, Andrea and the boys managed to attend Julia’s graduation from Loyola Marymount University in Los Angeles. Phil, however, had to remain with Dana, whose sodium levels had suddenly climbed precariously high and required a few days’ hospitalization.
Family vacations have been out of the question. A few years ago the Marellas did manage to arrange for a few days of splashing in the waves at Virginia Beach before heading to Bethesda, Maryland, for a National Institutes of Health (NIH) observational study of Niemann-Pick kids, but even that required the kind of planning that goes into a military operation. A hospital bed, for example, had to be brought to their hotel room for Dana before they arrived. And every morning, Phil and Andrea would administer her treatments and medications while the other kids waited. When they finally got around to hitting the beach, Phil and Andrea would take turns accompanying the boys down to the water —Julia was away at college—and wheeling Dana along the boardwalk.
After Dana died last summer, the family planned to take a much-deserved respite in Bermuda. The night before they were to depart, Andrew suffered a series of seizures, which he had never experienced before, and the trip was called off. Whether the attacks were related to Niemann-Pick or other causes, such as anxiety, remains unclear. What was obvious, however, was that the disease remains a tangible threat.
The stresses have been considerable. “Basically, we are Christians,” says Andrea. “We truly believe that you pray and God gives you the strength that you need. Sometimes I say, ‘That’s enough, God,’ but truly that’s what’s gotten us through all this.”
Rather than dwell on their lot, the Marellas are more likely to be found consoling others. Andrea’s friend Maria Pope Kessel, a former Greenwich resident who now lives in Bedford, remembers being at an event-planning meeting at the couple’s house with some other mothers one day last winter when word came down that Greenwich High School was being locked down because a student was (mistakenly) said to be wielding a gun. Several of the moms, including Andrea, had kids in the school, and they rushed over, as close to the scene as the police would allow.
Coming on the heels of the killings in Newtown, everyone was on edge. “This is what strikes me about Andrea,” says Maria. “Her son Philip was a junior at the high school. She was nervous and upset like everyone else, but she was the one comforting everybody, hugging everybody and saying, ‘No matter what, we’ll get through this.’”
The Search for Answers
Dana’s diagnosis was probably the greatest shock the couple experienced. Phil and Andrea had known something was wrong with their daughter for several years. She was showing problems with balance, which they initially wrote off to clumsiness. But then she began having learning difficulties as well. She was also slightly slurring her words. And one of her feet was turned inward.
So began three years of looking for answers. One doctor after another told them not to worry, that they couldn’t find anything wrong, that Dana would outgrow this or that problem. Someone thought Lyme disease might be the culprit. And though Dana was indeed found to have Lyme, that wasn’t the answer. Another doctor misdiagnosed her as having a form of mental retardation.
Finally, someone referred them to Dr. Marc Patterson, then director of pediatric neurology at New York- Presbyterian Hospital and now with the Mayo Clinic in Minnesota. When they brought Dana to see him toward the end of 2001, Dr. Patterson required but a few minutes to come up with a preliminary diagnosis of Niemann-Pick Type C. The key indicator, and one that most doctors wouldn’t know to connect with this rare disease, was her inability to rapidly move her eyes up and down, or what is known as vertical supranuclear gaze palsy. A subsequent skin-cell test confirmed the bad news a few months later.
Niemann-Pick Type C is a recessive genetic disease. (Although it was once thought to be a variation of Niemann-Pick Type A and B, those are now known to be unrelated illnesses.) For a child to be affected, both parents must carry the mutation. For each child such a couple might have, the odds are one in four that their offspring will have the disease.
“We didn’t ask Dr. Patterson initially about the details,” remembers Andrea. “But my husband found information about it online, which probably was the worst thing to do, because I remember it was around Christmastime, and it was the worst Christmas, to read those words—‘Children rarely live past their early teens.’ Because then we knew what was in our future.
“Dana was seemingly healthy other than being clumsy and falling a lot. She was a chatty little girl, beautiful, went on play dates, had tons of friends, did karate, ballet. I would look at her and it was hard to imagine that was all going to be taken away from her. I remember putting her to bed at night and just staring at her while she was sleeping, thinking how could I ever imagine that this was all going to go away. And little by little it does.”
Andrea recalls sobbing for nights on end after the initial diagnosis. It was during this time that she and Dana traveled to Lourdes, France, as part of an annual pilgrimage with the Order of Malta, a lay Catholic group, to give the legendary curative waters a try. (Five years later, Phil and Andrew took the same journey.)
Not long after Andrea and Dana returned, the couple decided that they wanted to do more than simply hurt. “Finally we had to take control and just do something,” Andrea says.
That spring the kids at Parkway School, where Dana was a student, donated money for research to the Marellas, which led to the creation of the charity. In looking for how best to contribute to the fight, Phil and Andrea contacted the Ara Parseghian Medical Research Foundation and the National Niemann-Pick Disease Foundation, the biggest and best-established nonprofits devoted to finding a cure. (The former’s namesake is the onetime Notre Dame football coach who lost three grandchildren to the disease.)
Living in Greenwich, the couple thought fundraising seemed an obvious way that they could help. They also had skills that they felt could smooth the way in dealing with the powers-that-be to move research and other processes along. Naming the group Dana’s Angels, meanwhile, helped to personalize, and localize, their efforts. After all, people around town knew the Marellas and their kids. This was no distant and faceless disease. It was here, affecting a real child, right now.
The couple started small, holding a gathering at a private home to introduce themselves and their cause, then putting on some fundraising runs and a show and raffle at the school. Their first big benefit, in 2005, was a one-man show by Richard Dreyfuss, which was followed over the years with concerts by Rob Mathes, Smokey Robinson, Frankie Valli, Natalie Cole and last spring, the Beach Boys.
Pushing the Science Forward
Phil and Andrea divide the labor of running their charity, with each of them taking on the role that plays to their strengths. “Phil is the medical expert,” says Andrea. “He missed his calling. He should have been a doctor. And he puts it in words that everybody can understand. As the mom, I deal with people and try to encourage support and share our story. I’m more on the emotional side of what this disease does, and he’s more focused on the science.”
Which gives them both plenty to do in pressing their fight against Niemann-Pick Type C, whether it’s deploying volunteers to put on their fundraiser each year—this year’s Beach Boys concert raised more than $365,000—or persuading researchers and scientific institutions that are used to proceeding at their own measured pace into streamlining and stepping up their work.
“I see my role as constantly prodding the scientists to move from the lab to the bedside,” says Phil. “Scientists are incredibly detail-oriented. They discover something and they want to know why it works, to the tiniest detail. We don’t necessarily need that. There are a number of drugs out there in the world today that we believe are safe and we believe they are effective and don’t have a clue why they work….
“It’d be nice to really drill down and try to figure out why exactly a drug is working. But we can figure that out later, and sometimes you can’t. But right now if we really do think it’s safe and we do really think it’s effective, we need to get a trial designed and in front of the FDA and approved and going.”
Among other accomplishments, DART helped get clinical trials for Zavesca, the first drug to effectively delay symptoms of the disease, to move forward eight years ago when the studies became stalled by changes in the drugmaker’s ownership. Growing increasingly frustrated, Phil finally reached out to some members of the state’s congressional delegation, who in turn made inquiries with the company. “We didn’t want to irritate anybody,” Phil says. “But we knew that if the right people expressed some interest, that they were looking out for their constituents, that people would take notice.”
Dana’s Angels works with other nonprofits and family foundations involved in the fight against Niemann-Pick Type C in a group called Support of Accelerated Research (SOAR). Its purpose is to collaborate with researchers to speed the process of finding a cure, encouraging openness and cooperation between scientific camps and serving as a resource. Among other endeavors, SOAR is funding the efforts of four laboratories—Albert Einstein College of Medicine, Icahn School of Medicine at Mt. Sinai School, Washington University School of Medicine and Oxford University—to develop and proceed with a “cocktail” treatment of several drugs to combat Niemann-Pick Type C. In turn, those institutions have agreed to work closely together, sharing information and brainpower.
After some delays, the NIH has been slated to commence human trials of the drug cyclodextrin this fall, and the Marellas’ son Andrew is enrolled in the study. Cyclodextrin is not expected to be a cure per se, but holds the potential as the best therapy yet to halt Niemann-Pick Type C in its tracks, to help people manage the disease. The drug, which was to be administered through a spinal tap, has proven successful in animal studies at reducing the debilitating cholesterol and lipids that have built up in brain cells. Whether it will show beneficial results in humans is an open question.
In studying genetics at school, Andrew surprised his parents by writing a paper about the upcoming trials and his faith that the drug would keep him healthy. “This one actually has the potential of stopping the progression of the disease,” says his mother. “So there’s a lot of hope for his future.”
Hope. That’s the key word behind the Marellas’ work. Even with the loss of their daughter, Phil and Andrea are unbowed in their determination to fight on. “Dana’s death has just made us realize that our mission has to be even stronger,” says Andrea. “Now more than ever we have to make sure no more children die from this disease. Dana should be the last.”