Children’s Health—Care | Minnesota Medical Foundation

Never accepting defeat

2011-12-05T17:59:14Z

Florida State coach and family raise awareness and funds for Fanconi anemia research at the U

Jimbo Fisher has a microphone and he’s not afraid to use it.

When the Florida State University football coach and his wife, Candi, learned earlier this year that their youngest son, 6-year-old Ethan, has a rare blood disease called Fanconi anemia, they dealt with the devastating news in private.

Then they decided to use their visibility in the media to raise awareness of the disease, an inherited blood disorder that can affect all systems in the body and leads to bone marrow failure.

The Fishers also established the Kidz1stFund™ to raise money through online donations and T-shirt and hat sales for Fanconi anemia research at the University of Minnesota, a national leader in blood and marrow transplantation, and they are promoting the fund through a campaign called OnaKwest for a Cure. Jimbo Fisher is donating all fees from his public speaking engagements to the Kidz1stFund™ as well.

They’ve already raised more than $400,000 for this work.

“One thing I’ve learned as a football coach is to never accept defeat,” Jimbo Fisher says. “We are in this to win the fight against Fanconi anemia on behalf of all the children who share this struggle with Ethan.”

For decades, Fanconi anemia was thought to be untreatable, but promising advances in medical research have improved the prognosis.

Many of those advances have happened at the University of Minnesota. That’s why the Fisher family’s search for the country’s foremost Fanconi anemia team led them to University of Minnesota Amplatz Children’s Hospital.

Today most children who have Fanconi anemia will need a transplant of stem cells, derived either from bone marrow or umbilical cord blood, to extend their lives. University of Minnesota physician-scientists performed the world’s first successful bone marrow transplant in 1968 and have been blazing new trails in the field since.

“Through research, improvements are made each year in treating patients with Fanconi anemia — improvements that have changed the survival rate after unrelated donor bone marrow transplant for this disease from less than 30 percent to greater than 80 percent in the last 15 years,” says Margaret MacMillan, M.D., Ethan’s doctor and a leading Fanconi anemia researcher. “But there is much more to do. We will not stop until we have 100 percent survival.”

Determination is just one of the qualities the Fishers liked about MacMillan when they met her. Candi Fisher says MacMillan spent time generously with the family to help them understand what they were facing and what treatment options they had for Ethan.

“One thing that she gave us was hope, and that was something at that moment that we were desperately striving for,” adds Candi Fisher. “It made us feel like this was something we could beat, with her help and with the University of Minnesota. … They have invested so much of themselves in this research, and that made us feel like we weren’t alone.”

The Fishers’ goal for the fundraising and awareness campaign is not a dollar figure. It’s a cure, so that Ethan and other kids with Fanconi anemia may lead full lives.

“We’re just hoping that we could make a difference in some way,” says Candi Fisher.

By Nicole Endres

Visit www.kidz1stfund.com to make a gift to this research.

You can make a difference

Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

Because with your support, anything is possible.

Coach and family raise awareness and funds for Fanconi anemia

2011-10-24T16:57:48Z

When Florida State University football coach Jimbo Fisher and his wife, Candi, learned earlier this year that their son Ethan has a rare, life-threatening blood disorder called Fanconi anemia, they felt compelled to take action that would help not only Ethan but other children, too.

So they established the Kidz1stFund™ to raise money for Fanconi anemia research at the University of Minnesota. The University is a leader in discovering better ways to treat the disorder and in the pursuit of a cure.

A hereditary disease, Fanconi anemia can affect all systems in the body and leads to bone marrow failure. For decades, it was thought to be untreatable, but advances in medical research have improved the prognosis for people with the condition.

“One thing I’ve learned as a football coach is to never accept defeat,” says Jimbo Fisher. “We are in this to win the fight against Fanconi anemia on behalf of all the children who share this struggle with Ethan.”

The family and their supporters already have raised more than $400,000 for the University’s work. Six-year-old Ethan’s physician is University of Minnesota Amplatz Children’s Hospital blood and marrow transplant physician Margaret MacMillan, M.D.

“Through research, improvements are made each year in treating patients with Fanconi anemia — improvements that have changed the survival rate after unrelated donor bone marrow transplant for this disease from less than 30 percent to greater than 80 percent in the last 15 years,” MacMillan says. “But there is much more to do. We will not stop until we have 100 percent survival.”

Visit www.kidz1stfund.com to make a gift to this research.

You can make a difference

Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

Because with your support, anything is possible.

A place of their own

2011-10-12T20:15:54Z

Amplatz Children’s Hospital’s new children-only emergency department delivers exceptional care with a unique mission

When University of Minnesota Amplatz Children’s Hospital opened the doors of its emergency department for the first time on April 30, the breakthrough pediatric medicine offered there became easier to find.

The brand-new hospital, located on the University’s Riverside campus, now has a welcoming and easy-to-find emergency department dedicated solely to children. Amplatz Children’s Hospital’s former setup as a “hospital within a hospital” on the University’s East Bank campus meant that all patients needing emergent care—whether children or adults—entered through one set of emergency department doors at University of Minnesota Medical Center, Fairview.

The culture of the new emergency department reflects the entire hospital’s attention to family-centered care and to delivering the very best experience for children. “There’s nothing in here that isn’t state-of-theart,” says Anupam Kharbanda, M.D., research director for the Division of Pediatric Emergency Medicine.

Treatment spaces are large enough that parents can stay by their child’s side. Children and adolescents with behavioral or mental health needs have a room specially designed to help them feel safe. And two fully equipped trauma bays allow emergency staff to care for the most severely ill or injured children.

“Being able to influence emergency pediatric care for the better—perhaps around the world—through our work at Amplatz is what it means to be part of the University of Minnesota,” says assistant professor Tom Hellmich, M.D., who joined the Department of Pediatrics’ emergency medicine team after 18 years practicing locally elsewhere. “That opportunity inspires me to be a better physician and educator.”

Setting the standard

Leading-edge emergency facilities demand leading-edge emergency care. The gold standard today is “evidence-driven.” In the emergency department, this means following carefully researched protocols that provide consistent, proven treatments in a fast-paced environment. Many of those protocols are designed at academic hospitals like Amplatz.

“We want to ensure that all children in the ED get the same excellent care for their particular injury or illness,” says Ron Furnival, M.D., codirector of the Division of Pediatric Emergency Medicine. “Evidence-driven care allows us to do that. For example, if a child comes in with diabetic ketoacidosis, we’ve designed a protocol that nurses can get started immediately that is the same for every patient, every time.”

Furnival believes that being part of an academic medical center gives the emergency department a distinct advantage when designing these protocols. Here physicians are responsible for educating residents and medical students, so they must be attuned to the latest research—and may have conducted some of it themselves.

Kharbanda, for instance, explored ways to evaluate children with possible appendicitis without automatically ordering a CT scan. The result of his work is an online evaluation tool for physicians that suggests a course of action for the child. This method can be shared with emergency departments around the country.

“Designing evidence-driven care protocols can be time-consuming,” says Kharbanda. “But the University supports its physicians in this work and holds to the larger vision of improving care for everyone.”

Better care for all children

Combining teaching and research with first-rate innovative care is the essence of academic medicine. Sharing the knowledge gained in these pursuits is its mission—and it sets Amplatz Children’s Hospital’s emergency department apart from the pack.

According to Mark Roback, M.D., who codirects the Division of Pediatric Emergency Medicine with Furnival, 90 percent of children in the United States are seen in emergency departments not connected to a children’s hospital.

“It’s important that we take care of the patients we have at Amplatz, but it’s just as important for us to be involved in making sure that children, regardless of where they are seen, receive a similar level of care,” he says. “That’s the responsibility of academic medicine.”

Access to excellence

Since Amplatz Children’s Hospital’s new emergency department opened its doors, it has seen a 46 percent increase in patients over the combined pediatric visits to the two University of Minnesota Medical Center emergency departments on its Riverside and East Bank campuses during the same timeframe last year.

Children arrive at the emergency department with a myriad of emergencies: everything from head injuries to stomach aches to complications from bone marrow transplants. They come from the city and the suburbs, from the Dakotas and from Amplatz’s new Riverside neighborhood, with its Somali, East African, Vietnamese, Central American, and Mexican families.

These children not only have 24-hour access to the expertise of pediatric emergency medicine physicians, but also to those trained in other subspecialties, like pediatric ophthalmology, dentistry, and infectious diseases.

“Our job—and what we love—is to take care of children,” says Roback. “No matter what brings them to us, our emergency department and the entire hospital will serve them with unmatched care.”

You can make a difference

Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

Because with your support, anything is possible.

Center offers a family support system

2011-10-12T20:02:26Z

Rich Kaplan, M.D., M.S.W., was deliberate about the words he chose when he named the University of Minnesota’s Center for Safe and Healthy Children five years ago.

“The goal of the center is to keep children safe and to support families so they can raise healthy kids,” says Kaplan, who also founded the center and is one of only two physicians in Minnesota (and one of fewer than 200 in the United States) board-certified in child abuse pediatrics.

Kaplan works closely with the physicians at University of Minnesota Amplatz Children’s Hospital’s emergency department, a group he calls “the best in the state.” That group, along with Kaplan and pediatric trauma surgeons at the hospital, designed a strict protocol to follow when an injured child comes into the emergency department.

If there is not “a clear, corroborated mechanism of injury,” meaning the cause of the child’s injury is in question, Kaplan says, or if the child has significant injuries and is under two years old, the center receives a call.

But when possible, Kaplan prefers to be proactive and reach out to families before any child suffers maltreatment.

“We really want to help families so they don’t fail, rather than wait until they do fail and then call in child protection,” he says. “That’s the old model.”

The family-centered culture and quiet spaces at Amplatz Children’s Hospital provide a place for Kaplan and members of his staff to meet with families under stress, many of them with critically or chronically ill children, in a nonthreatening way.

“One thing families feel they’re not allowed to do is to say how awful and hard it is to have a sick child,” he says. “We try to give them permission to say that and then find them support.”

To learn how you can support the Center for Safe and Healthy Children, contact Courtney Billing at 612-626-1931 or c.billing@mmf.umn.edu.

You can make a difference

Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

Because with your support, anything is possible.

Winning CF team helps keep Kyle on the ice

2011-10-12T18:35:56Z

Something wasn’t right with Kyle Schwendemann. He came back from an ice fishing trip with pale skin and purple lips, recalls his mother, Melissa. She knew his cystic fibrosis was behind it.

Cystic fibrosis is a hereditary disease that causes thick mucus to accumulate in a person’s lungs and sinuses. The disease also makes it difficult for the body to absorb nutrients from food.

Kyle, who was diagnosed when he was 3 months old, was no stranger to University of Minnesota Amplatz Children’s Hospital. His cystic fibrosis landed him there a couple of times every year, even though he has kept up with his therapies, which include taking a handful of vitamins and enzymes daily and wearing his lung-clearing chest compression vest three or four times a day.

But he was in good hands. Thanks to research conducted at the University over the last 50 years, the life expectancy for patients of the Minnesota Cystic Fibrosis Center is a full decade longer than the national average.

The University is also the birthplace of the widely used chest compression vest, invented and perfected by pioneering physician-scientist Warren Warwick, M.D., who recently retired after five decades as a leader in his field.

So when Kyle checked into the hospital in March 2009 to have a part of his right lung removed—the part where mucus kept getting stuck, causing most of his hospitalizations—his family knew he was in the right place.

Since recovering from that surgery, 14-year-old Kyle is back on skates where he belongs.

“Kyle is a hockey player first, a young man with cystic fibrosis second,” says Terri Laguna, M.D., Kyle’s pulmonologist at Amplatz Children’s Hospital. “Since his surgery, he has spent more time on the ice and less in the hospital.”

You can make a difference

Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

Because with your support, anything is possible.

U.S. News & World Report "Best Hospital"

2011-06-30T19:14:15Z

University of Minnesota Amplatz Children’s Hospital now ranks among the nation’s top 50 children’s hospitals in eight medical specialties, according to U.S. News & World Report—an all-time high for our hospital. This also marks the fourth consecutive year that the hospital’s cancer program and the third consecutive year that its kidney care program have been ranked among the country’s best. Learn more.

You can make a difference

Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

Because with your support, anything is possible.

Supporting body and spirit

2011-06-15T21:00:00Z

Amplatz Children’s Hospital inspires giving from the heart

By Martha Coventry

Of all the things a teenage boy might choose to do with his bar mitzvah money, giving a portion to medical research might seem low on the list. After all, there are Xboxes and iPods and skateboards to buy. But when Matthew, 13, gave his money to a research program led by John Wagner, M.D., at the University of Minnesota, he was sharing a heartfelt thanks.

“His research saved my life, and I wanted to help him save other lives,” Matthew says.

When Matthew was a week shy of his eighth birthday, he was diagnosed with acute myeloid leukemia. As he was undergoing chemotherapy in Miami, where he lives, his parents, Marcy and Harry, were busy researching treatment options in case he relapsed. Matthew had no family or non-related bone marrow match. His best hope, they learned, was a transplant of publicly donated blood from two umbilical cords.

Wagner, director of the University’s Blood and Marrow Transplantation Program and clinical director of the Stem Cell Institute, had pioneered the world’s first umbilical cord blood transplant for leukemia in 1990. Later, he found that, for older children and adults, co-infusing units from two different donors instead of one led to faster recovery and a markedly lower risk of leukemia relapse.

Aware of Wagner’s successes, Matthew’s parents contacted him to learn more about his work. He got back in touch immediately. “The University was the only place willing to offer Matthew a double cord blood transplant if he needed it,” says Marcy.

Within a year, Matthew’s leukemia had indeed relapsed. He came to University of Minnesota Amplatz Children’s Hospital in April 2007 and became the first child in the world to receive a double cord blood transplant specifically to reduce the chance that his leukemia would ever recur again.

Today, Matthew has a clean bill of health, and his parents have made their own financial gifts to the University to support medical care and research. “One of the things that we particularly like about the University is that we know our gifts will go to developing therapies, like Matthew received, that will be brought to the bedside as soon as possible,” says his mom.

A cascade of names

Matthew says he’s excited about seeing his name on the digital donor roster in the lobby the next time he visits University of Minnesota Amplatz Children’s Hospital, which opened a new state-of-the-art facility on April 30. Located on the University’s West Bank, the new hospital stands out because of its inviting, colorful, and “green” design — inside and out — and for its many other special features that accelerate healing and make young patients and their families feel at home.

Cascading down the large digital screen like a waterfall are the names of those who have contributed to the Children’s Health Campaign at the University of Minnesota, which aims to raise $175 million for the hospital building project, as well as pediatric research, education, and care. Thanks to Matthew, his parents, and many others like them, more than half of that amount — $98 million — had been raised by mid-May.

The gift of solace

Ted Thompson, M.D., and his wife, Lynette, have given a lasting — and growing — place of healing and comfort to Amplatz Children’s Hospital.

Dr. Thompson has been in the Department of Pediatrics at the University of Minnesota since 1975. When he comes home after caring for ill or premature newborns, his wife’s garden offers him respite. Lynette, a certified master gardener and former adult intensive care and coronary care unit nurse, knows the consoling power of flowers and plants. The Thompsons wanted to make that solace available to patients and families at Amplatz Children’s Hospital, as well as to the hospital’s physicians, nurses, and other staff.

Near the hospital’s main entrance is the healing garden they have funded and endowed. Paths leading off from the main garden have benches under the trees to create a feeling of peaceful, private space.

“One of the most stressful things in life is to have your child in the hospital,” says Thompson. “We wanted to provide a place where people can go to get away from that stress for a moment, to think and to contemplate.”

Thompson says he is proud to help bring about a dream he and his colleagues have shared for decades: a top-notch facility at the University that gathers the very best of pediatric care, research, and education all under one roof.

To learn about additional recognition opportunities at University of Minnesota Amplatz Children’s Hospital and the many ways to support the Department of Pediatrics, contact the Minnesota Medical Foundation’s Children’s Health Team at 612-626-1931 or childrenshealth@mmf.umn.edu, or visit UofMHope.org.

To become part of our Partners in Care program, contact Jen Foss at 612-626-5276 or j.foss@mmf.umn.edu.

You can make a difference

Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

Because with your support, anything is possible.

A family crusade

2011-06-15T20:00:00Z

An extended family bands together to support the U’s neurofibromatosis research

By Robyn White

Nine-year-old Zachary “Zak” Bartz isn’t your typical second-grader — to many, he’s an inspiration.

Zak has a disorder called neurofibromatosis type 1 (NF1), which has caused tumors to grow in his brain and for which there is no known cure. Zak has endured multiple surgeries, countless rounds of chemotherapy, and 30 radiation treatments — all conducted at clinics associated with the Masonic Cancer Center, University of Minnesota.

Although the treatments are difficult, Zak doesn’t let NF1 get him down, says his grandpa Harvey Bartz. Zak still finds the time and energy to “coach” his 13-year-old brother’s hockey team. “He goes into the locker room … and gives them a pep talk,” says Bartz. “He gets them all psyched up.”

Family support

Seeking a cure and grateful for Zak’s care at the University, Bartz in 2009 contacted the Minnesota Medical Foundation to find out how he could speed up NF1 research. After talking with MMF, he decided to create the Zachary Neurofibromatosis Research Fund at the University.

“What I really wanted was to find someplace where every dollar I raised would go to research,” says Bartz.

Bartz and his wife, Janet, along with Zak’s other grandparents, Dave and Darlene Rudolph, contributed initial gifts to establish the fund. Both sets of grandparents and other family members — Zak’s parents, Carol Ann and Nathan Bartz, Zak’s aunt Sandy Bartz, and many others — have contributed to the fund through various marketing efforts, fundraisers, and direct donations. To date, the fund has raised nearly $82,000.

Advancing NF research

The University is home to a clinic for NF1 patients and to two nationally known experts in NF — pediatric hematologist-oncologist Christopher Moertel, M.D., and cancer geneticist David Largaespada, Ph.D. Together Moertel and Largaespada lead the Minnesota Neurofibromatosis Clinic Without Walls, which provides care to NF patients from infancy through adulthood and maintains strong ties to research in the field.

Currently, they are working on three research projects studying NF1-related cancer. NF1 is one of two types of NF. The disorder is not a type of cancer, but it can cause cancer.

“The patient experience and laboratory experiments merge at the University of Minnesota so that we can learn more about NF1 and how to improve treatment in patients like Zachary,” says Moertel, who is Zak’s doctor.

Largaespada is identifying gene pathways related to NF1 cancers and testing new drug combinations in mouse models of these cancers. He hopes to soon move into a clinical trial with patients, working with Moertel.

Zak and Harvey Bartz recently visited Largaespada’s lab to see how the research was taking shape. “It was very illuminating for us to know that we were making a direct difference,” says Bartz.

Zak’s visit made an impression on the researchers, too, says Largaespada. “It really drove home that what we’re doing could have an impact on people’s lives.”

To support the University’s NF research, contact Kathy Beenen at 612-625-6495 or give to the Zachary Neurofibromatosis Research Fund at www.mmf.umn.edu/giveto/zachary.

To become part of our Partners in Care program, contact Jen Foss at 612-626-5276 or j.foss@mmf.umn.edu.

You can make a difference

Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

Because with your support, anything is possible.

A 'top-shelf' care team

2011-05-14T18:31:12Z

Susan Doherty calls her 13-year-old son’s experience with hepotoblastoma—a rare pediatric liver cancer— a “life-altering experience.”

Eighteen months ago, she brought Elliott to an emergency room for severe abdominal pain, which she thought was appendicitis. Instead, the physician found an 11-centimeter mass on his liver. “As a parent, it was terrifying,” she says. Three days later, Elliott underwent surgery at University of Minnesota Amplatz Children’s Hospital.

Elliott’s care team—Donavon Hess, M.D., Ph.D., M.B.A., Joseph Neglia, M.D., M.P.H., Timothy Pruett, M.D.,William Payne, M.D., and Nissa Erickson, M.D.—are all “top-shelf” people, says Doherty. “We would have come to the University from no matter where we lived.”

Unfortunately, that first surgery was just the beginning. Elliott’s cancer returned to his liver, lighting up several spots on a PET/CT scan. Four rounds of chemotherapy followed, but it became evident that a new liver would give him the best chance at survival.

Amazingly, on the ideal day he was eligible to receive a transplant, a donor liver became available. Although the surgery went well, Elliott suffered severe complications and underwent another liver transplant just 13 days later, in addition to completing two final courses of chemotherapy.

Fortunately, the cancer is gone, Elliott has gained 20 pounds, he’s on the track team, and he’s made the seventh-grade honor roll.

Pulling Elliott and his family through were not only the physicians, but many Amplatz team members. “A lot of the care is [delivered] by the support people. All of these people kept us connected and pulled us along,” Doherty says.

“When people give money to Amplatz Children’s Hospital, they’re giving money to provide that level of care and survivorship,” she adds. “They’re giving it to the people who choose to commit their lives to finding innovative treatments and ultimately caring for real children with very serious health issues.”

You can make a difference

Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

Because with your support, anything is possible.

Center of attraction

2011-04-25T21:07:56Z

The new University of Minnesota Amplatz Children’s Hospital stands out for all the right reasons, but child-centered care tops the list

By Martha Coventry

Dr. Joseph Neglia’s job just got easier. As the Department of Pediatrics head works to recruit topflight experts in children’s health, he can point to the extraordinary new University of Minnesota Amplatz Children’s Hospital as a very visible symbol of the University’s dedication to children. “Having the Amplatz Children’s Hospital will go a long way to attracting the most talented faculty members and residents,” says Neglia, who also is the hospital’s physician-in-chief. “They will see that the University not only has a verbal commitment to caring for children, but that it, along with Fairview Health Services [which owns the hospital], has also put resources into creating something exceptional.”

Pulling in the best

Pediatric emergency medicine physician Anupam Kharbanda, M.D., who joined the Department of Pediatrics last year, is just the type Neglia wants to attract. Kharbanda grew up in Minneapolis, graduated from Carleton College, and attended medical school at the University of Iowa. While chief resident at New York-Presbyterian Morgan Stanley Children’s Hospital- Columbia University, he moved along with the rest of the pediatrics department into a newly built child-dedicated facility where he stayed for five years as an attending physician.

Several years ago, he and his wife thought of returning to Minneapolis to raise their children. Kharbanda was attracted to academic medicine, but he wanted to work in a hospital where every single person was committed to children and adolescents. At the time, the University’s children’s hospital was on the fifth floor of its adult medical center.

“To have a truly child- and adolescentcentered hospital, all of the services — clinical, child-family life, radiology, pharmacy, etc. — have to be aligned toward a common cause and, ideally, under one roof,” he says.

When Kharbanda heard that the University and Fairview were going to build a hospital dedicated to that kind of singular care, he reconsidered.

Building quality from the start

The new hospital rises above the more staid buildings on the West Bank campus of the University of Minnesota Medical Center, Fairview. Already a landmark, its shimmering skin of anodized steel changes color in the light. It stands out. And that’s a good thing. The University and Fairview want the new children’s hospital to be easy to find and easy to get to.

As the signature pediatric hospital of the state’s only academic health center, University of Minnesota Amplatz Children’s Hospital must play a crucial role in teaching and in conducting research. It must also help the University convey to the public what pediatric research is and how it connects to a child’s care. Aaron Friedman, M.D., vice president for health sciences and dean of the Medical School, believes the hospital’s obvious presence will help build that understanding.

“What the Department of Pediatrics does no longer happens behind the walls of the University, it happens in a very noticeable building that’s part of a neighborhood,” says Friedman, who was in charge of the department until he assumed his new roles in January. “When someone points at the hospital and asks what goes on in there, we hope it will become easier to answer in a way that helps people see how research leads to extraordinary care.”

The best of care for all children

In the century since the University admitted its first pediatric patient in 1911, the Department of Pediatrics has become one of the best in the nation, making discoveries that have changed the face of medicine worldwide. Among its many groundbreaking achievements, the University performed the world’s first successful pediatric bone marrow transplant and open-heart surgery using cross-circulation between a child and parent, as well as Minnesota’s first pediatric living-donor liver transplant and infant heart transplant.

Such breakthroughs are in the works every day both in the hospital and in University labs. For example, by applying research that started in the lab, University doctors recently performed the world’s first stem cell transplant to treat recessive dystrophic epidermolysis bullosa, a usually fatal skin disease. Currently, this novel approach is the only viable treatment for children who have this devastating disease.

The University’s children’s hospital has always been the place for kids with the most difficult health issues and it will continue to be. But the new facility cements its role as a full-service children’s hospital that manages the continuum of pediatric illnesses, from tonsillitis to diabetes to rare cancers, with an equally high level of care.

“In order to be the center for training the majority of pediatricians in the state, and to be more accessible to referring physicians, we need to be able to treat common pediatric illnesses as well as the complex,” says Abe Jacob, M.D., director of the Pediatric Hospital Medicine Program.

Doing what’s needed for care and comfort

Some of the sickest children treated at University of Minnesota Amplatz Children’s Hospital must stay in the hospital for weeks or months at a time.

For those families, the hospital room becomes a home away from home. In the former children’s hospital, the rooms were small, they had no comfortable place for parents to sleep, and they were often shared between two patients.

If the new hospital was going to set the standard for familyand child-centered care, that had to change. When it came time to design the rooms for the new hospital, patients, former patients, and their families met with the architects and select physicians and staff.

“We said to the children and their families, ‘Tell us how to build a hospital,’” says Neglia. “Then we just listened and learned.”

Based on their advice and needs, Fairview committed to building large, cheerful rooms, all private, with a wall of windows and space for parents to sleep, store their clothes, work on a computer, and fix and share simple meals. These exceptional rooms are designed to improve outcomes and accelerate healing. Through the Adopt A Room program, individuals and companies can cover the cost of more special features that let patients control many aspects of their rooms and stay connected with school, friends, and family. Other design elements keep disruption of the family and patients to a minimum. The hospital has lounges for parents, family education spaces, and playrooms on nearly every floor.

“It’s really important to have families stay with their children,” says pediatric resident Katie Larson, M.D. “It helps with healing, and parents gain the knowledge and confidence to care for their child when they go home.”

In fact, at University of Minnesota Amplatz Children’s Hospital, parents are considered a crucial part of the care team, and physician rounds literally put them at the center of their children’s care.

“With family-centered rounds, we’ve essentially moved all of our discussions about the child from the conference room to the bedside, with the family present,” says Jacob. “It’s been proven that this type of care improves a family’s experience of the hospital stay and engages families more in the care of their child.”

Family-centered rounds also enrich the learning experience of medical students and residents, as do better, larger education spaces in the new hospital.

An exceptional message

Primary physicians in the community and around the country refer children to the University’s hospital for the treatment of many challenging diseases. According to Jacob, the new hospital’s location, facilities, philosophy, and technology will strengthen that connection.

“We deal with many referring physicians, who care deeply about their patients’ well-being,” says Jacob.

“With the new hospital, we can now assure them of four major things: that their patients and families will have easy access to our facility, that they are getting the most innovative care experience, that the care delivery will be family-centered, and that we will continually communicate with them about their patients.”

Those messages reflect a philosophy Kharbanda endorses and make him happy that he signed on with the University of Minnesota.

“With the Amplatz hospital, there’s the monetary commitment on the part of the University and Fairview, which is crucial, but equally important is their long-term commitment to making everything and everybody child- and family-focused,” he says. “This approach forges a different type of team, a children’s team, and I’m glad to be part of it.”

Martha Coventry is a freelance writer and editor who’s interested in medicine and the natural world.

To find out how you can make every chance possible for a child, please contact the Minnesota Medical Foundation’s Children’s Health Team at 612-626-1931 or childrenshealth@mmf.umn.edu or visit uofmhope.org.

Web extras

Video: Because children deserve every chance

Video: Amplatz Children’s Hospital tour

Companion story

Delivering novel cancer therapies. Read more.

Companion story

Through the emergency department doors. Read more.

You can make a difference

Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

Because with your support, anything is possible.

Healthy kids at home and abroad

2011-04-25T19:00:36Z

The International Adoption Clinic celebrates 25 years

Ten-month-old Margo Freeman couldn’t have looked any healthier. Yet, when Don and Robyn Freeman learned that their adoptive baby girl was waiting for them in Ethiopia, they sought an expert evaluation of her medical records and advice on what to expect when adopting a baby from a foreign country.

“We were already in love with her just from seeing her picture, so there was probably nothing that would have changed our minds,” says Robyn Freeman. Still, they wondered what medical challenges might lie ahead.

They turned to the International Adoption Clinic (IAC) at the University of Minnesota. Now celebrating its 25th anniversary, the clinic was the first of its kind in the country and helped to establish the field of adoption medicine as a specialty in pediatrics. It has become a model for other clinics around the country, garnered an international reputation, and through extensive research, has created a greater understanding of the special medical and developmental needs of adopted children.

Even before they went to Ethiopia, the couple sent the medical information they had about Margo to the clinic for a pre-adoption assessment. The IAC staff conducts roughly 1,500 of these annually and sees 300 to 400 children in the clinic each year.

A wealth of expertise

One problem for adopting families is that medical information is inconsistent from country to country, and a number of factors — different calendars, incomplete paperwork, and translation issues, for example — can paint a confusing and misleading picture of a child’s health.

As it turned out, Margo had tuberculosis, which was treated successfully in Ethiopia. Says Freeman, “The IAC did a great job of ensuring that the TB was completely treated and performed many follow-up tests that gave us peace of mind.”

“It’s important to have the expertise to view medical reports in the context of each child’s country of origin,” says associate professor Cynthia Howard, M.D., the clinic’s director since 2005 and also the proud mother of two daughters adopted from Democratic Republic of Congo. “We often know the orphanage and the medical staff personally.

“We like to see kids in the clinic two to three weeks after they arrive home,” she adds. “We do baseline screening to address any immediate physical problems as well as long-term cognitive and attachment concerns.”

Depending on the child’s needs, the clinic draws from the University’s wealth of expertise across the medical spectrum — ophthalmology to occupational therapy — and coordinates the child’s care among all of these specialties. According to Howard, the sooner they can address a child’s needs, the greater the chances for the child’s success, not just medically, but also psychologically, socially, and academically.

Concerns shift with geography

Parents wishing to adopt a child internationally haven’t always had these medical resources. In 1986, when the clinic was founded, Americans were adopting about 8,000 international children per year, mostly from Korea. The number of Korean adoptions dropped by half in the early ’90s, but new regions and countries such as Russia, Romania, China, South America, and sub-Saharan Africa opened for adoption.

This change brought children with entirely new medical issues. In Eastern Europe, for example, children live in orphanages rather than foster homes, which has profound implications for their development; whereas children in Ethiopia are at risk for infectious diseases such as malaria and tuberculosis. It is difficult for primary care providers in the United States to be entirely familiar with the health risks that affect children in these parts of the world.

The clinic’s research has pinpointed some of the physical and developmental challenges that many of these children face, including growth delays, attachment difficulties, sensory processing disorders, and learning problems.

From the IAC’s work, a broader view of clinical support necessary for internationally adopted children like Margo has evolved. Over its first 25 years the IAC has improved care and supportive strategies not only for children at the clinic but also from around the world.

Margo is a happy and thriving 18-month-old now. And throughout her childhood, says her mom, “the International Adoption Clinic will continue to play a key role in her development.”

By Terri Peterson Smith, a freelance science writer and editor

Web Extras

Video: The Goldman Family celebrates 25 years of the IAC

Video: Interview with Judith Eckerle Kang, M.D.

Video: Interview with Dana Johnson, M.D.

Video: Interview with Cynthia Howard, M.D.

IAC’s founding father

Few people have had a greater impact on the world’s children than International Adoption Clinic (IAC) founder Dana Johnson, M.D., Ph.D. A professor in the University’s Department of Pediatrics, Johnson adopted his own son, Gabriel, from an orphanage in Calcutta, India, in 1985.

“There wasn’t a lot of information about the medical issues in international adoption then. I talked to a lot of adoptive parents who were concerned,” recalls Johnson. He responded by starting the IAC to counsel families before an adoption, provide care after adoption, and establish a research program to further characterize international adoptees’ problems and needs.

Johnson has since become one of the world’s foremost experts on health and developmental issues affecting the institutionalized child. He turned the leadership of the clinic over to Cynthia Howard, M.D., and now takes his message around the world to advocate for adoption and to change the system of institutional care.

“We intuitively knew families were important,” Johnson reflects. “Through this work we’ve learned just how devastating it can be to be without a family, in spite of advances in medicine.”

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Delivering novel cancer therapies

2011-04-25T16:41:37Z

With crucial philanthropic support from individuals and organizations such as Children’s Cancer Research Fund, research discoveries made at the University of Minnesota have helped increase survival rates for childhood cancer from 10 percent in 1959 to nearly 80 percent today. But Department of Pediatrics faculty and leaders realized that if the University wanted to continue as a leader in the fight against pediatric cancers, it needed better facilities.

That’s why nearly a third of the new University of Minnesota Amplatz Children’s Hospital is dedicated to serving children who have cancer. The larger space will allow the University to expand the number and types of therapies it can offer, says pediatric oncologist Brenda Weigel, M.D. Neuroblastoma is a type of childhood cancer that develops from nerve tissue. In the last decade, doctors have made a major advance against these tumors using an IV delivered radioactive compound. A child who receives this therapy emits radiation and can only be treated if he or she stays in a lead-lined room. There were none of these rooms in the former University children’s hospital, but the new hospital has built one.

“There are only six places in United States that will be able to offer this therapy, and now we’re one of them,” Weigel says. “If the new hospital hadn’t built this room, we would have been a decade behind in this kind of care, which will be the therapy of choice for high-risk patients within five to 10 years.”

Weigel notes that another feature of the new hospital — a combined hematology/oncology and blood and marrow transplant clinic — will allow doctors to offer better cancer care and make treatments easier for patients and families.

Web Extras

Read more about how the new University of Minnesota Amplatz Children’s Hospital stands out for all the right reasons.

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Through the emergency department doors

2011-04-25T12:44:04Z

As codirectors of the Division of Pediatric Emergency Medicine at University of Minnesota Amplatz Children’s Hospital, Ronald Furnival, M.D., and Mark Roback, M.D., share a big job. They are building an emergency department that promises to become one of the best in the country.

Their role is especially important because a significant number of patients will come to the new children’s hospital via its pediatric-only emergency department.

Most of these children will be seen for common problems such as trauma, asthma, fever, and abdominal pain, as well as serious flare-ups of chronic conditions. “Research is vital to discern the ideal way to treat these acute conditions in the emergency department setting,” says Anupam Kharbanda, M.D., who recently joined the pediatric emergency medicine team as director of research and assistant professor.

Kharbanda has developed an algorithm to determine a child’s risk for appendicitis when he or she comes into the emergency department with acute abdominal pain. “The goal is to reduce our reliance on imaging without missing an appendicitis,” he explains, noting that the same approach could potentially be adopted for assessing other illnesses.

“We are an academic children’s hospital,” says Roback. “One of the things that’s so exciting about working here is to witness how teaching and research are helping us continually provide better emergency department care for children.”

Web Extras

Read more about how the new University of Minnesota Amplatz Children’s Hospital stands out for all the right reasons.

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Bridging research and care: University's new children's hospital to open its doors April 30

2011-04-12T19:32:35Z

Coming soon to a neighborhood near you: a state-of-the-art children’s hospital in a vibrant package.

It’s hard to miss the new University of Minnesota Amplatz Children’s Hospital along Riverside Avenue in Minneapolis. With its special anodized steel exterior, the building changes color throughout the day depending on how the light hits it. This material has been used on only one other building nationwide.

Beyond its physical brilliance, University of Minnesota Amplatz Children’s Hospital will become a beacon of hope for children and their families when it opens its doors to patients on April 30.

“We’ve tried to create, throughout the hospital, little moments of ‘Wow!’—where a child will see something and, even if it’s for a split second, they forget they’re a patient and just feel like a child,” says Russ Williams, vice president of professional services at the hospital.

Special care for special kids

Because it’s affiliated with the state’s only academic health center, University of Minnesota Amplatz Children’s Hospital plays a crucial role in conducting research focused on finding better ways to treat, cure, and prevent childhood disease.

The treatment protocols and devices developed by our physician-researchers are adopted by community hospitals and pediatricians throughout the country and the world. And because these advances happen here, Minnesota patients benefit from them first.

As a result, families with the sickest children often come to University of Minnesota Amplatz Children’s Hospital for the treatment they can’t get anywhere else.

Designed to foster healing

Some ill children must stay in the hospital for weeks or months at a time. For these children and their parents, the hospital room becomes the family’s home away from home.

That’s why hospital leaders believed it was so crucial to ask patients and their families what they wanted in a hospital—and then build the safe, healing environment that the kids deserve.

In the new hospital facility, private, cheerful rooms that are 35 percent larger than the national standard include a wall of windows and space for parents to sleep, store their clothes, work on a computer, and fix and share simple meals.

These exceptional rooms are designed to improve outcomes and accelerate healing and can be sponsored by individuals or companies through the Adopt A Room program.

A cheerful environment

Outside of each individual patient room, the hospital hallways and common spaces incorporate a “Passport to Discovery” theme, which is meant to tie into the University’s mission of being “Driven to Discover.”

For starters, there’s a large compass mosaic on the lobby floor. Then each level of the hospital highlights a different type of wildlife habitat and animal mascot. The third floor, for example, is “grasslands,” decorated with warm orange-yellow paint and giraffe and elephant animal mascots.

Each habitat theme is reflected in several places throughout the floor—from the backlit scenes that greet people coming off of the elevator to the animal silhouettes on patients’ room signs.

Most important, the new building fills a need for a space that is truly focused on children and families, says Joseph Neglia, M.D., M.P.H., physician-in-chief of University of Minnesota Amplatz Children’s Hospital and chair of the Medical School’s Department of Pediatrics.

“We now have an outstanding physical space that reflects the quality of care we have been providing for years,” he says. “This new facility lets us offer families a whole new level of service.”

Slideshow

See more of the new University of Minnesota Amplatz Children’s Hospital.

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A global impact: University physician-researchers' work is making a difference far beyond Minnesota's borders

2011-04-12T19:25:00Z

There are places in the world where nearly seven in 100 newborns do not live more than a month, where a vast majority of births take place without skilled birth attendants, and where one in five children never lives to see his or her fifth birthday.

These stark realities fuel the drive of physicianscientists in the Department of Pediatrics at the University of Minnesota Medical School.

“When you actually visit these countries and see firsthand the tremendous health issues children face, it’s life-changing,” says Chandy John, M.D., M.S., director of the department’s Division of Global Pediatrics. “You can’t do this kind of work and not be moved by it.”

Understanding malaria

Working with colleagues in Uganda and Kenya, John and his team have made the University an international leader in the study of malaria, a disease that accounts for approximately one in five of all childhood deaths in Africa.

“We’re seeking to find answers to basic questions—such as how malaria occurs and progresses, whether it can be prevented, and how it’s best treated,” says John.

In Kenya, his research is focused on understanding why malaria is more prevalent in the country’s highlands than in other regions.

“These areas don’t have year-round transmission where people are exposed to malaria every day, but they can have severe epidemics,” he says. “Determining why epidemics occur should provide more clues about what we can do to prevent them.”

In Uganda, John is studying cerebral malaria, an acute type of malaria that affects the brain. His studies have shown that one in four children with this type of malaria develops long-term cognitive impairment.

Exactly how this happens and what can be done to reverse or minimize the damage is an emerging area of study. If researchers can identify treatments that help protect children’s cognitive abilities, John says, they’ll have a better quality of life and a greater chance of reaching their full potential as contributing members of society.

Making diabetes manageable

While the widespread impact of malaria in developing countries demands considerable resources, other diseases that are generally considered manageable in the United States contribute to the global children’s health crisis.

One such illness is type 1 diabetes. With the leadership and expertise of University pediatric endocrinologist Toni Moran, M.D., a long-term, collaborative relationship with Mulago Hospital in Kampala, Uganda, is addressing the need for better diagnosis and treatment of type 1 diabetes in children.

The actual incidence of diabetes in Uganda is likely five times higher than what is reported because children aren’t always brought to medical facilities, Moran says.

“The majority of children with diabetes are dying without being treated,” she says. “A modest amount of education can make a huge difference.”

At Mulago Hospital, University physicianresearchers are helping to create health care teams to treat chronic illnesses, train providers to become diabetes educators, establish pediatric insulin protocols, and develop basic medical record systems.

“Our work indicates that physicians from developing nations can make a difference in improving the care of chronic illness without an unrealistically large investment of time or money,” says Moran.

Surely that’s an investment worth making.

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A resource at home and abroad, U's International Adoption Clinic celebrates 25 years

2011-04-12T19:23:10Z

“We were already in love with her just from seeing her picture, so there was probably nothing that would have changed our minds,” says Robyn.

Still, they wondered, based on the medical information they received, what challenges might lie ahead?

They turned to the International Adoption Clinic (IAC) at the University of Minnesota. Now celebrating its 25th anniversary, the clinic was the first of its kind in the country and helped to establish the field of adoption medicine as a specialty in pediatrics. It has become a model for other clinics around the country, garnered an international reputation, and created a greater understanding of the special medical and developmental needs of adopted children from foreign countries.

The IAC staff sees 300 to 400 children in the clinic each year and conducts roughly 1,500 pre-adoption health assessments annually.

As it turned out, the Freeman family learned that Margo had tuberculosis, which was treated successfully in Ethiopia. Today Margo is a thriving 17-month-old. “As she continues to grow, the IAC will play a key role in her development,” Robyn says.

You can make a difference

Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

Because with your support, anything is possible.

A Rosie conclusion to a long journey

2011-04-12T19:19:50Z

Any child diagnosed with cancer faces the battle of her life. But what if her family lives in an unstable country and cannot see a doctor? That’s what happened to Rosie Jones.

Albert and Koliju Jones knew something was wrong with their baby girl in early 2004.

“Her abdomen was getting large and very firm on the right side, and she complained of increasing pain,” recalls Albert.

The family lived in the West African nation of Liberia. “Our country was in civil unrest and medical services were not available,” he says. “We were just living with [the problem] until we had the opportunity to come to America.”

The luck of an immigration lottery gave them their opportunity. In June 2004, the Joneses moved to Thief River Falls, Minnesota, where other family members had settled.

After an exam at a local medical center, Rosie was referred to University of Minnesota Amplatz Children’s Hospital. At just under 2 years old, Rosie was diagnosed with Wilms’ tumor, an aggressive kidney cancer, which had spread to her lungs and liver.

Under the care of pediatric cancer specialist Brenda Weigel, M.D., Rosie went through an intense 16-month treatment program that included surgery, chemotherapy, and radiation.

Rosie finished treatment in October 2005. Her family remained strong and hopeful throughout the battle. “There is always hope, even when there seems to be none,” says Albert. “We never knew we were going to come to the U.S.A., but we had hoped that a cure was possible.”

Today Rosie is a healthy third grader who excels in school and loves ballet dancing and singing.

Her parents credit Rosie’s recovery to her health care providers—from Weigel and the entire medical staff to the social workers, occupational and physical therapists, and lab technicians.

“The treatments were just blessings,” Albert says.

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A special gift

2011-04-12T19:10:58Z

Family creates Ben & Bob’s Room to honor loved ones and make the hospital more like home for young patients

Bob Calmenson lived decades longer than doctors predicted. But when he died at 60 in 2009, his life seemed far too short to family and friends. “He left us too soon,” say his sisters, Margie Howell and Janet Lesgold.

Yet the spirit of Bob, and his father, Ben, who died at age 90 just 13 days before Bob died, will live forever in Ben & Bob’s Room, one of the patient rooms designed to feel more like home under the Adopt A Room program at the new University of Minnesota Amplatz Children’s Hospital.

Though Ben lived to age 90, it was Bob who truly beat the odds. He was born with a congenital heart defect and not expected to make it past age 20. However, surgery as an adolescent at the University’s former Variety Club Heart Hospital helped extend his life.

In gratitude, Ben and his wife, Vivian, established the Robert L. Calmenson Pediatric Cardiology Research Fund when Bob turned 50. Now members of the entire Calmenson family have given $200,000 to create Ben & Bob’s Room. It and other privately funded “adopted rooms” promise to provide a vastly different experience than Bob and his family had when he was hospitalized.

Vivian remembers small, shared rooms and restricted visiting hours. Family members were not allowed to stay overnight. The situation left a lot to be desired. And that’s why the Adopt A Room program appealed so much to the Calmensons.

“This is a way of paying it forward,” says Laurie Calmenson, Bob’s widow.

Adopted rooms will be private and large, with mini-fridges, microwaves, tables for eating together and doing homework, and sleeper sofas. These rooms also will feature technology that allows children to control the lighting, TV, inter-room communications, and video conferencing that keeps them connected to school, family, and friends.

Both men surely would approve of Ben & Bob’s Room, says Lesgold. “If they’re looking down on us, they would be very pleased.”

To learn more about how you or your company can sponsor an Adopt A Room, contact Lauren Moore at 612-626-7946 or l.moore@ mmf.umn.edu.

To become part of our Partners in Care program, contact Jen Foss at 612-626-5276 or j.foss@mmf.umn.edu.

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Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

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Turning loss into hope

2011-03-25T17:28:29Z

Family hosts community fundraiser, creates a pediatric heart research fund in daughter’s memory

When Jaclyn and Tony Doffin found out that they were having triplets, they got busy planning for their new life with three infants. One of the triplets was diagnosed with a heart condition prior to birth, but the family never anticipated the devastating complications that their baby would have as a result.

When the triplets, Tyler, Sophia, and Grace, were born on September 8, 2008, doctors quickly determined that Grace had a more severe case of hypoplastic right heart syndrome than originally thought. “She was born with three [heart] chambers,” explains Tony. “She was missing the one that pumped [blood] to the lungs.”

In her first few months of life, Grace had two heart surgeries at University of Minnesota Amplatz Children’s Hospital, and she spent more than two months in the neonatal intensive care unit—most of that time on a ventilator. She went home before Thanksgiving, but while awaiting her third heart surgery, Grace died unexpectedly on July 7, 2009.

Compelled to create some good out of their tragic loss, the Doffins created the Grace Doffin Pediatric Heart Research Fund through the Minnesota Medical Foundation to benefit pediatric heart research at the University of Minnesota. They raised money for the fund through a community fundraiser they organized called Hope in Grace.

“You can do something good with it. Life is what you make of it,” Jaclyn explains.

Superior care at the University

The Doffins say that the high level of care Grace received at University of Minnesota Amplatz Children’s Hospital inspire them to give back to the University. “We had great experiences with the doctors and nurses,” says Jaclyn.

Their situation was particularly complex, she explains, because they had two babies at home while Grace was still in the hospital—resulting in many trips back and forth between the hospital and their home in Blaine, Minnesota.

“It was very hard to not be able to be by Grace’s side constantly,” Jaclyn says. “We found out that a lot of nurses and staff members were coming in to hold Grace when we couldn’t be there. That was really awesome.”

Helping babies like Grace

In the fall of 2009, the Doffins started planning the first Hope in Grace Family Fun Run, which took place in their Blaine neighborhood in April 2010. “This was an opportunity for other people with healthy children to give back,” Jaclyn says.

And give back they did.

The run and silent auction drew more than 300 participants and 50 volunteers, and far exceeded the Doffins’ expectations—raising $17,000 for the Grace Doffin Pediatric Heart Research Fund. “It was really great to see neighbors afterwards wearing the T-shirts with Grace’s name on them,” Jaclyn recalls.

The run’s success has encouraged the Doffins to make it an annual event. They recently gained nonprofit status for their Hope in Grace organization and are preparing for the next run, set for April 16, 2011. That will be a busy time for the Doffins, who are expecting twins in June.

Tony says that he’s hopeful that the money raised will help others like Grace. “Maybe with research they could find a fix for it,” he says of Grace’s condition.

Adds Jaclyn, “If it helps even one child that would be rewarding.”

Hope in Grace Family Fun Run

Learn more about the event at www.hopeingrace.org

You can make a difference

Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

Because with your support, anything is possible.

Finding hope

2010-12-21T17:19:21Z

U researchers’ stem cell breakthrough treatment for a fatal skin disease began with philanthropy

For the first time ever, physician-scientists at the University of Minnesota have demonstrated that a lethal skin disease can be successfully treated with stem cell therapy.

Medical School researchers John E. Wagner, M.D., and Jakub Tolar, M.D., Ph.D. — in collaboration with researchers in Oregon, the United Kingdom, and Japan — used stem cells from bone marrow to repair the skin of patients with a fatal skin disease called recessive dystrophic epidermolysis bullosa (RDEB).

It’s the first time researchers have shown that bone marrow-derived stem cells can repair the skin and upper gastrointestinal tract and alter the natural course of the disease.

Until now, bone marrow has only been used to replace diseased or damaged marrow.

“To understand this achievement, you have to understand how horrible this disease actually is,” says Wagner. “From the moment of birth, these children develop blisters from the slightest trauma, which eventually scar. They live lives of chronic pain, preventing any chance for a normal life.”

That was reality for the Liao family of New Jersey. Theresa Liao tracked down Wagner at a medical conference in New York in 2004, literally thrust her then-2-year-old son, Jacob, at him, and begged him to save her child. It was a promise Theresa had made to her son at birth.

“I used to hold him in my arms and cry,” she says. “I told him I was going to make a difference, and that we were going to fix it or make it better or at least go down swinging.”

The encounter launched Wagner’s work on the disease. The Liao family raised enough money for his team to start a laboratory study with mouse models of RDEB. In 2007 the team found that a rare subpopulation of marrow stem cells could repair the mice’s skin.

With additional philanthropic support, Wagner and Tolar launched a clinical trial to find out if the therapy also could work in humans.

Since that study began at the University in 2007, 10 children with the most aggressive forms of EB have received transplants at University of Minnesota Amplatz Children’s Hospital. Although all of those children have responded to the therapy, the magnitude of each response has varied.

“While the treatment offers a chance for a better life, it comes with significant risk,” Tolar says. “Two children have died from complications related to the treatment, so refinements are needed.”

Jake Liao was one of the two children who didn’t make it. His younger brother, Nate, who also was born with EB, however, has improved significantly since his transplant.

For Theresa Liao, the success is bittersweet.

“I miss my best friend,” she says of Jake, “but I wasn’t afraid, because one way or the other I knew what the outcome was going to be if we didn’t give him a chance.”

And that’s precisely what motivates both Wagner and Tolar to keep improving the therapy.

“My hope is to do something that might change the natural history of this disease and enhance the quality of life of these kids,” Wagner says.

By Nicole Endres

To support this research, contact Elizabeth Patty at 612-625-6136 or e.patty@mmf.umn.edu.

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Help the University of Minnesota save lives, inspire hope, and prepare the world’s future health care leaders. Make a gift today.

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