Children’s Health—Research | 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.

Streamlining science

2011-10-25T01:01:05Z

The U’s Clinical and Translational Science Institute puts focus on bench-to-bedside research

By Dan Haugen

When Dorothy Hatsukami, Ph.D., began her University of Minnesota research career, investigators had to be “extraordinarily resourceful” to find everything they needed to conduct a study, from laboratory equipment to advice on filling out regulatory forms.

“Individual researchers had to do pretty much everything on their own,” says Hatsukami, a professor of psychiatry and director of the University’s Transdisciplinary Tobacco Use Research Center. It took time and sleuthing to get questions answered, forms completed, and studies set up and running. “There wasn’t one place that you could go to ask questions,” she recalls.

Today, 30 years later, that “one place” is finally becoming a reality for University researchers.

The Clinical and Translational Science Institute (CTSI), launched in April 2009, aims to be a one-stop shop for clinical and translational researchers. It’s doing so by centralizing research services that were previously spread out across several departments, as well as by investing in new, shared programs and technology that will be made available to all researchers, including offsite collaborators.

“The idea is integration for the purposes of highest efficiency, greatest patient safety, and optimum faculty and staff productivity,” says Bruce Blazar, M.D., the CTSI’s founding director and a Regents professor of pediatrics.

That may sound like a yawner, but it’s a big deal for scientists, doctors, and patients. That’s why the National Institutes of Health (NIH) just awarded the University a $51 million federal grant to accelerate the CTSI’s work, which also includes boosting community engagement and training the next generation of researchers and clinicians.

With the grant, the University joins a consortium of 60 Clinical and Translational Science Award (CTSA) health research centers across the country that are sharing resources and best practices in an effort to speed the translation of research discoveries into better patient care. The bottom-line outcome: more real-world cures, treatments, and best practices, says Frank Cerra, M.D., former senior vice president for health sciences and one of the CTSI’s leading proponents.

“It’s the infrastructure that will support an increased volume, on a shorter time line, of translational research that gets new knowledge into new treatments, new cures, and new companies,” he says. “That’s what this is about.”

Siloed approach in the past

Clinical and translational research at most institutions has tended to occur in silos and at a slow pace; on average, it takes 17 years for a lab discovery to actually benefit people, say CTSI leaders. All of the day-to-day and administrative tasks related to conducting studies have usually landed on the staff or department of the individual investigator, often on the shoulders of just one or two people for each area.

“As investigators, we were left to our own devices, so sometimes there would be duplication of effort in overcoming obstacles or challenges necessary to bring research forward,” says Blazar, who is a pediatric blood and marrow transplant physician.

In October 2005, the NIH announced it wanted to see a new, more efficient approach to clinical and translational research at the nation’s academic health centers. “We are truly at a crossroads in medicine,” the institutes’ then-director, Elias Zerhouni, said at the time. “The scientific advances of the past few years, such as the completion of the Human Genome Project, dictate that we act now to encourage fundamental changes in how we do clinical research, and how we train the new generations of clinician scientists for the medical challenges of this century.”

The NIH unveiled the CTSAs — large, multiyear grants to academic health centers and their community partners that can demonstrate a strong plan for reorganizing the way they support clinical and translational research. Among its priorities: lowering barriers across scientific disciplines, improving community engagement, and training the next generation of clinical and translational research scientists.

The University’s initial applications for the award fell short. After revamping the interdisciplinary research section and bolstering the Academic Health Center’s health informatics program, it was successful this year. In June, the NIH announced a five-year, $51 million award — the largest single-institution NIH award the University has ever received.

Growing data and complexity

The University has long been a leader in clinical and translational research, says Aaron Friedman, M.D., vice president for health sciences and dean of the Medical School. He points to the school’s history of developing new cancer treatments and transplant procedures, for example. The CTSA will help the University continue that success into the future, he says. “These awards provide the infrastructure to be able to do that kind of work the way it actually needs to be done in the 21st century.”

In the past, a well-organized spreadsheet and file cabinet might have been all the tools necessary to manage and analyze the data coming out of a clinical study. Today, though, both the volume and complexity of information researchers have to deal with has grown exponentially. “The amount of data has just exploded,” says Connie Delaney, Ph.D., R.N., dean of the School of Nursing and director of the Academic Health Center’s Biomedical Health Informatics program.

That data explosion has been driven by factors such as our knowledge of the human genome, the use of medical imaging technology, and the electronic health record. “Those are expansive amounts of data that, when used for discovery, enable us to deliver better care,” says Delaney. The availability of these data also requires clinical researchers to track, manage, and analyze far more information. Add the electronic medical record and a neverending stream of new scientific literature, and the data management burden on small research teams or departments gets to be overwhelming.

These trends make sharing and collaboration increasingly critical, something the NIH recognized when it designed the CTSA program.

Shared technicians, technology

The CTSI provides à la carte services for clinical researchers, from injecting medication and collecting blood samples to preparing grants and analyzing statistics.

As a senior researcher three decades into her career, Hatsukami has her own staff to manage most of the tasks related to running a research project. However, she still turns to the CTSI for help with certain functions. She conducts her clinical studies of tobacco users at the CTSI’s Delaware [Street] Clinical Research Unit, where the institute provides exam and observation rooms and CTSI staff can assist as needed with blood draws and other tasks. “Everything is here, in one place, for us to conduct studies,” says Hatsukami.

Meanwhile, the CTSI, integrally linked to the Institute for Health Informatics, led by Delaney, is investing in a suite of software, such as clinical trials management systems, that will be available for use by any University investigator. The goal is to establish a common and secure platform for managing clinical data, which should relieve the tech support burden on individual teams and departments, as well as make de-identified patient data easier to share with colleagues and collaborators across departments or multiple sites.

The ability to share technology and connect collaborators is critical to a current study by Aaron Kelly, Ph.D., an assistant professor of pediatric cardiology. He’s working with clinicians at Children’s Hospital and Clinics of Minnesota on a pediatric obesity study that is exploring the potential of an adult diabetes drug to help treat extreme obesity in adolescents. The study is funded by the CTSI, but it also relies on a piece of software provided by the institute called REDCap, which allows Kelly and his collaborators to collect and securely input data into the same database from either site.

“It’s important to have a secure system, and one that can be easily accessed and used across multiple centers,” says Kelly. “For a lot of us, setting up databases is not our expertise, so that kind of support is invaluable.”

Shared software and common practices for inputting data should also make it easier to share with other researchers, whether across campus or across the country.

Data + ideas + people

One of the CTSI’s primary goals is to build more cross-disciplinary collaborations among researchers and better connect researchers to communities outside the University’s walls.

In August, the institute launched a social network for University researchers called UMN Profiles, which can be used to help people find other investigators with like or complementary research interests and expertise.

The CTSI is also working with several community-led committees charged with developing partnerships among University researchers and communities across Minnesota. The committees — focused on child health, health disparities, rural health, and health care systems — will help to identify priority research topics, design studies, and disseminate the results of completed research.

“They’re going to help us identify the ways in which researchers from the University should approach and work with community partners,” says Andrea Leinberger-Jabari, coordinator for the CTSI community engagement efforts. “These leaders are closer to the community that they’re serving, so they can help researchers set the agenda and conduct research that ultimately helps build healthier communities.”

Mitchell Davis Jr., who chairs the University’s Community Health Advisory Council, one of the four committees, says he sees opportunity for a two-way conversation. The committee can call attention to new or overlooked research priorities and share advice on approaching people in their community. It can also play a role in distributing research results. “[Researchers are] not necessarily the ones who will know … who the influencers are,” says Davis.

Training the next generation

In addition to creating a shared infrastructure and improving community connections, the CTSI will stress training the next generation of clinical researchers and technicians. Plans call for developing new programs and courses to expose students to research careers earlier on and throughout their programs as well as continuing to provide mentorship opportunities.

Medical student Emily Mood says most of her exposure to clinical research has been either introductory or aimed at how to read and use research in practice. For some of her classmates, that was enough. But for her and others who entered the program with an interest in clinical research, there’s room for more emphasis.

“My friends who were interested in research and I thought it should have been given much more priority, and I think my friends who were less interested in research thought it was plenty the way it was,” says Mood. She’s spending her fifth year (through the Flexible M.D. program) in Mexico City working on a clinical environmental health study that will explore the interaction of lead exposure and maternal stress on childhood development.

Sarah Cusick, Ph.D., an assistant professor of global pediatrics, will be working to establish her research career over the next few years through the CTSI’s KL2 Scholars Program. The mentoring and career development program will pay 75 percent of her salary for the next three years and give her regular contact with three mentors, one of whom must be a statistician. Cusick, one of five KL2 scholars chosen this year, will be studying the interaction between infection and nutrition.

“It would be a lot more stressful right now” without the program, says Cusick. “I have this three-year breathing space. … I’m definitely learning the procedure for making research ideas become reality, but also learning to expand and enhance those research ideas, looking for outcomes that I might not have previously known to look for.”

Cultural shifts needed, too

CTSI leaders say they’ve made considerable progress in putting the institute’s pieces in place. Some functions are still ramping up and some software and technology still need to be purchased, but the $51 million NIH grant will accelerate the transition they started just over two years ago.

“We’re looking at a single clinical enterprise for translational research,” says Blazar. “We’re going to try to develop an approach that meets the needs of our investigators in a way that we haven’t been able to do before.”

Money can go only so far. For the CTSI and its grant to be successful, a cultural change will be required as well, says John E. Wagner, M.D., codirector of the University’s Center for Translational Medicine, now part of the CTSI (see sidebar).

The problem is that today, developing new therapies is too complex and too expensive to do alone, and the University can’t afford to duplicate services, Wagner says. “We have to really espouse the team culture — that the team is greater than the sum of its parts.”

Dan Haugen is a Minneapolis freelance writer who covers business, technology, and sustainability.

Web Extras

Video: Shifting research benefits to patients

Video: UMN Profiles - Clinical & Translational Science Institute

Video: Aaron Kelly uses the CTSI in obesity research

Audio

Center for translational medicine: bridging the gap

The translational “pipeline” — the path science takes from bench to bedside — starts with basic science and drug discoveries and ends in clinical trials and regulatory approval.

A few years ago, John E. Wagner, M.D., and Bruce Blazar, M.D., both clinical researchers and professors in the Department of Pediatrics, spotted a clog in the University’s pipeline.

“You can get funding for the very basic research, and you can get funding for the clinical trials, but trying to get funding to support this in-between section is impossible,” says Wagner.

What happens in between are less glamorous, preclinical studies, including proof-of-principle testing and animal toxicology studies, that are required before a treatment is approved for human trials.

Wagner and Blazar had developed some expertise in this area, and in 2007 they helped launch the Center for Translational Medicine (CTM). Now part of the Clinical and Translational Science Institute, the center offers University researchers expertise, technicians, facilities, and funding to support translational research and prepare treatments for clinical trials

While the Clinical and Translational Science Institute focuses on the entire pipeline, CTM zeroes in on the gap between basic science and clinical studies. It helps to design and conduct studies, plan project timelines, and, if and when the time comes, manufacture treatments for use in Phase I clinical trials.

“This is … bridging the gap between basic science and clinical medicine,” says Wagner. “That’s what the Center for Translational Medicine is.”

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.

Harnessing crucial cells to save lives

2011-10-12T16:37:50Z

It has been more than 40 years since University of Minnesota physicians performed the world’s first successful pediatric bone marrow transplant, and researchers here have never stopped trying to find better ways to secure long and healthy lives for children who have cancer.

Physician-scientist Heather Stefanski, M.D., Ph.D., echoes the dedication of her colleagues past and present when she says of her young patients, “I have to make life better for them.”

Today two major research efforts are exploring how to manipulate immunity-boosting T-cells—before and after an umbilical cord blood or bone marrow transplant—to save a child’s life.

Helping the body do its job

When a child receives a blood or marrow transplant, immune system T-cells play a central role. These sophisticated cells are educated as youngsters in the thymus—the “T” in T-cell—and develop into mature T-cells ready to protect the body against invaders.

One of the things to guard against after a transplant is graft-versus-host disease (GVHD), which occurs when T-cells in the graft (the transplanted blood or bone marrow) attack the patient’s body (the host) as “foreign.”

Bruce Blazar, M.D., director of the University’s Clinical and Translational Science Institute, wants to find a way to thwart GVHD by boosting a child’s supply of a type of cell called regulatory T-cells, whose job is to suppress fellow T-cells if they become too aggressive.

Growing regulatory T-cells in a lab is difficult, but Blazar’s research—supported by the National Institutes of Health (NIH), Leukemia and Lymphoma Society, and Children’s Cancer Research Fund—has replicated these cells in vitro up to 50-million-fold compared with only 70-fold previously. In animal models and limited human trials, University studies have shown that fortifying the body with these cells before a transplant is effective against GVHD.

“The ability to deliver such large quantities of these cells to patients before they undergo transplantation significantly reduces the chances of graft-versus-host disease,” says Blazar.

John Wagner, M.D., director of the University’s Blood and Marrow Transplantation Program, is now evaluating the promise of Blazar’s work through a clinical trial.

No time to spare

If overactive T-cells in the graft pose a threat to the child, not having enough T-cells in the host—the child’s body—can be lethal.

“Until a year after a transplant, children don’t have enough of their own T-cells,” says Stefanski, an assistant professor of pediatrics in the Division of Hematology, Oncology, and Blood and Marrow Transplantation. “Our patients can be cured of their leukemia and yet die from a common cold.”

Stem cells in the transplanted blood or marrow will, over time, develop into a child’s own protective T-cells, but these patients cannot afford to wait that long, Stefanski says. To better defend children against infections in that critical first year, she is looking for a way to augment their immune systems with young T-cells.

Working under an NIH project grant secured by Blazar and Wagner, Stefanski is growing these cells in her lab until they are strong enough to be delivered to a child. The hope is that enough of them will then migrate to the child’s thymus and grow more quickly into protective T-cells.

In a year or two, Stefanski believes she’ll be ready to transplant umbilical cord blood into mice and then introduce enough budding T-cells to prevent life-threatening infections. If her methods are successful, a human clinical trial may be possible in five years.

“One of the most heartbreaking aspects of my job is when a child dies because of an overwhelming infection due to the transplant,” she says. “We do everything we can, and it’s still not good enough. This is unacceptable to me and the reason that I’m doing my 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.

U researchers advance crucial stem cell technology

2011-07-12T19:21:57Z

University of Minnesota researchers have developed a new method for creating induced pluripotent stem cells (iPS), which can differentiate into many different types of the cells in the body and are used in medical research focused on diabetes, cancer, and many other diseases. This new process will dramatically speed up the creation of iPS cells and improve their quality, which could accelerate the treatment of many otherwise incurable diseases.

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.

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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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.

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.

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.

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.

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.

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.

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 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.

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 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.

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.

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.

Donors accelerate autism research

2010-11-30T18:33:10Z

Philanthropy makes a real difference in the lives of children with debilitating diseases and disorders.

Because of Alfred and Ingrid Lenz Harrison’s $1 million challenge gift to the University of Minnesota’s Autism Spectrum Disorders (ASD) Initiative in 2007, for example, researchers here are digging deeper into the causes and possible therapies for autism and related conditions.

Some of the research initiatives made possible by the Harrisons and the generous group of donors who contributed to the challenge fund include efforts focused on:

Improving attention among people with ASDs
Identifying infants at risk for ASDs
Understanding speech and voice processing in the autistic brain
Using stem cell infusions in treating autistic children with gastrointestinal inflammation, a common effect of autism
Determining whether congenital cytomegalovirus is linked to autism
Uncovering the roles of serotonin signals in early forebrain development
Obtaining metabolomic profiles of people who have ASDs

To make a gift in support of autism research at the University of Minnesota, contact Lauren Moore at 612-626-7946 or l.moore@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.

U researchers make headlines around the world

2010-11-30T18:27:02Z

Physician-scientists at the University of Minnesota have for the first time 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 disease called recessive dystrophic epidermolysis bullosa (EB).

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.

Tolar and Wagner’s research, published in the New England Journal of Medicine, was featured in hundreds of local, national, and international media, including the USA Today, CNN, Fox News, and MSNBC.

“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.

This research is supported in part by grants from the National Institutes of Health; the Ministry of Health, Labor, and Welfare of Japan and the Ministry of Education, Culture, Sports, Science, and Technology of Japan; and the University of Minnesota Academic Health Center, Epidermolysis Bullosa (Liao Family) Research Fund, Sarah Rose Mooreland EB Fund, and Children’s Cancer Research Fund.

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 revolutionary decision

2010-11-16T15:52:58Z

Ten years after Molly Nash became the first Fanconi anemia patient to survive following a controversial transplant, the ethical debate continues

In most ways, 16-year-old Molly Nash is a typical teenager. She argues with her parents. She bickers with her younger brother and sister (but admits to loving them, too). And she is a budding actress, recently portraying Chip the teacup in Beauty and the Beast.

The science that came together 10 years ago to give Molly these opportunities was revolutionary, controversial, and for her family, intensely personal.

“You never thought beyond milestones. Can we make it a month, six months, the first year?” recalls Molly’s mom, Lisa Nash. “Ten years ago we never would have imagined that we would ever reach this mark.”

At birth, Molly was diagnosed with Fanconi anemia (FA), a usually fatal genetic disease. Her only hope was a blood and marrow transplant. But survival rates for those without a matched sibling donor were a dismal 16 percent. And Molly had no siblings.

University of Minnesota pediatric hematologist/oncologist and umbilical cord blood transplant pioneer John E. Wagner, M.D., wanted better numbers. He worked with in vitro fertilization and preimplantation genetic diagnosis (PGD) experts to create better odds.

Ultimately, the Nashes would become the first to use PGD to have a child who was guaranteed to be free of FA and an exact blood match for a sibling.

“We knew this was going to be a hotly contested ethical issue. But we also knew that it was Molly’s best chance,” Wagner says.

In the spotlight

To start the process, Lisa Nash’s eggs were extracted and fertilized. Genetics specialists tested the resulting embryos to be sure they were disease-free and a match, and the selected embryos were implanted.

It doesn’t always work immediately—it took the Nashes five tries before a successful pregnancy. Adam was born on August 29, 2000, and Molly received his cord blood on what her family calls her “second birthday,” September 26, 2000.

“We recycled Adam’s cord blood—he didn’t need it anymore—and gave Molly life,” Lisa Nash says.

There were critics—vocal critics—who accused Wagner of playing God and manufacturing designer babies. Others took issue with selecting embryos for a trait such as blood match that was not of benefit for the resulting child. Through the firestorm of worldwide publicity, however, the overriding response was supportive.

The Nashes found the international attention overwhelming. “We weren’t doing it for the world’s approval or disapproval,” Lisa says. “We were flat out doing it for our family, so we could have a family.”

But it wasn’t a decision that the Nashes or their doctors took lightly. Wagner consulted with Jeffrey Kahn, Ph.D., M.P.H., director of the University’s Center for Bioethics.

“We made a conscious effort to be public and discuss this in a transparent way,” Kahn says.

A lesson in living

Ten years later, the ethical debate continues. Many embryos are created to get one perfect genetic match for patients in need of a transplant. Alternatively, before preimplantation genetic screening was an option, some families chose to abort healthy but non-matched fetuses, Kahn says.

From a scientific perspective, much has changed. “This technology is now being used for many different diseases, and many places offer it,” Wagner says. “While the debates continue, we use these technologies for good.”

And the FA survival rates are vastly improved—about 90 percent for transplants involving unrelated donors and 100 percent when the donor is a matched sibling.

Meanwhile, Molly Nash, who will always be a “first,” goes about her typical life.

“Molly’s taught us to live life,” Lisa Nash says. “To this day we live life to the fullest because we still have no idea how long we will have her on this earth, so we make every second special.”

By Sara E. Martin

Web Extras

Companion Story: Finding hope

Laurie Strongin’s uneventful pregnancy belied the reality of her firstborn’s medical condition. Born in 1995, Henry had Fanconi anemia, and Laurie and her husband, Allen Goldberg, quickly learned that a matched sibling blood and marrow donor was his only hope. Read more here.

Excerpt from Saving Henry: A Mother’s Journey by Laurie Strongin

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.

SPH survey finds fewer uninsured U.S. kids

2010-10-01T18:13:37Z

Since Congress launched the State Children’s Health Insurance Program (SCHIP) in 1997, the number of uninsured children in this country has declined by more than 20 percent, according to a new study from the State Health Access Data Assistance Center (SHADAC).

The survey finds that 8 million U.S. children are without health insurance, down from 10 million in the late 1980s. The researchers credit SCHIP, which uses a mix of state and federal dollars to provide coverage for children in families who earn too much to qualify for Medicaid but cannot afford private insurance.

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Did you know? Famous firsts at U of M Amplatz Children's Hospital

2010-10-01T17:18:29Z

University of Minnesota Amplatz Children’s Hospital has one of the nation’s top 15 pediatric research programs. Partnering with the University’s Medical School and Department of Pediatrics, the hospital has been a part of numerous medical breakthroughs, including:

The first successful pediatric blood and marrow transplant to treat childhood cancer.
The first infant heart transplant in Minnesota.
The first cochlear ear implant surgery for a child.
The first pediatric kidney biopsy.
The first blood and marrow transplant to treat a lethal skin disease in children.

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.