Children's Cancer Research Fund provides critical funding to the University of Minnesota Cancer Center for new cancer research and training related to the prevention, treatment and cure of childhood cancers. The success of the research is directly impacted by the quality of the researchers. We strive to support, recruit and retain the brightest minds in the field.
Cancer is the leading cause of death by illness among children, but doctors and researchers supported by Children’s Cancer Research Fund are working hard to change that statistic.
Meet some of the doctors conducting this breakthrough research and see how your contributions are helping find a cure for childhood cancer.
John E. Wagner, M.D.
Pediatric cancer research is at a pivotal junction. Breakthroughs are being discovered at increasing frequency.
What is your current research focus?
My research is focused on the development of new treatment approaches for life-threatening disease for which conventional treatments are unsatisfactory. While most of my work historically is in the setting of leukemia and bone marrow transplantation, new possibilities now exist allowing us to consider moving outside this area - such as the area of cardiovascular disease, diabetes and neurological diseases.
Why are you committed to or why did you choose to focus on childhood cancer?
When I look back, there were two pivotal points that led to my career as someone who treats children with potentially incurable cancers using experimental therapies. The first event occurred in July of 1978. As a second year medical student, I was awarded a scholarship to spend two months during summer break working on the cancer ward at the Children's Hospital of Philadelphia. On my very first day, I arrived early where I found a distinguished woman in the waiting room of the pediatric oncology clinic inside a floor-to-ceiling bird cage. After she finished her morning chore of feeding the birds, she introduced herself and surprisingly, kissed me on the cheek. Why? She looked me in the eye and told me this was going to be a great day - a spectacular day - because one of her patients had achieved an unexpected remission. While I didn't know it at that moment, this woman happened to be Dr. Audrey Evans, a world renowned oncologist and pioneer in the development of various treatment regimens for children with cancer. To me she was just a doctor who was thrilled 'to the bones' that her patient was doing well. I was captivated.
It was a transforming moment. While I had imagined myself being a cancer doctor, it was at that moment that I knew I wanted to be just like her - a researcher and clinician - a doctor focused on treating children with cancer. But more importantly, I wanted to have a career that was as exciting the last day as it was on the very first. My course was set.
Five years later, a second event occurred. I was halfway through my first year as a pediatric oncology fellow at The Johns Hopkins School of Medicine in Baltimore. On December 15, 1984, I set foot, for the first time, on the bone marrow transplant unit. From the moment I arrived, it was clear that this was going to be a very long rotation. Ont he BMT unit, we had 14 patients, children to adults up to 40 years of age, all with leukemia. On Christmas Eve, I spent the night because on that night it was possible that every single patient could die. For days, I lived in the hospital, sitting at the bedside for hours managing ventilators and medicines minute by minute. I saw things that were unimanginable - like the day I took a 30 year old mother of two kids to say goodbye to them as she was about to die from graft rejection. But the 'goodbyes' were through a two-way mirror; she didn't want her children to remember her the way she looked at that moment. It was simply horrible.
At the end of my rotation, Dr. George Santos, who is considered to be one of the 'fathers of bone marrow transplantation' sat me down, told me to go home, and come back to him after a few days of rest. As it would turn out, it was the second transforming event of my careeer. We talked for an hour about this seemingly horrible treatment, but I walked away knowing it was a great frontier to be explored. To him and me, it meant hope for people that had none otherwise.
That afternoon, I received a call from the head nurse on the transplant unit asking me to come and say goodbye to two patients. As it would turn out, the only two that survived those six weeks. While it was a celebration for the patients, it was also a surprise party for me. The nurses, residents and families of the survivors and those that did not, came to the party. It had been a long six weeks and as a gift, they gave me a book - Osler's Textbook of Medicine written at the turn of the 20th century. On the inside cover were the names of all the nurses with notes and well-wishes. To this day, it remains the single best acknowledgement I've ever had. On that day, I knew 'to the bones' that this is what I should do. And it has and continues to be the career I had hoped for since the first day in the clinic - next to the bird cage.
How has Children’s Cancer Research Fund made a difference in your work?
The impact of Children's Cancer Research Fund remains to be fully determined. While it may be measured by the number of papers written, number of grants received, or even the number of new ideas that become reality, the organization's greatest legacy is in the number of lives it has saved and hope it has brought.
Clearly at a time when grant dollars are shrinking, it is critically important to our success. CCRF funds allow us to investigate new treatment avenues, to fill gaps between grants and to train new doctors. But, CCRF is much more. It is composed of a group of tremendously dedicated staff who work day and night and self-less volunteers.
Certainly, we know we are lucky and fortunate. Without CCRF we would be far less than what we are today. When we look into the eyes of the kids with cancer and those of their mothers and fathers, we know there is a team behind us. In every way possible, CCRF is motivating us and supporting us.
If asked how CCRF has specifically helped my own research, we can take cord blood as an example. I used CCRF funds initially in the mid-1990's. Work was just beginning and survival rates were good but not great. In a series of clinical trials, we were able to move cord blood from second line source of bone marrow transplant to first line. At the University of Minnesota, we have tripled survival in adults. The double cord blood approach, which gave this result in adults, is now being tested in children. The NIH is supporting this multi-institutional/multi-national trial in both children and adults soley based on our results.
But there are other examples. CCRF has supported studies in Fanconi anemia. Still today, the only curative therapy is marrow transplantation. In 1995, results were dismal; the overall survival rate was 18% at two years after transplantation. Today, four trials later, survival exceeds 80%. Today, more than half of all children with Fanconi anemia come to the University of Minnesota for care.
We have recently used marrow stem cells to treat a life threatening skin disorder called Epidermolysos Bullosa. Without CCRF, this could never have occurred. Together with funds that the family generated, Jakub Tolar and Bruce Blazar were able to obtain sufficient data to justify such a risky treatment. Four weeks ago, we performed the first such therapy in the world. In several weeks, we may know its true benefit. CCRF funds permitted this to happen. The impact of having funds immediately available to jump-start such studies is immeasurable.
So, how has CCRF helped my research? Time will tell - there is much more to come.
Julie Ross, Ph.D.
The research being done today will save the children of tomorrow.
What is your current research focus?
Many of my studies investigate the causes of childhood leukemia. I also direct a laboratory that focuses on understanding genetic and environmental factors important in childhood cancer overall. I lead the Epidemiology Committee in the Children's Oncology Group, a consortium of hospitals and institutions in North America that treat approximately 90% of all children diagnosed with cancer. A focus area involves the establishment of a new North American pediatric cancer research registry called the Childhood Cancer Research Network (CCRN), which will be launched in 2008. The CCRN will enroll children and their parents in the United States and Canada in order to facilitate studies that investigate the causes and consequences of childhood cancer.
Why are you committed to or why did you choose to focus on childhood cancer?
While children with cancer represent only a small proportion of the general population with cancer, several important discoveries in understanding how cancer occurs have been made by studying childhood cancer. However, there is still much more to be learned. The window of opportunity for childhood cancer to occur is much narrower than for adult cancer. We need to focus on why kids get cancer, particularly since some childhood cancers are increasing in number over time.
How has Children’s Cancer Research Fund made a difference in your work?
Children’s Cancer Research Fund provided the seed money for my investigations of childhood leukemia. The data generated from CCRF funded pilot studies led to grant funding from the National Cancer Institute in excess of $5 million over a 10 year period. CCRF is also instrumental in supporting students who have a specific interest in understanding the causes and consequences of childhood cancer. These students represent the next generation of researchers, and CCRF has been instrumental in helping us hire the best and brightest.
Joseph Neglia, M.D., M.P.H.
As recently as the 1950’s, a diagnosis of cancer was a virtual death sentence for a child. Today, 8 out of 10 children with cancer can be successfully treated.
What is your current research focus?
My current research focuses on the outcomes of children we have treated for cancer. We are looking at not only the physical health of these children, but their educational, social and emotional outcomes. We are also working on understanding the best ways to make our long-term survivors informed and effective self-advocates as they move into adulthood.
Why are you committed to or why did you choose to focus on childhood cancer?
First and always, it is the children. With full lives ahead, the potential of any child is both unknown and wondrous. The opportunity to develop a career that would take on a life-threatening disease in a child is something that has allowed me to do something I believe in every day. It is certainly one with great highs and great lows, but in balance we continue to make progress and make a difference for our children and their families.
How has Children’s Cancer Research Fund made a difference in your work?
Children’s Cancer Research Fund helped fund my training here at the University of Minnesota, but then went on to help fund some of my early research in the causes of childhood cancer and the late effects of cancer that we study now. This investment has allowed me to develop research that has now been supported by the National Cancer Institute, the American Cancer Society, the Lance Armstrong Foundation and many others. This wouldn't have happened withouth CCRF. More than this, the broad scope of work that CCRF supports lets me honestly tell every family there is hope and we will not only cure a disease, but really restore health for their child.
Brenda Weigel, M.D.
CCRF gives us the opportunity to pursue incredibly promising ideas in childhood cancer.
What does your research focus on?
My research focuses on new immunotherapy strategies for pediatric cancers especially focused on rhabdomyosarcoma and AML. My team is working on identification of promising new agents that may improve the treatment of childhood cancers. Using laboratory models, these agents can be identified and screened, then translated into clinical trials for patients.
Philip McGlave, M.D.
…long-standing collaborations with pediatric oncologists have been incredibly fruitful.
What is your current research focus?
My interest lies in cell and immune-based therapy of blood cancers (such as leukemia) and solid tumors (such as breast cancer)
Why are you committed to childhood cancer?
I am an internist/cancer specialist, not a pediatrician, but I am committed to finding cures for cancer in adults and children through innovative clinical research and therapy. My long-standing collaborations with pediatricians such as John Kersey, Norma Ramsay, John Wagner, Bruce Blazar and other pediatric cancer specialists have been incredibly fruitful.
How has Children’s Cancer Research Fund made a difference in your work?
CCRF has provided grants for many faculty who are just starting their careers. Such grants have supported developments in blood cell transplant such as umbilical cord blood transplant, which have proven revolutionary for the therapy of both children and adults with cancer.
K. Scott Baker, M.D.
In medical school I became fascinated with childhood cancer, the kids, their families and how amazingly well they did with their diagnosis and treatment.
What is your current research focus?
My main research activities are in the area of short and long term effects of blood and marrow transplantation. Investigations are underway related to the incidence, risk factors, and characteristics of cardiopulmonary, renal, endocrine, reproductive late effects and quality of life outcomes in long-term childhood cancer survivors.
Why are you committed to childhood cancer?
I have always had an interest in cancer, but it was not until my third year of medical school that I even really knew that kids got cancer, too. I did an elective rotation in pediatric oncology and was fascinated with the diseases, the kids, their families and how amazingly well they did with their diagnosis and treatment. I was also drawn to pediatrics, and subsequently pediatric oncology, as none of the diseases that we treat in kids are “self-induced” as is so often the case with many diseases in adults, including some cancers.
How has Children’s Cancer Research Fund made a difference in your work?
I have received several CCRF pilot grants that have jump started my research career in cancer survivorship. For me, these CCRF grants have provided seed money that has allowed me to do smaller preliminary studies and this data could then be included in larger grants submitted to the National Institutes of Health. Recently, a $40,000 pilot grant from CCRF generated data that was used to secure an NIH grant valued at $2.5 million.
I also direct the Hematology/Oncology Blood and Marrow Transplant Fellowship Training program and funds from CCRF are utilized to help support this fellowship training program. Today, there are very few funding sources for these training programs and this support has allowed us to offer one of the top training programs in the country drawing applicants from around the world. Past University of Minnesota Fellows are leaders in the field of pediatric oncology across the United States and also in several countries outside the U.S. Through support of this program, Children’s Cancer Research Fund is active globally in the fight against childhood cancer.
Bruce Blazar, M.D.
We need to find innovative ways to treat childhood cancer outside of conventional chemotherapy and radiation therapies.
What is your current research focus?
My current focus is to get early phase therapies into the clinic. Areas that are currently under investigation include:
- Prevention of graft-versus-host-disease, a common side-effect after blood or marrow transplantation.
- Development of new strategies to enhance immune recovery after transplantation.
- Prevention of tumor and leukemia relapse.
- Gene therapy and tissue repair.
Why are you committed to or why did you choose to focus on childhood cancer?
Although some types of childhood cancer can be readily treated using chemotherapy drugs alone, other types will need innovative approaches that are developed from the laboratory. When I began my career in pediatric oncology and blood and marrow transplantation, it was with the hope and expectation that I might make a difference by investigating the basic principles needed for treating childhood cancers and translating those into the clinic.
How has Children’s Cancer Research Fund made a difference in your work?
CCRF has had a tremendous impact on my research, providing the critical seed money needed to develop innovative concepts and approaches for childhood cancer therapy and then translating these to the clinic. CCRF has enabled our division and program to create a powerful team dedicated to treating childhood cancer using all available techniques and approaches to do so. Without question, CCRF is a major reason that we have been successful in accomplishing this goal and for remaining at the University of Minnesota to continue these efforts.
Daniel Mulrooney, M.D., M.S.
We need to educate and empower childhood cancer survivors so they are better able to advocate for themselves and lead full and productive lives.
What is your current research focus?
My research focuses on the long-term follow-up care of childhood cancer survivors. When we understand the long-term effects, we can focus on maximizing cure and minimizing side-effects.
Paul Orchard, M.D.
CCRF has been instrumental in supporting basic research that has furthered our understanding of mesenchymal stem cells, a versatile and potentially powerful tool in the fight against childhood cancer.
What is your current research focus?
I am interested in the use of blood cell transplantation for inherited metabolic and storage diseases, including new therapies to prevent neurologic deterioration during the transplant process. This includes new transplant techniques to decrease the dangers of transplant on the developing brain, and the use of anti-oxidant therapy. These techniques may have important implications to other children undergoing transplantation. A particular interest has been osteopetrosis, an inherited disorder leading to increased density of bone, which is also amenable to treatment with transplantation.
Why are you committed to childhood cancer?
I am fascinated by using blood and marrow transplantation as a means of therapy. There are amazing possibilities including modifying the transplant process to make it more specific to the patient’s particular situation.
How has Children’s Cancer Research Fund made a difference in your work?
The Children’s Cancer Research Fund has been invaluable in allowing our group to develop innovative therapy, and to perform the clinical research required to move the field ahead. However, it is difficult to obtain the funding to provide the necessary data to document whether new therapies are successful. In this regard CCRF has been especially important in allowing us to develop our new approaches.
Logan Spector, Ph.D.
CCRF allows continued exploration into genetic and environmental factors and their relationship to childhood cancer.
What is your current research focus?
My research has focused on the causes of childhood leukemia, hepatoblastoma, and osteosarcoma. In addition, I conduct research that helps improve the methods used to study childhood cancer.
Why are you committed to or why did you choose to focus on childhood cancer?
I was drawn to studying childhood cancer for several reasons. First of all, there are not a lot of people studying it compared to cancer in adults. I thought that needed to change. Also, childhood cancer is an amazing intellectual challenge to me because there’s just not a lot known about what causes it.
How has Children’s Cancer Research Fund made a difference in your work?
CCRF has been incredibly important in funding the preliminary studies that have helped me obtain large, national grants. With support like that you can do anything you put your mind to.
Jakub Tolar, M.D., Ph.D.
I‘m driven in pursuit of a cure for childhood cancer because of the high price we pay when we fail.
What is your current research focus?
In the laboratory, I have focused on stem cell gene therapy for tissue healing, on side effects of stem cell therapy, on novel non-viral gene therapy for correction of immunodeficiencies, and on biology of adult stem cell. In the clinic, our team has described improved conditioning for aplastic anemia and osteopetrosis, novel therapy for adrenoleukodystropy, and we’re focusing on reviewing data on transplant with adult stem cells for metabolic disease.
Why are you committed to or why did you choose to focus on childhood cancer?
I have been driven to childhood cancer by the high price we pay when we fail, and by the intense focus needed to limit the risk of such failure. Despite major advances by the gifted scientists and clinicians who gave us a glimpse of success, there is no reason to believe that the search for a cure of childhood cancer has been completed. The ideal of cure is real, but only on the understanding that it is real with, still unknown, complications.
How has Children’s Cancer Research Fund made a difference in your work?
CCRF has made a difference by supporting the high-quality products of creative impulse, which is aimed solely at improvement in the care of children with cancer. They have provided:
- Seed money which often develops into larger translational grants
- Smaller grants aimed at answering a specific clinically relevant question
- Funds for new equipment needed to advance childhood cancer research
Michael Verneris, M.D., Ph.D.
Only basic and clinical research will provide answers that will ultimately translate to safer and more effective methods of treating childhood cancer.
What is your current research focus?
Currently, I am focused on the following projects:
- The effect of an immune suppressive drug (cyclosporine A) on natural killer cell function. This drug is commonly used during bone marrow transplant and whether it impairs immune recognition of leukemia is hotly debated.
- Generation of NK cell from bone marrow stem cells.
- Characterization of NK cell receptors on cord blood T cells. Such receptors are uniquely expressed on cord blood T cells and may improve their ability to recognize leukemia.
- Investigating the impact of “double UCB transplantation” on acute leukemia relapse. We have found a lower rate of leukemia relapse using this approach.
- The use of bone marrow irradiation (instead of total body irradiation) for pre-transplant therapy. Such an approach has the potential to increase the dose of radiation to sites of disease while sparing non-involved sites. This is expected to increase remissions and reduce side effects.
Why are you committed to or why did you choose to focus on childhood cancer?
It is a privilege to be able to be involved in the care of children with cancer. My choice to pursue pediatric blood and marrow transplantation grew out of a combination of my love of children, their innocence and incredible resilience, and the inherent need to improve the lives of such children. Additionally, it is well established that many of the advances in the treatment of such children have been through both organized clinical and laboratory research. I wanted to be able to contribute to this.
How has Children’s Cancer Research Fund made a difference in your work?
Simply put, CCRF has made it all possible.
- Possible for me to be recruited to UMN
- Possible for me start my current research laboratory
- Possible for me to continue this work
Moreover, with the seed money that I’ve received from CCRF, I’ve been able to successfully compete for national grants. Without this generous support, I would spend more time writing grants and less time concentrating on the actual research and clinical trials.
Margaret MacMillan, M.D.
We’ve made a lot of progress in pediatric oncology research, but kids with cancer still need us to do more.
What is your current research focus?
My current research focus is on the development and implementation of novel therapeutic strategies for preventing the early toxicities of allogeneic hematopoietic stem cell transplantation. In collaboration with Bruce Blazar, M.D., I am studying the safety and efficacy of T regulatory cells to prevent graft-versus-host disease, a major cause of morbidity and mortality after transplantation. I am also the co-director of the University of Minnesota Fanconi Anemia Comprehensive Care Clinic, which follows the largest number of Fanconi anemia patients in the world. We are currently investigating whether we can successfully transplant Fanconi anemia patients using a reduced radiation dose protocol to help prevent toxicities.
Why are you committed to or why did you choose to focus on childhood cancer?
The initial interest was inherent. As a young child when someone asked me what I wanted to do when I was older, I always replied, “Cure cancer”. I’m not really sure where this came from as no one in my family had cancer and no one was in medicine. Nevertheless the goal never abated. I feel fortunate to have the ability to help take care of children with cancer. They are in such need for help. I can’t imagine turning my back on them.
How has Children’s Cancer Research Fund made a difference in your work?
The Children’s Cancer Research Fund allows me to give children with cancer hope for a future. CCRF provides vital funding to initiate novel research. This leads in turn to preliminary data which then can be used to pursue larger national grants.
John Kersey, M.D.
The lessons we’ve learned from successfully treating leukemia are now being applied to breast cancer, lung cancer, colon cancer and more.
What is your current research focus?
My current research is focused on leukemia in infants and children, particularly the leukemias caused by an abnormality of a gene called MLL. We study this abnormality in both human and mouse models.
Why are you committed to childhood cancer?
This form of leukemia in infants and children has a very poor outcome with therapy. I want to change that.
How has Children’s Cancer Research Fund made a difference in your work?
CCRF has provided grants that allow us to obtain larger grants. Children’s Cancer Research Fund allowed us to develop the world’s first mouse model for this rare and deadly form of leukemia.
John Ohlfest, Ph.D.
Once I learned that brain tumors are the leading cause of cancer-associated death in people under the age of 35, it was clear that childhood cancer was the battleground I would bring my war against cancer to.
What is your current research focus?
The focus of our research is harnessing the precision of the immune system to treat brain tumors. We know that brain tumor cells can be killed by the immune response, but in most children this process is inadequate, the immune response needs help. We are developing new approaches to increase the number of tumoricidal immune cells and sustain them for longer periods of time to fight brain tumors. By combining immunotherapy with other therapeutic advances, such as novel small molecule drugs, we are getting progressively closer to achieving our goal of a world free of brain tumors.
Why are you committed to childhood cancer?
I knew from the time I entered graduate school that I wanted to dedicate my career to developing tumor-specific brain cancer therapy. Once I learned that brain tumors are the leading cause of cancer-associated death in people under the age of 35, it was clear that childhood cancer was the battleground I would bring my war against cancer to.
How has Children’s Cancer Research Fund made a difference in your work?
We know that incremental thinking will led to incremental progress. Children's Cancer Research Fund (CCRF) has provided the crucial funding to get our wildest, highest-risk ideas to the proof-of-concept stage. CCRF allows me to think outside of the box, to hit brain cancer as hard as I possibly can every day; and, if I fail, to learn from it and come back smarter the next time.