March 10, 2002
Pediatric oncology: Enhancing the lives of cancer's youngest
patients
Adams Fenelon, 4, cruises down the hall as his nurse, Amy Gage, RN, looks on.
When Sidney Farber, MD, envisioned a Boston-based pediatric cancer institute in the 1940s, childhood leukemia was uniformly fatal. From the moment of diagnosis, all that families could hope for was minimal suffering in the remaining months of a child's life.
Today, more than 80 percent of children treated at Dana-Farber Cancer Institute for acute lymphoblastic leukemia (ALL) — the most common childhood cancer — are cured. This dramatic change stems from various forms of chemotherapy, pioneered by Farber and refined by subsequent generations of physician-scientists. At the same time, the quality of life for pediatric patients has markedly improved, thanks to advances in technology and a greater understanding of the challenges faced by cancer patients and their families.
Since its formation in 1974, the Pediatric Oncology Department at DFCI has been at the forefront of numerous innovations through its outstanding research, clinical, and training programs. Its roughly 250 physicians, nurses, fellows, psychologists, technicians, and other staff treat and investigate all forms of pediatric cancer — including leukemias, brain tumors, and other solid tumors — while probing the biological mechanisms behind them.
"We're as poised to take basic research findings and translate them into new concepts and new therapies as any place in the world," states Department Chair Stuart Orkin, MD. "Our fellowship program has trained many leaders in the field, and patients seen in our Jimmy Fund Clinic receive superb, compassionate care from day one."
Patient sibling Ian MacLean gives Patricia Dwyer, LICSW, a "checkup."
Pediatric cancer is rare. Of the nearly 1.3 million new cancer diagnoses likely to occur in the United States during 2002, only 9,100 are expected in children 14 and younger, according to the American Cancer Society. These figures are a mixed blessing for caregivers.
"One of the greatest things about being a pediatric oncologist is that there are not that many children with cancer," says Holcombe Grier, MD, associate chief of pediatric clinical oncology at DFCI. "However, it's much harder to conduct a clinical trial because you don't have as many human subjects. Our ability to answer scientific questions is severely curtailed."
Despite its small size compared to adult oncology, however, pediatric oncology plays a large role in the overall field of cancer medicine.
"Our discipline is critical because so many of the breakthroughs in cancer research have had their origins in pediatric oncology," observes David E. Fisher, MD, PhD, whose work at Dana-Farber on pigment cells appears to shed light on melanoma and bone metabolism in both children and adults. Another example of shared knowledge is Alan D'Andrea, MD's, exploration of the rare childhood condition Fanconi anemia, which has yielded insights into breast cancer — a major cause of cancer death among women.
"On the therapy side, pediatrics is leading the way as well," Fisher adds, "because of what we learn from successfully treating tumors. Although children are generally more resilient than adults and their diseases are different, many of the treatments given to adults are built upon protocols first found to work in children."
Sidney Farber, MD, with a patient
Dr. Farber's dream
Such success can be traced to the vision of Dr. Farber, who developed the Children's Cancer Research Foundation more than a half century ago. (Now known as Dana-Farber Cancer Institute, the foundation began treating adults as well in 1969.) Farber believed young patients would benefit if all their caregivers worked in one clinic. "Things had always been balkanized in medicine, with specialists in different places," recalls DFCI President Emeritus David G. Nathan, MD. "Sidney brought them all together."
This was accomplished with the completion of the Jimmy Fund Building, which this year celebrates its 50th anniversary. "It was Sidney's hope," explains Nathan, "that up on the fifth floor of the building, somebody would yell, 'Aha!' and rush down and give medicine to a child and cure him or her."
Farber also successfully advocated for an inpatient oncology unit next door at Children's Hospital Boston, where youngsters now receive multi-day chemotherapy, surgery, bone marrow transplants, and other services requiring overnight stays. After several decades of informal partnership, the two institutions cemented their relationship in 2000 with the creation of Dana-Farber/Children's Hospital Cancer Care, a joint program in pediatric oncology and hematology.
"Many steps have been taken to create as much cohesion as possible, including streamlining the admission process and creating a computerized system for ordering chemotherapy that is used by both hospitals," affirms Patricia Branowicki, MS, RN, who oversees pediatric oncology nursing at Children's and Dana-Farber. "We are committed to creating a seamless system for our patients and families."
A young patient meets with his care provider.
Enhancing quality of life
Many of the therapies pioneered during the 1950s and early 1960s are still being used today, with refinements made through clinical trials. However, much of the care given to young DFCI patients has changed in recent years. This is thanks to specialized treatment programs such as the Stop & Shop Family Pediatric Brain Tumor Clinic, expanded support services, and more focus on quality-of-life issues.
Anesthetic cream to numb the skin, intravenous "ports" that allow medications to be given and blood to be drawn more easily, and anti-vomiting drugs have helped make procedures more tolerable. Additional social workers and other support staff, along with two pediatric resource rooms filled with books, videos, and other sources of cancer information, have also enhanced care.
Among efforts to improve the cancer experience are the Back to School Program, which aims to educate patients' schoolmates and teachers about the illness, and the David B. Perini, Jr. Quality of Life Clinic, which helps survivors of childhood cancers deal with the long-term effects of radiation, chemotherapy, and other regimens.
"Treatment doesn't end when a child's cancer is gone," notes Lisa Diller, MD, medical director of the Perini Clinic, which currently follows about 650 patients of all ages. "The child cured of a brain tumor first becomes an adolescent concerned about school performance because of therapies that might affect memory skills, then becomes an adult wondering if infertility will result from the cancer treatment received years earlier. It is a lifelong process."
Rosalind Segal, MD, PhD, and David Fisher, MD, PhD, benefit from collaboration.
Leader in research
The Perini Clinic also conducts research to support current and future cancer patients, including studies on the benefits of genetic counseling and the prevention of late complications for Hodgkin's lymphoma survivors. Its work represents some of the many studies under way in the Pediatric Oncology Department that are funded by federal grants, gifts to the Institute, and other sources.
Among the advances making recent headlines is the translational research of Scott Armstrong, MD, PhD, and his colleagues, who announced in December they had used gene profiling to identify a unique type of leukemia that strikes infants. They hope the finding will lead to treatments targeting the deadly disease.
Another example of such translational research — which applies discoveries from the laboratory and clinic to one another — is the work of pediatric oncologist Eva Guinan, MD, director of DFCI's Stem Cell Transplant Program, whose team found a way to reduce the chance of graft-versus-host disease in some bone marrow transplants.
The department is also at the forefront of clinical research, which tests new therapies in patients through trials, and of basic research that expands knowledge about nature. To remain current, many of the physician-scientists collaborate with colleagues in their department, around the Institute, and at other Harvard-affiliated institutions.
One of the multiple hats donned by the MDs and PhDs in Pediatric Oncology is that of teacher, and the department's fellowship program, run jointly with Children's Hospital Boston, is considered one of the most sought-after in the country.
"We have trained leaders in the field for the last 30 years," states Chief of Staff Stephen E. Sallan, MD, a graduate of the program that boasts alumni heading pediatric oncology departments at Stanford University, Johns Hopkins, the University of Chicago, and other prestigious institutions.
Contributing to the program's strength is that graduate fellows hone their clinical and interpersonal skills by caring for patients throughout their three years of training. Each fellow also has a clinical mentor and a panel to help review his or her laboratory progress, and is encouraged to seek further training beyond the Institute's walls.
Samuel E. Lux IV, MD, who co-heads the fellowship program, and department Chair Stuart Orkin "know researchers throughout the Boston area and can help our fellows get a breadth of experience in other laboratories," notes Grier. "That's something you can't find in many other places."
Brittany Hardiman, BSN, RN, CPON, confers with Patricia Branowicki, MS, RN
Challenges and rewards
The pediatric fellows represent the next generation of pathbreakers in the lab, classroom, and clinic at DFCI and beyond. One of the department's major goals is to make cancer therapy more effective and less toxic. "We'd like our treatments to be more specific, using drugs that kill only cancer cells," says Sallan. "We would like treatments to be freer of unpleasant side effects like hair loss and nausea. We've made huge strides in end-of-life care, and symptom management — but we can always do better."
That "do better" mantra seems to resonate through the department and Jimmy Fund Clinic, where staff members often are asked how they handle the sometimes-heartbreaking work.
When Fisher has to deliver sad news to families, he focuses as much as possible on tangible, practical information-sharing. "In my experience, there's nothing more scary than our imaginations," he reflects. "So being able to convey information can be very comforting. I try to help families in some measurable way; that's why I'm a doctor.
"Pediatric oncologists are self-selecting," he continues. "We don't have good work hours, and it's not a glamorous occupation. Yet the challenges in this field are greater than almost any I could ever imagine. So are the rewards."
(Paths of Progress, Winter/Spring 2002)
