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JUST SUPPOSE THE SIDNEY KIMMEL COMPREHENSIVE CANCER CENTER AT JOHNS HOPKINS THE STORY IS TOLD OF A LITTLE BOY WHO FINDS THOUSANDS OF STARFISH WASHED UP ON THE BEACH AFTER A BIG STORM. HOW COULD HE SAVE THEM ALL? AFTER ALL, WHAT DIFFERENCE COULD ONE PERSON POSSIBLY MAKE? HE REACHED DOWN AND THREW ONE FISH BACK IN, THEN ANOTHER... “WELL, I MADE A DIFFERENCE TO THIS ONE, AND THIS ONE...” JUST SUPPOSE OUR COMMUNITY SAW CHILDHOOD CANCERS THE SAME WAY. IMAGINE THE KIND OF DIFFERENCE WE COULD MAKE. PLEASE JOIN US IN CHANGE HOW THESE ARE TARGETED AND ONE CHILD AT A HELPING CANCERS TREATED TIME. WHY JOHNS HOPKINS? “WE’RE THE ONES LEADING THE WAY —FINDING THE GENES, STUDYING WHAT THEY DO IN TERMS OF THE BIOLOGY OF A PARTICULAR PEDIATRIC CANCER, AND DEVELOPING MOLECULARLY TARGETED THERAPY AGAINST IT.” Donald Small, M.D, Ph.D. Kyle Haydock Professor and Director of Pediatric Oncology The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Cancer strikes more than 12,000 children each year, claiming the lives of more children than any other disease. But there is good news. We understand the urgency of a search for the cure. Every child we treat represents a future filled with possibility and promise. At Johns Hopkins, we are pioneers in all aspects of pediatric cancer research and clinical care: • For 21 consecutive years, the Johns Hopkins Hospital has been ranked #1 in U.S. News and World Report. • We are recognized leaders in the research and care of all pediatric cancers, including leukemia, lymphoma, brain cancer, sarcomas, and Wilms tumor. • Johns Hopkins Pediatric Oncology is a national referral center for stem cell and bone marrow transplantation. Our patients have access to the latest treatments offered through cutting-edge clinical trials. The Johns Hopkins Pediatric Survival Clinic is a national model for care. Our team of dedicated researchers and clinicians: • train the next generation of pediatric oncology clinicians and scientists in a joint fellowship program with the National Cancer Institute. • are leaders in translational medicine, turning laboratory breakthroughs into new treatments. • are on the forefront of developing targeted therapies, the next generation of cancer treatment. Our scientists deciphered the genetic code of medulloblastoma, one of the most common types of pediatric brain cancer. A Johns Hopkins researcher cloned the FLT3 gene, responsible for many treatment-resistant leukemias, and identified new drugs to block its effect. These kinds of advances—based on decades of research—are what set Johns Hopkins Pediatric Oncology apart and bring real hope to our patients and families. WHY NOW? A GENERATION AGO, FEW CHILDREN SURVIVED A DIAGNOSIS OF CANCER. TODAY, THE OPPOSITE IS TRUE—MOST CHILDREN HAVE A GOOD CHANCE FOR SURVIVAL. THIS IS THE KIND OF CHANGE THAT REFLECTS THE POWER OF RESEARCH, AND IT IS THE HALLMARK OF JOHNS HOPKINS. Now, we have the opportunity to make even greater progress. For the first time in the history of medicine, scientists are discovering how mutations in DNA cause cancer. With this new knowledge, we can develop molecularly targeted therapies that treat the cancer without harming the child. It’s an exciting time for pediatric oncology researchers and clinicians at Johns Hopkins. Deadly cancers like chronic myeloid leukemia can now be controlled with daily medicines that have virtually no side effects. Each advance leads us closer to the future we envision—one where cancer no longer threatens the life of any child. Just suppose we were able to expand our research effort, accelerating laboratory breakthroughs, creating new therapies against cancer even more quickly, and conducting clinical trials to learn how to use them best. Support for Johns Hopkins Pediatric Oncology does more than help the children we see and treat in our hospital today. It helps our scientists and clinicians develop the treatments that will benefit children everywhere, long into the future. We are the home to 21st century pediatric cancer medicine. The Charlotte R. Bloomberg Children’s Center is the home of Johns Hopkins Pediatric Oncology. This new building offers the latest, most technologically advanced treatments in a welcoming, family friendly setting, providing a hospital experience only equaled by the excellence of Johns Hopkins science and medicine. • The 11th floor of the Bloomberg Children’s Center is dedicated exclusively to the treatment of children with cancer. • We have 20 private inpatient rooms, a playroom for children, and a separate lounge for teens. Our goal is to create a $30 million research fund to facilitate monumental leaps in knowledge and new cures for children everywhere. Leukemias, sarcomas, lymphomas, brain tumors and other forms of pediatric cancer affect thousands of children each year. All too often, however, research funds neglect childhood cancers to focus on more common adult diseases. At Johns Hopkins Pediatric Oncology, we understand that even one child suffering from cancer is one too many. Our research efforts are directed entirely toward the cure of childhood cancers. • Amenities for families include a sleeper sofa in each room, lounges, showers, laundry facilities, and 24-hour food service. • Our outpatient unit provides private exam rooms and infusion areas. • We have an on-floor pharmacy serving only pediatric oncology patients. Help us turn science into survival. Support the research that is making a difference in pediatric cancer. JUST SUPPOSE ... YOU COULD HELP A CHILD WITH CANCER. YOU CAN. “I HAVE HOPE THAT THE DOCTORS WILL FIND A CURE FOR CANCER, AND EVERYONE WILL BE HEALTHY AND WELL IN THE FUTURE.” ZOE, 13 LEUKEMIA SURVIVOR FUTURE RADIOLOGIST JUST SUPPOSE WE COULD MAKE ZOE’S HOPE A REALITY. WE CAN. JUST SUPPOSE hopkinsmedicine.org/kidscancer Z O E ’ S S TO RY ZOE BOONE’S NATURAL BEAUTY , easy smile and confident manner make her one of those rare 14-yearolds who somehow escaped the awkwardness of adolescence. What she hasn’t escaped, however, is an incredibly challenging medical history, one that would have left most adolescents defeated. Not Zoe. It’s probably no mistake that the eighth grader’s favorite position to play on her school’s soccer team is defender. She’s had a lot of practice. Zoe’s problems began in the womb, when her mother developed breast cancer. She began treatment before Zoe was born, which bought her some time— she passed away six years later. But Zoe’s exposure in utero predisposed her to developing cancer. When Zoe was 9, she was diagnosed with a rare form of acute myeloid leukemia (AML). Fortunately her sister Maxine proved to be a perfect match for a bone marrow transplant. After the transplant came a period marked by nausea, weakness, and worries about infections attacking Zoe’s immune-suppressed body. When Zoe finally returned to school the following year, she initially had to wear a mask to reduce the chance of infections. At one point, she required a feeding tube— tough stuff for a 10-year-old. Zoe hasn’t let cancer curtail her dreams for the future. Instead, she says, the experience has given her inspiration. She’d like to become a radiologist. C H I LD H O O D CAN C E R LEUKEMIA Leukemia is among the most common of the pediatric cancers, and Kimmel Cancer Center researchers have been on the forefront of discoveries that have dramatically altered its course. More than three decades ago, our researchers introduced refinements in therapy for acute lymphocytic leukemia (ALL), the most common type of childhood leukemia, which resulted in a remarkable increase in cure rates, and these advances continue. identified and cloned the FLT3 gene and linked it to a treatment resistant type of acute myeloid leukemia (AML). Based on his work, our scientists have developed drugs that block the gene and make the cancer more responsive to chemotherapy. The discovery is now also providing a desperately needed new clinical approach for infant ALL, which currently has the lowest survival rate of all the childhood leukemias. DR. DONALD SMALL For many patients, bone marrow transplant (BMT) offers the best chance of a cure. An innovative new approach, called halploidentical (half-identical) BMT is making this therapy available to many more patients. The treatment is only available at Johns Hopkins. In a recent groundbreaking discovery, a pediatric oncology researcher transformed a blood stem cell into a beating heart cell. He believes the plasticity of the blood stem cell provides important new clues about how leukemia originates and how it can be controlled. L U C A’ S S TO R Y LUCA ASSANTE is the quintessential 3-year-old boy. Whether he’s pretending to be Superman or ‘helping’ at his grandfather’s Italian restaurant and deli, Luca is always on the go. Not even rhabdomyosarcoma—a fast-growing cancer of the muscles—could slow him down. “He’d be at the hospital for treatment and running around the hospital halls,” recalls Lucia Assante, Luca’s mom. Despite spending half of his young life in treatment for his cancer, Luca was blissfully unaware of the severity of his illness. The sudden diagnosis and related treatments, however, left his family reeling. His parents, who thought Luca’s discomfort stemmed from a bad case of diaper rash, were stunned to learn he had cancer. Initially, even aggressive chemotherapy did not stop Luca’s stubborn tumor from growing. Ultimately, the perseverance of his Hopkins team won out. The tumor was now small enough to be taken out with surgery. Today, Luca remains cancer-free. He is happy to be back in the routine of attending preschool and spending time with his two sisters—and, of course, helping out at his grandfather’s restaurant. The Assante family credits Johns Hopkins with making their ordeal bearable. “You couldn’t ask for a better group of people to care for your child. In a horrible situation, it was the best experience,” says Luca’s mom. “They gave us hope.” C H I LD H O O D CAN C E R P E D I AT R I C S A R C O M A S Treatment for pediatric sarcomas has not changed much since the 1980s. Our experts believe that future advances will come only from a deeper understanding of the biology of these tumors, and the translation of this understanding into clinical trials. Our researchers are driving this progress. As the leaders in deciphering the genetic code of cancer, Johns Hopkins researchers are bringing 21st century-science to the fight against sarcoma. They are working to understand the molecular biology of the cancer and the genetic differences that make some tumors more responsive to treatment than others. Another exciting new area of research is in cancer stem cells. These cells represent a rare and stealth population of cells that often escape the assault of anticancer drugs and drive the growth of tumors. Like the typical stem cell that helps form and regenerate tissue and cells, cancer stem cells are capable of limitless growth, self-renewal, and the generation of new tumors. Drugs that target cancer stem cells are currently being studied in Ewing’s sarcoma and rhabdomyosarcoma. With promising early laboratory data, the team is working to move quickly to clinical trials. N I C K ’ S S TO RY NICK SELTZER’S MOTHER describes her son as ‘an old soul.’ Nick was an only child who enjoyed the company of adults, had a knack for golf, and possessed a hearty sense of humor. When he was just 8 years old, he received a shocking diagnosis of brain stem glioma, a relentless type of childhood brain tumor. Currently, there is no cure for this cancer, but Johns Hopkins pediatric oncologists, who specialize in all types of brain tumors, are working towards one. The brain stem connects the brain to the spinal cord. It is the command center of the brain. All information going to and from the body passes through the brain stem. Experts at Johns Hopkins likened Nick’s cancer to ivy growing around the trunk of a tree. With a cancer so entwined in his brain stem, surgery is dangerous and of little benefit. Current anticancer drugs do little to fight it. Radiation therapy temporarily staves off the inevitable, but eventually the tumor continues to grow. Nick passed away on Father’s Day, weeks shy of his tenth birthday. His mother marvels at how, even in the later stage of his illness, Nick stayed positive and held onto his sense of humor. She recalls one evening, when complications from Nick’s illness rendered him unable to speak. Friends were visiting, and Nick was trying desperately to relay something to them. “He was laughing hysterically because we couldn’t figure out what he was saying,” his mother recalls. “Up until the end, he was happy.” C H I LD H O O D CAN C E R BRAIN STEM GLIOMAS There is no question that brain stem gliomas are the most challenging of all the pediatric cancers, but they may have met their match in Ken Cohen. He is a hero among pediatric oncologists, one of the few willing to dedicate his career to thwarting these and other pediatric brain cancers. “It is unbelievably difficult to look at a mother and tell her there is nothing you can for do for her child,” he says. “I don’t accept that.” Working with basic scientists at the Kimmel Cancer Center, he has developed a novel twodrug approach that targets and shuts down a gene pathway involved in the growth of these tumors. The new therapy, pioneered at Johns Hopkins, is now being used to help children with brain stem gliomas and high-grade astrocytomas, another deadly form of brain cancer. Cohen sees hope on the horizon. “This is a new era in cancer research where, for the first time, I believe we have the opportunity to offer the chance of a cure to patients who before could not be cured,” says Cohen. “We can conquer these cancers. We will conquer these cancers.” L AU R E N ’ S S TO RY received the devastating news that she had a golf-ball sized cancerous tumor in her brain. The pediatrician who found Lauren’s tumor gave her mother some crucial advice. She said, “Take her, right now, to Johns Hopkins.” Combined treatments of surgery, chemotherapy, and radiation therapy cured Lauren. Six years ago, when she learned her diagnosis, Lauren recalls wondering, “Am I going to die?” Today she is a college student studying public relations and American Sign Language. She is a voice for all children with cancer, testifying before Congress with the Children’s Cause of Cancer Advocacy and calling for increased funding for basic research, drug discovery, and ways to study and manage the long-lasting side effects of treatment. AT 13, LAUREN MURPHY FAST FACT: In 2010, Kimmel researchers became the first to decipher the genetic blueprint of a pediatric cancer when they mapped the genetic sequence of medulloblatoma. C A R L A’ S S TO R Y 15-year-old, when a fall down the steps revealed that she had osteosarcoma, bone cancer, in her leg. Her doctor sent her straight to Johns Hopkins Hospital. Carla underwent multiple rounds of chemotherapy, but ultimately her leg had to be amputated just above the knee. She faced a lengthy physical and emotional recovery in which she had to come to terms with losing her leg and learning how to walk with a prosthetic. Now, most people don’t even notice that she has a prosthetic leg. Just five years later, she has graduated from the Community College of Baltimore County and plans to attend the University of Maryland at Baltimore County to pursue a career in veterinary medicine. When I look forward in life,” Carla says, “I see myself as a person who can take chances, who can become a leader, who can understand what people are going through, because I’ve been through a difficult situation. I’ll never forget what happened, but I won’t let it affect the person I’ve become.” CARLA WAS AN ACTIVE