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Ewings sarcoma, the second most common bone cancer in children and young adults, is very aggressive. Current treatments are not very effective at curing the disease and patients often experience a recurrence of their cancer. Dr. Van Meir is looking for new and more effective treatments for this type of tumor. His laboratory has found a small molecule (KCN1) that they believe may reduce the growth of Ewings sarcoma. KCN1 binds with EWS-FLI1 which is known to stimulate the growth of Ewings sarcoma. Earlier attempts at using a recombinant EWS-FLI1 to produce targeted therapy have been difficult due to the lack of information about the structural makeup of EWS-FLI1. In this project researchers are investigating 1) How the binding occurs between EWS-FLI1 and KCN1, 2) Whether the KCN1 reduces the expression of the EWS-FLI1 gene that causes Ewings and 3) Does KCN1 interfere with Ewings sarcoma development.

Therapy for children with leukemia is difficult, prolonged, toxic and carries long term-side effects. Previous studies clearly show that the immune system can recognize and destroy leukemia. However, this process is inefficient. Dr. Verneris is working to design drugs that bring elements of the immune system in close contact with the leukemia. This has the immediate effect of killing the leukemia and the long-term potential of "training" the immune system to recognize and remember the leukemia. These drugs are now being tested in clinical trials with impressive results and this research will create more effective forms of this therapy.

Children with advanced cancer experience significant suffering. To improve their comfort, Dr. Wolfe aims to understand how distressing symptoms are evaluated and treated, and also to identify attitudes and behaviors in clinicians and families that may interfere with optimal symptom control. To do so, this project is carefully following what happens when a child reports high distress from pain and other common symptoms during clinic visits, as well as interview providers and families, and review charts. The results will help researchers improve symptom control and will be used to design the PediQUEST Champions intervention aimed at easing suffering in children with cancer.

This research is funded by P.A.L.S. Bermuda with funds raised through the St. Baldrick's Foundation.

Dr. O'Bryan is studying the signals that contribute to the development and growth of neuroblastoma tumors. His lab has found a new gene that is important for tumor growth. Although the gene is not useful for targeting treatment, the researchers are studying the ways that this gene works within the cell. With this information, they may be able to identify new drugs that interfere with its function thus interfering with the growth of neuroblastoma cells. This information may be useful in the development of new, more effective treatments for neuroblastoma patients.

Current treatments for kids with brain tumors don't work as well as they should and they have toxic side effects. Often these brain tumors and also other kinds of tumors outside the brain in kids are caused by mutations in a gene called N-Myc. Since researchers don't understand very well how N-Myc causes cancer, they can't fix it. The goal of this project is to test the idea that N-Myc mutations create bad cells called "cancer stem cells" that are like seeds that grow brain tumors. By testing this idea, Dr. Knoepfler and his lab hope to develop new treatments that are better and safer.

Despite major improvement in outcomes for children and older adults with cancer over the past three decades, there has been little or no improvement in survival among adolescent and young adult (AYA) cancer patients (ages 15-39 years). The reasons for this disparity are not completely understood and likely include many factors, including differences in tumor biology, insurance coverage, clinical trial participation and adherence to treatment. This research aims to produce detailed information about the factors affecting AYA cancer incidence and survival that will help doctors target care and close this gap. The project will also produce detailed information for local cancer care service providers on where to target their efforts, and information that will help clinicians recruit AYA patients most in need of help to clinical trials to address their needs.

Having friends is vitally important for all children. Children surviving brain tumors often have physical problems (jerky movements, slurred speech, etc.) and cognitive delays caused by their disease or treatment. These cancer survivors are frequently described by peers as "not well liked," "having few friends" and "isolated". This puts them at risk for being bullied, dropping out of school, becoming anxious or depressed, and being less likely to marry or have good jobs as adults. Dr. Noll is conducting a research-backed, school-based project to support brain tumor survivors' social involvement by training classmates to be more inclusive of others viewed as "different."

Dr. Phipps is studying what happens to children who were treated with bone marrow transplant for cancer many years ago. This is a very challenging treatment that can cause problems for survivors in a number of areas, including their thinking and learning ability, their physical health, and their ability to have children. He is learning how these children and young adults are faring relative to their healthy peers, to identify factors that lead to better or poorer outcomes so that researchers can develop treatments to prevent any negative effects.

Childhood cancer survivors are at risk for developing long term health problems including heart disease. Dr. Ruble's research finds ways to make survivors healthy by participating in physical activity. Currently less than 50 percent of survivors participate in adequate amounts of exercise, which increases the risk of developing health problems. This project aims to find out the best way to support survivors in changing behaviors and being healthy.

Even though more and more children are being cured of cancer, the treatment can cause major life-long health problems for survivors. Unfortunately, most adult survivors don't seek appropriate medical care to monitor and care for these problems. This project is to study whether or not adult survivors referred to adult-focused follow-up care actually engage in such care and what might get in the way of doing so. The results of the study will help medical providers better prepare young adult survivors to engage in adult-focused follow-up care to assure that they stay as healthy as possible.

Cancer therapy for childhood leukemia is often successful, but not without side effects. Pancreatitis, characterized by severe abdominal pain and inability to digest food, is a severe side effect seen in some children but not others who are similarly treated for leukemia. This side effect is sometimes fatal, and very often causes significant delays in continuing with treatment. This research is to learn the reasons why some children are more likely to develop this side effect than others, with the goal of helping all patients avoid it.

About 80% of children and adolescents diagnosed with cancer become long-term survivors. About half of them develop therapy-induced hearing loss that is cumulative and irreversible. Most at risk are patients with brain tumors, neuroblastomas, osteosarcomas, soft tissue sarcomas, retinoblastomas, hepatoblastomas, or germ cell tumors who need to be treated with cisplatin, combinations of cisplatin and carboplatin, radiation to the head and neck, or combinations of platins and radiation. Therapy-induced hearing loss adversely affects speech and language development, reading ability, communication, academic performance and psychosocial development. It frequently causes stress, social isolation, low self-esteem, and low overall quality of life. This project explores the use of moderate to high doses of dietary selenium as means to reduce or prevent cisplatin-induced hearing loss.

Although most children with leukemia can be cured, chemotherapy frequently causes permanent brain dysfunction in survivors. With previous support from St. Baldricks, Dr. Cole's lab identified promising protective interventions, and he will now test them among those children at greatest risk for brain damage. To identify this population researchers are studying whether variation in neurocognitive function among leukemia survivors is related to specific inherited differences in genes related to repair mechanisms or to metabolism of drugs thought to cause cognitive deficits.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Comprehensive Approach to Improve Medication Adherence in Pediatric ALL. For a description of this project, see the consortium grant made to the lead institution: the University of Alabama at Birmingham, Birmingham, AL.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Comprehensive Approach to Improve Medication Adherence in Pediatric ALL. For a description of this project, see the consortium grant made to the lead institution: the University of Alabama at Birmingham, Birmingham, AL.

Rhabdomyosarcoma is the most common pediatric soft tissue sarcoma. The two main subtypes, embryonal and alveolar, are defined by their appearance under the microscope. However, identification of a unique gene mutation in alveolar rhabdomyosarcoma identifies a subgroup with worse prognosis. Future studies may use this gene mutation rather than microscopic appearance to determine risk stratification and treatment, but before instituting this strategy researchers must confirm the role of fusion status in low-risk and metastatic rhabdomyosarcoma. This consortium is examining low-risk and high-risk clinical trials of the COG (Children's Oncology Group) to confirm the importance of fusion status in determining treatment for patients with rhabdomyosarcoma. Awarded at Oregon Health & Science University and transferred to Seattle Children's Hospital.

One of 5 children with acute lymphoblastic leukemia suffers a relapse within 5 years of diagnosis. Also, Hispanic and African American children are more likely to relapse. Research has shown that 44% of the children are non-adherent to oral chemotherapy and that non-adherence is associated with an increased risk of relapse. This consortium has created a comprehensive package of interventions to improve adherence to oral chemotherapy. It is believed that this improvement in adherence will directly translate into an improvement in survival and a higher number of children cured of their cancer. Awarded at the Beckman Research Institute of the City of Hope and transferred to the University of Alabama at Birmingham. Funds administered by the University of Alabama at Birmingham.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Prognostic significance of gene fusion in low or high risk rhabdomyosarcoma. For a description of this project, see the consortium grant made to the lead institution: Seattle Children's Hospital, Seattle, WA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Comprehensive Approach to Improve Medication Adherence in Pediatric ALL. For a description of this project, see the consortium grant made to the lead institution: the University of Alabama at Birmingham, Birmingham, AL.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Prognostic significance of gene fusion in low or high risk rhabdomyosarcoma. For a description of this project, see the consortium grant made to the lead institution: Seattle Children's Hospital, Seattle, WA.