2006 Grant Recipient - The Sunshine Project, Pediatric Cancer Foundation

The Joanna McAfee Childhood Cancer Foundation announces its grant recipients for 2006. Barb Rebold, executive director of the the Pediatric Cancer Foundation in Tampa, FL was awarded a $2000 grant to continue her work on  ”The Sunshine Project”

 

Pediatric Cancer Foundation

14005A North Dale Mabry Highway

Tampa, FL 33618

(813) 269-0955

(813) 269-8016 (fax)

w w w . p c f u s a . o r g

 

OVERVIEW OF THE RESEARCH PROJECT

Childhood Cancer. It is every parents worst fear, every doctors dreaded diagnosis. Currently, the Pediatric Cancer Foundation is one of a handful of organizations nationwide that have focused their efforts 100% on pediatric cancer research. Many people consider pediatric cancer to be a rare disease. Yet, cancer remains the #1 killing disease of our children. What is rare however, is research focused on finding a cure for these children. According to a 2005 report of the Institute of Medicine and National Research Council of The National Academies, The near absence of research in pediatric cancer drug discovery threatens to halt the progress in childhood cancer treatment achieved during the past four decades. A recent report by the American Cancer Society concurs, stating that research is at a near standstill for childhood cancer drugs. In response to the current state of pediatric cancer research, the Pediatric Cancer Foundation has created The Sunshine Project, a novel collaborative research initiative that not only addresses the issues that are currently prohibiting the development and testing of new drugs for children with cancer, but provides a way to streamline the research process that will help us save precious lives.

The Sunshine Project consists of a coalition of the nations leading pediatric oncologists, pathologists, biologists and medical information technology specialists who will collaborate over a period of three years. Their organizations include the most prestigious medical and research institutions in the United States, including Moffitt Cancer Center, City of Hope Cancer Center, Memorial Sloan Kettering, MD Anderson Cancer Center, All Childrens Hospital, Nemours Jacksonville, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University Medical Center and Vanderbilt University Medical Center. The researchers will work as a single, cooperative virtual team, providing a comprehensive study of the disease from each scientific perspective. They will simultaneously implement the most promising therapeutic trials at an accelerated pace, then fast track these treatments to clinical application. The Sunshine Project will concurrently conduct three phases of research including Basic Science, Translational Research and Clinical Trials, thereby speeding up the process and changing the way pediatric cancer research is conducted in this country. The new research model will be applied to developing treatments that target only specific cancer-causing molecules in the body, unlike more traditional treatments that often prove toxic to healthy cells.

The missions of the Sunshine Project are to develop new and more effective therapies for pediatric cancer and to establish new methods to accelerate drug discovery and treatment.

SIGNIFICANCE

Conservative estimates indicate that, in 2005, 12,000 Americans will be diagnosed with any of the sarcoma types and 5,000 will die of the disease. Furthermore, sarcomas will account for approximately 15 % of pediatric malignancies.

In children and adolescents, osteosarcoma, the most common primary malignant bone tumor, is followed in frequency by the family of Ewings Sarcoma. Sarcomas of the soft tissues constitute 5% of all malignant childhood neoplasms and, of these, rhabdomyosarcoma (RMS) is the most common. 

Sarcomas mortality rate remains disproportionately high compared with that of other cancers common in this age group, such as testicular cancer and Hodgkins disease. Treatment options for patients with advanced sarcomas are limited, and often therapies do not follow a well established standard of care. This results in widely variable outcomes and complicates the assessment of therapeutic efficacy.

In recent years, tremendous progress has been made in our understanding of the molecular basis of cancer, and molecular-targeted therapies, based on these molecular insights, are beginning to have an impact on the bedside for a small subset of neoplasms.

Two major recent developments are expected to accelerate the identification of new potential molecular targets and the application of efficacious molecular-targeted therapies. First are the technological breakthroughs associated with functional genomics, such as gene expression profiling by microarray analysis and proteomics. Second is the increasing realization that a dynamic interaction between the tumor cell and its microenvironment has a profound impact on cancer progression and response to therapy, since cancer cells do not exist in a vacuum within the organism and tumor cell-microenvironment interactions modulate expression of genes that influence tumor progression and drug response.

The Sunshine Project will address directly these important aspects of oncogenesis via the integration of Research Projects, Cores, and hypothesis-driven Clinical Trials. The proposed studies will involve not only mechanistic approaches, through application of conventional cell and molecular biology techniques, but will also incorporate new functional genomics technologies, including microarray gene expression profiling and proteomics. Results of these studies will begin to define the molecular signatures of tumor cells in their natural microenvironment that impact on tumor progression and response to therapy. These insights in turn will be applied toward the development of new therapeutic approaches through collaborative interactions among the Project components (Basic Science, Translational Research and Clinical Trials), facilitated by the Project Cores (Administration, Pathology and Biostatistics/Bioinformatics).

OBJECTIVES

1. To complete novel early-phase clinical trials in children and adolescents (5-25 years of age) diagnosed with metastatic or high-risk bone or soft tissue sarcoma.

2. To identify new molecular targets specific for drug development using sarcoma cell lines and patient tumor biopsies, and to validate these targets in the clinical trials.

3. To perform pre-clinical evaluations of several new classes of anti-tumor agents in human sarcoma cell lines and mouse xenograph models in order to identify candidate drugs suitable for clinical trials in sarcoma patients

LEADERSHIP

Douglas Letson, MD. As the overall leader of the Sunshine Project Dr. Letson will oversee all operations and will maintain continuous relationships with the leadership of the Pediatric Cancer Foundation. He will be assisted by the Co-leader of the Sunshine Project, Dr. Richard Jove.

Richard Jove, PhD. As Co-leader of the Sunshine Project and Leader of the Basic Science component, Dr. Jove will oversee the scientific approaches that constitute the foundation for clinical trials and correlative studies.

Daniel Sullivan, MD. As leader of the Translational Research component of the Sunshine Project, Dr. Sullivan will oversee all clinical trials and all associated regulatory issues, as well as the interactions with Pharmacological companies. He will also supervise all experiments designed to test drugs in vitro. He will be assisted by Chris Cubbitt, PhD and Richard Lush, PhD.

Marilyn Bui, MD. As leader of the Pathology Core and co-leader of the Basic Science component, Dr. Bui will coordinate the projects logistics related to tissue collection and distribution, and will oversee all correlative studies in cells and tissues in support of the Basic Science and Clinical Trials components. She will also interact with the leader of the Biostatistics Core to ensure the proper analysis of data.

Steve Hunger, MD. As Co-leader of the Clinical Trials Component, Dr. Hunger will coordinate clinical efforts with Dr. Sullivan and investigators.

Michael Nieder, MD. As Co-leader of the Clinical Trials Component, Dr. Hunger will coordinate clinical efforts with Dr. Sullivan and investigators.

Tanya Trippett, MD. As leader of the Biostatistics/Bioinformatics Core, Dr. Trippett will ensure the integration and analysis of data obtained in clinical trials and correlative studies.

 

INSTITUTIONAL ENVIRONMENT AND RESOURCES

H. Lee Moffitt Cancer and Research Institute. The H. Lee Cancer Moffitt Center & Research Institute, a not-for-profit institution, is a free-standing NCI-designated Comprehensive Cancer Center located on the campus of University of South Floridas Health Sciences Center. It includes a Hospital with a large inpatient and outpatient capacity, a Research Institute consisting of approximately 150 scientific members, and a wide array of outreach and educational activities for the general public and underserved populations. As part of an elite group of National Cancer Institute (NCI) Comprehensive Cancer Centers, Moffitt focuses on the development of early stage translational research aimed at the rapid translation of scientific discoveries to benefit patient care. It includes private patient rooms, the Southeasts largest Blood and Marrow Transplant Program, outpatient treatment programs that record more than 150, 000 visits a year, the Moffitt Research Center, the Moffitt Clinic at Tampa General Hospital, and the Lifetime Cancer Screening Center. In 2003, Moffitt opened its doors to the new Moffitt Clinic and the Vincent A. Stabile Research Building. These two buildings dramatically increase the existing research and outpatient clinical space to better serve our patients.

All Childrens Hospital. All Children's Hospital is a leading center for pediatric treatment, education and research. All Children's provides specialized care for children of all ages, from newborns through teens. It is one of two freestanding children's hospitals in the state of Florida, and the only one on Florida's west coast. Patients come to All Children's from throughout Florida and from many other states and foreign countries. The Neonatal Intensive Care Unit accommodates 60 premature and at-risk infants. Two pediatric intensive care units provide critical care staffing to children who are acutely ill and patients who are recovering from complex surgeries. Two-thirds of the surgeries performed yearly in our eight operating suites are outpatient procedures. The Short Stay Unit makes it possible for many children to have surgery or diagnostic procedures without the anxiety and expense of an overnight hospitalization.

Nemours Childrens Hospital. Nemours mission is to provide leadership, institutions, and services to restore and improve the health of children through care and programs not readily available, with high standard of quality and distinction regardless of the recipient's financial status. Nemours physicians and surgeons cared for more than 227,000 children with complex or chronic

medical conditions during 2003. Nemours is one of the nation's largest pediatric subspecialty group practices. Employing 445 pediatric subspecialty physicians at sites in Delaware and Florida, Nemours is dedicated to providing treatment and curative services to acutely and chronically ill children. This is in addition to the many ongoing research programs that are also conducted at Nemours. There are a number of research projects underway in areas such as spinal trauma, gait analysis, and bone density. Nemours owns and operates the Alfred I. Du Pont Hospital for Children in Wilmington, Delaware, as well as four major Children's specialty centers in Wilmington, Jacksonville, Orlando, and Pensacola. The Hospital features the nation's largest academic pediatric orthopedic practice, as well as a specialized cancer program for children.

University of Florida  Shands Childrens Hospital. Shands Children's Hospital at the University of Florida is the State's premier children's health center, providing innovative and comprehensive care for children at the highest standards of quality and service, in partnership with their families, healthcare teams and communities. Operational since 1958, Shands admits more than 8,000 pediatric patients annually. In addition, more than 99,000 children are followed as outpatients each year. Shands Children's Hospital has many statewide affiliations, including associations with more than 500 outreach clinics stretching from the Georgia border to Miami. Shands offers one of the most comprehensive pediatric services and outreach programs in the state, including a 24-bed Pediatric Intensive Care Unit, one of seven children's lung centers in the nation and a camp in Florida for children with cancer. As Florida's leading referral center for organ transplantation, Shands sets the pace in the procurement and transplantation of organs of all types. Our programs include heart, liver, kidney, bone marrow, lung, cornea and bone transplant.

City of Hope National Medical Center and Beckman Research Institute. Located northeast of Los Angeles in Duarte, California, City of Hope is a most unusual and remarkable place, where compassion and leading-edge care go hand-in-hand. Here, some of the worlds most knowledgeable physicians, researchers, scientists and health care professionals join forces to treat and to one day cure cancers and other life-threatening diseases. City of Hope is one of just a handful of NCI designated Comprehensive Cancer Centers in the United States, and a founding member of the National Comprehensive Cancer Network (NCCN). It is here that more than 300 physicians and scientists and over 2,500 employees work to find the causes of and cures for cancer. City of Hope has 165 licensed beds, 39 of which are devoted to bone marrow transplantation patients. More than 290,000 volunteers and donors worldwide support City of Hope and one out of every $5 of City of Hopes operating budget comes from private philanthropic support. The Sunshine Project has a very specific mission  to increase the survival rate of children battling cancer as quickly as possible. This project will take place over a span of three years, beginning this December with our first clinical trial. Utilizing the resources of the institutions in our collaboration, we will track every aspect of this project. We will measure the success of this project against the number of new and successful treatment options that are introduced and by the number of lives we save. In developing cures for pediatric cancers, we will be helping the children of today, right here in the Tampa Bay area and across the country, that are in desperate need of new treatment options. We will be helping the children of tomorrow who have yet to be diagnosed, but will be able win their battle against the disease and live full and healthy lives because of this research.