|Making a Mark
Making a Mark is published bi-annually by the CHOC Foundation. It features CHOC and children?s healthcare news, patient highlights, hospital updates, board member spotlights, and community involvement stories and is mailed to donors who support CHOC with a gift of $250 and more.
CHOC Research Institute at the Forefront of Clinical Research
Forty years ago, Acute Lymphoblastic Leukemia (ALL) had a cure rate of only 4%; today, the cure rate is 94%. Thanks to years of progressive research, scientists were able to turn the tide, resulting in more children surviving ALL and other childhood diseases than ever before. CHOC was one of only five institutions in the United States to receive IMMUNE AND INFLAMMATORY DISEASE “ CHOC is one of 15 institutions worldwide selected as a “major affiliate” CANCER Through its Children’s Oncology Group (COG) membership, BIOMEDICAL OPTICS
THE CHOC RESEARCH INSTITUTE
Children’s Hospital of Orange County (CHOC) is committed to both basic science and clinical research in an ongoing effort to unravel the complex underpinnings of diseases that affect children. This commitment keeps CHOC at the leading edge of technology, meaning children seen at CHOC benefit from the latest in scientific thinking with access to the most up-to-date treatment options.
The Institute’s state-of-the art, basic science laboratory centers support six primary areas of research: Molecular Neurobiology; Immune and Inflammatory Disease; Bleeding Disorders; Cancer; the identification, isolation and characterization of non-fetal, federally-approved stem cells; and Biomedical Optics.
MOLECULAR NEUROBIOlOGY AND STEM CELL RESEARCH
Scientists at the CHOC Research Institute have made significant advancements toward understanding stem cells. By studying the role of various cell types in the nervous system, scientists at the Institute are able to identify and isolate stem cells that might someday be cultured and used to replace tissue destroyed by disease or injury.
Experiments primarily focus on understanding the cellular and molecular biology of neural stem cells in an effort to develop new approaches for treatment of diseases like cerebral palsy and multiple sclerosis.
a grant from the National Institutes of Health to teach qualified
scientists from around the world how to culture and use non-fetal,
federally-approved stem cells.
Many devastating childhood diseases are autoimmune—when the body’s immune system turns against itself in diseases like juvenile rheumatoid arthritis, type 1 diabetes and lupus.
Immune and inflammatory disease studies at the CHOC Research Institute focus on understanding cytokines—proteins produced by immune system cells that play a key role in keeping the immune system in balance. Knowing how to control cytokines will someday lead to better treatment for a wide range of chronic and debilitating illnesses.
of the Type 1 Diabetes TrialNet Network, which studies the development,
prevention and early treatment of type 1 diabetes. ”
Experts at the CHOC Research Institute believe that cancer stem cells (CSC) play a key role in the development of many types of cancer, including leukemia and solid tumors of the brain. Understanding the unique characteristics of CSCs will help determine better and more effective treatment for CSC-derived cancers.
Researchers also look at the blood supply and molecular factors that support tumor growth in an attempt to block the development of these cancers. They also study the role of a certain type of blood stem cell isolated from bone marrow or umbilical cord blood in reducing rejection and preventing new disease
CHOC is recognized by the Centers for Disease Control and Prevention and the Maternal and Child Health Bureau as the western regional coordinating center for the treatment of pediatric hemophilia and other bleeding disorders. Experts actively engage in research using the latest scientific approaches to identify the genetic and molecular causes of these diseases.
The CHOC Hemophilia Treatment Center (HTC) is one of the first outpatient programs of its kind in the state to participate in an innovative program designed to give the HTC the ability to maintain and grow the services it provides to patients without relying on funds from the federal government.
CENTER FOR CLINICAL RESEARCH
The Center for Clinical Research, as part of the CHOC Research Institute, supports Phase I through IV clinical trials in nearly all subspecialty clinical areas. This allows physicians to provide up-to-theminute treatment options to CHOC’s patients. This is perhaps most important to children who are failing conventional therapy, or to children with illnesses for which there is no established standard therapy.
CHOC is also the only approved full-member institution without an academic affiliation in the Children’s Oncology Group (COG). COG has approved CHOC as both a Phase I Clinical Trial and Bone Marrow Transplant institution.
The open COG protocols at the Institute offer pediatric cancer patients access to the latest and most current treatment options. The CHOC Research Institute is currently developing advanced molecular diagnostic capability using state-of-theart gas chromatography, tandem mass spectrometry, gene array and proteomic technology.
CHOC received a prestigious Phase 1 clinical trial designation,
making it one of only 21 institutions in North America able to
offer innovative, experimental treatments.
The many and varied properties of light have numerous applications in medicine. Light on a clip attached to the finger allows for an easy way to determine blood-oxygen saturation in almost any clinical setting; and the use of different kinds of lasers is common in many medical specialties. In the Biomedical Optics laboratory at CHOC—its newest translational research center—Ramen spectroscopy and fluorescence imaging are being studied as the basis for the development of real-time, less-invasive technologies for distinguishing between normal and abnormal or diseased tissue. So far, one of the applications developed in this laboratory has shown promising results in non-invasively identifying joint bleeds in kids with bleeding disorders.
Another promising application is the use of this technology for real-time determination of the borders between cancer and normal tissue during surgery. Successful application of this technology would allow surgeons to more precisely determine the borders of cancerous tumors, permitting more exacting removal while preserving normal, healthy tissue.
THE CHOC NURSING RESEARCH FELLOWSHIP PROGRAM
CHOC also has a Nursing Research Fellowship program, which enables nurses to develop research skills and carry out research projects that contribute to the growing local and national body of knowledge.
THE PROMISE OF RESEARCH
The level of research being done at CHOC reflects its commitment to providing leading-edge care for the region’s children. It is also important for attracting and keeping top-level researchers, physicians and nurses. Every time a child’s life is saved by a new treatment at CHOC, many more children can be helped and the importance of research is realized.
Forty years ago, Acute Lymphoblastic Leukemia (ALL) had a cure rate of only 4%; today, the cure rate is 94%. Thanks to years of progressive research, scientists were able to turn the tide, resulting in more children surviving ALL and other childhood diseases than ever before.
CHOC was one of only five institutions in the United States to receive
IMMUNE AND INFLAMMATORY DISEASE
“ CHOC is one of 15 institutions worldwide selected as a “major affiliate”
Through its Children’s Oncology Group (COG) membership,