Cancer and Blood Disease Institute

Dramatic advances in cancer research and treatment over the past two decades have made it possible for more children to survive this disease. This reality fuels the lifesaving scientific research and clinical trials conducted at the Cancer and Blood Disease Institute (CBDI) of Children’s Hospital Los Angeles. The Institute has pioneered many of the diagnostic and treatment advances that have improved survival rates.

Our Institute offers access to the latest treatment options being tested in clinical trials through our participation in national and international research consortia, including Children's Oncology Group (COG). Each year, the CBDI enrolls more than 1,612 children in approximately 180 clinical trials and other research studies. The Institute offers early phase trials, which are open to enrollment.

We have one of the largest programs of clinical trials for children with cancer and blood disorders in the U.S. The Institute’s Clinical Trials Program gives our patients access to the most current and promising therapies. We are home to multiple leading international pediatric consortia, including:

• Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL)
• Pediatric Blood and Marrow Transplantation Consortium (PBMTC)
• New Approaches to Neuroblastoma Therapy (NANT)

Cancer research in the CBDI focuses on neural tumors, such as brain, neuroblastoma and retinoblastoma, acute leukemia and sarcomas. Cancer develops within a specific environment of surrounding cells and molecules, called the tumor microenvironment, which is involved in the growth and spread of cancer and the development of resistance to treatment. A major research goal is to better understand the interactions of malignant cells with host cells of the tumor microenvironment, and to then translate this knowledge into clinical applications in areas of diagnosis, risk assessment and therapy.

The Institute’s hematology investigators are conducting pioneering research into the measurement and management of iron overload, the basic pathology of sickle cell disease, and the treatment of childhood bleeding and clotting disorders.

Our cancer research program is also exploring the human genome for answers to today’s deadliest diseases, using information about an individual’s genetic makeup to unravel the biology of his or her tumor. Creation of a personalized cancer screening and treatment program will help determine a person’s risk of developing cancer, match patients with treatments that are most likely to be effective, and predict which patients are at the highest risk for recurrence of the disease. CHLA is the first institution to offer a new whole-gene sequencing panel to patients with retinoblastoma and family members who may also have inherited the gene mutation responsible for the development of this cancer.

The Cancer and Blood Disease Institute is an internationally renowned, innovative hub for research and treatment of pediatric oncological and hematological diseases. The Institute’s director is Alan S. Wayne, MD, who joined CHLA and the University of Southern California (USC) in July 2013. Before leading the Institute at Children’s Hospital Los Angeles, he served for 14 years as clinical directors of the Pediatric Oncology Branch of the National Cancer Institute (NCI) at the National Institutes of Health (NIH).

Children’s Hospital Los Angeles is one of the top pediatric cancer research and teaching programs in the world. The Institute has pioneered many of the diagnostic and treatment advances that have improved survival rates.

We provide care for more than 1,600 new patients each year and see approximately 35,000 outpatient visits annually. Our pediatric Hematology-Oncology division is among the largest of its kind in the western U.S., serving as the leading referral centers in the region.

Many landmarks in childhood cancer and blood diseases have been achieved at CHLA and the CBDI, including:

• A founding member of the nation’s first pediatric cancer clinical trials group
• Identified a class of noncoding RNA, found to be the single most significant predictor of diagnosis or outcome in certain childhood cancers
• First in the western US to provide modern chemotherapy to children with leukemia
• Targeted leukemia cells with antibodies
• Found that fat cells promote leukemia drug resistance
• Established one of the first dedicated pediatric radiation therapy programs in the US
• Developed one of the nation’s first comprehensive psychosocial support programs tackling the consequences of childhood and adolescent cancers and blood diseases
• Reported the first successful noninvasive test to measure tissue iron levels in patients with sickle cell disease and other blood disorders
• Became the first place to offer a new whole-gene sequencing panel to evaluate children with retinoblastoma and their family members who might also have inherited the gene mutation, placing them at high risk of developing this malignant eye cancer
• Combined laser technology and chemotherapy for treatment of retinoblastoma – heralded by clinicians as the most significant therapeutic advance in the treatment of retinoblastoma in 25 years
• Discovered that bone marrow can be removed, purged of cancer cells and then returned to the patient to successfully treat metastic neuroblastoma

Housed at one of the top pediatric research and teaching institutions in the world, the Cancer and Blood Disease Institute has pioneered advances that have led to dramatic improvements in survival rates and quality of life for children with cancer and blood diseases.

Dramatic advances in cancer research and treatment over the past two decades have made it possible for more children to survive a cancer diagnosis. This reality fuels the lifesaving scientific research and clinical trials conducted at the Cancer and Blood Disease Institute. Our goal is to discover, develop and deliver new approaches to curing pediatric cancer and blood diseases.

Treating a child or adolescent who has cancer or a blood disorder is different than treating an adult. We were the first to identify a component of the cells that can be used to predict the diagnosis or outcomes in certain childhood cancers. Each decision must be designed with the child’s development in mind. With 50-plus years of pediatric experience, the Cancer and Blood Disease Institute strives to help each child return to as normal a life as possible, as soon as possible. 

Blood and Marrow Transplantation Advances

• Pioneering reduced toxicity regimens for children with non-malignant disorders that will decrease late effects and improve fertility
• Researching advanced methods of minimal residual disease testing which will allow us to define which children with ALL can go without radiation in their transplant therapy, thus decreasing risks of late effects and second tumors
• Testing methods of selection of donors which will allow for increased chances for a cancer cure in combination with advanced approaches to T-cell depletion that allow minimal risks of graft versus host disease
• First pediatric institutions in the world to offer a comprehensive program using different approaches to chimeric antigen receptor T-cell (CAR-T) therapy aimed at curing patients with highly resistant leukemias

Leukemia Advances

• First in the western US to provide modern chemotherapy to children with leukemia
• Targeted leukemia cells with antibodies
• Found that fat cells promote leukemia drug resistance

Bone and Soft Tissue Advances

• A multidisciplinary approach for treating pediatric sarcomas of the bladder and prostate which yields a much better survival rate and reduces the need to remove these organs, leaving the child anatomically intact
• The first pelvic bone salvage protocols for children with bone tumors, leading to pioneering limb-saving techniques

Neuroblastoma Advances

• Developed a neuroblastoma treatment regimen that has increased survival rates to 65%. This treatment is now used nationwide and includes a high-dose chemotherapy and local irradiation protcol - total body irradiation is not used
• Use of retinoic acid as a post-transplant therapy for neuroblastoma, a treatment that was found to decrease recurrence of disease
• Use of gene therapy for recurring brain tumors
• Developed the CEM (carboplatin, etoposide, melphalan) regimen for myeloablative transplant, the elimination of total body irradiation from the transplant regimen, and using isotretinoin as maintenance therapy after transplant
• Discovered that bone marrow can be removed, purged of cancer cells and then returned to the patient to successfully treat neuroblastoma, which is brain and nerve cell cancer

Retinoblastoma Advances

• Combining laser technology and chemotherapy for treatment of retinoblastoma, significantly reducing the need for enucleation. Clinicians have heralded this as the most significant therapeutic advance in the treatment of retinoblastoma in 25 years.

Radiation Oncology Advances

• One of the few pediatric radiation oncology programs in the nation that is focused exclusively on children
• First institutes worldwide to use intensity-modulated radiation therapy, which concentrates the radiation dose on the tumor and directs it away from sensitive nearby tissues

Support Programs

We were the first institution to develop a comprehensive psychosocial support program specifically geared towards the consequences of childhood and adolescent cancers and blood diseases.

The Cancer and Blood Disease Institute is educating the next generation of pediatric oncologists, radiation oncologists and hematologists.

Fellowships

We offer a Pediatric Hematology-Oncology Fellowship approved by the Accreditation Council for Graduate Medical Education and postgraduate subspecialty fellowships in blood and marrow transplantation, coagulation, neuro-oncology and adolescent and young adult oncology. Programs for general and subspecialty pediatric residents, radiation oncology residents, medical students and behavioral science trainees (neuropsychology, psychology and social work) are also offered.

Research Training

We provide research opportunities in hematology and oncology for postdoctoral fellows. A fellowship training grant from the California Institute for Regenerative Medicine supports post-doctoral scholars exploring therapeutic uses of stem cells.

Summer Research Program

For more than 40 years, our Summer Oncology Research Fellowship Program – sponsored by Children's Hospital and the USC Keck School of Medicine – has attracted top students from universities and medical schools nationwide. Our NIH-supported Summer Oncology Research Fellowship Program for US medical students is a full-time 10-12 week program in the summer(s) between school years in medical school. It is designed for students who are considering a biomedical career that involves cancer research. Students participate in clinical or laboratory research studies. This research fellowship takes place at two sites: Children’s Hospital Los Angeles (CHLA) and the Norris Comprehensive Cancer Center and Keck School of Medicine of USC.

International

The Institute mentors trainees from around the world, including Poland, Russia, China, Italy and Brazil, who then take their knowledge back to their countries to help treat and cure children. We have cooperative training and research programs with major pediatric medical institutions in China, Russia, Brazil, Mongolia, Italy and Poland. Our faculty routinely lecture in other countries, and we welcome medical observers from abroad.

The Cancer and Blood Disease Institute is supported by a range of hospital diagnostic and support services.

Blood Donor Center

The hospital's Blood Donor Center collects blood and platelet donations at the hospital and in community blood drives to help children who need these vital blood components. Transfusion medicine specialists prepare blood for use, test for compatibility and manage the platelet, red cell and plasma inventory. Each year, caring for children hospital-wide requires 20,000 units of blood or blood components. 

Cancer Support Resources

The HOPE Behavioral Health, Neuropsychology and Education Service provides compassionate psychosocial care and support for patients diagnosed and treated in our Cancer and Blood Disease Institute. 

Dentistry

The Division of Dentistry and Orthodontics is aligned with the acclaimed USC School of Dentistry to provide routine and specialized dental and orthodontic care to 3,500 children and adolescents each year, including those with medically complicating conditions such as cancer and hemophilia. Many of our patients are unable to see a general pediatric dentist in the community. The Division’s expert staff rounds out our program of care.

Pain Management

When necessary, we call upon the expert services of the Comfort, Pain Management and Palliative Care Program. It provides round-the-clock consultation on appropriate pain management therapies and the use of such complimentary medicine modalities as massage, pet therapy and other wellness techniques.

Pathology

The Department of Pathology and Laboratory Medicine reinforces our capabilities for accurate and timely diagnoses. A team of clinical and anatomical pathologists, led by an expert in cancer cell biology, is specially trained to test for illness and identify disease or abnormal growths in tissues. Among their advanced diagnostic techniques is molecular genetics, which studies the structure and function of genes at a molecular level.

Pharmacy

Clinical pharmacists located within our clinic on the 5th floor assist with formulations for drug treatments, using protocols for best practice cancer care. Pharmacists optimize the protocol based on each child’s height, weight and kidney function.

Surgical Expertise

When surgery is an appropriate treatment option, patients are seen by the expert surgeons in the Department of Surgery. This busy, accomplished department performs some 14,000 pediatric surgeries a year, nearly twice that of its peers. Surgeons are highly experienced in treating children of all ages – from infants and school-aged children to young adults – and in intervening for complicated cancers.

Oncologists and surgeons meet in weekly sessions to ensure that any surgical plan is integrated into a patient’s overall plan of care. Surgeons from different disciplines become involved in oncology cases and routinely exchange ideas on treatment approaches. These include the latest in minimally invasive techniques, one focus of research and innovation for this accomplished Department.

Hospital surgeons are members of oncology study groups in the nationwide Children’s Oncology Group (COG), a source for new treatment protocols – knowledge they bring to benefit our young patients.

Third-Party Support

Ronald McDonald House Charities®

Many of our patients’ families stay at the Los Angeles Ronald McDonald House™ while their children receive treatment at Children's Hospital Los Angeles. Programs of Ronald McDonald House Charities® of Southern California also include Camp Ronald McDonald for Good Times, which provides free, medically supervised, residential camp sessions.

Padres Contra El Cáncer

Founded in 1985, the nonprofit Padres Contra El Cáncer provides primarily Spanish-speaking families with the resources to combat cancer – the only Latino program of its kind in the U.S. Its services include counseling, transportation, social services assistance and emotional support in a linguistically and culturally effective manner.

Members of major clinical trials consortia:

Children’s Oncology Group (COG)

COG Phase 1 Institution

Pediatric Brain Tumor Consortium (PBTC)

Pacific Pediatric Neuro-Oncology Consortium (PNOC)

Pediatric Cancer Immunotherapy Trials Network

Collaborative Ependymoma Research Network (CERN)

Neurofibromatosis Consortium

Sarcoma Alliance for Research Through Collaboration (SARC)

Sunshine Pediatric Cancer Foundation

SunCoast Community Clinical Oncology Program

Primary Immune Deficiency Treatment Consortium

Center for International Blood and Marrow Transplantation Research (CIBMTR)

Childhood Cancer Survivor Study (CCSS)

North American Pediatric Aplastic Anemia Consortium

Mount Sinai Acute GVHD International Consortium

Federally Designated Hemophilia Treatment Center

American Thrombosis and Hemostasis Network