For decades, UCSF researchers have been discovering solutions for childhood and adolescent health problems, both common and rare. A long list of internationally renowned basic scientists and clinical researchers have effectively translated science and influenced the way care is delivered for afflicted children around the world.

Pediatric Asthma

To decrease the incidence of, and lessen the effect on, the more than 10 million children in the United States who suffer from asthma, UCSF researchers attack the disease from multiple angles.

Robert H. Lustig, MD, left, director of the Weight Assessment for Teen and Child Health Program at UCSF, is a leading authority on sugar and obesity.

For example, a UCSF pediatrics team based at the UCSF-affiliated San Francisco General Hospital (SFGH) is engaged in the YES WE CAN Urban Asthma Partnership, a research collaboration that has led to individualized plans that help children control their asthma more successfully than most areas of the country.

Among other ongoing asthma studies, Michael Cabana, MD, MPH, chief of the Division of General Pediatrics, is evaluating whether infants who receive probiotic supplements — live microorganisms like the bacteria found in yogurt — are less likely to develop asthma than those receiving a placebo.

“It is possible that stimulating the immune system at an early age may help prevent allergic diseases,” says Cabana. “Today’s babies are exposed less often to various types of infections at an early age, and this may be one reason we’re seeing more asthma today.”

Children and Obesity

Obesity is another illness that affects millions of US children and adolescents, and is a factor in chronic conditions — including heart disease, diabetes and depression — that can haunt people throughout their lives. Because obesity can arise from medical, psychological and social factors, the UCSF Weight Assessment for Teen and Child Health (WATCH) Clinic uses teams comprising several types of specialists to treat the condition and to conduct research exploring causes and treatments.

“We’ve learned a great deal about the links between biochemistry and behavior and their effect on obesity,” explains WATCH Clinic Director Robert Lustig, MD, whose work on the role of fructose in weight gain has garnered national attention.

Lustig is also part of the UCSF Center for Obesity Assessment, Study and Treatment, known as COAST, believed to be the first in the country to focus on the impact that stress has on eating and weight gain. In addition to other projects, he is doing leading research on the biochemical factors that contribute to obesity in children who have undergone treatment for a brain tumor.

Other UCSF projects addressing childhood obesity in the community include research by Kristine Madsen, MD, MPH, an assistant adjunct professor of pediatrics, who is conducting a National Institutes of Health-funded evaluation of an after-school soccer program to improve childhood fitness and decrease obesity rates. 

Anisha Patel, MD, assistant adjunct professor of pediatrics, is looking at early childhood eating habits by working with the Bay Area community to study how changing the beverages offered at day care centers can affect future nutritional habits in kids and families and improve community obesity rates.

Cancer Care

UCSF’s Kate Matthay, MD — one of the world’s leading childhood cancer researchers — was the first to demonstrate the importance of high-dose therapy supported by bone marrow transplant in the treatment of high-risk neuroblastoma, an aggressive tumor that begins in an infant’s sympathetic nervous system. This approach is now the standard of care throughout the world. She also leads a consortium of 16 institutions to deliver new, targeted medications to patients with advanced cancer.

Matthay, a professor in pediatrics, recently completed an eight-year clinical trial that found intermediate-risk neuroblastoma can be fought successfully with far less chemotherapy than previously believed, thus avoiding harmful side effects caused by cancer drugs. One of the key factors in determining safe use of less chemotherapy for a patient was gaining an understanding of the individual’s genetic makeup.

Kate Matthay, MD, a professor of pediatrics, is one of the world’s leading researchers in new treatments for neuroblastoma, the most common extracranial solid cancer of infancy and early childhood. 

Genetic understanding is also the basis of groundbreaking UCSF work on childhood leukemia. Pediatric cancer specialist Mignon Loh, MD, recently discovered mutations in a gene that can be inherited in families and can predispose children to a specific type of deadly leukemia.

Kevin Shannon, MD, Roma and Marvin Auerback Distinguished Professor in Pediatric Molecular Oncology — who specializes in the treatment of myeloid leukemia, a cancer characterized by abnormal growth of white blood cells — is testing novel treatments based on the underlying mutations. “By exploring the genetics of leukemia patients, we are beginning to unravel the pathways and mechanisms that cause leukemia to develop,” he says.

In bone cancer research, Richard O’Donnell, MD, chief of Orthopaedic Oncology, and pediatric oncologists Steven DuBois, MD, and Robert Goldsby, MD, are involved in clinical investigations to improve outcomes in children, adolescents and young adults with malignant bone tumors, including osteosarcoma and Ewing’s sarcoma. The spectrum of studies ranges from evaluation of the molecular biology of these cancers to multi-institutional clinical trials of new treatments.

UCSF is one of 10 institutions in the United States selected to participate in the Pediatric Brain Tumor Consortium, sponsored by the National Institutes of Health. The goals of this consortium are to better understand pediatric brain tumor biology and to develop effective, new treatments for children with malignant brain tumors.

Policy That Saves Lives

Severe combined immunodeficiency (SCID) is a rare condition in which inherited defects in the immune system make children extremely vulnerable to life-threatening infections. If untreated, children with SCID die before their first or second birthday. However, when they are diagnosed early and treated with bone marrow transplants, the cure rate is more than 90 percent.

With that in mind, UCSF’s Jennifer Puck, MD, an internationally recognized expert in immune disorders, has developed a simple-to-administer blood screening test for SCID that led to approval by the California Department of Public Health of a pilot program to screen all newborns in the state.

“That’s more than 500,000 babies per year,” says Puck, who is working with the state to follow up and diagnose babies with abnormal screening results. Puck presented her data to a national committee advising the secretary of the US Department of Health and Human Services, leading to the 2010 recommendation that SCID screening be adopted nationwide.

The screening project, a collaborative effort with Morton Cowan, MD, chief of the UCSF Pediatric Blood and Marrow Transplant Program, also was initiated in the Navajo Nation, where there is a very high incidence of SCID. UCSF Benioff Children’s Hospital has been designated as the primary transplant center for Navajo children diagnosed with the disease.

UCSF research on methods that have the potential to better address neurological complications also is the driving force behind the pioneering Neuro-Intensive Care Nursery at UCSF Benioff Children’s Hospital.

“Our clinical research through UCSF’s Clinical and Translational Science Institute was crucial,” says Donna Ferriero, MD, chief of child neurology. “Among other things, it led to creation of a mobile, MRI-compatible incubator [pioneered by UCSF’s James Barkovich, MD, chief of Pediatric Neuroradiology], where we place an at-risk infant into a temperature-controlled environment, monitor and ventilate the patient as necessary, and safely conduct neurological imaging.”

The imaging enabled by this unit has opened new doors for researchers in brain development.

Ongoing Research

Other areas of significant research include:

  • Fetal Intervention: UCSF is the birthplace of fetal intervention, a process whereby a team of specialists corrects a life-threatening complication in the fetus while it remains positioned in the mother’s uterus. Current research focuses on congenital heart disease and on a range of birth defects, including myelomeningocele, a form of spina bifida in which the backbone and spinal canal do not close before birth.  Surgeon in Chief Diana Farmer, MD, recognized around the world for her groundbreaking work on spina bifida, is conducting a clinical trial to establish the safety and efficacy of in utero correction of the condition. “We hope it will lead to improved quality of life for these patients,” says Farmer.
  • Heart Disease: Deepak Srivastava, MD, is a UCSF professor of pediatrics, biochemistry and biophysics, and cardiology, and director of the UCSF-affiliated Gladstone Institute of Cardiovascular Disease. He studies the causes of heart disease with the goal of developing novel therapies that can prevent congenital defects and treat acquired heart disease. One recent study demonstrated that the functional cells that make up cardiac muscle can be directly reprogrammed and possibly used to repair damaged heart muscles.
  • Pediatric Devices: Michael Harrison, MD, known as the father of fetal surgery, heads the Pediatric Device Consortium, which develops medical devices to correct birth defects. One such device, currently in use, is a magnet that treats sunken chest syndrome by gradually pulling out the breastbone. The consortium also is developing a motorized magnetic device to correct scoliosis, a curving of the spine that, in severe cases, can be disabling.
  • Advancing Adolescent Medicine: Since 1993, UCSF has been home to the National Adolescent Health Information Center, which aims to improve the health of adolescents by serving as a national resource. Funded by the US Department of Health and Human Services, the center collects, analyzes and disseminates information on adolescents and young adults, translating key information on health behavior, health access, health insurance, and innovative practices for policymakers and decisionmakers at the local, national and international levels.  The center’s researchers — including Director Charles Irwin, MD, and Executive Director Claire Brindis, DrPH — are internationally recognized pioneers in adolescent and young adult health. 
  • Understanding Child Health and Safety: The UCSF School of Nursing supports a number of research projects aimed at improving the health and safety of young people. Nursing school epidemiologist Abbey Alkon, RN, PhD, directs the California Childcare Health Program, a statewide program that creates and distributes an array of research-based services, health information and health education for child care providers and families. Alkon also is working on a long-range research program aimed at understanding the links between stress and children’s physical and mental health.