Tiare Dunlap 4/29/19
Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have received a $4.6 million grant from the California Institute for Regenerative Medicine, known as CIRM.
The funds will support a phase 1 clinical trial of a treatment for advanced sarcomas and other cancers with a specific tumor marker called NY-ESO-1. The research, led by Dr. Theodore Scott Nowicki in collaboration with Dr. Antoni Ribas, will test a therapeutic approach that genetically engineers each patient’s own blood-forming stem cells to produce cancer-fighting immune cells called T cells. [read full story]
Mirabal Vogt-James 1/10/2019
UCLA researchers led by Dr. Donald Kohn have created a method for modifying blood stem cells to reverse the genetic mutation that causes a life-threatening autoimmune syndrome called IPEX. The gene therapy, which was tested in mice, is similar to the technique Kohn has used to cure patients with another immune disease, severe combined immune deficiency, or SCID, also known as bubble baby disease. The work is described in a study published in the journal Cell Stem Cell.
IPEX is caused by a mutation that prevents a gene called FoxP3 from making a protein needed for blood stem cells to produce immune cells called regulatory T cells. Regulatory T cells keep the body’s immune system in check; without them, the immune system attacks the body’s own tissues and organs, which is known as autoimmunity. [read full story]
Denise Heady 11/1/2018
Something as simple as a change in diet can potentially help to increase the cancer survival rate of obese children with acute lymphoblastic leukemia, the most common childhood cancer, according to a new study by UCLA scientists.
The research team, led by Dr. Steven Mittelman, chief of pediatric endocrinology at UCLA Mattel Children’s Hospital and member of the UCLA Jonsson Comprehensive Cancer Center, administered the chemotherapy drug vincristine to obese and non-obese mice with leukemia. Researchers discovered that if they switched the obese mice from a high-fat to a low-fat diet immediately before starting chemotherapy, the mice had a dramatically improved outcome. The mice on the low-fat diet had a five times higher survival rate than the mice in the high-fat diet group. [read full story]
Elaine Schmidt 10/29/2018
Students from lower-income neighborhoods who attended one of five high-performing Los Angeles County high schools were less likely to abuse marijuana than those who weren’t offered admission, UCLA researchers found.
“We concluded that schools play an important role in influencing adolescent behavior,” said the study's first author, Dr. Rebecca Dudovitz, an assistant professor at the David Geffen School of Medicine at UCLA and pediatrician at UCLA Mattel Children’s Hospital. “Investing in schools offers a tool for improving teen health. [read full story]
First CDC guidelines for treating children's concussions offer valuable tools for doctors, UCLA researchers report
Elaine Schmidt 9/17/2018
For the first time, the Centers for Disease Control and Prevention has issued guidelines for diagnosing and treating children who have suffered a concussion, also known as a mild traumatic brain injury. These guidelines will provide doctors with the tools they need to deliver the best outcomes for young patients with concussion, said two UCLA co-authors of the report, recently published in JAMA Pediatrics.
Dr. Meeryo Choe and Dr. Christopher Giza are co-authors of the report. “Children have been overlooked in research and clinical protocols for concussion, but their smaller size doesn’t make them less important,” Giza says. [read full story]
Parents who have had severe stresses, trauma in childhood more likely to have kids with behavioral health problems
Amy Albin 7/08/2018
A new study finds that severe childhood trauma and stresses early in parents’ lives are linked to higher rates of behavioral health problems in their own children.
The types of childhood hardships included divorce or separation of parents, death of or estrangement from a parent, emotional, physical or sexual abuse, witnessing violence in the home, exposure to substance abuse in the household or parental mental illness.Dr. Adam Schickedanz led the study, which showed that household medical costs were 30 percent higher when an adult had lived through three or more adverse experiences during childhood. [read full story]
Ryan Hatoum 2/20/2018
Dr. Steven Mittelman, chief of pediatric endocrinology at UCLA Mattel Children’s Hospital, is among those looking for clues to improve children’s survival and recovery from cancer. But unlike most other researchers, Mittelman explores the connection from the perspective of the fat cell, not the cancer cell; he’s trying to understand the environment in the body that fat itself cultivates that leaves some people more vulnerable to cancer. [read full story]
Amy Albin 10/17/2017
Dr. Neal Halfon, the founding director of the UCLA Center for Healthier Children, Families and Communities, received the C. Anderson Aldrich Award in Child Development at the American Academy of Pediatrics’ national conference held in Chicago in September.
The award, which recognizes outstanding contributions to child development, was established in 1964 by the American Academy of Pediatrics Section on Child Development. Halfon directs the Child and Family Health Leadership and Training Program in the UCLA Fielding School of Public Health and is a professor of pediatrics at the David Geffen School of Medicine UCLA; health policy and management in the school of public health, and public policy at the Luskin School of Public Affairs.
Enrique Rivero 6/21/2017
HIV-positive women with cytomegalovirus, or CMV, in their urine at the time of labor and delivery are more than five times likelier than HIV-positive women without CMV to transmit HIV, the virus that causes AIDS, to their infants, according to a UCLA-led study. The research also found that they are nearly 30 times likelier to transmit cytomegalovirus to their infants.
Dr. Karin Nielsen, a professor of clinical pediatrics in the division of infectious diseases at the David Geffen School of Medicine at UCLA and CDI faculty researcher, is the senior author of the study, which was published today in the journal Clinical Infectious Diseases. [read full story]
Ryan Hatoum 5/10/2017
About 750 guests attended the event, "Light: A Celebration of Discovery and Innovation," on May 6 in Culver City.
Over the past five years, the benefit events have raised more than $10 million for UCLA Mattel Children's Hospital and UCLA Children's Discovery and Innovation Institute. The gifts have led to advancements in treatment and care for pediatric patients around the world, and provided support for high-priority clinical programs and multidisciplinary research in children’s health. [read full story]
Elaine Schmidt 5/2/2017
Up to 10 percent of pregnancies worldwide are affected by intrauterine growth restriction, which means a baby weighed less than 90 percent of babies at the same gestational age. The condition increases the risk of a wide range of serious health problems, but the cause remains poorly understood.
Lead by the Children's Discovery and Innovation Institute's Executive Director, Sherin Devaskar, UCLA scientists have discovered specific genetic changes in the placentas of women who gave birth to growth-restricted infants. These changes appear to sabotage the ability of the placenta to grow blood vessels and adequately nourish the fetus, interfering with the infant’s growth in the womb. [read full story]
Tiare Dunlap 5/10/2017
UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated
In a phase 2 clinical trial led by UCLA Professor and pediatric scientist at the UCLA Children's Discovery and Innovation Institute Dr. Donald Kohn, who is also at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, all nine babies were cured. A 10th trial participant was a teenager at the time of treatment and showed no signs of immune system recovery. Kohn’s treatment method, a stem cell gene therapy that safely restores immune systems in babies with the immunodeficiency using the child’s own cells, has cured 30 out of 30 babies during the course of several clinical trialnary research in children. [read full story]
Elaine Schmidt - 3/07/2017
In a study published today in Science Signaling, UCLA researchers have discovered that after the initial hug, T cells become more gregarious, giving something more like a bear hug to any cell presenting its antigen. These larger hugs help to activate the T cell, equipping it to go out into the body and coordinate multi-cellular attacks to fight infections or cancers. The UCLA team learned that how stiff or soft T cells are controls their response — the cells react slowly when they are stiff and trigger easily when they are soft.
T cells are like the shy person at the office holiday party who acts stiff until they loosen up a bit and then are all over the dance floor,” said CDI scientist Dr. Manish Butte, associate professor of pediatrics and microbiology, immunology and molecular genetics at the David Geffen School of Medicine at UCLA and the study’s senior author.
Enrique Rivero- 2/14/2017
Mother-to-child transmission of human immunodeficiency virus type1 poses a serious health threat in developing countries, and more effective interventions are needed. Previous studies determined that strains of HIV that infect both adults and infants almost always use the CCR5 co-receptor for infection and also that that infection is often established by a single viral variant rather than by multiple variants. Additionally, previous studies have shown some of the features of the transmitted viruses.
In the transmission of HIV-1 from mother to child only a subset of a mother’s viruses infects their infants either in utero or via breastfeeding, and the viruses that are transmitted depend on whether transmission occurs during pregnancy or through breastfeeding, according to UCLA-led research from Professor of Pediatrics and CDI scientist, Grace Aldrovandi. [read full story]
Amy Albin - 06/13/2016
Many physician-scientists have ideas for research that could lead to new treatments or cures for the patients they treat. But getting federal funding to support that research isn’t always easy.
That’s why a “giving circle” at UCLA Mattel Children’s Hospital has so much impact. It offers pediatric physician-scientists the opportunity to pitch their ideas, win grants and change young lives.
The circle, officially known as the Today’s and Tomorrow’s Children Fund, is made up of community members who are interested in medical research. Each year, they pool their donations of $5,000 per person. The members listen to presentations by doctors and then democratically select how to distribute the funds among the grantee or grantees. [read full story]
Amy Albin - 10/13/2015
Harry Winston, Inc., the international fine jeweler and watchmaker, and the UCLA Children’s Discovery and Innovation Institute have named the new recipients of the 2015-2016 Harry Winston Fellowships. The fellowships support the work of young pediatric physician-scientists from UCLA Mattel Children’s Hospital who are conducting research to prevent, treat and cure disease and illness in children. Harry Winston, Inc. donated a five-year, $1 million contribution to launch the fellowship program last year.
Harry Winston Fellows are chosen annually by an internal selection committee led by Dr. Sherin Devaskar, the Mattel Executive Endowed Chair of the department of pediatrics at UCLA Mattel Children’s Hospital and executive director of the UCLA Children's Discovery and Innovation Institute.
“We are excited to name our second group of Harry Winston Fellows. These are some of the best and brightest subspecialty physicians who will go on to become exceptional academic physician-scientists,” said Devaskar. “We are grateful to Harry Winston, Inc. for supporting their training and we can all look forward to seeing the contributions they will make in the field of medicine.” [read full story]
Amy Albin - 09/29/2015
The National Institutes of Health has awarded UCLA a $4.2 million grant to study how environmental pollution negatively affects how the placenta develops in pregnant women, ultimately contributing to poor pregnancy outcomes.
Building upon earlier work that has examined the impact of air pollution on pregnancy, Kyung Sung, Dr. Sherin Devaskar and Dr. Carla Janzen, along with Dr. Beate Ritz (not pictured), will study how exposure to pollution affects the placenta.
Dr. Sherin Devaskar, the Mattel Executive Endowed Chair of the department of pediatrics at UCLA Mattel Children’s Hospital, will lead a team of researchers that will build upon earlier work that has examined the impact of air pollution on pregnancy.
“While we know there is a link between airborne pollution and poor birth outcomes, we don’t know the mechanisms — how it occurs,” said Devaskar, who is also the executive director of the UCLA Children’s Discovery and Innovation Institute. “The aim of the study is to develop and evaluate new magnetic resonance imaging (MRI) technologies to assess the impact of environmental pollution exposure so that we can accurately predict placental insufficiency; that is, the insufficient blood flow and thereby nutrients to the placenta during pregnancy.” [read full story]
Mirabai Vogt-James - July 28, 2015
Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have been awarded $7.4 million to lead a clinical trial that will utilize a stem cell gene therapy to correct a genetic defect associated with an immunodeficiency disorder. Chronic granulomatous disease, which prevents white blood cells from killing foreign invaders such as bacteria, fungi and other microorganisms, is an inherited blood disorder affecting approximately 1 in 200,000 persons in the United States. The disorder is usually diagnosed before age 5 and can cause severe, persistent and untreatable tissue infections that lead to the formation of granulomas (a mass of tissue produced in response to infection). Without treatment, children often die within the first decade of life.
The financial award from the California Institute for Regenerative Medicine will fund a phase I/II clinical trial led by Dr. Donald Kohn, professor of pediatrics in the David Geffen School of Medicine at UCLA and the principal investigator. “CIRM has provided us with a very exciting opportunity to assess the viability of a potential cure for this devastating disease using a novel therapy that, if successful, could possibly be utilized in other diseases in the future,” said Kohn, who is also a UCLA professor of microbiology, immunology and molecular genetics and a member of the UCLA Children's Discovery and Innovation Institute (CDI) at Mattel Children’s Hospital.
“We will start by treating the most common form of the disorder, X-linked CGD,” said Dr. Caroline Kuo, clinical instructor in the Division of Allergy & Immunology, Department of Pediatrics at UCLA and chair of the study. “Our hope is that this treatment will provide a lasting cure for X-linked CGD patients, whose quality of life is greatly impacted by the disorder. If it works, the treatment could theoretically be expanded to treat all forms of CGD.” [read full story]
Shaun Mason - 06/19/2014
UCLA researchers led by Dr. Brigitte Gomperts have discovered the inner workings of the process thought to be the first stage in the development of lung cancer. Their study explains how factors that regulate the growth of adult stem cells that repair tissue in the lungs can lead to the formation of precancerous lesions. Findings from the three-year study could eventually lead to new personalized treatments for lung cancer, which is responsible for an estimated 29 percent of U.S. cancer deaths, making it the deadliest form of the disease.
The study was published online June 19 in the journal Stem Cell. Gomperts, a member of the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and the UCLA Jonsson Comprehensive Cancer Center, collaborated with Manash Paul and Bharti Bisht, postdoctoral scholars and co-lead authors of the study.
Gomperts, who also is an associate professor in the department of pediatrics at UCLA and a scientist at the UCLA Children's Discovery and Innovation Institute, said "Loss of this ROS regulation leads to precancerous lesions. Now, with this precancerous model in place, we can begin looking for what we call 'driver mutations,' or those specific changes that take the precancerous lesions to full-blown cancer." Gomperts said that because many different factors - including cigarette smoke, smog and inflammation - could potentially trigger an increase in ROS in the airway stem cells, researchers might eventually be able to customize treatments based on the cause. "There are likely multiple ways for a person to get to a precancerous lesion, so the process could be different among different groups of people. Imagine a personalized way to identify what pathways have gone wrong in a patient, so that we could target a therapy to that individual." [read full story]
Amy Albin - 06/16/2014
UCLA study suggests new models for more efficiently delivering preventive care to low-income families. In a year-long study led by Dr. Tumaini Coker, an assistant professor of pediatrics at UCLA Mattel Children's Hospital, researchers developed a new design for preventive health care for children from birth through age 3 from low-income communities. The team partnered with two community pediatric practices and a multi-site community health center in greater Los Angeles.
"The usual way of providing preventive care to young children is just not meeting the needs of the low-income families served by these clinics and practices," said Coker, who also is a researcher with the hospital's UCLA Children's Discovery and Innovation Institute. "Our goal was to create an innovative and reproducible - but locally customizable - approach to deliver comprehensive preventive care that is more family-centered, effective and efficient." [read full story]
Steve Tisch donates $10M to UCLA program devoted to researching, treating sports concussions, especially in youth
Elaine Schmidt - 05/28/2014
The BrainSPORT (Brain Sports concussion Prevention Outreach Research and Treatment) Program was founded in 2012 by UCLA's Dr. Christopher Giza, who is scheduled to participate in the White House briefing. Integrating the expertise of clinicians and scientists at the UCLA Brain Injury Research Center and in pediatric neurology, neuropsychology and sports medicine, the program provides research-based treatment for sports concussions in school-age to professional athletes.
As director of the UCLA Steve Tisch BrainSPORT Program, Giza's immediate goal is to develop an age-appropriate concussion-evaluation tool that blends baseline testing, recordings from advanced biomechanical sensors, and expert neurological and cognitive exams. The tests will measure a concussion's severity, determine the treatment and guide plans for the affected athlete's return to competition.
"Mr. Tisch's generous gift will be an enormous game-changer, enabling us to create diagnostic tools customized to younger athletes," said Giza, who is a professor of pediatric neurology and neurosurgery at the Geffen School of Medicine and UCLA Mattel Children's Hospital. "Currently, young athletes are assessed with adult tests - but kids aren't little adults. With the right diagnosis and personalized care, kids can recover completely from concussion." Dr. Giza is also a neuroscientist in the UCLA Children's Discovery and Innovation Institute. [read full story]
Amy Albin - 05/03/2014
Teens caught drinking or using marijuana at school should be screened for exposure to trauma, mental health problems and other serious health risks, according to a study presented May 3 at the annual meeting of the Pediatric Academic Societies in Vancouver, Canada. UCLA researchers found that the use of substances at school was associated with significantly increased odds of serious problems such as depression, being the victim of intimate-partner violence and attempting suicide.
"At-school substance use is not just an isolated event requiring simple disciplinary action but an important signal identifying teens in need of urgent psychosocial assessment and support," said lead author Dr. Rebecca N. Dudovitz, an assistant professor of pediatrics at UCLA Mattel Children's Hospital and the UCLA Children's Discovery and Innovation Institute.
Dudovitz and colleagues analyzed data from the 2011 Youth Risk Behavior Survey, a nationally representative survey of more than 15,000 U.S. high school students. The Centers for Disease Control and Prevention conducts the survey every two years to monitor conditions and behaviors that impact adolescent health. [read full story]
Amy Albin | April 21, 2014
Harry Winston, Inc., recently announced a $1 million pledge to the UCLA Children's Discovery and Innovation Institute to establish the Harry Winston Fellowships.
As the first corporate sponsor of UCLA Mattel Children's Hospital's innovative pediatric fellowships, Harry Winston, Inc., an international fine jeweler and watchmaker, will support the work of young pediatric physician-scientists who are conducting research to prevent, treat and cure disease and illness in children.
Three outstanding fellows, Dr. Mark Hanudel, Dr. Kevin Quinn, and Dr. Caroline Kuo, whose appointments as the 2014 Harry Winston Fellows are underway, were recognized at Mattel Children's Hospital's second annual Kaleidoscope Ball on April 10 at the Beverly Hills Hotel. Proceeds from the gala benefit the UCLA Children's Discovery and Innovation Institute. [read full story]
Amy Albin - December 11, 2013
For youth dealing with obesity who need extra help losing weight, experts suggest a multidisciplinary approach in which care is provided by several health specialists. However, the logistics of traveling to multiple appointments, even if just across town, can be a barrier to receiving care, especially for low-income families.
UCLA researchers at the UCLA Children's Discovery and Innovation Institute who work with this patient population set up a pilot program using telehealth technology - a secure system that allowed patients to see and speak with their health care providers at UCLA over a computer from their local health clinic - to evaluate if such a system could be an effective strategy to help overcome these issues. Their study of the program found that the great majority of pediatric patients - approximately 80 percent - were satisfied with their telehealth appointment, saying it was just as good as talking to the doctor in person, that it was easier to go to the local clinic than to the UCLA campus in Westwood, that they felt comfortable and that their privacy was protected.
In addition, 80 percent said they would participate in a telehealth appointment again. Responses from the health care providers were similarly positive. The results of the project were presented at the Southern California Public Health Association Conference on Dec. 9.
"One surprise was how natural it was to talk with each other through the telehealth system, even though we never met the patients in person," said lead author Dr. Wendy Slusser, medical director of the Fit for Healthy Weight program at UCLA Mattel Children's Hospital and director of pediatric wellness programs at the Venice Family Clinic. "The interaction was very much like being in the same room together. Some kids even thought it was fun to see themselves on the screen." [read full story]
Amy Albin, [email protected] August 14, 2013
Children who suffer from intestinal failure, most often caused by a shortened or dysfunctional bowel, are unable to consume food orally. Instead, a nutritional cocktail of sugar, protein and fat made from soybean oil is injected through a small tube in their vein.
For these children, the intravenous nutrition serves as a bridge to bowel adaptation, a process by which the intestine recovers and improves its capacity to absorb nutrition. But the soybean oil, which provides essential fatty acids and calories, has been associated with a potentially lethal complication known as intestinal failure-associated liver disease, which may require a liver and/or intestinal transplant. Such a transplant can prevent death, but the five-year post-transplant survival rate is only 50-70 percent. Previous studies have shown that replacing soybean oil with fish oil in intravenous nutrition can reverse intestinal failure-associated liver disease. However, the necessary duration of fish oil treatment had not been established in medical studies.
Now, a clinical trial conducted at the Children's Discovery and Innovation Institute at UCLA Mattel Children's Hospital has found that, compared with soybean oil, a limited duration (24 weeks) of fish oil is safe and effective in reversing liver disease in children with intestinal failure who require intravenous nutrition. The researchers believe that fish oil may also decrease the need for liver and/or intestinal transplants - and mortality - associated with this disease. [Read full story]