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CDI in the News

CDI Grant to help teens and young adults with cancer

November 4, 2014

Dr. Theodore B. Moore

Mattel Children's Hospital UCLA and the UCLA Children's Discovery and Innovation Institute (CDI) have received a one-year, $50,000 grant from the St. Baldrick's Foundation, a volunteer-driven and donor-centered charity dedicated to raising money for childhood cancer research. The grant will support research staff with expertise in implementing clinical trial participation for teens and young adults with cancer who are inpatients at the hospital.

Studies have shown that outcomes for this group of patients are much lower than for young children and older adults, partly because their disease is misdiagnosed or diagnosed late. Once diagnosed, many received inconsistent care. Relatively low numbers of patients in this age group participate in oncology-related clinical trials, compared to children and older adults.This funding will allow researchers to develop and implement best practices for the clinical management, treatment and survivorship care of teens and young adults with cancer so that their quality of life outcomes can be enhanced.

"We are tremendously appreciative of the St. Baldrick's Foundation, its donors and volunteers for the support we have received for research," said Dr. Theodore B. Moore, chief and clinical director of pediatric hematology-oncology and director of the Pediatric Blood and Marrow Transplantation Program at UCLA. "As we move to build our program for adolescents and young adults, this support will provide increasing access to state-of-the-art clinical trials that bring with them the hope of improving care and finding a cure for cancer."

CDI Today's and Tomorrow's Children Fund 2015 Awards Announced.

November 4, 2014

The annual Today's and Tomorrow's Children Fund (TTCF) awards were presented to three pediatric faculty researchers from the UCLA Children's Discovery and Innovation Institute (CDI) on November 4, 2014.  The awardees are shown above with some of the TTCF members. Since 2006, the TTCF group has made a significant impact supporting innovative research at Mattel Children's Hospital, and at the UCLA's CDI Institute since it was established in 2012. Applications were reviewed by a scientific committee led by Sherin Devaskar, M.D., Executive Chair of the UCLA Department of Pediatrics and the CDI Institute. Top finalists were presented to the TTCF philanthropic members who voted for final selection for funding and level of funding. Research funding period:  January 1 to December 31 of 2015.

Martin Martin, M.D., M.P.P.; Professor, Pediatric Gastroenterology; "Development of a porcine model of bioengineered intestines"

Eileen Tsai, M.D.; Assistant Professor, Pediatric Nephrology; Medical Director, Pediatric Kidney Transplant Program; "Innovative approaches and treatments of antibody-mediated rejection in children"

Laura Wozniak, M.D., M.S.; Assistant Clinical Professor, Pediatric Gastroenterology; "Donor specific antibodies following pediatric liver transplantation"

CDI Scientists' clinical trial shows favorable outcomes of gene therapy for X-linked severe combined immunodeficiency

October 9, 2014 [publication date]

   [L to R: Kohn, De Oliveira]

Pediatric scientists from the UCLA Children's Discovery and Innovation Institute, Donald Kohn, M.D. and Satiro De Oliveira, M.D., recently co-authored a scientific paper in the prestigious New England Journal of Medicine reporting favorable results of a phase 1|2 clinical trial in nine boys who were diagnosed with X-linked severe combined immunodeficiency (SCID-X1) and underwent gene therapy at 4-10 months of age.  [Hacein-Bey-Abina S. et al.  A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency. N Engl J Med 2014;371:1407-17]. Brief excerpts from the paper are summarized below.

SCID-X1 is caused by mutations in the gene encoding the interleukin-2 receptor γ chain with resultant immunodeficiency effects that often lead to death from community-acquired or opportunistic infection within a year of age unless therapy bone marrow transplant is given. In this clinical trial, in which an SIN γ-retrovirus vector with deletion of strong viral enhancers was used to treat SCID-X1, eight of the nine patients survived, and seven patients had clearance of infection, recovery of gene-marked T cells, evidence of naive T-cell generation, and a diverse T-cell receptor repertoire. Future studies could compare T-cell recovery in the full cohort with that among recipients of alternative types of allogeneic transplants, to determine whether either approach leads to more rapid recovery of immunity. Of note, at the time the paper went to press, the patients had been followed for a median of 33 months (range, 16 to 43) without any occurrence of leukemia. If long-term safety is confirmed, consideration might be given to the inclusion of low-dose pre-transplant marrow conditioning, to enhance the likelihood of engraftment of gene-corrected stem cells and sustained B-cell and NK-cell reconstitution.

CDI Harry Winston Fellows announced for 2014-2015 

Amy Albin - 10/07/2014

[L to R, listed below]

Harry Winston, Inc., the international fine jeweler and watchmaker, donated a five-year, $1 million contribution to support the work of young pediatric physician-scientists from the UCLA Children's Discovery and Innovation Institute (CDI) and Mattel  Children' s Hospital UCLA who are conducting research to prevent, treat and cure disease and illness in children. 

Kristina Adachi, MD - Pediatric Infectious Diseases
"Investigating the impact of bacterial STIs on perinatal transmission of HIV and/or Cytomeglavirus (CMV)"

Leslie Kimura, MD - Pediatric Endocrinology
"Cytoprotective effects of Humanin on myoblast survival and differentiation against oxidative stress"

Edward Talya, MD - Pediatric Gastroenterology, Hepatology and Nutrition
"Efficacy of glucagon like peptide-1 agonist exenatide in the management of pediatric short bowel syndrome"

Harry Winston Fellows will be chosen annually by an internal selection committee led by Dr. Sherin Devaskar, Pediatrics Chair and executive director of the UCLA Children's Discovery and Innovation Institute (CDI) which promotes "scholarship and lifelong learning at all levels of career development," said Devaskar. "The Harry Winston Fellowship Fund will support the best and brightest subspecialty fellows toward becoming exceptional academic physician-scientists who will go on to collaborate and establish networks locally, nationally and globally."  [read more] 


UCLA scientists identify link between stem cell regulation and the development of lung cancer

Brigitte GompertsShaun 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]


Redesigning the well-child checkup

Tumaini CokerAmy 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 Mattel Children's Hospital UCLA, 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

Christopher GizaElaine Schmidt - 05/28/2104

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 Mattel Children's Hospital UCLA. "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]


Using substances at school may be cry for help, UCLA researchers say

Rebecca DudovitzAmy 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 Mattel Children's Hospital UCLA 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]

Jeweler makes $1 million gift to establish pediatric fellowships

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 Mattel Children's Hospital UCLA'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]


Pilot program study finds that pediatric obesity patients like telehealth services

 Wendy Slusser

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 telehelath 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 Mattel Children's Hospital UCLA 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]


Six months of fish oil reverses liver disease in children with intestinal failure, study shows

Kara Calkins

Amy Albin, aalbin@mednet.ucla.edu     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 Mattel Children's Hospital UCLA 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]