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UCLA Clinical Updates bladder and bowel dysfunctions and chronic pelvic pain. Minimally Invasive Surgery for GERD in Pediatric Patients Mattel Children’s Hospital UCLA pediatric surgeons employ an advanced, minimally invasive technique called laparoscopic Nissen fundoplication to treat infants and children diagnosed with severe gastroesophageal reflux disease. Learn about the Latest Advances from UCLA Head and Neck Cancer Program Cellular and Gene Therapy for Pediatric Diseases Developing the most effective treatment plan for head and neck cancers involves input from a variety of specialists. In order to optimize their consultations at UCLA, patients meet with many members of our multidisciplinary team during a single visit. Mattel Children’s Hospital UCLA is developing and conducting several groundbreaking gene-therapy clinical trials. Therapies that use the patients own cells can avoid the graft-versus-host disease risk of bone-marrow transplantation. Immunotherapy for Advanced Melanoma A novel immunotherapy drug that was tested at UCLA has gained FDA approval. It is the first in a new class of immune-system modulators that are expected to dramatically improve survival in patients with unresectable or advanced disease. Clinical Trial for Brain Aneurysm Device UCLA is taking part in a study on the use of the FRED™ stenting system for wide-necked or large intracranial aneurysms. The new device holds the promise of improved clinical outcomes and fewer procedure-related complications. Female Athlete Triad Hypertrophic Cardiomyopathy Some girls and women who exercise intensely are at risk for this medical condition encompassing three interrelated components: low energy availability with or without disordered eating, menstrual dysfunction and low bone-mineral density. Offering the most advanced therapies — including myectomy and alcohol septal ablation — UCLA physicians are able to restore a more normal blood flow to patients who have become obstructed with excess muscle tissue in the septal walls. In addition to caring for children with congenital abnormalities — such as cleft lip or palate — UCLA doctors treat pediatric patients with trauma-related injuries and vascular anomalies and reconstruct children’s faces disfigured by cancer removal or treatment. At UCLA, a collaborative, multidisciplinary team of specialists provides coordinated consultation and treatment to offer a full range of care, including the most advanced and innovative therapies available. Pelvic Floor Dysfunction Physical therapy can be an effective, conservative treatment for pelvic floor conditions, including A novel immunotherapy drug that was tested at UCLA and 11 other sites has gained U.S. Food and Drug Administration approval for use in patients with advanced melanoma. The drug, pembrolizumab (brand name Keytruda), is an antibody that inhibits the programmed death-1 receptor, or PD-1, to help expose cancer cells to a patient’s immune system. The medication represents the first in a new class of immune system modulators that are expected to dramatically improve survival in patients with unresectable or advanced disease. Rates of melanoma have been rising for more than three decades. The disease now accounts for more than 9,700 deaths annually in the United States. Melanoma that has metastasized presents a particular challenge, with five-year survival rates of 15 to 20 percent among patients with stage IV disease. Immunotherapeutic agents such as interleukin-2 have produced responses in patients with melanoma, however at extremely low response rates. Pembrolizumab produces a dramatically high, durable response rate that represents a significant treatment advance. Releasing the immune response Immunologic checkpoints such as the PD-1 pathway play an essential role in immune system function by preventing excessive immune response. However, some tumors exploit these checkpoints. UCLAHEALTH.ORG 1-800-UCLA-MD1 (1-800-825-2631) UCLAHEALTH.ORG “This drug is a game changer, a very significant advance in the treatment of melanoma,” says Dr. Ribas, a professor of medicine in the division of hematology/ oncology at the David Geffen School of Medicine at UCLA. “For patients who have not responded to prior therapies, this drug now provides a very real chance to shrink their tumors and the hope of a lasting response to treatment.” uclahealth.org/clinicalupdates Innovative cellular and gene therapy programs treat pediatric diseases Lowering transplant rejection risk “The goal of the newly reorganized Head and Neck Cancer Program at UCLA is to provide the highest-quality care while providing the best possible patient experience,” says Maie St. John, MD, PhD, co-director of the program. The standard of care for a number of childhood illnesses is bone marrow transplantation, in which a patient’s diseased blood cells are replaced with healthy donor cells. Transplants are most likely to be successful when the donor cells comes from a close relative. Unfortunately, 70 percent of patients don’t have a familial match. “The odds of graft-versus-host disease, a complication in which donor cells attack the recipient’s body, go up dramatically when using cells from an unrelated donor,” says Ami J. Shah, MD, director of the Program for Cellular Therapeutics at Mattel Children’s Hospital UCLA. “On their first visit, patients can be seen by an expert team, including those who will assist them with qualify-of-life issues,” says Elliot Abemayor, MD, PhD, co-director of the program. “Each patient’s initial visit is used to identify the best therapies available for them. We want to give patients options, and allow them to make choices after they’ve been informed.” The UCLA Head and Neck Cancer Program provides comprehensive, personalized care for benign and malignant tumors of the head and neck — tumors arising within the oral cavity, larynx, pharynx, salivary glands, thyroid, nose and sinuses. UCLA is one of the nation’s most active head and neck cancer treatment centers. The head and neck consultation clinic sees more than 50,000 patient visits each year, many of which are for the care of benign and malignant problems. The range and experience of the head and neck specialists allows us to offer the most comprehensive and advanced evaluations and treatments. Our specialists are highly experienced in the early diagnosis and accurate staging of head and neck cancers — from common types to extremely rare ones — and are dedicated to providing outstanding medical care that preserves speech, swallowing, hearing and facial appearance. Team treatment for more efficient care Developing the most effective treatment plan for head and neck cancers involves input from a variety of specialists. In order to optimize their consultations, patients meet with as many members of our multidisciplinary team as possible during a single visit. UCLAHEALTH.ORG 1-800-UCLA-MD1 (1-800-825-2631) 1-800-UCLA-888 (1-800-825-2888) Treatment options at the UCLA Head and Neck Cancer Program extend beyond current standards of treatment. Our nationally recognized research program is pioneering new ways to treat and monitor head and neck cancers. Ongoing research initiatives available to patients include methods to improve tumor margin delineation and the use of polymer drug delivery systems to ensure that medications reach only the intended target and with controlled release to minimize side effects. Republished with permission of American Society of Hematology, from ASH Image Bank; permission conveyed through Copyright Clearance Center, Inc. Giving patients the most options For several decades, physicians have had little to offer patients with advanced melanoma. However, recent advances in understanding the biological mechanisms of the disease as well as a deepening knowledge of immune regulatory checkpoints has ushered in a new era of targeted drug development. Dr. Antoni Ribas (right) with Tom Stutz, whose health improved dramatically after treatment with the newly approved drug. To download these and other clinical advances at UCLA Health, go to: Personalized, Coordinated Care for Head and Neck Cancer A pivotal advance in melanoma treatment Pembrolizumab overcomes the natural tendency of cancer cells to shield themselves from immune cells by expressing the ligand to PD-1 (known as PD-L1), thus overcoming a major obstacle in the evolution of effective cancer immunotherapeutics, says Antoni Ribas, MD, PhD, a principal investigator of the phase 1, fast- track trial of pembrolizumab. FDA Approves Lung-Cancer Drug The Food and Drug Administration has approved a new drug to treat non- small-cell lung cancer (NSCLC). The drug, ramucirumab, was tested on more than 1,200 people with NSCLC whose cancer worsened during or after first-line chemotherapy. The research was conducted as part of a multiyear, Phase 3 clinical trial at UCLA and other centers in 26 countries on six continents. uclahealth.org/lungcancerdrug Protein Plays Important Role in Blood Stem Cell Replication UCLA scientists have identified a protein that plays a key role in regulating how blood stem cells replicate in humans. This discovery could lead to the development of more effective therapies for a wide range of blood diseases and cancers. uclahealth.org/stemcellprotein Pediatric Craniofacial Clinic UCLA Cardiovascular Center New Immunotherapeutic for Advanced Melanoma Represents a Major Advance News from UCLA Health Bone marrow sample from a patient with Gaucher disease stained to show the cell nuclei. Ever since UCLA opened one of the nation’s first bone marrow transplant units in 1973, doctors in the gene and cellular therapy programs have been leading the way in researching treatments and cures for debilitating childhood diseases. Today, Mattel Children’s Hospital UCLA is a world leader in developing and conducting several groundbreaking gene-therapy clinical trials. The Program for Cellular Therapeutics in the Division of Pediatric Hematology/ Oncology is working in conjunction with the UCLA Human Gene and Cell Therapy Program, the UCLA Children’s Discovery & Innovation Institute and the Pediatric Bone Marrow Transplant Program to develop safer and innovative treatments for children who suffer from these life-threatening diseases. Severe combined immunodeficiency (SCID) UCLA’s Program for Cellular Therapeutics has been conducting gene-therapy clinical trials for adenosine deaminase (ADA) deficiency or ADA-SCID. This “bubble-baby disease” weakens the immune system, making it difficult for children to fight off even mild infections. ADA-SCID is lethal if left untreated. Donald B. Kohn, MD, director of the UCLA Human Gene and Cell Therapy Program, and his colleagues performed the basic laboratory research to improve gene delivery to blood-forming stem cells, developed clinical protocols and received all local and national regulatory approvals. UCLAHEALTH.ORG 1-800-UCLA-MD1 (1-800-825-2631) Cellular and gene therapies use the patient’s own (autologous) cells. “There is less risk of rejection when using a patient’s own cells,” explains Dr. Shah. “The cells are genetically modified in a laboratory and then transplanted back into the patient where it is hoped that they will multiply and start to kill diseased cells.” Thanks in large part to their dedicated research into cellular and gene therapies, UCLA doctors have been tapped to spearhead several ground- breaking clinical trials aimed at treating genetic diseases. Some Men Receive Prostate-Cancer Treatment Contrary to Guidelines National guidelines recommend that men with low- and intermediate-risk prostate cancer who have life expectancies of fewer than 10 years should not be treated with radiation or surgery, since they are unlikely to live long enough to benefit from treatment. Yet a new study by UCLA researchers found that more than half of such men are receiving these aggressive treatments, putting them at risk for potentially debilitating side effects. uclahealth.org/ prostatecancertreatment