World-renowned cancer investigator Tom Curran, PhD, FRS, was honored by the American Association for Cancer Research as it inaugurated the first class of the fellows of the AACR Academy.

Dr. Curran, who is also the deputy scientific director of the CHOP Research Institute, was formally inducted into the Academy on April 5 in Washington, D.C. He was one of 106 fellows from across the country to receive the honor of induction into the AACR Academy.

The Academy was created to recognize and honor distinguished scientists whose major scientific contributions have propelled significant innovation and progress against cancer. The fellows were selected through a rigorous peer review process that evaluates individuals on the basis of their stellar scientific achievements in cancer research.

Dr. Curran, a past president of the AACR, studies brain development and pediatric brain tumors, with an eye toward identifying molecular changes and potential drug targets. He also investigates the mechanism of action of anticancer drugs in tumor cells and cancer models.

Specifically, Dr. Curran discovered the Fos oncogene and its binding partner from another oncogene called Jun. He later showed that these two oncogenes regulate gene expression associated with cell proliferation and differentiation, cell death and neuronal activation. This work illuminated the pathways that go awry in cancer cells, and initiated the use of Fos as a marker for activity-dependent changes in the nervous system.

Dr. Curran recently united his interests in cancer and neurobiology to study children’s brain tumors. He developed a high-incidence model of pediatric medulloblastoma that he used to demonstrate how orally bioavailable, small molecule inhibitors of Hedgehog signaling rapidly eliminate even large tumors in mice. This work led to clinical development of inhibitors of Smoothened for the treatment of basal cell carcinoma and medulloblastoma.

Additionally, Dr. Curran considers among his greatest achievements his contribution to the development of a drug that is now in pediatric trials — Erivedge, which in 2012 was approved by the FDA to treat cancer in adults.

In addition to serving as the Deputy Scientific Director at CHOP Research, Dr. Curran is a Professor of Pathology and Laboratory Medicine and Professor of Cell and Developmental Biology at University of Pennsylvania’s Perelman School of Medicine. He is also the associate director of Translational Genomics at the Penn Genome Frontiers Institute in Philadelphia.

Children’s Hospital investigator Claudio Giraudo, PhD, of the Department of Pathology and Laboratory Medicine, joined the prestigious community of Pew Scholars in 2013. Previous Pew Scholars have included Nobel laureates, MacArthur fellows, and many others who were awarded Pew grants to help launch their careers.

Dr. Giraudo was one of just 22 investigators to be selected out of 134 nominated for the Pew Scholars Program in the Biomedical Sciences. The Pew Scholars were each awarded $240,000 over four years to conduct their research.

The Pew Scholars Program “identifies and invests in talented researchers in medicine or biomedical sciences,” enabling “promising scientists to take calculated risks and follow unanticipated leads to advance human health,” according to the Pew website. Since 1985, Pew has awarded more than 500 investigators over $130 million in research funding.

Dr. Giraudo’s research interests center on intracellular membrane trafficking and calcium-regulated exocytosis in eukaryotic cells, particularly how immune cells secrete granules that destroy infected cells.

With the support of the Pew Scholar grant, Dr. Giraudo said he hopes “to identify the protein machinery cytotoxic T lymphocytes (CTLs) use to secrete granules that break down cells.” A type of white blood cell, CTLs target and kill other cells, including cancer cells and cells that have been infected with viruses.

“This award will give me the chance to study what happens at the cell membrane during the body’s immune response, which could be therapeutically targeted to improve prognosis and quality of life of patients,” Dr. Giraudo said.

In addition to better understanding cell-mediated killing, the Pew-supported research project could also shed light on how similar processes lead to disease, such as in diabetes and brain disorders, he added.

Paul A. Offit, MD, was lauded for his more than three decades of vaccine research and advocacy with back-to-back honors. The blog Vaccine Nation named him one of the 50 most influential people in vaccines, and he was also awarded the 2013 Maxwell Finland Award for Scientific Achievement, given annually by the National Foundation for Infectious Diseases (NFID).

Along with late Children’s Hospital researcher Fred C. Clark, DVM, PhD, and University of Pennsylvania Emeritus Professor Stanley A. Plotkin, MD, Dr. Offit is a co-creator of the rotavirus vaccine, Rotateq. Prior to the invention of Rotateq, thousands of children in the U.S. were hospitalized with rotavirus each year, and the drug is credited with saving hundreds of thousands of lives a year around the world.

Dr. Offit, director of CHOP’s Vaccine Education Center and chief of the Division of Infectious Diseases, is also an ardent champion of the safety and necessity of vaccinations, and his willingness to speak his mind has earned him many vocal critics over the years.

Vaccine Nation conducted a survey of its subscribers, LinkedIn group members, and contacts to compile its list of the top 50 people in vaccines. Dr. Offit, who was named the sixth most influential person, is joined on the list by Dr. Plotkin, as well as the philanthropists Bill and Melinda Gates.

The NIFID award recognized Dr. Offit and other “who have made outstanding contributions to the understanding of infectious diseases or public health.” Past winners of the award include Dr. Plotkin and former U.S. Surgeon General C. Everett Koop, MD.

Calling Dr. Offit “an impassioned advocate for immunizations,” the NFID award citation noted that he “has rallied the scientific counteroffensive against those who would denigrate the power and worth of vaccines.”

A groundbreaking clinical trial of gene therapy for a form of congenital blindness conducted by Children’s Hospital and the University of Pennsylvania was recognized with the Distinguished Clinical Research Achievement Award from the Clinical Research Forum, an organization of clinical research centers, industry, and volunteer groups.

That award is the second highest in the CRF’s annual Top 10 Clinical Research Achievement Awards. Recognizing studies published in 2012, the CRF focused on a Feb. 2012 article in Science Translational Medicine, co-authored by researchers from CHOP and the Perelman School of Medicine at the University of Pennsylvania. The authors reported on the most recent phase of a clinical trial for Leber’s congenital amaurosis (LCA), a rare retinal disease that progresses to total blindness by adulthood.

The study team reported on further improvements in vision in three adult patients previously treated in one eye who then received the same innovative gene therapy in the second eye.

This LCA research is an ongoing collaboration among Jean Bennett, MD, PhD, F.M. Kirby professor of Ophthalmology at the University of Pennsylvania School of Medicine; CHOP’S Katherine A. High, MD, HHMI, director of the Center for Cellular and Molecular Therapeutics (CCMT); and Albert M. Maguire, MD, of Penn Medicine and CHOP.

Dr. High, a pioneer of gene therapy, directs the CCMT, which sponsors the clinical trial in LCA and manufactured the genetically engineered virus used to carry the therapeutic gene. Dr. Maguire, a retina specialist, injected the corrective gene into the eyes of adult and pediatric study subjects at Children’s Hospital.

As widely reported in October 2009, this clinical trial of gene therapy achieved dramatic results in children with LCA. Building on their previous work, the research team is now conducting the first Phase 3 gene therapy study for genetic disease in the U.S. This is also the world’s first Phase 3 gene therapy study for a non-lethal disorder. If successful, it could lead to the first approved gene therapy product in the United States.

Renown AIDS researcher Robert W. Doms, MD, PhD, joined Children’s Hospital in September 2012 as pathologist-in-chief and chair of Pathology and Laboratory Medicine.

Much of Dr. Dom’s work as an investigator has focused on AIDS pathogenesis and how viruses enter cells, but more recently his lab has also studied West Nile Virus and other emerging pathogens. He has won a number of awards for his work, including the Elizabeth Glaser Scientist Award from the Pediatric AIDS Foundation.

In his role as the new Chief of Pathology, Dr. Doms plans to work to integrate next generation sequencing (NGS) techniques into the Department of Pathology’s work, a project he calls a “big challenge.” By reducing sequencing time and costs, NGS can help researchers and clinicians to efficiently identify the genetic variants underlying diseases through whole-exome or whole-genome sequencing.

“I am very pleased that Dr. Doms has agreed to join us at CHOP, and we look forward to his contribution to an exceptional Pathology and Laboratory Medicine program,” said Children’s Hospital Chief Executive Officer Steven M. Altschuler, MD. “Bob is internationally known and recognized for his discoveries in HIV/AIDS research, and his reputation for excellence will certainly enhance our efforts at CHOP.”

Before joining Children’s Hospital, Dr. Doms spent more than two decades at the University of Pennsylvania, where he retains a dual appointment.

During her decades-spanning career, Beverly J. Lange, MD, played a number of important roles both at Children’s Hospital and in the scientific community at large. After four decades of leadership at Children’s Hospital — more than 35 of which she spent working on childhood cancers — Dr. Lange retired in FY13.

As part of a celebration of her career, in March Dr. Lange presented a talk called, “Survival on the Yellow Brick Road,” in which she highlighted some of her many accomplishments while touching on her recent work helping to better understand and alleviate the myriad side effects of cancer treatment.

Dr. Lange worked on many aspects of pediatric oncology over her career, including groundbreaking work on acute myelogenous leukemia (AML). While AML is the second most common form of leukemia in adults, the disease is relatively rare in children, with only 500 to 600 children diagnosed each year.

AML survival rates have greatly increased over the last several decades, with the 5-year survival rate now around 85 percent.

Dr. Lange’s research interests also focused on how cancer therapy impacts pediatric patients’ cognitive function. Cancer-related cognitive dysfunction (CRCD), also known as “chemo brain,” can lead to learning and memory deficits as well as problems later in life. CRCD is therefore particularly worrisome in young children who are still developing,

Dr. Lange has advocated for improved CRCD assessments as well as more feasible trials to better understand the effects of CRCD, and encouraged clinicians and investigators to make CRCD a priority for families.

In addition, Dr. Lange had an interest in the mitigation and prevention of other effects of cancer treatment. For example, she and her colleagues recently completed a trial aimed at preventing hearing loss associated with treatment with cisplatin, a drug used to treat a variety of cancers and which can cause a number of side effects in addition to irreversible hearing loss.

In honor of her work and many accomplishments in pediatric cancer, Dr. Lange was honored in July 2012 with the “Pitcher of Hope” award during an Alex’s Lemonade Stand Foundation (ALSF) fundraising event held. The “Pitcher of Hope” award is presented annually to a Children’s Hospital professional who shows extraordinary commitment to caring for children with cancer.

In addition to her role at Children’s Hospital, Dr. Lange held a number of prestigious appointments outside CHOP over the years, including leadership roles in the American Society of Pediatric Hematology and Oncology and membership in the Children’s Oncology Group. She has also received a host of awards and honors, including a 2006 Temple University Alumni Achievement Award, as well as a lifetime achievement award from the Children’s Oncology Group in 2008.

Dr. Lange, who now splits her time between Philadelphia and Venice, Italy, called her work with cancer and leukemia “the love of my life.”

Three decades of innovative research led the Council for the Advancement of Nursing Science to honor nurse-scientist Barbara Medoff-Cooper, PhD, RN, FAAN, with its Outstanding Nursing Scientist Award.

The Council is the country’s leading scientific organization for nurse-scientists. The award acknowledges Council members whose research studies have had significant impact on nursing and healthcare knowledge and practice.

Dr. Medoff-Cooper is a nationally and internationally known expert on developmental outcomes, feeding behaviors and infant temperament in high-risk infants. She has received six grant awards from the National Institutes of Health to support her research, including her latest study that uses state-of-the art telehealth technology for in-home monitoring of infants who underwent neonatal cardiac surgery.

“This ongoing study has the potential to change the paradigm of home monitoring care for extremely vulnerable populations,” said Dr. Medoff-Cooper, who has published more than 70 journal articles and is the co-inventor of Neonur, a feeding device allowing researchers and clinicians to objectively measure feeding behaviors in neonate and young infants.

Her work extends outside of patient care and includes participation in many NIH review groups and serving as the chair of a national task force to develop protocols for the care of preterm infants.

Dr. Medoff-Cooper holds the Ruth M. Colket Endowed Chair in Pediatric Nursing at CHOP, the first endowed chair for nursing in a children’s hospital. She also serves as a professor at the University of Pennsylvania School of Nursing and previously served as the director for the Center for Nursing Research at Penn.

A pediatric oncologist with the Hospital’s Cancer Center received a national award highlighting his lifetime research on neuroblastoma, the most common solid tumor of childhood. The American Society of Clinical Oncology conferred one of its highest awards on Garrett M. Brodeur, MD, who received the Pediatric Oncology Award and delivered the Pediatric Oncology Lecture.

The Award and Lecture, held during the ASCO annual meeting in Chicago, recognizes “outstanding scientific work of major importance to the field of pediatric oncology” during the course of a career.

A cancer of the peripheral nervous system that typically appears as a tumor in a child’s abdomen or chest, neuroblastoma varies greatly in severity, ranging from forms that spontaneously disappear to high-risk subtypes that are difficult to cure. Because of this variability, researchers have sought ways to predict the course of disease in order to select the most appropriate treatment for each patient. The underlying assumption of this approach is that better understanding of the biology of this form of cancer will allow pediatric oncologists to avoid undertreating or overtreating a child.

Over his career, Dr. Brodeur has focused on identifying the genes, proteins and biological pathways that give rise to neuroblastoma and drive its clinical behavior. He also has built on this knowledge to develop more effective and less toxic treatments for children by targeting specific pathways.

His research first demonstrated in the 1980s that when neuroblastoma cells developed multiple copies of the MYCN gene — a process called amplification — a high-risk subtype of neuroblastoma occurs, requiring more aggressive treatment. This discovery ushered in the current era of genomic analysis of tumors, both in adult and pediatric oncology. Profiling specific molecular alterations in a given patient’s tumor helps oncologists to predict that patient’s outcome and select the most appropriate treatment.

Dr. Brodeur and his colleagues also identified deletions of important genes on chromosomes 1 and 11 as markers of high-risk neuroblastoma. He has collaborated with other CHOP investigators who identified the ALK gene as the gene responsible for most cases of hereditary neuroblastoma.

Another major focus of his research has concerned receptor tyrosine kinases, a family of signaling proteins that control the clinical behavior of neuroblastomas. His preclinical work led to a clinical trial with a novel drug that selectively blocks TRK signaling. He is now working on second-generation TRK inhibitors, as well as on nanoparticle delivery systems to treat patients more effectively, and with less toxicity.

Dr. Brodeur has been a member of the CHOP medical staff since 1993 and holds the Audrey E. Evans Endowed Chair in Pediatric Oncology at the Hospital. He also is a professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania, where he is an associate director of the Abramson Cancer Center.

Chromosome 22q.11.2 deletion syndrome is a congenital disorder that occurs when a portion of the DNA on chromosome 22 is missing. The loss of genetic material has multiple effects, which may include abnormalities in the immune system, the heart, the endocrine system, facial features, and cognitive abilities.

Over the years, researchers have found that deletions on this section of chromosome 22 are an underlying cause of various clinical diagnoses, known by such names as DiGeorge syndrome, velocardiofacial syndrome, and conotruncal anomaly face syndrome.

In recognition of her years of work on chromosome 22 deletions, Donna McDonald-McGinn, MS, CGC, received the Angelo DiGeorge Medal of Honor.

The Angelo DiGeorge Medal recognizes outstanding contributions to understanding and/or treatment of chromosome 22q.11.2 deletion syndrome, a relatively common multisystem genetic disorder. The International 22q11.2 Deletion Syndrome Consortium established the award in 2010 to commemorate the life and work of the late Dr. DiGeorge, a pediatrician who described aspects of the syndrome nearly 50 years ago.

Ms. McDonald-McGinn, associate director of Clinical Genetics and program director of the Hospital’s “22q and You” Center, is only the second person to receive this highly esteemed honor.

In addition to publishing more than 80 articles on this deletion syndrome, she has served as a tireless advocate for children and families, and has spent countless hours working on support and educational events related to this condition.

While presenting the award, Dr. Peter Scambler of Great Ormond Street Hospital for Children in London praised her “singular breadth of achievement and dedication.” In particular, he singled out her recent work co-authoring an important scientific article that presents best practice recommendations for patients with this syndrome.

Since its inception in 1988, the Hospital’s Endowed Chair program has supported pioneering investigators working to understand and treat childhood diseases. Named for an honoree, endowed chairs provide crucial, assured funding to more than 100 CHOP investigators.

Center for Outcomes Research director Jeffrey Silber, MD, PhD, was named the first Nancy Abramson Wolfson Endowed Chair in Health Services in FY13. A pediatric oncologist, cancer survivorship researcher, and health care economist, Dr. Silber is the first researcher to hold this endowed chair.

The new Endowed Chair is named for longtime Children’s Hospital Board of Trustees member Nancy Abramson Wolfson, who has served as the chair of the Hospital’s annual fundraising Daisy Day Luncheon for the past 15 years. Since its inception, the luncheon has raised more than $14 million to support care and research at CHOP, and the 2013 Daisy Day Luncheon raised more than $1.5 million to support Children’s Hospital’s Pain Management Program.

The Endowed Chair was created to honor Nancy Abramson Wolfson’s contributions, and to ensure that the Hospital will be able to continue to recruit and support top researchers and pediatricians like Dr. Silber.

An authority on outcomes measurement, Dr. Silber created the Failure-to-Rescue quality of care measures, which were subsequently adopted by the National Quality Forum, a nonprofit organization that works to improve healthcare.

Dr. Silber also leads the Hospital’s Center for Outcomes Research, which aims to improve healthcare by using healthcare outcomes research and by teaching and mentoring clinicians. The Center has been instrument in looking at the impact of restricting residents’ work hours on errors and quality of care, obesity and surgical outcomes, and the effect obstetric unit closures have had on pregnancies.

More recently, Dr. Silber has contributed to studies of race and obesity in operative procedure length, and to an investigation of the association between acute kidney injury and obesity.