TAG: "Awards & honors"

UC Davis Children’s Hospital ranked among top in nation for quality of care


University HealthSystem Consortium ranks it in top five based on quality of pediatric care.

For the first time, UC Davis Children’s Hospital ranks as one of the top five U.S. hospitals based on the quality of pediatric health care it provides to infants, children and adolescents admitted to the hospital.

A ranking by the University HealthSystem Consortium (UHC) compares UC Davis Children’s Hospital to all other university-based, academic children’s hospitals based on a careful review of information about safety and quality. This top-tier ranking recognizes that teams caring for children at UC Davis work together across a wide range of services, from complicated surgery to intensive care to a brief overnight stay to treat asthma.

Gary Raff, co-chair of the Children’s Hospital Quality and Safety Committee, said, “Our nurses and doctors are working together to put patient care first. Whether it’s something as simple as careful handwashing or as complicated as advanced heart surgery, we’re all committed to quality.”

The rankings address Americans’ increasing awareness that hospitals are not only places of healing, but also of risk for problems such as complications of surgery or serious infections, said JoAnne Natale, co-chair of the Children’s Hospital Quality and Safety Committee.

“Nothing is more important to a family than the swift recovery of their sick or injured child, and every family wants to know their hospital is working hard to prevent such problems,” said Natale.

The federal Agency for Healthcare Research and Quality (AHRQ) has identified eight different measures of problems that might arise for hospitalized children. UC Davis Children’s Hospital is ranked number five on the UHC overall pediatric quality composite indicator, based on these measures from October 2013 to September 2014.

“At UC Davis, we carefully review any quality concerns that emerge, track data, and rapidly undertake changes to provide the highest quality care,” said Natale.

Judie Boehmer, Director of Patient Care Services, said, “All of us listen carefully to parents and other family members who help us understand how they think our care can be improved.”

Boehmer said that UC Davis Children’s Hospital particularly prides itself on the way that all those providing care — nurses, doctors, pharmacists, respiratory therapists and many others — work together as a team focused on providing quality care for every child.

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UCSF professor wins international award for protein research


William DeGrado to receive Stein and Moore Award.

William DeGrado conducts research at his UCSF lab while a colleague looks on. (Photo by Elisabeth Fall)

A UC San Francisco researcher has won a prestigious award from the Protein Society, an international organization dedicated to supporting protein research.

William DeGrado, Ph.D., a professor in the UCSF School of Pharmacy’s Department of Pharmaceutical Chemistry, won the Stein and Moore Award, which is given to leaders in protein science who have made sustained, high impact research contributions to the field.

“It is exciting to me, because it represents recognition from respected peers in my own area of research. It’s an honor to have been selected,” he said. “UCSF’s Peter Walter, Ph.D., has also received this award, although there are many others here who are highly qualified. I am sure we will see other faculty members receive the Stein and Moore in the future.”

DeGrado’s research – spanning decades – focuses on small molecule and protein design as an approach to understanding macromolecule structure and function. He learned that proteins can be rationally designed in a staged modular manner based on simple chemical and conformational principles, and that both de novo and biologically inspired functional elements also can be installed.

His work has also taught us the pitfalls in rational design, and has provided a battery of biophysical and biochemical methods that can be powerfully applied to validate and understand the structure of these designs. His pioneering work has shown that simple chemical principles can be rationally applied to highly complex systems to both understand them and create new materials and potential therapeutics.

The Stein and Moore Award is named for Nobel laureates Dr. William Stein and Dr. Stanford Moore. DeGrado will receive his award at the 29th Annual Symposium of The Protein Society on July 22-25 at Barcelona, Spain.

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UC Davis scientist receives International Sisley-Lejeune Foundation Award


Randi Hagerman honored for her work developing treatments for fragile X syndrome.

By Phyllis Brown, UC Davis

Randi Hagerman receiving the International Sisley-Lejeuen Award 2014 in Paris.

Randi Hagerman, medical director of the UC Davis MIND Institute, has received the prestigious International Sisley-Lejeune Award 2014 from the Paris-based Jérôme Lejeune Foundation, for her groundbreaking work developing targeted treatments for individuals with fragile X syndrome, a leading cause of intellectual disability and the leading single-gene cause of autism spectrum disorder.

The award recognizes significant accomplishments in therapeutic research for Down syndrome or other genetic intellectual disabilities for researchers who have ‘contributed to creating knowledge of these pathologies and the discovery of treatments and cures.’

The Jérôme Lejeune Foundation was founded by Jérôme Lejeune, discoverer of the cause of Down syndrome, which in 1958 he renamed trisomy 21, to accurately describe the genetic abnormality. Hagerman received the award in Paris on March 10, in an event timed to coincide with World Down Syndrome Awareness Day. The award comes with a €30,000, or $33,855 prize, and a lecture.

“I am very positive about new targeted treatments that have the potential to reverse intellectual disability at a variety of ages, including childhood, adolescence and even in adulthood. My work will continue for this very important goal,” said Hagerman, distinguished professor of pediatrics and Endowed Chair in Fragile X Research and Treatment.

Hagerman is one of the world’s leading physician-scientists investigating fragile X-related disorders, including fragile X syndrome and fragile X-associated tremor/ataxia syndrome, or FXTAS, a condition affecting primarily older men that she discovered with her husband, Paul Hagerman, in 2001. In 1984 she founded the National Fragile X Foundation in the United States.

Hagerman said during her acceptance speech that she is hopeful that her ongoing trials of investigational drugs, such as ganaxolone, mGluR5 antagonists, minocycline, and sertraline, eventually may improve language, attention, socialization and learning in people with fragile X syndrome and other genetic conditions, such as Angelman syndrome. This work, she said “leads the way for treatments that may reverse neurobiological abnormalities in autism and many other neurodevelopmental disorders.” These treatments must be coupled with educational programs to ensure that patients may take advantage of their improved cognitive strengths.

Hagerman is director of the MIND Institute’s Fragile X Research and Treatment Center. She is the recipient of numerous honors, including the Jarrett Cole Clinical Award from the National Fragile X Foundation for dedicated service to families in the worldwide fragile X community; the Bonfils-Stanton Foundation Award for her pioneering work in fragile X disorders; and the Distinguished Scholarly Public Service Award from UC Davis. In 2008 she received a Lifetime Achievement Award from the National Fragile X Foundation and, in 2014, she received the C. Anderson Aldrich Award from the American Academy of Pediatrics, for her outstanding contributions to the field of child development.

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Scientist awarded international prize in epilepsy research


Jeanne Paz with UCSF-affiliated Gladstone Institutes to receive prestigious Michael Prize.

By Dana Smith, UC San Francisco

Jeanne Paz, Ph.D., an assistant investigator at the UC San Francisco-affiliated Gladstone Institutes, has been awarded the prestigious Michael Prize, recognizing her significant contributions to the field of epilepsy.

The international prize is given every two years for the best clinical and scientific advancements in epilepsy research and is one of the most highly regarded awards in the field.

Paz will be presented with the award at the International Epilepsy Congress in Istanbul in September.

“I feel very happy to receive this most prestigious international award. It is an immense pleasure when your work is recognized. It encourages me to pursue my work and makes me think I must be doing something right,” said Paz, an assistant professor in the UCSF Department of Neurology. “My peers who received this award are amongst the brightest scientists in the epilepsy field and in neuroscience in general. Therefore, I feel extremely honored to receive this award. I look forward to joining the Michael Foundation family in September.”

The prize celebrates Paz’s discovery that epileptic seizures can be stopped in real-time in rodents using optogenetics — a tool that enables scientists to turn certain cells in the brain on or off with light. Using this technology, Paz identified key neural circuits that can prevent seizure activity.

“Jeanne’s receipt of the Michael Prize is a great achievement, acknowledging her pioneering analysis of the role of circuit mechanisms in epilepsy,” said Lennart Mucke, M.D., director of the Gladstone Institute of Neurological Disease. “We are very proud of her accomplishment.”

Before coming to Gladstone in 2014, Paz was a postdoctoral researcher in the labs of Karl Deisseroth, M.D., Ph.D., and John Huguenard, Ph.D., at Stanford University.

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UC San Diego Health System named one of nation’s best


Truven Health Analytics includes UC San Diego on list of nation’s 100 Top Hospitals.

By Jackie Carr, UC San Diego

UC San Diego Health System was named one of the nation’s 100 Top Hospitals by Truven Health Analytics. This is the third time UC San Diego Health System has been recognized for this prestigious honor.

The Truven Health 100 Top Hospitals study identifies hospitals and leadership teams that provide the highest level of value to their communities based on a national balanced scorecard. The scorecard measures overall organizational performance across 11 key analytic measures including patient care, operational efficiency and financial stability.

“It is an incredible honor for UC San Diego to be named a top performing health system in the country,” said Paul Viviano, CEO, UC San Diego Health System. “This national recognition is the result of the extraordinary devotion of our staff and faculty to achieve excellence for our patients through improved safety, shorter hospitals stays, increased satisfaction, lower costs and demonstrably superior clinical outcomes.”

UC San Diego Health System is listed as a top hospital among 3,000 U.S. hospitals and is recognized among the nation’s most prominent teaching institutions, including Duke University Hospital, Emory University Hospital and Stanford Hospital.

To conduct the 100 Top Hospitals study, Truven Health researchers evaluated close to 3,000 short-term, acute-care, nonfederal hospitals. Risk-adjusted methodologies were used to analyze public information — Medicare cost reports, Medicare Provider Analysis and Review (MEDPAR) data, and core measures and patient satisfaction data from the Centers for Medicare & Medicaid Services (CMS) Hospital Compare reports. Hospitals do not apply, and winners do not pay to market this honor.

“This year’s 100 Top Hospitals represent the highest national standards in hospital care and management today. They set the benchmarks for peers around the country to follow — consistently delivering outstanding quality of care, satisfaction and community value at a reasonable cost,” said Jean Chenoweth, senior vice president for performance improvement and the 100 Top Hospitals program at Truven Health Analytics. “The majority of the 2015 award winners have produced year-to-year performance improvement, as well. This speaks to the consistent focus on excellence by the entire organization and the men and women who serve patients.”

The study shows that if all hospitals in the U.S. performed at the level of this year’s winners:

  • 126,471 additional lives could be saved
  • 108,926 additional patients could be complication-free
  • $1.8 billion in inpatient costs could be saved
  • The average patient stay would decrease by half a day
  • Episode-of-illness expense would be 2 percent lower than the peer average

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Nursing science program ranked among best in West


UC Irvine honored by NurseJournal.org.

UC Irvine’s Program in Nursing Science has been ranked in the top 5 percent of 200 four-year bachelor’s programs in the western U.S. by NurseJournal.org in its annual survey of America’s best nursing schools.

Among comparable programs, it’s 10th in quality, seventh in satisfaction and fifth in value. Overall, it’s the highest-rated nursing program in Orange County.

“These rankings reflect the hard work and investment of our program’s dedicated faculty and staff,” said interim Director Alison Holman, a Robert Wood Johnson Foundation Nurse Faculty Scholar. “We are poised to launch into the next phase of our development with many new, exciting initiatives and programs on our horizon. We have a bright future ahead as we continue to focus on achieving excellence in research and teaching.”

Launched in 2007, the program provides academic and professional education in nursing and offers bachelor’s, master’s and doctoral degrees.

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UC Davis Huntington’s Disease Center receives Level 1 designation


Joins UCLA, UC San Diego as one of 21 centers designated by Huntington’s Disease Society.

By Phyllis Brown, UC Davis

The UC Davis Huntington’s Disease Center, whose compassionate patient care and research expertise have made it a beacon of hope for people with Huntington’s disease throughout Northern California and beyond, has been acknowledged by the national society committed to the values that it so ably upholds  the Huntington’s Disease Society of America — as a Level 1 Huntington’s Disease Center of Excellence.

The acknowledgement recognizes the center’s service to over 300 families coping with Huntington’s disease and its plan to provide both medical and social services to a large number of underserved families coping with Huntington’s disease who are affiliated with Kaiser Permanente in Northern California.

Huntington’s disease is an inherited, degenerative brain disorder for which there is no cure and only one Food and Drug Administration-approved treatment, Xenazine (tetra benazine). The condition slowly diminishes the affected individual’s ability to walk, talk and reason. They eventually become completely dependent upon others for their care.

“The Huntington’s Disease Society of America was impressed with the scope of UC Davis’ plan to provide expert care to the large number of underserved families who use the Kaiser Permanente insurance system,” said Louise Vetter, chief executive officer of the Huntington’s Disease Society of America. “UC Davis has our continued thanks for the center’s support of the Huntington’s Disease Society of America’s mission and all that it does to provide exemplary care to families impacted by Huntington’s disease.”

The UC Davis Huntington’s Disease clinic has been designated as one of 21 Huntington’s Disease Centers of Excellence in the United States since 2001. There are three other centers of excellence in the western United States, at UCLA, UC San Diego and the University of Washington in Seattle, which is also a Level 1 center.

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UC leads nation in NIH biomedical research funds


UC system received $1.8B in 2014 contract and grant funding from NIH.

UC San Francisco and UC Newsroom

The University of California system received more than $1.8 billion in 2014 contract and grant funding from the National Institutes of Health (NIH), making it the leading recipient for high-caliber biomedical research that is driving advances in science and breakthroughs in health.

UC placed three campuses in the top 20 funding recipients and five in the top 50, according to annual NIH figures. UC San Francisco was second overall in NIH funding, and for the second year in a row its four schools — dentistry, medicine, nursing and pharmacy — led the nation in federal biomedical research funding in their fields.

These highly competitive NIH funds enable UC scientists to advance understanding of the underlying causes of cancer, cardiovascular disease, diabetes, HIV, Parkinson’s and Alzheimer’s disease, and others, and work to develop improved therapies for them.

“The projects these funds support at UCSF are driving advances in the biomedical sciences, from fundamental, curiosity-driven science to the application of those discoveries to patient care and populations, to training the next generation of scientists and health care professionals to carry those innovations forward,” said UCSF Chancellor Sam Hawgood, M.B.B.S. “NIH funding is the lifeblood of that research and the heart of efforts by our faculty, staff, students and trainees to collectively transform health.”

The UCSF School of Medicine topped the list of NIH funding for medical schools for the third year in a row, at $480.6 million for fiscal year 2014, supporting research, graduate-student training and fellowships for postdoctoral scholars.

The UCSF schools of pharmacy, dentistry and nursing also ranked first in their fields in NIH grants for 2014: Pharmacy for the 35th consecutive year, with $31.8 million in grants; dentistry for the 23rd year, with $15.5 million; and nursing for the 10th time in the last dozen years, with $10.1 million.

All told, UCSF received more than $538.1 million total in grants, with an additional $8.5 million in NIH contracts, for which UCSF scientists compete to perform specific research projects for the national institutes. Johns Hopkins University was the top organization with $618.6 million in overall NIH funding.

NIH funding not only supports powerful scientific advances in research institutions nationwide, it buoys local and regional economies, as the scientists purchase materials and instruments and employ laboratory staff. Other economic engines include patents and scientific advances generated by NIH-funded research and related industries, such as biotechnology, as well as the entrepreneurs who launch companies based on research conducted on campus. These grants also play a tremendous role in helping innovative young scientists launch their research careers.

“In the past year, our graduate students and postdocs played key roles in discoveries in areas like the human microbiome, cancer immunotherapy and genome editing,” said Elizabeth Watkins, Ph.D., dean of the UCSF Graduate Division and vice chancellor of student academic affairs. “These funds are critical in supporting the early careers of those future scientific leaders, and instrumental in supporting the discoveries they make.”

By the numbers

Here is a UC campus/location breakdown of National Institutes of Health funding for 2014:

  • UC Berkeley: $122.5M
  • UC Davis: $189.3M
  • UC Irvine: $105.3M
  • UCLA: $373.1M
  • UC Merced: $4M
  • UC Riverside: $15.4M
  • UC San Diego: $398.7M
  • UCSF: $546.6M
  • UC Santa Barbara: $18M
  • UC Santa Cruz: $30.1M
  • UC Office of the President: $216,000
  • Berkeley Lab: $26M
  • Livermore Lab: $2.6M
  • Los Alamos Lab: $5.2M

UC system total: $1.8B

Related link:
UCSF schools lead nation again in NIH biomedical research funds

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UC Davis Center for Pain Medicine named Clinical Center of Excellence


Designation is highest recognition that a pain center can receive nationally.

By Charles Casey, UC Davis

The American Pain Society has selected the UC Davis Center for Pain Medicine as a 2015 Clinical Center of Excellence. The award recognizes pain-care teams that provide outstanding, exemplary care for those with chronic pain disorders, acute pain after surgery or trauma, as well as in palliative care settings for pain from cancer and other terminal conditions.

Designation as a clinical center of excellence is the highest recognition that a pain center can receive nationally. UC Davis also was recognized with the honor in 2010.

Known for taking on the most difficult cases of acute, cancer-related and chronic pain in adults and children, UC Davis pain-treatment specialists work with patients and their primary-care clinicians and referring physicians to provide for those suffering from all forms of pain. The Center for Pain Medicine emphasizes a comprehensive, patient-centered approach to care along with innovative research, clinical education and training, and patient advocacy to improve public policy.

“Being named a national center of excellence is truly an ensemble award and a great honor for our program,” said Scott Fishman, professor and chief of the UC Davis Division of Pain Medicine. “The award recognizes the impressive breadth of work UC Davis is doing to help patients cope with and overcome pain. Our pain medicine team is able to provide seamless, evidence-based care, including medical, surgical, psychiatric, psychological, social and alternative forms of pain management.”

Diverse expertise in pain management

The UC Davis team is distinguished from other programs around the country by their diverse backgrounds that bring a wide range of clinical professions and disciplines to bear on the care of patients in pain (including internal medicine, anesthesiology, physical medicine and rehabilitation, psychiatry, addiction medicine, pediatric pain, hospice and palliative care, radiology, psychology, pharmacy, physical therapy, social work, bioethics, acupuncture/oriental medicine and meditation-based stress reduction).  This diversity spills over into their research and advocacy work.

Interprofessional education

One example is the center’s collaboration with the Betty Irene Moore School of Nursing at UC Davis — as well as the Mayday Fund, the Macy Foundation and the Milbank Foundation — to develop pain-education competencies across all health care professional schools. With its goal to increase clinicians’ basic skills in pain management, the initiative has received international recognition.

“Pain is an ideal topic to address from an interprofessional perspective,” said Heather M. Young, associate vice chancellor for nursing and dean of the Betty Irene Moore School of Nursing. “Every health professional comes in contact with individuals and their family members coping with pain. We all need to have a basic understanding of pain so we can provide appropriate care. By working together to develop these basic skills, we gain multiple perspectives on a very complex topic.”

Innovative behavioral pain-care team

Pain management is well integrated within UC Davis Health System’s primary and specialty services in both its hospital and outpatient clinical settings. It is designed to address the broadest possible physical, psychological and social needs of patients. Among the center’s innovative initiatives was the establishment of a behavioral pain-care team led by a psychiatrist and internist, a psychologist and social worker, all of whom work full time within the pain center to offer individualized care as well as group-setting opportunities for cognitive behavioral therapy, resilience and lifestyle education, and general support.

“Every person’s battle with pain is unique,” added Fishman. “The best way to help patients regain control of their lives is to provide comprehensive expertise and services. Effective pain management that increases function and decreases pain requires an individualized-care approach that taps into the wide variety of resources and specialists we have here.”

Sharing pain management expertise with others

UC Davis pain specialists also consider each clinical encounter an opportunity to guide and educate the referring clinical team, including those outside of Sacramento. As an example, UC Davis is in the midst of a special training project for rural Medicaid-based health systems using the university’s renowned telehealth expertise. The program is funded through the California HealthCare Foundation and harnesses video-conferencing technologies to provide “Telementoring” educational opportunities for rural health centers. The goal is to help develop primary-care centers of excellence in remote communities that currently have minimal pain management resources.

“Pain management expertise must not reside only in large urban medical centers,” said Fishman. “We have a long tradition of providing pain education and training throughout our health system as well as sharing that expertise with other health providers and professional organizations. Advancing high quality pain management, locally, regionally and nationally has been a central mission at UC Davis. This national designation as a center of excellence not only reflects the passion of our center for safe and effective pain relief for all, but also highlights a health system that has uniquely committed to interprofessional and multidisciplinary integration to offer real hope for improving quality of life.”

About the Clinical Centers of Excellence Awards Program

The American Pain Society established its Centers of Excellence program in 2006 to showcase progressive teams of health professionals who address critical, and sometimes unmet, needs in pain management. The program also aims to identify U.S.-based, multidisciplinary teams providing distinguished, comprehensive pain care to serve as examples to other pain management programs.

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UC San Diego professor recognized for pioneering gene therapy research


Theodore Friedmann receives prestigious Japan Prize.

Theodore Friedmann, UC San Diego

By Scott LaFee, UC San Diego

Theodore Friedmann, M.D., professor in the Department of Pediatrics at University of California, San Diego School of Medicine was named today (Jan. 29) one of three recipients of the 2015 Japan Prize, a prestigious international award honoring laureates whose “original and outstanding achievements in science and technology have advanced the frontiers of knowledge and served the cause of peace and prosperity for mankind.”

Friedmann is being recognized for his pioneering research and contributions to the development of gene therapy, a new field of medicine which in significant ways originated at UC San Diego. The sponsoring Japan Prize Foundation describes Friedmann as “the father of gene therapy.”

Sharing the 2015 Japan Prize “in the field of medical science and medicinal science” with Friedman is Alain Fischer, M.D., Ph.D., director of immunology at the Necker Hospital in Paris, France. Fischer is credited with demonstrating the clinical efficacy of gene therapy by successfully treating children suffering from a severe genetic disorder that renders them extremely vulnerable to infections.

The third 2015 Japan Prize laureate is Yutaka Takahasi, Ph.D., professor emeritus at the University of Tokyo, who is being honored in the “field of resources, energy and social infrastructure” for his contributions to river basin management and reducing water-related disasters.

Each laureate will receive a certificate of recognition and commemorative gold medal. A cash award of approximately $416,600 will also be given to each prize field. Since its inception in 1985, 83 laureates from 13 countries have received the Japan Prize in a variety of fields and disciplines. Several have subsequently become Nobel Prize laureates as well.

In 1972, Friedmann, then a visiting scientist at the Salk Institute for Biological Sciences in La Jolla, and Richard Roblin, also at the Salk Institute and a postdoctoral fellow out of James Watson’s lab at Harvard University, published a foundational article in the field, a paper in the journal Science under the heading “Gene therapy for human genetic disease?”

Though posed as a question, Friedmann and Roblin firmly believed the answer was yes, citing emergent thinking, new studies and growing data that suggested “good DNA” could be used to replace defective DNA in people with inherited conditions.

“In our view,” they wrote, “gene therapy may ameliorate some human genetic diseases in the future. For this reason, we believe that research directed at the development of techniques for gene therapy should continue.”

Though Friedmann said initial response to the paper was “not overwhelming,” it’s now commonly cited as a major milestone in the scientific beginnings of gene therapy research, though Friedmann said it was the Asilomar conference three years later (scientists set safety standards for recombinant DNA technology) where interest really “exploded.”

The idea of gene therapy, which quickly captured the public imagination, was fueled by its appealingly straightforward approach and what Friedmann has described as “obvious correctness”: Disarm a potentially pathogenic virus to make it benign. Stuff these viral particles with normal DNA. Then inject them into patients carrying abnormal genes, where they will deliver their therapeutic cargoes inside the defective target cells. In theory, the good DNA replaces or corrects the abnormal function of the defective genes, rendering previously impaired cells whole, normal and healthy. End of disease.

It’s not quite that simple, of course, something Friedmann and Roblin had cautioned in their 1972 paper. Despite progress in the understanding of cellular functions, the roles of DNA and a series of experimental and clinical advances, the history of gene therapy has been marked by distinct highs and lows.

In 1968, Friedmann, working at the National Institutes of Health in Bethesda, Maryland, with the late Jay Seegmiller (a founding faculty member of the School of Medicine) and others, showed that by adding foreign DNA to cultured cells from patients with Lesch-Nyhan syndrome, they could correct genetic defects that caused the rare but devastating neurological disorder. The condition was first described by William Nyhan, M.D., a UC San Diego professor of pediatrics, and medical student Michael Lesch in 1964.

The feat was a powerful proof-of-concept, but subsequent efforts to advance the work to human clinical trials stalled. “We began to realize that it would be very complicated to take this idea and make it work in people,” Friedmann said, who joined the School of Medicine faculty in 1969.

In 1990, a 4-year-old girl with a congenital disease called adenoside deaminase (ADA) deficiency, which severely affects immunity and the ability to fight infections, became the first patient treated by gene therapy. White blood cells were taken from her, the normal ADA gene was inserted into them using an engineered and disabled virus and the cells re-injected. Despite initial claims of success, Friedmann said the experiment was eventually deemed a failure. The girl’s condition was not cured, and the research was found wanting.

A report commissioned by National Institutes of Health Director Harold Varmus, M.D., was highly critical of the entire gene therapy field and the ADA effort in particular, chiding investigators for creating a “mistaken and widespread perception of success.” Friedmann says he took the Varmus report “personally. I felt awful. It almost made me feel like I had been deceiving myself and my colleagues for more than two decades about the promise of gene therapy.” But he also knew there were “many more good people doing gene therapy research than rogues” and continued diligently and conscientiously to pursue his own research.

Nonetheless, media attention and hype about gene therapy continued to be rampant, fueled in part by over-enthusiastic opinions by some scientists. Things crashed in 1999 when an 18-year-old patient named Jesse Gelsinger, who suffered from a genetic disease of the liver, died during a clinical trial at the University of Pennsylvania. Gelsinger’s death was the first directly attributed to gene therapy. Subsequent investigations revealed numerous problems in the experimental design.

Friedmann said he has long realized that eventual success of gene therapy could not stop with a good idea and with good laboratory results but would require truly careful, rigorous and imaginative clinical studies. “That’s happened – and the field has moved greatly in the past decade.” Friedmann’s efforts have focused on development of improved virus gene transfer tools. He is pursuing studies on the application of current genetic knowledge and stem cells models to try to understand the basis for Lesch-Nyhan and identify more accessible treatment targets for gene-based and other forms of therapy. He continues to be a staunch advocate for rigorous gene therapy science elsewhere.

“Technology has gotten better. New kinds of viruses, such as the lentiviruses (a more efficient and safer gene delivery vector) were created. Disease models expanded. The science got more rigorous. I think the Japan Prize Foundation’s decision to honor Dr. Fischer and myself is even greater recognition of the field itself, how far it has come and how much promise it holds.”

In recent years, researchers in Europe and elsewhere have reported successfully treating children with SCID, a dramatic genetic disorder characterized by the lack of an immune response. These patients are extremely vulnerable to infectious diseases; previously, children with the condition had to live in highly sterile environments to avoid exposure to life-threatening infections or to undergo bone marrow transplantation. Friedmann noted that clinical trials for other conditions, such as forms of blindness, degenerative brain diseases, hemophilia and some metabolic diseases, are also proving effective and evolving rapidly into important treatments.

He added that the basic concept gene therapy – genetic correction – is now being expanded to include even more definitive methods to correct the abnormal spelling of mutated, disease-causing genes – an approach called “genome editing.”

“We’re well past the stage of having to prove the concept of gene therapy and have finally overcome our history of perhaps promising too much too soon. We’re at the point where we can truly begin to deliver real treatments to real people.”

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UCSF professor wins Vilcek Prize in Biomedical Sciences


Peter Walter honored for making major contributions to science as an immigrant.

Peter Walter, UC San Francisco

By Pete Farley, UC San Francisco

Peter Walter, Ph.D., professor of biochemistry and biophysics, has received the 2015 Vilcek Prize in Biomedical Sciences, which recognizes major contributions to science made by immigrants to America.

Walter, professor of biochemistry and biophysics, was born in West Berlin, Germany, during the Cold War era. He joined the UCSF faculty in 1983. A Howard Hughes Medical Institute investigator since 1997, Walter was honored for two main achievements. In the 1980s, while working in the Rockefeller University laboratory of Günter Blobel, Ph.D., Walter discovered a molecular apparatus in cells called the signal recognition particle, which facilitates the transport of newly minted proteins across a cellular compartment called the endoplasmic reticulum by homing in on address tags on the proteins.

Since establishing his own lab at UCSF, Walter has done groundbreaking work on a cellular quality-control system known as the unfolded protein response, or UPR. Found in organisms ranging from yeast to humans, the UPR is crucial to life, and disruptions in its workings are believed to play a role in neurodegenerative diseases, cancer, diabetes and other illnesses.

Last year, Walter was the recipient of both the Shaw Prize in Life Science and Medicine and the Albert Lasker Basic Medical Research Award.

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UCSF professor wins NAS award for scientific discovery


Jonathan Weissman honored for helping develop a technique called ribosome profiling.

The National Academy of Sciences has bestowed UC San Francisco’s Jonathan Weissman, Ph.D., its the inaugural NAS Award for Scientific Discovery – presented in the field of chemistry, biochemistry or biophysics.

“I was thrilled and deeply honored when received word of the award,” said Weissman, a professor in the Department of Cellular and Molecular Pharmacology, and a Howard Hughes Medical Institute investigator. “I think the award reflects the growing appreciation of the critical role of translational control and the ability of the ribosome profiling approach to monitor translation with unprecedented precision.”

In 2009, Weissman and colleagues at UCSF developed a technique called “ribosome profiling.” This method allows researchers to sequence chunks of messenger RNA (mRNA) that ribosomes are decoding, giving a snapshot of the genes being translated within a cell.

First applied to yeast, ribosome profiling has been since been extended to many other organisms, including humans. It has been used to identify new proteins and peptides, investigate the process of translation, measure gene expression in cells and determine rates of protein synthesis. In addition, Weissman and his team have employed ribosome profiling to make important insights into the critical role that protein synthesis plays in cell growth and differentiation.

The NAS Award for Scientific Discovery is awarded every two years to recognize an accomplishment or discovery in basic research within the past five years. The fields of science for each presentation will rotate from among chemistry, biochemistry, biophysics, astronomy, physics, and materials science. Endowed in 2014 through a gift from the Research Corporation for Science Advancement (RCSA) in honor of John P. Schaefer. This award is presented with a medal, $50,000 cash prize, and $50,000 to support the recipient’s research.

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