Appendix A: Thumbnail Sketches of Key CPMCRI Investigators and Clinical Research Programs
California Pacific Medical Research Institute
Key CPMCRI Investigators and Clinical Research Programs
Approximately 60 principal investigators, both laboratory and clinical researchers, including molecular
biologists, immunologists, pharmacologists, biochemists, physicists, epidemiologists, behavioral
scientists, biostatisticians, and computer scientists work within the Research Institute and the Medical
Center. Biomedical research is conducted in such diverse areas as aging, arthritis, epilepsy, diabetes,
neurobiology of pain, cardiovascular disease, osteoporosis, organ transplantation, mechanisms of drug
addiction, neurodegenerative diseases (e.g. amyotrophic lateral sclerosis), cancer, AIDS, hepatitis and
other infectious diseases.
Laboratory Based Research Programs
Cancer: Brain Tumors
Principal Investigator: Charles Cobbs, M.D.
Liliana Soroceanu, M.D., PhD
Dr. Cobbs’ laboratory focuses on understanding the molecular mechanisms that lead to the
development and progression of malignant gliomas, which are the most lethal form of brain cancer,
often killing patients within months of diagnosis. Recent results from Dr. Cobbs’ work have identified
the human cytomegalovirus (CMV) in tissue samples from patients with malignant gliomas. While CMV
normally causes a common and harmless infection in humans, it may also “push” precancerous cells
into an unstable state and thus promote tumor formation. Another example of cancer-promoting
viruses comes from the human papilloma virus (HPV) which has been shown to cause cervical cancer.
The studies focus on deciphering the pathways that “reactivated” CMV (which may exist dormant
within the brain for decades) to drive tumor growth. When the CMV link to cancer is indeed proven,
Dr. Cobbs will investigate the possibility of using anti-viral agents for treatment of glioma patients,
which would offer new hope for an otherwise devastating disease.
Cancer: Culturing Cells for Targeted Breast Cancer Drug Development
Principal Investigator: Shanaz Dairkee, PhD
In researching life-threatening diseases such as cancer, human experimentation is unethical and
impractical. Thus scientists rely heavily on small laboratory-bred animals, mainly mice and rats for
testing hypotheses related to the development of cancer, and strategies for its prevention and
treatment. Although animal ‘model’ systems have indeed played a key role in advancing our
knowledge, due to significant differences in the biology of humans and rodents, improved
experimental systems are necessary, wherein the application of new information towards cancer
control in patients can be validated. In this regard, getting cancer cells from clinical samples to survive
and proliferate outside the body is recognized as a major technical hurdle. This obstacle has been
overcome in the Dairkee laboratory by novel approaches, such as simulation of the tumor
microenvironment in Petri dishes. Moreover, using genetic engineering techniques, they routinely
generate an unlimited supply of patient tumor cells for comprehensive studies. Focusing primarily on
human breast cancer cells, detailed molecular analyses are being conducted in the laboratory to
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compare them with normal, healthy breast cells from cancer-free individuals as well as between
tumors with markedly different degrees of aggressiveness. They anticipate that the characteristics of
cultured cancer cells will assist in identifying new approaches for biologically targeted drug
development. Their human tumor model systems are also providing information about which drugs
work in different types of breast cancer and which don’t, and are therefore a rich testing ground for
new, potential pharmacologic therapies.
Molecular comparisons of cells in live clinical samples have also facilitated their studies of the very
early stages of breast cancer. Here their approach has been to first expose non-cancerous breast cells
to known carcinogens and then evaluate whether genetic changes that occur over successive
generations reflect those observed in cancerous tissue. It was through studies like this, for example,
that they found non-malignant precursor cells that mirror genetic anomalies found in nearby malignant
breast cells. In an extension of this work, they are presently studying the biological response of live
non-malignant breast cells isolated from women residing in California counties with high breast cancer
incidence, to determine whether estrogen-like agents in the environment are a potential risk factor.
Among their future goals, an opportunity to apply breakthroughs in breast cancer model system
development to other tumor types is a high priority.
Cancer: Breast Cancer Disease Progression
Principal Investigator: Pierre Desprez, Ph.D.
The Desprez research group’s goal is to understand the reasons why some cancers remain localized,
and others spread (metastasize), a step of cancer progression generally fatal for cancer patients. This
group is particularly interested in breast cancer and they have isolated some important genes
(conductor genes) that regulate breast cancer progression to metastasis. They have determined that
some of these "conductor genes" can serve as diagnostic as well as prognostic markers for cancer
progression in women with breast cancer. In addition, they were also able to inhibit metastasis in preclinical models in the laboratory by blocking the expression of these genes. The lab now focuses on
discovering new drugs and reagents that can also block the same genes and therefore the spread of
metastatic cancers in human patients.
Cancer: Breast Cancer Disease Progression
Principal Investigator: John Muschler, Ph.D.
In breast cancer the cells grow as disorganized masses, attachment process is often absent, and growth
control is not regulated. Specific molecular information on breast cell attachment is one potential key
to controlling disease progression, and even for finding ways to prevent breast cancer. Dr. Muschler
recently discovered that a molecule called "dystroglycan" (DG) is important for cell attachment and
signaling growth arrest in breast cells. It appears that dystroglycan is non-functional in the majority of
breast tumor cells. Most importantly, introducing dystroglycan function into breast cancer cells can
restore many normal properties. In the early stages of breast cancer, there is considerable evidence
that restoring critical attachment function will serve to reverse the disease. Evidence indicates that loss
of DG function is one part of cancer progression. Nothing is currently known about how dystroglycan
functions in normal breast cells and how its function is undermined in cancer cells. Dr. Muschler’s lab
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studies: (i) how dystroglycan acts in normal breast cells; and (ii) which functions of DG are critical to
activate its tumor suppressor function. The long-term aim of this study is to understand how
dystroglycan function is undermined in tumor cells, and identify methods to restore DG function to
these cells.
Cancer: Cancer and Cannabinoids
Principal Investigator: Sean McAllister, PhD
We have discovered that non-psychotropic cannabinoid constituents, of the plant Cannabis sativa, can
inhibit the growth of and kill aggressive human breast cancer cells in culture. These compounds can
also inhibit the properties of breast cancer cells that allow them to spread to other parts of the body
(i.e., they can inhibit metastatic properties). An anticancer agent with a low toxicity profile that can
both inhibit cancer cell growth and metastasis would be extremely valuable clinically. Cannabinoid
compounds have a low toxicity profile and have been demonstrated to inhibit a broad range of human
cancers. Dr. McAllister’s laboratory is currently determining the mechanism(s) by which cannabinoids
interact with aggressive breast and brain cancer cells to produce thier anticancer activity.
Understanding the mechanisms behind the anticancer activity of cannabinoids may also lead to the
discovery of a new biological target for the development of diagnostic tools and additional therapies
for the treatment of cancer.
Cancer: Drug Development of Novel Compounds for Improved Treatment
Principal Investigator: Li-xi Yang, M.D.
Metastatic cancers, particularly breast and lung, represent an ongoing treatment challenge. Currently
available standard chemotherapeutic drugs have limited effectiveness on metastatic tumors and carry
significant toxicity for patients. Dr. Yang’s laboratory investigates the therapeutic properties/action at
the cellular level of current chemotherapeutic drugs, with the goal of altering their composition to
develop a second generation drug that may provide a drug with enhanced effectiveness and less
toxicity.
Cancer: Complementary Medicine
Principal Investigator: Garret Yount, Ph.D.
Dr. Garret Yount is exploring how promising complementary and alternative medical therapies might
be integrated with conventional medicine in the treatment of patients with cancer. His laboratory
applies state-of-the-art molecular biology tools to assess the efficacy of Chinese herbs and Qigong
under rigorously controlled experimental conditions. Another focus of Dr. Yount’s laboratory is the
invasive behaviors of cancer cells. Using time-lapse microscopy, he recently discovered that fragments
of cancer cells are able to pinch off from the cell body and migrate independently. He is exploring
whether these independent tumor cell fragments will prove useful as effective delivery systems for
gene therapies because of their ability to mimic tumor cell migration.
Cancer: Gene Therapy
Principal Investigator: Robert Debs, MD
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Despite all currently-available therapies, the great majority of patients who develop metastatic cancer
still die from their disease. Over the last 20 years, it has become clear that the development of specific
genetic abnormalities produce cancers and other common fatal human diseases. Although our
understanding of the genetic causes of human cancer has improved dramatically, the inability to
correct these underlying genetic abnormalities in patients has prevented the development of truly safe
and effective therapies for cancer and other genetic diseases. The research in my laboratory focuses on
the development of safe and effective methods that specifically correct abnormal, cancer-causing
genes directly in patients. The gene therapy approaches we are developing would, if successful,
effectively treat a broad range of human diseases, including cancer, heart diseases, infectious diseases
and neurodegenerative diseases. Several prior approaches developed in the Debs laboratory have
already been translated into gene therapy trials in human patients with cystic fibrosis or with
metastatic cancer. Earlier, an approach developed in his laboratory led directly to the development of
the second drug approved by the FDA for use in AIDS patients, and the first therapy that prolonged life
in these patients.
Cancer: Melanoma
Principal Investigator: Mohammed Kashani-Sabet, MD
Malignant melanoma is the fifth most common malignancy in the United States, and its incidence is
increasingly more rapidly than any other cancer. Dr. Kashani utilizes a large patient data set for analysis
of diagnostic and prognostic factors for patients with melanoma and cutaneous lymphoma to test
novel molecular diagnostic and prognostic factors derived from various laboratory-based analyses that
may either yield independent information regarding diagnosis or prognosis or that may explain the
basis of known factors, such as ulceration and vascular invasion. Understanding the relative impact of
such histologic and molecular factors will result in homogeneous patient groups from which to base
treatment decisions, possibly resulting in different treatment algorithms from those currently in use.
Cell Biology
Principal Investigator: Giuseppe Inesi, MD, PhD
Dr Inesi's laboratory is engaged in a long term research project on Cardiac Molecular and Cell Biology.
This research will clarify the mechanism involved in transport of calcium across cell membranes, as
well as the involvement of these proteins in cardiac failure. Disturbances of calcium distribution in
heart muscle cells is one of the main causes of heart attack and weakened heart muscle. This research
is also contributing to our understanding of how calcium blocking cardiac drugs work, and will open up
the way to development of new pharmacological agents to treat heart disease.
Neurobiology of Disease
Principal Investigator: Dmitri Leonoudakis, PhD
During the aging process, molecular and biochemical changes in the brain lead to impairments in
cognitive and motor function and are major risk factors for neurological disorders such as Alzheimer
disease (AD) and Parkinson disease (PD). The Leonoudakis laboratory is testing the hypothesis that
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dystroglycan (DG) function is altered during aging where it contributes to neuroinflammation,
neurodegeneration and cognitive decline. Loss of functional DG may allow leakage of inflammatory
factors into the brain substance. Other studies in the laboratory have shown that the postinjury
inflammatory response in the brain produces excitotoxicity by excessive signaling in nerve cells
mediated by the inflammatory cytokine tumor necrosis factors.
Immunology: Infection/Host Response and Liver/Pancreas Biorepository
Principal Investigator: Stewart Cooper, MD
The research aim of Stewart Cooper, MD, MRCP, sounds straightforward enough. “We want to
establish the mechanisms of protective immunity against hepatitis C virus infection,” he says. “If we
understand the type of immune response leading to spontaneous clearance of virus, then we can
figure out which part of the immune system to stimulate in order to achieve protective vaccination.”
While this overall research goal has not changed much in 20 years, Cooper is now taking some new
paths to reach that goal. In this next phase of his research, he is directing a new basic liver research
program at California Pacific Medical Center Research Institute Cooper is now laying the groundwork
for several innovative studies to track actual human immune responses to initial HCV infection.
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Clinical Research Programs
San Francisco Coordinating Center
Director: Steven Cummings, MD
CPMRI Investigators: Peggy Cawthon, PhD , Katie Stone PhD, Gregory Tranah PhD, Esther Wei, ScD,
Warren Browner MD, MPH
The San Francisco Coordinating Center uniquely combines scientific expertise with broad experience in
managing multi-center studies, and offers access to a network of high quality, experienced clinical
centers. The mission of the SFCC is to conduct novel clinical research that sets new standards for
excellence and efficiency. A joint venture between CPMCRI and UCSF, the SFCC works selectively with
industry and NIH to develop and conduct studies that have a critical impact on clinical research and
public health. CPMCRI’s investigators at the SFCC are extraordinarily prolific, with more than 40
scientific papers in 2010 alone. Areas of ongoing research at SFCC include women’s health,
aging/longevity, cognitive function/dementia, cardiovascular disease, breast cancer prevention, colon
cancer screening and prevention, osteoporosis, arthritis, endocrine disorders, insomnia/sleep
disorders, genetics, neonatology, and research methods in biostatistics and study design.
Breast Health Study and San Francisco Mammography Registry
Principal Investigator: Steven Cummings, MD
Project Leader: Alice LaRocca
The Breast Health Study is compiling a databank of mammograms and blood samples that, in
combination with new technologies, may help clinicians predict postmenopausal women's risk of
developing breast cancer. All of this will eventually allow clinicians to see if combinations of a women's
breast density, age, family history, hormone levels, and genetics will give us an accurate assessment of
her breast cancer risk. Compounds have become available that may reduce a woman's risk of
developing breast cancer by as much as 70 percent, so it will be critical to develop a personalized
medicine approach to identify which patients may benefit and what treatment they should receive.
Roughly 45,000 CPMC patients per year have had breast-density mammograms since 2005 and more
than 20,000 have given blood samples.
Cardiology:
Principal Investigators: Richard Shaw, PhD and Ernest Haeusslein, MD
Dr. Shaw’s research focuses on the impact of treatment guidelines on quality of care, risk reduction
strategies, and analysis of large datasets to determine risk predictions for different interventions. In
addition to heart transplant research, Dr. Haeusslein studies pharmacological and mechanical
circulatory device therapies to improve end-stage heart disease management.
Gastroenterology: Interventional Endoscopy
Principal Investigators: Kenneth Binmoeller, MD, Janek Shah, MD and Antonio Quiros, MD
Interventional endoscopy allows the gastroenterologist to not only diagnose, but to perform surgical
procedures. A channel within the endoscope provides a conduit to introduce miniature instruments
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for the endoscopist's use during examination and procedures. Miniature endoscopes can be passed
through the channel enabling endoscopic procedures in the bile and pancreatic ducts. Tiny ultrasound
transducers are even used to examine tiny lesions within the gastrointestinal tract. Drs. Binmoeller
and Shah have been on the forefront of clinical research as the first endoscopists to research and
clinically utilize many of the new micro-instruments for detecting and treating disorders. Examples of
device clinical trials they have embarked upon include: tissue sampling devices (brush, forceps and
aspiration needles), needle and wire “knives,” injection catheters, clip, band, suture, and stent
applicators, thermal coagulation probes, balloons and baskets, miniature endoscopes, and miniature
ultrasound catheters.
Gastroenterology: Neurogastroenterology and Motility Disorders
Principal Investigators: William Snape, MD and Katerina Shetler, MD
Approximately 35 million Americans suffer from gastrointestinal motility disorders. These disorders
range from gastroparesis, affecting a small number each year, to common gastrointestinal disorders irritable bowel syndrome (IBS), gastroesophageal reflux disease (GERD), and fecal incontinence.
Motility disorders can involve any part of the gastrointestinal (GI) tract including the esophagus,
stomach, small intestines and colon. Dr. Snape is the Principal Investigator of a nation-wide, federally
funded study with the National Institutes of Diabetes and Digestion and Kidney Diseases. The
Gastroparesis Registry (GpR) is an observational study to clarify the epidemiology, natural history,
clinical course, and other outcomes of gastroparesis. The Gastroparesis Registry will also provide a
resource to inform the development of clinical trials and ancillary studies of the epidemiology, etiology,
pathophysiology, and impact of gastroparesis . Clinical studies By Drs. Snape and Shetler are already
underway which focus on nausea, diabetes control, aspiration risk after extubation, and pharmalogical
interventions.
Gastroenterology: Pediatric
Principal Investigator: Antonio Quiros, MD
Dr. Quiros has an active pediatric research program, examining the immunology, genetic predictors,
and treatment of pediatric inflammatory bowel diseases, and the use of endoscopic devices.
Mind-Body Medicine
Principal Investigators: John Astin, PhD and Cassandra Vieten, PhD
Drs Astin and Vieten's research sutdies how the mind and its attendant thoughts, feelings,
psychological constructs, and levels of stress can impact our physical health. They are studying the
potential value of meditation-based interventions to help prevent relapse from addictive disorders. In
this work, they are testing the theory that by developing the capacity for greater mindfulness, i.e., the
ability to attend to one's present moment experience (thoughts, feelings, sensations, circumstances)
with greater openness and acceptance and in turn less habitual reactivity and judgment, then
recovering addicts will be better able to manage distressing emotional states and thereby lessen their
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habitual tendency to use drugs or alcohol to manage and cope with those powerful emotions or
thoughts when they arise. Other research tests the potential value of mindfulness-based interventions
to reduce stress and negative affect during pregnancy. A large body of data has linked stress during
pregnancy to a host of negative outcomes including greater likelihood of complications during labor
and delivery; higher incidence of post-partum depression; and impaired fetal/infant development. Drs
Vieten and Astin are examining if mindfulness-based interventions can help ameliorate or prevent
some of these problems from occurring.
Dr. Astin, in collaboration with CPMCRI Scientific Director Michael Rowbotham, is studying how to
improve the informed consent process for enrolling participants in human research studies. An ongoing
study compares an interactive consent form presented on an iPad using technology developed by
CPMCRI’s Dr. Steven Cummings with the traditional paper-based method.
Neuroscience: Forbes Norris MDA/ALS Research and Treatment Center
Principal Investigators: Robert Miller, MD, Jonathan Katz, MD and Susan Woolley-Levin, PhD
Drs. Miller and Katz have an international reputation for their leadership in studies of potential
therapies for amyotrophic lateral sclerosis (Lou Gehrig’s Disease). The Forbes Norris MDA/ALS
Research and Treatment Center at CPMC has been a part of essentially every significant study of how
to treat ALS conducted over the past 15 years, including the recent studies of lithium for ALS. Dr. Miller
and Dr. Katz are active in many ALS research consortia: CPMC is one of five hub sites for the MDA-ALS
consortium; CPMC is the western regional leader for PAC-10; Dr. Katz is President of the ALS Research
Group; and CPMC is also active in the Northeast ALS Consortium and the Western ALS Group.
Neuropsychologist Dr. Susan Woolley-Levin is part of the NINDS-funded Common Data Elements
research project for ALS. The CPMC group is active on research into other neuromuscular diseases,
such as myasthenia gravis and muscular dystrophy. Dr. Katz has a particular interest in peripheral
neuropathies and is part of GBS/CIDP International; CPMC is one of 15 centers in the USA. This group
has completed several national collaborative projects focused on immune neuropathy outcomes and
criteria. At any one time as many as a dozen studies are enrolling subjects, with funding from the NIH,
the Muscular Dystrophy Association, and industry.
Neuroscience: Sutter Pacific Epilepsy Program
Principal Investigators: Kenneth Laxer, MD, David King-Stephens, MD and Peter Weber, MD
Dr. Laxer has been involved in epilepsy research since the early 1970’s, playing an active role in the
development of neuroimaging techniques for the evaluation of epilepsy, surgical and non-surgical
temporal lobe epilepsy treatment, and vagus nerve stimulation for seizure control. Together with Drs.
King-Stephens and Weber, the Sutter Pacific Epilepsy Program maintains a comprehensive research
program with 13 active approved protocols ranging from studies of neuromodulation devices, new
anti-epileptic drugs, and brain pathology in patients with epilepsy.
Neuroscience: Pain Clinical Research
Principal Investigator: Michael Rowbotham, MD
Dr. Rowbotham’s research primarily focuses on chronic neuropathic pain, the pain that follows injury
to the peripheral or central nervous system. Current areas of interest include research into the clinical
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mechanisms underlying painful peripheral neuropathy from cancer chemotherapy, pain following
‘shingles’ (herpes zoster and post-herpetic neuralgia), and the transition from acute to chronic pain. Dr.
Rowbotham is also actively involved in the design and conduct of large scale clinical trials of new
analgesics. Dr. Rowbotham plays a leadership role in national and international clinical research and
education initiatives, such as the ACTTION public-private partnership that brings together the FDA,
NIH, academic investigators, and industry to accelerate treatment development by improving clinical
trials.
Neuroscience: CPMC Center for Stroke Research
Principal Investigators: David Tong, MD, Jack Rose, MD, Jeffrey Thomas MD, and Nobl Barazangi, MD
Dr. Tong’s work as an early leader in applying telemedicine to rapid evaluation and treatment of
patients with acute stroke has led to CPMC being a hub site for an NSA-funded telestroke trial,
Advancing Telestroke Care (ATC). Dr. David Tong of CPMC is on the board of the Western States Stroke
Consortium; collaborative projects are a goal of this consortium. Dr. Thomas is involved in several
studies of neurovascular interventions such as stents and other vascular reconstruction devices. As
part of the NINDS-funded national NETT (Neurological Emergency Treatment Trials) program, Dr.
Barazangi has lead CPMC’s participation in several important studies of seizure and stroke
management, such as RAMPART, POINT, and ALIAS. In addition, she and the rest of CPMC’s stroke
group have been involved in industry-sponsored trials of new therapies. Most recently, Dr. Barazangi
initiated a pilot study of her own design (PERFUSE; with support from SPMF and CPMCRI) examining
ways to extend the time window for patients with acute stroke to receive rTPA.
Oncology
Principal Investigators: Ari Baron, MD, Kathleen Grant, MD, Kevin Knopf, MD, Allan Kramer, MD, David
Minor, MD, Bert Tuan, MD, and Wei Wang, MD, PhD
Oncology clinical research at CPMCRI is robust, offering cancer patients entry into pharmaceutical and
biotech sponsored clinical trials as well as National Cancer Institute clinical trials. An oncologist may be
selected to be the Principal Investigator of the study and the others may be listed as co-investigators.
This means all oncologists may enroll into any open study at any time. Oncologists may enroll into any
NCI clinical trial once they have received certification from the NCI and consortiums to allow
participation.
Dr. Minor is the Principal Investigator for the majority of melanoma clinical trials conducted at CPMC.
The melanoma clinical trials complement the basic science research conducted by Dr. Kashani and the
surgical expertise of Drs. Leong and Singer. Combined, this team represents a comprehensive
melanoma program at CPMC. Dr. Minor is an expert in melanoma and a knowledge leader for the
recently approved FDA drug, ipilimumab. He has conducted phase 1 to pivotal phase 3 clinical trials of
ipilimumab and has published extensively in this area. Dr. Minor and Dr. Wang are working with
industry sponsors to move emerging therapies targeting specific genetic mutations into trials for
treating treat melanoma patients. These studies are entering the critical phase 3 process providing
additional optimism for advanced melanoma patients.
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Dr. Baron is the Principal Investigators on numerous clinical trials at CPMC. He has a special interest in
prostate cancer. In the past years, he has focused on prostate cancer of numerous Phase 2 and 3
studies. Participation in these studies has resulted in FDA approval for cabazitaxel (2010) and
abiraterone acetate (2011). As these drugs are approved, he continues to conduct clinical trials as our
industry sponsors work to fine tune dosing and minimize symptoms of newly approved drugs. He also
works with industry sponsors to identify new compounds for clinical research.
Dr. Baron is also the Principal Investigator for cancers of the colon, breast, lung, pancreas, and liver.
Working with surgeons and physicians at CPMC and in the Bay Area, the Oncologists receive numerous
referrals and upon consenting and qualification will enter patients into clinical trials suitable for their
disease. The oncologists work towards establishing a variety of clinical trials for each stage of disease
treatment.
Pharmacology: Addiction & Pharmacology Research Laboratory (APRL)
Principal Investigators: Gantt Galloway, PharmD and John Mendelson, MD
The disease of addiction is produced by interactions between individual biological organisms (people),
specific pharmacological agents (drugs), and the social, economic, cultural, biologic, and geographic
milieu (environment). The Addiction Pharmacology Research Laboratory is dedicated to
understanding how specific pharmacologic compounds (drugs) contribute to developing or treating
addictive disease. We believe that deciphering the biological effects of drugs is essential for developing
effective therapies to treat addiction. We exist to conduct human research on the effects of addictive
and abused drugs and to develop new medications and treatments for drug addiction. We are
dedicated to the safe and ethical human study of addictive and abused drugs, both in controlled
laboratory settings and in people living and functioning in the community. Ongoing research has a
special focus on Methamphetamine and MDMA (Ecstasy) abuse.
Transplant Program: Kidney Transplant
Principal Investigators: Ram Peddi, MD, Parul Patel, MD, and Kimi Ueda, PharmD
Kidney transplant research is integrated into patient care at CPMC. All patients listed for a transplant
are aware of the research program through receipt of research information from the research team
and conversations with the transplant nephrologists regarding clinical trials. Clinical trials may be
conducting in the operating room, immediately post-transplant while hospitalized, or several weeks or
months later to combat a graft-threatening virus. The majority of trials seek to improve
transplantation outcomes with better drug treatments. This team regularly prepares abstracts and
publishes manuscripts to describe innovations, observations and clinical trials they conduct.
Transplant Program: Liver Transplant and Hepatology
Principal Investigators: Maurizio Bonacini MD, Natalie Bzowej MD, PhD, Timothy Davern MD, Todd
Frederick MD, Jennifer Guy MD, Raphael Merriman MD, and Adil Wakil, MD
The Liver Disease Management and Transplant Program promotes clinical research into clinical care.
Areas of research interest include hepatitis B and C disease management, hepatoma, hepatitis and HIV
co-infection, nonalcoholic steatohepatitis, hepatorenal syndrome, pre and post-liver transplant and
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other complex liver diseases. After conducting both Phase 2 and pivotal phase 3 clinical trials, Dr.
Bzowej has recently received recognition with the recently FDA approved hepatitis C virus drug,
telaprevir. which lead to FDA approval. In addition to HCV clinical trials, Dr. Bzowej is a Principal
Investigator for a National Institute of Diabetes and Digestion and Kidney Diseases (NIDDK) trial
entitled, the Hepatitis B Research Network (HBRN). The purpose of this national network is to describe
participants with hepatitis B virus (HBV) infection and identify factors that may cause the disease to
activate or worsen. Dr. Jennifer Guy studies hepatocellular cancer, which is often associated with
chronic viral hepatitis.
Dr. Davern is a nationally-recognized expert on acute liver injury. He is the Principal Investigator of two
NIDDK clinical trials. One is the Drug Induced Liver Injury Network (DILIN). An objective of DILIN is to
develop standardized definitions and instruments to identify and fully characterize cases of druginduced liver injury. With a systematic way of classifying drug-induced liver injury, to enable the
analysis, epidemiology and clinical issues of liver injury and collect biological samples that can be used
to study the causes of liver toxicity using biochemical, serological, and genetic testing. Another
objective of DILIN is to establish a registry of patients who have experienced severe drug induced liver
injury. His second trial funded by the NIDDK is entitled, the Study of N-Acetylcysteine in Acute Liver
Failure (ALF). The purpose of this multi-center open label study is to determine if N-acetylcysteine has
any survival benefits in patients with ALF. Dr. Maurizio Bonacini is co-Investigator on the DILIN Network
grant and is an active investigator in the area of HIV-associated liver disease.
Dr. Merriman also has received funding from the NIDDK to enroll patients into the Nonalcoholic Fatty
Liver Disease (NAFLD) Adult Database 2. The NAFLD Database 2 will recruit at least 600 new adult
participants suspected or known to have NAFLD or NASH-related cirrhosis. The purpose of this study is
to elucidate, through the cooperative effort of a multidisciplinary and multicenter group of
collaborators, the etiology, natural history, diagnosis, treatment, and prevention of NAFLD, and in
particular its more severe form of NASH and its complications.
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