About the Tau Consortium
The Tau Consortium is a collaborative research program that is managed and funded by the Rainwater Charitable Foundation in partnership with other funders.
We commission world-class research and drug discovery to treat and prevent primary tauopathies like progressive supranuclear palsy, as well as secondary ones such as Alzheimer’s disease. Our strategy is to engage and support talented researchers who:
- have a proven track record and/or high potential for high impact research in neurodegeneration
- are dedicated to using their research to produce tangible benefits for patients
- are willing to work collaboratively across disciplines and institutions, and
- can engage with other partners who are helping to accelerate our shared progress.
We always act with urgency and with the patient in mind.
With about $100 million invested to date, the Rainwater Charitable Foundation (RCF) is a leader in the field of Tau research. Since founding the Tau Consortium in 2009, RCF provided about 65% of all US philanthropic funding for tau-related research, including 75% of all US philanthropic funding for treatment development.
Major advances have already been achieved. In just a few short years, the Tau Consortium has identified a key risk gene involved in tauopathies, discovered radically new disease mechanisms, and developed stem cell and animal models to enable drug discovery efforts. The Tau Consortium formed several exciting partnerships to drive additional advances in genetics, next generation diagnostics, and clinical trial methodologies.
Most importantly, we have helped to put eight treatments into human trials and are working on nearly two dozen others at earlier stages in the pipeline.
Increasingly, the Tau Consortium is recognized as the leading treatment effort in the world focused on tau aggregation in the brain.
What We Do
The Tau protein is present and has important functions in the brains of healthy people, but its abnormal build-up seems to be an important characteristic of Alzheimer’s disease, progressive supranuclear palsy, chronic traumatic encephalopathy, and more than 20 other serious neurodegenerative diseases. With its normal function unclear, and its dysfunctional state a consistent component of several neurodegenerative diseases, understanding Tau will bring us closer to effective treatments for patients suffering from these diseases.
Tau is a protein found in neurons, where it is thought to stabilize the shape and function of the cell. As part of normal function, proteins in the body fold, unfold and refold into different shapes in order to perform specific functions. When these proteins are no longer needed, they are broken down and recycled by the cell. In many neurodegenerative diseases, the normal folding and unfolding stop, and the misfolded protein begins to accumulate. This tau accumulation is thought to disrupt normal cellular function and possibly lead to neuronal death.
Our goal at the Tau Consortium is to establish and foster collaborative networks that seek to improve our understanding of tauopathies and develop effective treatments for patients.
In addition to our funded research programs, special initiatives, and strategic partnerships, we are in the early stages of planning an international conference open to everyone.
Additionally, The Rainwater Charitable Foundation launched its first ever prize program. Nominations began in November 2018 with an aim to encourage and accelerate scientific progress toward new treatments for neurodegenerative diseases. Click here to learn more about the Rainwater Prize Program.
Meet the Investigators
Click on the investigators below to learn more about their contribution to the Tau Consortium.
Michelle Arkin, PhD
Randall Bateman, MD
Boxer, MD, PhD
Adam Boxer, MD, PhDWebsite Papers
Dr. Boxer received his MD and PhD degrees as part of the NIH-funded Medical Scientist Training Program at New York University Medical Center. He completed an internship in internal medicine at California Pacific Medical Center and a residency in neurology at Stanford University Medical Center. He completed a fellowship in behavioral neurology at University of California, San Francisco and is a Professor of Neurology at the UCSF Memory and Aging Center.
Dr. Boxer directs the UCSF Memory and Aging Center’s Clinical Trials Program for Alzheimer’s disease and frontotemporal dementia (FTD). His observational research focuses on developing biomarkers for normal aging, mild cognitive impairment, Alzheimer’s disease, frontotemporal lobar degeneration, progressive supranuclear palsy (PSP), and corticobasal degeneration. He designed and led the first US multicenter, randomized, placebo-controlled, clinical trial of a therapeutic agent for frontotemporal dementia (memantine/Namenda®) and an international, phase 2/3, randomized, placebo-controlled trial of the microtubule stabilizing agent, davunetide (NAP, Al-108), for PSP. Dr. Boxer is a recipient of the Edwin Boldrey Award from the San Francisco Neurological Society, the John Douglas French Foundation Alzheimer’s Award, a Hellman Foundation Scientist Award and the Alzheimer’s Association’s Part the Cloud Award for Translational Research.
Giovanni Coppola, MDWebsite Papers
Dr. Coppola received his MD and completed his neurology residency at the Federico II University, Napoli, Italy, and is currently an Associate Professor of Psychiatry and Neurology at the University of California Los Angeles. The long-term goal of his work is to advance the understanding of the genetic architecture of neuropsychiatric disorders by adopting a combination of genetic (sequencing, genotyping), genomic (gene expression, epigenetics), and bioinformatic (integrative network analysis) approaches. As Director of the UCLA Semel Center for Informatics and Personalized Genomics he is interested in setting up an infrastructure for large-scale genomic studies and research access to the electronic medical record system.
Crary, MD, PhD
John Crary, MD, PhD
Cuervo, MD, PhD
Ana Maria Cuervo, MD, PhDWebsite Papers
Dr. Cuervo is co-director of the Einstein Institute for Aging Research. Her laboratory at Albert Einstein College of Medicine studies the role of protein degradation in aging and age-related disorders, with emphasis in neurodegeneration. Dr. Cuervo’s group is interested in understanding how altered proteins can be eliminated from the cells. Her group has linked alterations in lysosomal protein degradation (autophagy) with different neurodegenerative diseases including Parkinson’s disease, Alzheimer’s disease, and Huntington’s disease. They have also proven that restoration of normal lysosomal function prevents accumulation of damaged proteins with age, demonstrating this way that removal of these toxic products is possible.
Dr. Cuervo is considered a leader in the field of protein degradation in relation to biology of aging and has been invited to present her work in numerous national and international institutions. She has organized and chaired international conferences on protein degradation and on aging, belongs to the editorial board of scientific journals in this topic, and is currently co-editor-in-chief of Aging Cell and associate editor of Autophagy. Dr. Cuervo has served in NIH advisory panels, special emphasis panels, and study sections, and has been recently elected member of the NIA Scientific Council. She has received the 2005 P. Benson Award in Cell Biology, the 2005/8 Keith Porter Fellow in Cell Biology, the 2006 Nathan Shock Memorial Lecture Award, the 2008 Vincent Cristofalo Rising Start in Aging Award, the 2010 Bennett J. Cohen Award in Aging Biology, and the 2012 Marshall S. Horwitz, MD Faculty Prize for Research Excellence.
Marc Diamond, MDWebsite Papers
Dr. Diamond graduated from Princeton University in 1987 with a bachelor’s degree in history. He entered the UCSF School of Medicine in 1987 and subsequently completed two years of research under Dr. Keith Yamamoto as a Howard Hughes Medical Student Research Fellow. He graduated from UCSF in 1993. Dr. Diamond completed an internship and residency in neurology at UCSF in 1997 and was then a postdoctoral fellow in the laboratory of Dr. Yamamoto until 2001. He joined the faculty of the Department of Neurology at UCSF from 2002–2009, before moving to Washington University in St. Louis in 2009, as the David Clayson Professor of Neurology. He joined the faculty of UT Southwestern Medical Center in 2014 as the founding director of the Center for Alzheimer’s and Neurodegenerative Diseases.
Dr. Diamond’s laboratory is focused on basic research to identify therapeutic targets and develop small molecules to treat human disease. His group is especially interested in targeting abnormal protein conformational change, which plays a key role in neurodegenerative diseases such as Huntington disease (HD) and tauopathies. The tauopathies constitute a large family of neurodegenerative diseases that include dementias such as Alzheimer disease, motor neuron diseases, and progressive supranuclear palsy. All of these diseases feature deposition of aggregated tau protein and exhibit inexorable spread of pathology. Dr. Diamond’s work on tau suggests that the aggregates are not static within the cell. Rather, aggregates are taken up by vulnerable cells where they can trigger fibrillization of endogenous, natively folded protein. The newly formed aggregates are capable of moving to neighboring cells to spread pathology. Dr. Diamond’s lab is determining the molecular basis of these processes, and what their role is in vivo. These prion-like mechanisms of pathology represent a potentially new paradigm in our understanding of neurodegenerative diseases and could enable a host of new therapeutic strategies based on blocking propagation of misfolding.
Matthew Disney, PhDWebsite Papers
Dr. Disney graduated from University of Maryland with a BS degree in chemistry in 1999. He went on to receive a MS degree in chemistry and a PhD in biophysical chemistry at University of Rochester. Dr. Disney completed his postdoctoral training at Massachusetts Institute of Technology and at Swiss Federal Institute of Technology. He was an assistant professor at University of Buffalo before starting his laboratory at The Scripps Research Institute of Florida.
Dr. Disney’s group develops rational methods to target RNA with small molecules. Dr. Disney is especially interested in the development of computational tools to target RNAs based on their sequence and using multi-dimensional combinatorial screening of small molecule and RNA libraries to understand chemical and structural spaces that confer binding privileges. Understanding of the interplay between RNA structure and small molecule recognition of those structures allow rational design of novel small molecules to target specific RNA sequences implicated in human disease. Dr. Disney’s lab uses such methods to design novel antibacterial compounds and small molecules to target diseases such as myotonic dystrophy. Their recent work also focuses on targeting RNA that encode potentially toxic tau protein.
Karen Duff, PhD
Bess Frost, PhD
Dr. Frost obtained her B.S. degree in biochemistry and molecular biology from the University of Texas, Austin in 2004. She went on to earn her Ph.D. from the University of California San Francisco in the laboratory of Dr. Marc Diamond. As a graduate student, Dr. Frost pioneered work that ignited a now prominent area of research, which is that tau adopts prion-like characteristics that help explain its pathological spread through the brain and the diverse disease phenotypes of the human tauopathies. Dr. Frost performed her postdoctoral training at Harvard Medical School in the laboratory of Dr. Mel Feany, where she developed a multi-system approach to studying tauopathy, interweaving studies in Drosophila, mice and postmortem human brain tissue. Dr. Frost began her independent laboratory as an Assistant Professor at the Barshop Institute for Longevity and Aging Studies, the Glenn Biggs Institute for Alzheimer’s and Neurodegenerative Disorders, and the Department of Cell Systems and Anatomy at the University of Texas Health San Antonio in 2015.
The research focus of Dr. Frost’s laboratory revolves around the basic neurobiology connecting toxic forms of tau to neuronal death and dysfunction. Specifically, her group has found that the detrimental effects of pathogenic tau on nuclear and genomic architecture activate “jumping genes” and disrupt RNA trafficking. Through this work, Dr. Frost and her team have identified novel targets for therapeutic treatment of tauopathies, as well as compounds that interfere with these processes and suppress tau-induced neurotoxicity. Dr. Frost’s honors include an AFAR New Investigator in Alzheimer’s Disease award and designation as a “Rising STAR” in the University of Texas system.
Li Gan, PhDWebsite Papers
Dr. Gan received a bachelor’s degree in physiology from China’s Peking University and a PhD in cellular and molecular physiology from Yale University School of Medicine. She conducted postdoctoral research at Yale University School of Medicine, Harvard Medical School, and the Gladstone Institutes before joining the faculty at University of California, San Francisco and Gladstone Institute of Neurological Disease.
Dr. Gan studies the molecular mechanisms behind the loss of functional neurons in neurodegenerative diseases, including Alzheimer’s disease, frontotemporal dementia, and progressive supranuclear palsy. Her lab explores the relationship between the aging of neural circuits, the accumulation of toxic proteins and the subsequent activation of a chronic inflammatory response. Understanding how these processes become dysfunctional in neurodegeneration could lead to new therapeutic strategies. One aspect of Dr. Gan’s research focuses on why toxic proteins accumulate in patient brains. She and her team have discovered new cellular mechanisms that could lead to novel approaches to get rid of the toxic proteins from aging neurons. Dr. Gan’s research also explores stem cell-based regenerative approaches in Alzheimer’s disease—a promising yet highly challenging therapeutic direction. Her study showed that neural stem cells in the hippocampus of mice genetically modified to mimic Alzheimer’s symptoms develop abnormally and integrate poorly into the network of neural circuits. More importantly, Dr. Gan and her colleagues find that they can offset these deficits by manipulating electrical signals via pharmacological approaches. Their research provides important clues to encourage the development of new brain cells in those with Alzheimer’s disease.
Geschwind, MD, PhD
Daniel Geschwind, MD, PhDWebsite Papers
Dr. Geschwind completed an AB degree in psychology and chemistry at Dartmouth College. He obtained his MD and PhD at Yale University School of Medicine in 1991. He then completed his neurology residency and postdoctoral research at University of California, Los Angeles, where he joined the faculty in 1997. Dr. Geschwind holds the Gordon and Virginia MacDonald Distinguished Chair in Human Genetics.
Dr. Geschwind’s laboratory focuses on integrating basic neurobiology, genetics, and genomics with translational studies of human diseases. One area of basic investigation has been in the analysis of transcriptome data where his group has used network biology approaches to understand brain transcriptome organization. Professor Geschwind has also put considerable effort into fostering large-scale collaborative patient resources for genetic research and data sharing. He played a major role in the founding, and has provided continuing scientific oversight, of the Autism Genetic Resource Exchange (AGRE), the largest collection of multiplex autism families in the world. Dr. Geschwind has published over two hundred papers and review articles and serves as an editorial board member of several journals in his field. Dr. He sits on numerous scientific advisory boards, including the NIH Council of Councils, the Executive Council of the American Neurological Association (ANA), and Co-Chairs the neurogenetics section of the Faculty of 1000 Medicine. He received the Derek Denny-Brown Neurological Scholar Award from the ANA in 2004, the Scientific Service Award from Autism Speaks in 2007, and is an elected member of the Institute of Medicine of the National Academies.
Geschwind, MD, PhD
Michael Geschwind, MD, PhDWebsite Papers
Dr. Geschwind received his MD and PhD in neuroscience through the National Institutes of Health’s Medical Scientist Training Program at the Albert Einstein College of Medicine in New York. He completed his internship in internal medicine at University of California Los Angeles Medical Center, his neurology residency at Johns Hopkins University School of Medicine in Baltimore and his fellowship in behavioral neurology at the UCSF Memory and Aging Center (MAC). He is on the UCSF faculty as a Professor of Neurology.
Dr. Geschwind evaluates new patients in the MAC clinic and participates in the continued management and care for these patients in the continuity clinic. He is active in the training of medical students, residents and neurobehavior fellows at UCSF. Dr. Geschwind lectures both nationally and internationally on dementia including rapidly progressive dementias such as prion diseases. He edited the recent American Academy of Neurology (AAN) Continuum edition focused on dementia and is on the AAN’s committee for dementia criteria. Dr. Geschwind’s primary research interest is the assessment and treatment of rapidly progressive dementias, including prion diseases such as Jakob-Creutzfeldt disease (CJD). Dr. Geschwind helped establish an inpatient hospital program for the assessment of rapidly progressive dementias at UC San Francisco, the first of its kind in the country. He helped to run the first ever US treatment study for CJD. He also has an active research interest in cognitive dysfunction in movement disorders, such as Huntington’s disease, corticobasal degeneration, progressive supranuclear palsy, other parkinsonian dementias, and neurogenetic disorders.
Jason Gestwicki, PhDWebsite Papers
Dr. Gestwicki completed undergraduate studies at SUNY Fredonia in 1997 and earned his PhD from University of Wisconsin in 2002. He then performed postdoctoral studies at Stanford University prior to starting his independent group at University of Michigan in 2005. In 2013, his group relocated to the University of California, San Francisco, joining the Department of Pharmaceutical Chemistry and the Institute for Neurodegenerative Diseases.
The Gestwicki Laboratory is interested in how molecular chaperones regulate protein folding and homeostasis. They discover and develop new chemical probes that they use to disrupt chaperone function, revealing how these factors are involved in the triage of misfolded proteins. Misfolded proteins are known to accumulate in many disorders, including neurodegenerative diseases. Thus, by better understanding the roles of molecular chaperones in disease, the Gestwicki group hopes to identify new opportunities for drug discovery. Ultimately, they are working towards rebalancing protein homeostasis as a new treatment for neurodegenerative disorders. In their quest to understand chaperone function, the Gestwicki group primarily uses high throughput screening, molecular design, protein biochemistry and organic synthesis. They are particularly interested in finding ways of perturbing the function of traditionally “undruggable” proteins, by targeting protein-protein interactions and allosteric sites.
Alison Goate, DPhilWebsite Papers
Dr. Goate received her DPhil from University of Oxford and conducted postdoctoral research at Eleanor Roosevelt Institute for Cancer Research in Denver, Institute of Neurology in London, and Imperial College in London. She was a faculty member at Washington University of St. Louis School of Medicine before becoming the Willard T.C. Johnson Research Professor in Neurogenetics at the Icahn School of Medicine at Mount Sinai.
The principal focus of research in the Goate lab is the molecular genetics of neuropsychiatric diseases. The two major research areas are dementia (Alzheimer’s disease and frontotemporal dementia) and substance dependence (alcoholism and nicotine dependence). Research falls within two areas: gene discovery and modeling disease mutations. The lab is using genetic association and exome or whole genome sequencing to identify common and rare risk alleles predisposing to disease. These studies are using both diagnostic phenotypes and correlated endophenotypes to localize risk genes. Genome screens for association have been completed in which several novel candidate regions/genes were identified. Follow up studies have identified specific mutations in several genes that influence risk for disease. Functional studies are underway to determine the mechanisms of disease risk.
Grinberg, MD, PhD
Lea T. Grinberg, MD, PhDWebsite Papers
Dr. Lea Grinberg is a neuropathologist specializing in brain aging and associated disorders. She received her MD and PhD degrees in São Paulo, Brazil and then acquired expertise in neuroanatomy and tridimensional brain reconstruction at the University of Würzburg, Germany. Currently, Dr. Grinberg is an Associate Professor at the UCSF Memory and Aging Center. She was the recipient of the UNESCO-L’Oréal Award “For Women in Science” and the John Douglas French Alzheimer Foundation “Distinguished Research Scholar Award.” She is also the chairwoman of the HUPO Brain Proteome Project.
The Grinberg lab investigates the basic principles and pathogenesis of aging and early stage neurodegenerative disease using postmortem human tissue of normal controls and patients. In her Tau Consortium work, Dr. Grinberg’s team is characterizing brain changes and deposition of abnormal phospho-tau protein in areas involved in sleep regulation in patients with tauopathies.
Stephen Haggarty, PhDWebsite Papers
Dr. Haggarty is an Associate Professor of Neurology at Harvard Medical School, an Associate Neuroscientist at Massachusetts General Hospital, Director of the MGH Therapeutic Screening Core within the MGH Center for Human Genetic Research, Head of the Neuropharmacology Program for the MGH Psychiatry Center for Experimental Drugs & Diagnostics, and a Faculty Member in the Harvard Chemical Biology PhD Program. Dr. Haggarty is also a Senior Associate Member of the Broad Institute of Harvard & MIT. Dr. Haggarty received his PhD from Harvard University in the laboratory of Dr. Stuart Schreiber where he developed innovative strategies for cell-based screening and target identification leading to the discovery of small-molecule probes used by hundreds of laboratories and targets that are currently the focus of active therapeutic programs in oncology and neurology. Dr. Haggarty then joined the Broad Institute as a research Fellow in the Chemical Biology Program before beginning his current appointment on the faculty at Harvard Medical School.
Dr. Haggarty’s research program focuses on the discovery and characterization of novel targets for the treatment and prevention of neuropsychiatric disorders with a focus on memory and mood dysfunction. Central to this mission is an understanding of the molecular, cellular, and circuit level mechanisms of neuroplasticity that enable the human brain to sense, adapt, and respond to everyday and pathophysiological events. With the ability to now generate patient-specific stem cell models, his laboratory has been pioneering methods for the large-scale differentiation of defined neuronal subtypes that can form synapses and regulate gene expression in an activity-dependent manner. In combination with high-content imaging and other functional assays, these human cellular models provide new avenues for understanding the neurobiology of human disease as well as for discovering novel targets and lead compounds for therapeutic development.
Songi Han, PhD
Prof. Songi Han received her Doctoral Degree in Natural Sciences (Dr.rer.nat) from Aachen University of Technology (RWTH), Germany, in 2001. She pursued her postdoctoral studies at the University of California Berkeley sponsored by the Feodor Lynen Fellowship of the Alexander von Humboldt Foundation. Dr. Han joined the faculty at University of California Santa Barbara (UCSB) in 2004, received tenure in 2010 and was promoted to full professor in 2012. She is currently a Professor in the Department of Chemistry and Biochemistry and the Department of Chemical Engineering at UCSB. She is a recipient of the 2008 Packard Fellowship for Science and Engineering, the 2010 Dreyfus-Teacher Scholar Award, the 2011 NIH Innovator Award, the 2015 Bessel Prize of the Alexander von Humboldt Foundation, and the 2018 recipient of the Biophysical Society Innovator Award. Her research group focuses on the development of novel techniques relying on dual electron and nuclear spin magnetic resonance that enables the study of biomolecular structure, dynamics and association with unprecedented sensitivity, resolution and information content.
David Holtzman, MD
Dr. Holtzman attended the honors program in medical education at Northwestern University receiving his BS degree in 1983 and MD in 1985. He completed his medical internship and neurology residency at UC San Francisco in 1989. He then did postdoctoral research training in the lab of Dr. William C. Mobley at UCSF from 1989–1994. At UCSF, he also established the Memory and Cognitive Disorders Clinic and was an assistant professor from 1991–1994. He moved to his own laboratory at Washington University in 1994 and was named Associate Professor of Neurology in 2001, Professor in 2002, and the Andrew and Gretchen Jones Professor and head of the Department of Neurology in 2003.
Dr. Holtzman’s group is interested in understanding basic mechanisms underlying acute and chronic cell dysfunction in the central nervous system, particularly in the context of Alzheimer’s disease (AD) and injury to the developing brain. Abundant evidence suggests a central role for misfolded amyloid-β (Aβ) peptide in AD pathogenesis. The Holtzman lab is working to develop a better understanding of Aβ metabolism and toxicity in the central nervous system and of its regulation by factors such as ApoE and synaptic activity. They use a variety of transgenic and knockout mice as well as unique biological assays such as Aβ brain microdialysis to study mechanisms leading to AD pathology and cerebral amyloid angiopathy. Dr. Holtzman’s group also studies the metabolism of tau protein. Specifically, they have been able to assess extracellular tau by in vivo microdialysis and are interested in understanding the regulation of tau metabolism and how to block tau aggregation and its spread within the brain and spinal cord. Another major goal is to discover early biomarkers for AD to detect AD pathology prior to symptom onset so that treatments can be used to prevent and delay dementia. To this end, Dr. Holtzman’s lab conducts studies that monitor Aβ and tau levels in patient cerebrospinal fluid and plasma samples, and uses more traditional methods such as ELISA and mass spectrometry coupled with neuroimaging to develop new biomarkers.
Justin Ichida, PhD
William Jagust, MDWebsite Papers
Dr. Jagust obtained a BA degree in psychology from Reed College in 1974 and went on to complete his MD from SUNY Stony Brook in 1978. After completing his medical internship and residency in Boston, he was appointed assistant professor and eventually full professor at University of California, Davis where he was director of the UC Davis Alzheimer’s Disease Center and chair of the Department of Neurology. He left Davis in 2004 for UC Berkeley, where he is currently a Faculty Senior Scientist at Lawrence Berkeley National Laboratory as well as a Professor of Public Health and Neuroscience at the University of California, Berkeley.
Research in Dr. Jagust’s lab is aimed at understanding the structural, functional and biochemical basis of brain aging and neurodegenerative diseases associated with brain aging. His group uses techniques of positron emission tomography (PET), magnetic resonance imaging (MRI), functional MRI, and neuropsychology and cognitive neuroscience to study normal older people, and patients with neurodegenerative disorders of Alzheimer’s disease and related dementias and Parkinson’s disease. Studies are using PET imaging to detect the deposition of brain amyloid and are also looking at how changes in neurotransmitters are related to behavioral changes in aging. The Jagust Lab’s goals include early detection of Alzheimer-related changes with PET and MRI scanning, using imaging to track treatments in both Alzheimer’s and Parkinson’s disease, and understanding the fundamental mechanisms of cognitive change in the aging brain.
Martin Kampmann, PhDWebsite Papers
Dr. Kampmann is an Assistant Professor at the UCSF Department of Biochemistry and Biophysics and the Institute for Neurodegenerative Diseases (IND), and an Investigator at the Chan Zuckerberg Biohub. Dr. Kampmann received his BA degree in biochemistry from Cambridge University (UK) and his PhD in biophysics and cell biology from The Rockefeller University. As a postdoctoral fellow at UCSF, he spearheaded the development of a next-generation genetic screening platform for mammalian cells, which incorporates systematic genetic interaction maps and CRISPR-based gain- and loss-of-function screens.
The goal of Dr. Kampmann’s lab is to elucidate cellular mechanisms of human diseases and to develop new therapeutic strategies. For this purpose, the lab has pioneered CRISPR-based genetic screening technology in cell types derived from induced pluripotent stem cells (iPSCs). A major focus is the investigation of neurodegenerative diseases in human iPSC-derived neurons, astrocytes, and microglia. The technology platform co-developed by Dr. Kampmann positions him uniquely to uncover cellular mechanisms controlling these diseases. Dr. Kampmann has also successfully applied his technology to reveal the mechanism of action of new drugs and to identify biomarkers that predict the response of patients to a given therapy – a central goal of precision medicine.
Dr. Kampmann’s achievements were recognized by the NIH Director’s New Innovator Award (2015), the Paul G. Allen Family Foundation’s Allen Distinguished Investigator Award (2015), a Stand Up 2 Cancer Innovative Research Grant (2016) and a Chan Zuckerberg Biohub Investigator Award (2017).
Kao, MD, PhD
Aimee Kao, MD, PhDWebsite Papers
Dr. Kao studied neuroscience at Brown University prior to receiving her MD and PhD degrees from the University of Iowa. There, she trained in the laboratory of Dr. Jeffrey Pessin, studying the cellular and molecular biology of insulin receptor signaling and GLUT4 trafficking. She completed an internship in internal medicine at the Beth Israel-Deaconess Medical Center in Boston and then a neurology residency at the University of California, San Francisco where she was Chief Resident. She completed a one-year fellowship in behavioral neurology with Dr. Bruce Miller in the Department of Neurology and a postdoctoral research fellowship with Dr. Cynthia Kenyon in the Department of Biochemistry prior to starting her own group at UCSF where she is an Assistant Professor in the Department of Neurology.
The Kao Lab uses C. elegans and other model organisms to better understand and ultimately cure neurodegenerative disease such as Alzheimer’s and Parkinson’s diseases, with a particular interest in Parkinson’s-related dementias including multiple system atrophy, corticobasal degeneration, progressive supranuclear palsy and dementia with Lewy bodies. Dr. Kao’s group focuses on progranulin and tau in the regulation of programmed cell death, cellular stress responses, innate immunity, and other processes involved in neuronal health and integrity.
Celeste Karch, PhD
Joel Kramer, PsyDWebsite Papers
Dr. Kramer earned his doctorate in psychology at Baylor University and completed a postdoctoral fellowship in neuropsychology at Martinez VA hospital. He is a board certified clinical neuropsychologist and Professor of Neuropsychology in Neurology and the Director of the Memory and Aging Center Neuropsychology program at University of California, San Francisco.
Dr. Kramer has been extensively involved in studying the cognitive changes associated with brain disorders for the past three decades. He has co-authored widely used neuropsychological measures of memory and executive functioning. Much of his work has been devoted to identifying the different ways in which aging and neurodegenerative diseases affect memory and other abilities and in utilizing these differences to improve differential diagnosis in clinic. Presently, Dr. Kramer’s active areas of research use neuroimaging, neuropsychology, neuroimmunology, and genetics to study the underlying biological mechanisms of cognitive aging, the cognitive effects of cerebrovascular disease and frontotemporal dementia, and the relationships between cognitive functioning, behavioral control, and reward systems.
Anna Krichevsky, PhD
Anna Krichevsky is Associate Professor of Neurology at Brigham and Women’s Hospital and Harvard Medical School, Boston, USA. She received her Ph.D. degree from the Hebrew University of Jerusalem, Israel, and completed her postdoctoral training in Ken Kosik lab at Brigham and Women’s Hospital and Harvard Medical School in Boston. As a postdoctoral fellow, Anna pioneered the work that led to recognition of microRNA functions and RNA interference mechanisms in brain physiology and pathology. Specifically, she performed the first successful RNA interference in mammalian neurons; contributed to the identification of first miRNAs in mammalian brain; developed first high-throughput arrays for miRNA expression profiling; and discovered the first oncogenic miRNA, miR-21, in gliomas, which is today the most studied and one of the most promising miRNA targets for various human diseases. Krichevsky laboratory, established in 2006, has identified and investigated key miRNAs involved in human brain tumors and neurodegenerative disorders such as Alzheimer’s disease and multiple sclerosis. Her laboratory also pioneered the identification of miRNA biomarkers for diagnostics and monitoring of primary and metastatic brain tumors. Dr. Krichevsky’s work has been evaluated as the “Must read” by the Faculty of 1000 and featured in the Boston Globe, HealthCanal, Science Daily, Thomson Reuters, International Innovations, and other periodicals. All her recent projects were based on successful collaborations that involved building very diverse academic and industrial teams. Dr. Krichevsky gave numerous invited talks at various national and international meetings. She served as a reviewer for the NIH and other governmental and private foundations, in USA and other countries. She is also serving on the Executive Committee of the HMS Initiative for RNA Medicine, established to promote RNA research towards therapies and build an RNA Medicine Institute at HMS.
Suzee Lee, MDWebsite Papers
Dr. Suzee Lee is an associate professor of neurology at the UCSF Memory and Aging Center. Dr. Lee’s research program focuses on neuroimaging in families with neurodegenerative diseases, such as frontotemporal dementia and other tau-spectrum disorders. Her central goals are to understand the influence that genetic mutations and genetic risk factors play in the development of various clinical syndromes, and to develop neuroimaging biomarkers that improve the diagnosis and monitoring of preclinical and early-stage neurodegenerative disease.
Dr. Lee received a BA degree in English from Harvard and an MD from McGill University in Montreal, Canada. She then completed an internal medicine internship at Brown University and neurology residency at Mount Sinai Hospital in New York City, serving as Chief Resident in her final year. Dr. Lee completed a behavioral neurology fellowship at the UCSF Memory and Aging Center and continues to evaluate patients in her clinic and clinical research projects.
Miller, MD, PhD
Timothy Miller, MD, PhDWebsite Papers
Dr. Miller performed his graduate studies with Dr. Eugene Johnson from 1993–1997 and completed his MD/PhD at Washington University School of Medicine in 1998. He then trained as a neurology resident and neuromuscular fellow at the University of California, San Francisco from 1998–2003. Afterwards, Dr. Miller moved to the University of California, San Diego where he spent four years as part of the Mentored Scientist Clinical Training Program doing postdoctoral work on disease models of ALS. He is currently an assistant professor of neurology at Washington University in St. Louis and a faculty member of the Hope Center for Neurological Disorders. He is the first director of the Christopher Wells Hobler Laboratory for ALS Research.
Dr. Miller’s research focuses on methods to turn off harmful genes in the brain and spinal cord as a way to bring novel treatments to neurodegenerative diseases. In ALS, these efforts have resulted in a first-in-man clinical trial using antisense oligonucleotides in ALS patients with a genetic form of ALS. Dr. Miller has recently focused on tau as a therapeutic target for dementias including Alzheimer’s disease and frontotemporal dementia. His group is using antisense oligonucleotides to reduce levels of tau or change the ratios of different types of tau proteins.
Lennart Mucke, MDWebsite Papers
Dr. Mucke is a graduate of the Georg-August-University and the Max-Planck-Institute for Biophysical Chemistry (Neurobiology) in Göttingen, Germany. He trained in internal medicine at the Cleveland Clinic, in neurology at the Massachusetts General Hospital and Harvard Medical School, and in neuroimmunology and neurovirology at The Scripps Research Institute, where he was subsequently appointed to the faculty. In 1996, Dr. Mucke was recruited to head a program in molecular neurobiology at the Gladstone Institutes and the University of California, San Francisco. The progress made resulted in the establishment of the Gladstone Institute of Neurological Disease, which he has directed since its inauguration in 1998. He also holds joint appointments as the Joseph B. Martin Distinguished Professor of Neuroscience and Professor of Neurology at the University of California, San Francisco.
Dr. Mucke’s research focuses on processes that result in memory loss and other major neurological deficits, with an emphasis on Alzheimer’s disease and related disorders. He has generated informative experimental models of these conditions and used them to identify novel strategies to prevent neurological decline. As the director of the Gladstone Institute of Neurological Disease, he has established a vigorous interdisciplinary program for research and training in disease-focused neuroscience. For his contributions, Dr. Mucke has received the Potamkin Prize from the American Academy of Neurology, the MetLife Foundation Award for Medical Research, the Kalid Iqbal Lifetime Achievement Award, the Zenith Award from the Alzheimer’s Association, the American Pacesetter Award from the ARCS Foundation, and an Award for Excellence in Direct Teaching and Mentoring from the Haile T. Debas Academy of Medical Educators. Dr. Mucke has served on the National Advisory Council on Aging for the NIH and is a member of the American Neurological Association, the Association of American Physicians, and the Senate of the German Center for Neurodegenerative Diseases (DZNE).
Thomas Neylan, MDPapers
Dr. Neylan is the Director of the Posttraumatic Stress Disorders (PTSD) Clinical and Research Programs at the San Francisco Veterans Affairs Medical Center. He is a professor in the Department of Psychiatry at the University of California, San Francisco. Dr. Neylan has been an active researcher in the study of sleep and posttraumatic stress disorder for the past 19 years. He has been the principal investigator on multiple funded projects sponsored by the National Institutes of Health, the National Institute of Justice, the Department of Defense, and the Department of Veterans Affairs
Stanley Prusiner, MDWebsite Papers
Dr. Prusiner received his BS degree and subsequent MD from the University of Pennsylvania before completing his medical internship at University of California, San Francisco. He conducted research in the laboratory of Dr. Earl Stadtman at National Institutes of Health before returning to UCSF for his neurology residency. Since then, he joined the UCSF faculty and has held various faculty and visiting faculty positions at both UCSF and University of California, Berkeley. Dr. Prusiner currently heads the Institute for Neurodegenerative Diseases at UCSF.
Dr. Prusiner won the Nobel Prize in Physiology or Medicine in 1997 for his work in proposing an explanation for the cause of bovine spongiform encephalopathy (mad cow disease) and its human equivalent, Creutzfeldt-Jakob disease. In this work, he coined the term prion, which comes from the words “proteinaceous” and “infectious” to refer to a previously undescribed form of infection due to protein misfolding. His group continues to study prion disease, Alzheimer’s disease, and tauopathies.
Gil Rabinovici, MDWebsite Papers
Born and raised in Jerusalem, Dr. Rabinovici received his BS degree from Stanford University and MD from Northwestern University Medical School. He completed an internship in internal medicine at Stanford University, neurology residency (and chief residency) at UCSF and a behavioral neurology fellowship at the Memory and Aging Center, where he has remains on faculty as an attending neurologist.
Dr. Rabinovici’s research evaluates how structural, functional and molecular brain imaging techniques can be used to improve diagnostic accuracy in dementia and to study the biology of neurodegenerative diseases. His Tau Consortium project is focused on characterizing and translating promising Tau PET ligands in patients with a variety of tauopathies. Dr. Rabinovici’s work is supported by the National Institute on Aging, the Alzheimer’s Association, the Tau Consortium, and the John Douglas French Alzheimer’s Foundation. He is the recipient of the 2012 American Academy of Neurology Research Award in Geriatric Neurology and the 2010 Best Paper in Alzheimer’s Disease Neuroimaging: New Investigator Award from the Alzheimer’s Association.
Katherine Rankin, PhD
Rubinsztein, MB ChB, BSc(Med), PhD
David Rubinsztein, MB ChB, BSc(Med), PhDPapers
Dr. Rubinsztein is a Wellcome Trust Principal Research Fellow and Deputy Director of the Cambridge Institute for Medical Research. Dr. Rubinsztein earned his MB ChB, BSc(Med), and PhD degrees from University of Cape Town. He came to Cambridge in 1993 as a senior registrar in genetic pathology and started working on Huntington’s disease and developed an independent research group. David Rubinsztein was appointed to a personal readership at the University of Cambridge in 2003 and was promoted to Professor of Molecular Neurogenetics at the University of Cambridge (personal chair) in 2005. He was awarded the Graham Bull Prize for Clinical Science by the Royal College of Physicians in 2007. Rubinsztein is a Fellow of the Academy of Medical Sciences and a member of EMBO.
His research, which is based in the Department of Medical Genetics in the Cambridge Institute for Medical Research, is focused on trying to understand the processes underlying the pathology in Huntington’s disease and related conditions associated with intracellular aggregates. He is trying to use the understanding of the pathobiology of these diseases to develop therapeutic strategies and has a particular interest in autophagy in this context. His group uses a range of approaches to address these aims, from cell biology to fly and mouse models.
William Seeley, MDWebsite Papers
Dr. Seeley completed his undergraduate studies at Brown University and received his medical degree from University of California, San Francisco, where he first encountered patients with frontotemporal dementia during a research elective with Dr. Bruce Miller. He was a medical intern at UCSF and a neurology resident at Massachusetts General Hospital and Brigham and Women’s Hospital. Returning to UCSF for a behavioral neurology fellowship with Dr. Miller, Dr. Seeley developed expertise in the differential diagnosis and treatment of patients with neurodegenerative disease. He is currently a professor of neurology at the UCSF Memory and Aging Center, where he participates in patient evaluation and management. He is a 2011 MacArthur Fellow.
Dr. Seeley’s research in his Selective Vulnerability Research Laboratory concerns regional vulnerability in dementia, that is, why particular dementias target specific neuronal populations. Dr. Seeley addresses this question through behavioral, functional imaging, and neuropathology studies. The goal of his research is to determine what makes brain tissues susceptible or resistant to degeneration, with an eye toward ultimately translating these findings into novel treatment approaches.
Judith Steen, PhDWebsite Papers
Dr. Steen studied chemistry and biochemistry at University of Toronto. She then studied mass spectrometry under Peter Roepstorff at University of Southern Denmark and cell biology under Marc Kirschner at Harvard University. She holds an appointment in the neurobiology department at Boston Children’s Hospital.
Dr. Steen’s research is focused on four different areas: development of novel and improved mass spectrometric and proteomic methods for the analysis of complex protein mixtures and the detailed characterization of proteins within these mixtures, development of instrument platforms for the detection and identification of disease-markers with special emphasis on pediatric diseases, use of mass spectrometry to identify mechanisms of cell cycle control during neurogenesis, and development and applications of strategies to solve systems biological questions and problems at the proteome level. The Steen Lab uses proteomic and mass spectrometric technology in addition to biochemistry and molecular biology approaches, cell culture systems and animal models, including mouse, rat, and African clawed frog Xenopus laevis.
Sally Temple, PhDWebsite Papers
Dr. Temple received her undergraduate degree at Cambridge University, Cambridge UK, specializing in developmental biology and neuroscience. She continued her PhD work at University College London, UK and postdoctoral fellowship at Columbia University, NY where she focused on optic nerve development. She is the co-Founder, Principal Investigator and Scientific Director of the Neural Stem Cell Institute located in Rensselaer, NY. Dr. Temple is recipient of many prestigious awards including the Jacob Javitz Merit award from NIH (2003) as well as the MacArthur fellowship Award (2008) in recognition of her contributions to neural stem cell developmental biology.
Dr. Temple leads a team of 30 researchers focused on using neural stem cells to develop therapies for eye, brain and spinal cord disorders. Her research is focused on the question of how neural stem cells alter their developmental potential over time to generate diverse progeny, and it indicates that internal counting mechanisms, rather than external signals in the progenitors, determine the number of cell divisions they undergo. She has identified markers on progenitor cell lines and external signaling molecules that participate in maintaining and differentiating them. Dr. Temple’s studies suggest that the limited success to date of embryonic stem cell transplants to repair neural damage could be due to introduction of stem cells at the wrong stage of development. It may be that progenitor cells further along in their differentiation will prove more effective. Through her research on the differentiation of neural progenitors, Dr. Temple brings us closer to developing effective clinical treatments for central nervous system damage due to trauma, neurodegenerative diseases, malignancy, or stroke.
Christine Walsh, PhDWebsite Papers
Dr. Walsh received her BA degree in physiology from Trinity College Dublin. Dr. Walsh did her doctoral work at the University of Michigan studying the effects of REM sleep modulation on learning and memory. She also studied the neural correlates of cognitive aging. In 2011 Dr. Walsh joined the UCSF Memory and Aging Center where she has been studying sleep in both healthy older adults and in individuals with neurodegenerative diseases. Dr. Walsh is particularly interested in the contribution of sleep disturbance to cognitive decline.
Haung Yu, PhD
Jim Audia, PhD
Executive Director at Chicago Biomedical Consortium
Board Member Constellation Pharmaceuticals
Bradley Boeve, MD
Consultant in Neurology at Mayo Clinic
Chair, Behavioral Neurology
Everett Cook, MBA
Co-Founder and Managing Director at Pouschine Cook Capital Management, LLC
Feldman, MD, FRCP
Howard Feldman, MD, FRCP
Professor of Neurosciences, University of California San Diego
Kenneth S. Kosik, MDWebsite Papers
Dr. Kosik completed BA and MA degrees in English literature from Case Western Reserve University in 1972 and his MD from the Medical College of Pennsylvania in 1976. He served as a resident in neurology at Tufts New England Medical Center and was Chief Resident there in 1980. Beginning in 1980 he held a series of academic appointments at the Harvard Medical School and achieved the rank of full professor there in 1996. In 2005, he moved to the University of California Santa Barbara as the Co-Director of the Neuroscience Research Institute. Dr. Kosik serves as a co-director of the Tau Consortium.
Patrick May, PhD
President, Advantage Neuroscience Consulting, LLC
Bruce L. Miller, MDWebsite Papers
Dr. Miller holds the A.W. and Mary Margaret Clausen Distinguished Professorship in Neurology at the University of California, San Francisco (UCSF). He directs the busy UCSF Memory and Aging Center where patients in the San Francisco Bay Area receive comprehensive clinical evaluations for dementia. He oversees a program project on frontotemporal dementia (FTD), currently in its 11th year and the UCSF Alzheimer’s Disease Research Center, both funded by the National Institute of Aging. He is a behavioral neurologist focused in dementia with special interests in brain and behavior relationships as well as the genetic and molecular underpinnings of disease. Dr. Miller has been the recipient of numerous lectureships and prizes including the Potamkin Award from the American Academy of Neurology and the Gene D. Cohen Research Award in Creativity and Aging from the National Center for Creative Aging. Dr. Miller serves as a co-director of the Tau Consortium.
Nestler, MD, PhD
Eric Nestler, MD, PhD
Professor of Neuroscience
Director of the Friedman Brain Institute
Icahn School of Medicine at Mount Sinai
Maria Spillantini, PhD
Professor of Molecular Neurology
University of Cambridge
Hui Zheng, PhD
Professor, Huffington Center on Aging, Baylor College of Medicine
The Tau Consortium is currently staffed by the Rainwater Charitable Foundation.
Senior Director of Drug Discovery
Leticia Toledo-Sherman, PhD
Dr. Leticia Toledo-Sherman is Senior Director of Drug Discovery for the Tau Consortium and Adjunct Assistant Professor of Neurology at UCLA. In her role at the Foundation, she provides strategic direction and oversight of the drug discovery portfolio. Leticia brings critical expertise and best practices in neurodegenerative disease drug discovery to accelerate the development of therapies for Tauopathies. Previously, she was Director of Medicinal Chemistry and Computer-Aided Drug Design at the CHDI Foundation, leading drug discovery programs for therapeutic development in Huntington’s Disease (HD). Prior to joining CHDI, Dr. Toledo-Sherman was Executive Director of Chemistry at Lymphosign (Pharmascience Inc), leading the development of therapeutics for blood cancers. From 2000 to 2004, she led a multi-site, multidisciplinary team using chemical proteomics and bioinformatics to discover therapeutic targets and to investigate the mechanism of action of drugs. Dr. Toledo-Sherman started her professional career as a leading scientist at Kinetix Pharmaceuticals (acquired by Amgen), where she implemented an in silico platform aimed at rational drug design of kinase inhibitors targeting multiple therapeutic areas. She has served in the Science Advisory Board of the SGC-UNC Kinase Unit and is currently in the Science Advisory Board of The Chemical Probes Portal. Leticia is an advocate and supporter of open science at the pre-competitive drug discovery stage. Leticia received a PhD in Organic Chemistry from The State University of New York at Stony Brook and did postdoctoral research at the Massachusetts Institute of Technology and The Skaggs Institute for Chemical Biology at The Scripps Research Institute in La Jolla, California.
Patrick Brannelly, MBA
Patrick Brannelly has served as the Program Director of the Tau Consortium since 2014. In this role, he provides oversight for the Rainwater Charitable Foundation’s medical research efforts, including grantmaking and strategic partnerships. In the decade prior to joining the Tau Consortium, Patrick was the co-founder and CEO of NeoCORTA and the Director of Partnerships & Group Programs at Posit Science, both brain health technology ventures. Earlier in his career, he served in the US and Europe as a management consultant in the pharmaceutical, energy, and technology sectors. He is a former Assistant Professor in entrepreneurship and strategy at California State University, Fresno. He currently serves as a Board member of the FTD Disorders Registry, a Steering Committee member of the PSP Research Roundtable, and the Co-Managing Director of the PSP Genetics Consortium. He is a former Board member of the Alzheimer’s Association, National Capital Chapter. Mr. Brannelly holds a BA in Psychology from Harvard College and an MBA with Distinction from Harvard Business School.
Amy Rommel, PhD
Dr. Amy Rommel joined the Rainwater Charitable Foundation in 2018. She uses her scientific expertise to guide the foundation’s medical research programming, with a primary focus on the Rainwater prize program and other public outreach efforts. Prior to joining the Rainwater Charitable Foundation, Amy was a research scientist at the Salk Institute, where her collaborative research efforts to understand cancer have contributed to the development of new technology and potential new treatments for glioblastoma. She has been a featured speaker at numerous public events such as TEDxSanDiego. She has participated in several community programs and advisory efforts including those associated with the Cancer Moonshot program, World Economic Forum and AAAS. In addition, she continues to be an avid supporter and volunteer for STEM Education Outreach and science advocacy. Dr. Rommel holds a BS and MS in Biochemistry from the University of North Texas, and a PhD in Biomedical Sciences from UT Southwestern Medical Center.
Thank you for your interest in learning about the Rainwater Foundation’s medical research programs. Participation in the Foundation’s grant-making programs is by invitation only. We are happy to answer any questions you have or connect you to additional resources and insights as needed.
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