Reconfiguring the Landscape for the Alzheimer’s Challenge

Christopher P. Austin
Director, National Center for Advancing Translational Sciences

Christopher Austin is Director of the National Center for Advancing Translational Sciences (NCATS) at the U.S. National Institutes of Health. NCATS’ mission is to catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions. Before becoming NCATS Director in September 2012, he was Director of the NCATS Division of Preclinical Innovation, which focuses on translating basic science discoveries into new treatments, particularly for rare and neglected diseases, and developing new technologies and paradigms to improve the efficiency of therapeutic and diagnostic development. In this role, he founded and directed numerous initiatives including the NIH Chemical Genomics Center (NCGC), the Therapeutics for Rare and Neglected Diseases (TRND) program, and the Toxicology in the 21st Century (Tox21) program. In 2016, Dr. Austin was elected chair of the International Rare Disease Research Consortium (IRDiRC). Before joining NIH in 2002, Dr. Austin directed research programs genomics-based target discovery, pharmacogenomics, and neuropsychiatric drug development at Merck, with a particular focus on schizophrenia. Austin earned an A.B. in biology from Princeton University and an M.D. from Harvard Medical School. He completed clinical training in internal medicine and neurology at Massachusetts General Hospital, and a research fellowship in genetics at Harvard.

David Baltimore
Robert Andrews Millikan Professor of Biology and President Emeritus, Caltech

After serving as President of the California Institute of Technology for nine years, David Baltimore was appointed President Emeritus and the Robert Andrews Millikan Professor of Biology in 2006. Awarded the Nobel Prize at the age of 37 for research in virology, Dr. Baltimore has profoundly influenced national science policy on such issues as recombinant DNA research and the AIDS epidemic. He is an accomplished researcher, educator, administrator and public advocate for science and engineering and is considered one of the world’s most influential biologists.

Born in New York City, Baltimore became interested in biology during high school when he spent a summer at the Jackson Memorial Laboratory and worked with research biologists on mammalian genetics. He received his B.A. in Chemistry from Swarthmore College in 1960 and a Ph.D. in 1964 from Rockefeller University, where he returned to serve as President from 1990-91 and faculty member until 1994.

For almost 30 years, Dr. Baltimore was a faculty member at Massachusetts Institute of Technology where his early investigations examined the molecular processes underlying the ability of poliovirus to infect cells. This led him to work on other RNA viruses and then to a consideration of how cancer-causing RNA viruses manage to infect and permanently alter a healthy cell. He identified the enzyme reverse transcriptase in the virus particles, thus providing strong evidence for a process of RNA to DNA conversion, the existence of which had been hypothesized some years earlier. Dr. Baltimore and Howard Temin (with Renato Dulbecco, for related research) shared the 1975 Nobel Prize in Physiology or Medicine for their discovery, which provided the key to understanding the life-cycle of retroviruses such as HIV. In the following years, he has contributed widely to the understanding of cancer, AIDS and the molecular basis of the immune response.

His present research focuses on control of inflammatory and immune responses, on the roles of microRNAs in the immune system and on the use of gene therapy methods to treat HIV and cancer in a program called “Engineering Immunity”. He has become Director of the Joint Center for Translational Medicine, an activity that joins Caltech and UCLA in a program to translate basic science discoveries into clinical realities and where an active clinical program is under way.

Dr. Baltimore has several outstanding administrative and public policy achievements to his credit. In the mid-1970s, he played an important role in creating a consensus on national science policy regarding recombinant DNA research. He served as founding director of the Whitehead Institute for Biomedical Research at MIT from 1982 until 1990. An early advocate of federal AIDS research, Dr. Baltimore co-chaired the 1986 National Academy of Sciences committee on a National Strategy for AIDS and was appointed in 1996 to head the National Institutes of Health AIDS Vaccine Research Committee. Dr. Baltimore served as a member of the Independent Citizen’s Oversight Committee to the California Institute for Regenerative Medicine until 2007 and on the Board of Directors for both MedImmune until 2007 and Cellerant until 2008.

He has played an important role in the development of American biotechnology since his involvement in the 1970’s in the formation of Collaborative Genetics. He helped found other companies such as Calimmune and Immune Design and he presently serves on the Board of Directors at several companies and non-profit institutions including the Broad Foundation and Broad Institute, and Amgen and Regulus Therapeutics. He is a member of numerous Scientific Advisory Boards, including the Broad Institute, Ragon Institute, Regulus Therapeutics and Immune Design. He is a Scientific Partner to the venture capital firm, The Column Group, and was a Director of the Swiss investment company BB Biotech through 2011.

Dr. Baltimore’s numerous honors include the 1970 Gustave Stern Award in Virology, 1971 Eli Lilly and Co. Award in Microbiology and Immunology, 1999 National Medal of Science, and 2000 Warren Alpert Foundation Prize. He was elected to the National Academy of Sciences in 1974, and is also a fellow of the American Academy of Arts and Sciences, and a foreign member of both the Royal Society of London and the French Academy of Sciences. He is past-President and Chair of the American Association of the Advancement of Science (2007-2009) and was mostly recently named a Fellow of the American Association for Cancer Research (AACR). He has published more than 700 peer-reviewed articles.

Ed Boyden
Professor of Biological Engineering and Brain and Cognitive Sciences, MIT

Ed Boyden is a professor of Biological Engineering and Brain and Cognitive Sciences at the MIT Media Lab and the MIT McGovern Institute. He leads the Synthetic Neurobiology Group, which develops tools for analyzing and repairing complex biological systems such as the brain, and applies them systematically to reveal ground truth principles of biological function as well as to repair these systems. These technologies include expansion microscopy, which enables complex biological systems to be imaged with nanoscale precision, and optogenetic tools, which enable the activation and silencing of neural activity with light, amongst many other innovations. He co-directs the MIT Center for Neurobiological Engineering, which aims to develop new tools to accelerate neuroscience progress.

Amongst other recognitions, he has received the Breakthrough Prize in Life Sciences (2016), the BBVA Foundation Frontiers of Knowledge Award (2015), the Carnegie Prize in Mind and Brain Sciences (2015), the Jacob Heskel Gabbay Award (2013), the Grete Lundbeck Brain Prize (2013), the NIH Director’s Pioneer Award (2013), the NIH Director’s Transformative Research Award (twice, 2012 and 2013), and the Perl/UNC Neuroscience Prize (2011). He was also named to the World Economic Forum Young Scientist list (2013), the Technology Review World’s “Top 35 Innovators under Age 35” list (2006), and is an elected member of the American Academy of Arts and Sciences (2017).

His group has hosted hundreds of visitors to learn how to use new biotechnologies, and he also regularly teaches at summer courses and workshops in neuroscience, and delivers lectures to the broader public (e.g., TED (2011); TED Summit (2016); World Economic Forum (2012, 2013, 2016)). He received his Ph.D. in neurosciences from Stanford University as a Hertz Fellow, where he discovered that the molecular mechanisms used to store a memory are determined by the content to be learned. Before that, he received three degrees in electrical engineering, computer science, and physics from MIT. He has contributed to over 300 peer-reviewed papers, current or pending patents, and articles, and has given over 300 invited talks on his group’s work.

Katja Brose
Science Program Officer, Chan Zuckerberg Initiative

Katja Brose, Ph.D., is a Science Program Officer at the Chan Zuckerberg Initiative. The goals of Chan Zuckerberg Science Initiative are to support basic science and technology that will make it possible to cure, prevent, or manage all diseases by the end of the century. Before joining CZI, she was part of the editorial team at Cell Press for 17 years, where from 2004-20017 she was Editor-in-Chief of Neuron and a Publishing Director at Cell Press-Elsevier. During her tenure as Editor, Neuron undertook a major expansion of its scope building on its historical strengths in molecular and cellular neuroscience to cover all areas of neuroscience from molecular/cellular mechanisms to systems and cognitive neuroscience, genetics, neurological and psychiatric disease, theoretical neuroscience and emerging technologies. As Publishing Director, she was responsible for Cell Press strategy for review content, including oversight of the Trends family of review journals. She also led Cell Press’ efforts around rigor and reproducibility. She has been an active committee member at the Society for Neuroscience, as a member of the Professional Development and Neuroscience Training Committees. She speaks frequently on topics related to scientific publishing and communication, including publication ethics and rigor and reproducibility in science. She earned her undergraduate degree in 1990 from Brown University, with a double concentration in Biology and European History. She received her PhD in Biochemistry from the University of California-San Francisco (1994-2000). For her graduate work, she worked in the laboratory of Dr. Marc Tessier Lavigne focusing on axon guidance mechanisms in the developing spinal cord.

Miyoung Chun 
Former Executive Vice President of Science Programs, Kavli Foundation

Miyoung Chun’s career spans a wide range of experience in academia and industry. Her academic career began as an Assistant Professor of Biochemistry and a member of Whitaker Cardiovascular Institute at Boston University School of Medicine in 1995. There she taught in the areas of cell biology and molecular medicine, and conducted research in signal transduction of G-protein coupled receptors.

From 1999 to 2004 she worked for Millennium Pharmaceuticals Inc. as a scientist and project leader, where her research focused on genomics/functional genomics and on molecular imaging in drug discovery and development. She discovered and characterized novel genes that are important to inflammatory and cardiovascular diseases, and has over 30 U.S. and International issued/published patents.

In 2004 she moved back to academia as Assistant Dean of Science and Engineering at the University of California, Santa Barbara (UCSB), in particular serving the California Nanosystems Institute. She was also appointed Director of International Research Advancement at UCSB. In this role she was active in building partnerships among academia, government and industry around the globe.

Dr. Chun was Vice President of Science Programs at The Kavli Foundation since 2007 and was Executive Vice President of Science Programs from 2013 to 2017. She obtained her Ph.D. degree in Molecular Genetics from The Ohio State University in 1990 and was a Postdoctoral Fellow at MIT’s Whitehead Institute studying the cell and molecular biology of receptors.

Jean-Jacques Degroof
Entrepreneur and Investor

Jean-Jacques Degroof started his career in the financial services industry and, since 1999, has been an active venture investor in the Boston area and in Europe, including in the biomedical sector. During part of this period, Dr. Degroof also worked as a researcher at the MIT Industrial Performance Center. Earlier, he was a Sloan Fellow at the MIT Sloan School of Management and a BCG Fellow at the Mossavar-Rahmani Center for Business and Government at Harvard University. He obtained an M.S. and Ph.D. in Management from the MIT’s Sloan School of Management. Philanthropically, he has supported several initiatives at MIT, including the Laboratory for Financial Engineering’s application of financial tools to drug discovery and development, specifically related to Alzheimer’s disease. of cell biology and molecular medicine, and conducted research in signal transduction of G-protein coupled receptors.

Carole Ho
Chief Medical Officer and Head of Development, Denali Therapeutics

Carole Ho is the Chief Medical Officer and Head of Development at Denali Therapeutics in South San Francisco. Denali Therapeutics is dedicated to therapeutic discovery and development of treatments for neurodegenerative diseases. Prior to Denali, Dr. Ho most recently served as Vice President of Genentech Early Clinical Development. In that role, she led a team of Medical Directors responsible for delivery of pivotal trial-ready therapeutic candidates in Neurology, Ophthalmology, Immunology, and Infectious Disease and served as the Chair of the Development Review Committee. During her 8 year tenure at Genentech, Dr. Ho and her team led the successful development of multiple therapeutics that are currently in Ph3 studies or have been approved, including ocrelizumab for Multiple Sclerosis, lampalizumab for dry AMD, rituxan for Wegener’s granulomatosis and microscopic polyangiitis and crenezumab for Alzheimer’s Disease. Dr. Ho and her team also lead the Alzheimer’s Prevention Initiative with Banner Health to spearhead a landmark Alzheimer prevention study in cognitively healthy individuals who are destined to develop Alzheimer’s disease because of their genetic history.

She received her undergraduate degree in Biochemical Sciences from Harvard College and her M.D. from Cornell University. She completed her Medical Internship and Neurology residency at Harvard and served as Chief Resident of Neurology at the Massachusetts General Hospital / Brigham and Women’s Hospital. Prior to Genentech, Dr. Ho was on Faculty in the Department of Neurology at Stanford University and subsequently a Medical Director at Johnson and Johnson.

David Hung
CEO, Axovant

David Hung, M.D., founded Medivation (NASDAQ: MDVN) in October 2003, taking the company public through a reverse merger into a SPAC (Orion Acquisition Corporation) in December 2004, thereby completely bypassing the traditional venture capital funding route.  Medivation was founded to develop technologies that represented true “medical innovation” in particularly challenging disease indications of high unmet need, such as Alzheimer’s disease and prostate cancer.   Medivation ultimately developed and commercialized Xtandi for the treatment of metastatic and non-metastatic prostate cancer, driving the program from invention to FDA approval in 7 years, one of the fastest development times in biopharmaceutical history.  By 2016, Xtandi had become the world’s leading prostate cancer therapy, with nearly $2.5 billion in annual revenue.  Medivation was uniquely capital efficient in its development as Dr. Hung raised a total of $440MM in public offerings over the life of the company to ultimately reach a market capitalization of $14.3B, delivering for his investors a total return on investment of 21,000% in 13 years. Dr. Hung established partnerships with world-class pharmaceutical companies (Pfizer and Astellas) worth ~$1.5B in upfront payments and milestones, excluding profit splits and royalties, with both partners paying the majority of development costs. In October 2016, when Medivation was sold to Pfizer for $14.3B in an all-cash deal, it was the largest sale of a biopharma company ever by an actively-seated founding CEO.

Prior to Medivation, Dr. Hung was President & Chief Executive Officer of Pro•Duct Health, Inc., a venture-backed startup medical device company founded in 1998 which developed, manufactured, and commercialized a breast microcatheter — which he himself invented — for a minimally invasive procedure called ductal lavage for early breast cancer detection and risk assessment. After raising a total of $22MM in venture capital and completing a successful 507 patient high-risk breast cancer pivotal trial, Dr. Hung obtained FDA clearance for the ductal lavage catheter and sold Pro•Duct Health in 2001 for $168MM to Cytyc Corporation, the world’s leading maker of Pap smears for the cervix, delivering a 10X ROI for his Series A investors in 3 years.

Dr. Hung previously spent several years in pre-clinical research and development roles of increasing responsibility at Chiron Corporation, including as VP of New Projects and VP of Lead Discovery and Development. Earlier in his career, he was a practicing physician at the hospitals and clinics of the University of California, San Francisco, School of Medicine, holding positions of Division Chief, Transfusion Medicine Division, Attending Physician, Hematology-Oncology/Transfusion Medicine and Assistant Professor in Residence, Department of Laboratory Medicine. He also completed basic science molecular biology research fellowships at the Brain Tumor Research Center (BTRC) and the Cardiovascular Research Institute (CVRI), as well as three clinical fellowships in Hematology, Oncology and Transfusion Medicine.  He has been published in dozens of top peer-reviewed basic science and clinical journals including Science, Cell, Nature, The New England Journal of Medicine and The Lancet.

Dr. Hung is the recipient of numerous distinctions including the Ernst and Young Entrepreneur of the Year Award in the Life Sciences Category as well as Overall US Winner of the Ernst and Young Entrepreneur of the Year Award in 2014 (the world’s most prestigious award in entrepreneurialism), the Pfizer Scholar’s Award (awarded yearly to the top 5 junior academic faculty in a biomedical discipline), the Hulga Irene Duggan Scholar’s Award from the Arthritis Foundation, the Arlo Guthrie Award from the Huntington’s Disease Society of America and the Outstanding Teaching Award for three years in a row from the University California, San Francisco, School of Medicine.

He received his A.B., summa cum laude, in Biology from Harvard College, and his M.D., Alpha Omega Alpha, from the University of California, San Francisco, School of Medicine.

Dr. Hung is an avid violinist (formerly the Concertmaster of the Waltham Symphony Orchestra) and pianist, tennis player, former 7-handicap golfer, and former head chef at Marco Polo, a pop-up Italian-Asian fusion restaurant he started as a summer vacation project.

Currently, Dr. Hung is the CEO of Axovant Sciences (NASDAQ: AXON) in New York City, a position he assumed in April 2017.  Axovant is a late clinical stage neurology-focused biotech company with 3 different small molecule therapeutic candidates (intepirdine, nelotanserin and RVT-104) all targeting Dementia with Lewy Bodies (DLB), which accounts for up to 20% of all dementias worldwide, as well as potentially other dementias such as Alzheimer’s disease and Parkinson’s Disease.  Axovant recently (September 26, 2017) announced the failure of the phase 3 MINDSET trial of intepirdine in Alzheimer’s disease, a program that was initiated nearly 2 years prior to his arrival at the company.  However, Axovant continues to advance the intepirdine and nelotanserin programs in other potentially pivotal dementia studies, both with readouts in the next 6 months.  Additionally, Axovant remains focused on further building out its pipeline of potential therapeutic candidates.

Terrence J. Sejnowski
Investigator, Howard Hughes Medical Institute
Francis Crick Chair, Salk Institute for Biological Studies
Distinguished Professor of Biology, University of California San Diego

Terrence J. Sejnowski is a pioneer in computational neuroscience and his goal is to understand the principles that link brain to behavior. His laboratory uses both experimental and modeling techniques to study the biophysical properties of synapses and neurons and the population dynamics of large networks of neurons. New computational models and new analytical tools have been developed to understand how the brain represents the world and how new representations are formed through learning algorithms for changing the synaptic strengths of connections between neurons. He has published over 500 scientific papers and 12 books, including The Computational Brain, with Patricia Churchland. He is a member of the National Academy of Medicine, National Academy of Sciences and the National Academy of Engineering, one of only 10 living persons to be a member of all 3 national academies.

He received his PhD in physics from Princeton University and was a postdoctoral fellow in the Department of Neurobiology at Harvard Medical School. He was on the faculty in the Department of biophysics at the Johns Hopkins University before moving to La Jolla where he is now an Investigator with the Howard Hughes Medical Institute and holds the Francis Crick Chair at The Salk Institute for Biological Studies. He is also a Distinguished Professor of Biology at the University of California, San Diego, where he is co-director of the Institute for Neural Computation and co-director of the NSF Temporal Dynamics of Learning Center.

Dr. Sejnowski is the President of the Neural Information Processing Systems (NIPS) Foundation, which organizes an annual conference attended by over 4500 researchers in machine learning and neural computation and is the founding editor-in-chief of Neural Computation published by the MIT Press. He was elected to the American Academy of Arts and Sciences and is also a Fellow of the American Association for the Advancement of Science, a Life Fellow of the Institute of Electrical and Electronics Engineers and a Fellow of the Cognitive Science Society. He has received many honors, including the NSF Young Investigators Award, the Wright Prize for interdisciplinary research from the Harvey Mudd College, the Neural Network Pioneer Award from the Institute of Electrical and Electronics Engin eers, the Hebb Prize from the International Neural Network Society and the Rosenblatt Award from the Institute of Electrical and Electronics Engineers. He re ceived an honorary doctorate from the University of Zurich.

Dr. Sejnowski was instrumental in shaping the BRAIN Initiative that was announced by the White House in 2013 and served on the Advisory Committee to the Director of NIH for the BRAIN Initiative.

Li-Huei Tsai
Director, Picower Institute for Learning and Memory at MIT
Picower Professor of Neuroscience, MIT

Li-Huei Tsai combines molecular, genetic and circuit-based techniques for a “whole-systems” approach to understanding neurological disorders that affect cognition as we age. Landmark discoveries include pinpointing major genetic risk for Alzheimer’s disease to immune genes, identifying chromatin-modifiers and kinases that regulate brain flexibility and can be targeted to improve cognition in Alzheimer’s disease, and discovering that genomic integrity is critical for neuronal protection during both aging and neurodegenerative disease.

Her discoveries in Alzheimer’s disease research, including therapeutic reversal strategies for cognitive defects, have been highlighted in Nature, Cell, and Neuron and featured on National Public Radio’s RADIOLAB, and in The Atlantic, The Smithsonian, and BBC News. She is currently spearheading MIT’s new Aging Brain Initiative – a far-reaching, collaborative effort to integrate life sciences, physical sciences, information technology, engineering and disciplines beyond to meet the global challenges of dementia and Alzheimer’s disease.

Professor Tsai obtained her Ph.D. from the University of Texas Southwestern Medical Center and postdoc­toral training at Cold Spring Harbor Laboratories and Massachusetts General Hospital. Tsai became Assistant Professor of Pathology at Harvard Medical School and was promoted to tenure Professor at Harvard in 2002. She was recruited to MIT in 2006. She was an Investigator of the Howard Hughes Medical Institute from 1997 to 2013.

Dr. Tsai is a recipient of the Glenn Award for Research in Biological Mechanisms of Aging, the Javits Neuroscience Investigator Award, and the Mika Salpeter Lifetime Achievement Award for her research on brain development, neurological disorders, and Alzheimer’s disease. She is a Fellow of the American Association for the Advancement of Science, a member of the National Academy of Medicine, and an Academician of the Academia Sinica in Taiwan.

George Vradenburg
Chairman and Co-Founder, UsAgainstAlzheimer’s

George Vradenburg is the Chairman and Co-Founder of UsAgainstAlzheimer’s (UsA2), a disruptive and catalytic force committed to stopping Alzheimer’s by 2020. The UsA2 platform seeks to escalate the fight against Alzheimer’s through a broad range of powerful voices from various walks of life.

UsA2 serves as the convener of the only industry coalition dedicated to stopping Alzheimer’s—the Global CEO Initiative (CEOi) on Alzheimer’s—as well as the co-convener of a 60+ member coalition of the Alzheimer’s-serving community, Leaders Engaged on Alzheimer’s Disease. As a result of his UsA2 work, Mr. Vradenburg was appointed to the World Dementia Council by the UK Secretary of State for Health in March, 2014.

In 2011, the United States Secretary of the Department of Health and Human Services named Mr. Vradenburg to serve on the National Alzheimer’s Advisory Council on Research, Care and Services for the first-of-its-kind National Alzheimer’s Strategic Plan. In 2013 Mr. Vradenburg was appointed by Congress to the Long Term Care Commission charged with devising a comprehensive long term support and services plan for the United States.

Among other efforts, Mr. Vradenburg has testified twice before the U.S. Congress regarding the Global Alzheimer’s pandemic; conceived and supported the Alzheimer’s Study Group; and, through the Vradenburg Foundation, has supported the Alzheimer’s Disease International World Alzheimer’s Reports and the National Institute of Health’s Global Alzheimer’s Research Summit. Before his retirement, Mr. Vradenburg served in senior executive positions at AOL/Time Warner, Fox and CBS.

Mr. Vradenburg is a member of the Council on Foreign Relations and the Economic Club of Washington. He received his B.A. from Oberlin College, magna cum laude, where he was elected to Phi Beta Kappa, and his J.D. from Harvard Law School, cum laude.