Program

 

Monday, June 6, 2022 – all times in US Eastern Time

Session/Lecture

11:00 AM
Opening Remarks and ADDF Funding Opportunities
11:20
KEYNOTE: Prion Biology: Implications for Alzheimer's Disease Therapeutics
12:00 PM
SESSION 1: Novel Tools and Mechanistic Insights to Embark on a Drug Discovery Campaign
12:00
Session Overview
12:05
What Makes a Good Translational Drug Target?
12:30
"Big Data" Analytics to Enable Precision Medicine
12:55
Multi-organ Human-on-a-chip Model to Test Alzheimer’s Disease Drugs
13:20
AMP-AD and Multilevel Brain "omics" Resource to Feed a Computational Pipeline for Novel Drug Target Discovery
13:45
PANEL DISCUSSION
14:05
Break
14:15
SESSION 2: Accelerate Preclinical Studies
14:15
Session Overview
14:20
MODEL-AD: Model Organism Development and Evaluation for Late-onset AD
14:45
Efficacy, Target Engagement and Translatability of Preclinical Models of CNS Disease
15:10
Ask Your Chemist which Chemical Series Is Right for YOU!
15:35
Early Trial Design for Repurposed Drugs
16:00
Panel Discussion
16:20
End of Day 1

Chair/Speaker

11:00 AM
Howard Fillit, MD - Alzheimer’s Drug Discovery Foundation Read more
11:20
Stanley Prusiner, MD - University of California, San Francisco Read more
12:00 PM
CHAIRS: Angela Taylor - Lewy Body Dementia Association Read more
Douglas Galasko, MD - US San Diego Read more
12:00
Chairs
12:05
Samuel Hasson, PhD - Rgenta Read more
12:30
Rhoda Au, PhD - Boston University Read more
12:55
James Hickman, PhD - Hesperos Read more
13:20
David Bennett, MD - Rush University Read more
13:45
Chairs and Presenters
14:05
Break
14:15
CHAIR: Kristophe Diaz, PhD - CurePSP Read more
14:15
Chair
14:20
Gareth Howell, PhD - The Jackson Laboratory Read more
14:45
Isabel Gonzalez, PhD - 3D Consultants Read more
15:10
Amy Ripka, PhD - Lucy Therapeutics Read more
15:35
Howard Feldman, MD - UCSD Read more
16:00
Chair and Presenters
16:20
End of Day 1

Tuesday, June 7, 2022 – all times in US Eastern Time

Session/Lecture

11:00 AM
KEYNOTE:Open Science in Biomedical Venture Philanthropies: Pros & Cons
11:35
SESSION 3: Advances in Biomarkers and Disease Monitoring
11:35
Session Overview
11:40
Classification and Prediction of Clinical Alzheimer's Diagnosis Based on Plasma Proteins
12:05
Digital Biomarkers in AD and Related Dementia
12:30
Tau PET Imaging in Neurodegenerative Tauopathies
12:55
Translatable Biomarkers and Target Engagement in Early-phase Clinical Trials
13:15
PANEL DISCUSSION
13:25
Break
13:25
SESSION 4: Case Studies: Strategies for CNS Targets and Emerging Therapeutics
14:30
Session Overview
14:55
Inflammation, Immune Response, and Alzheimer’s
14:20
Blood-brain Barrier in Health and Disease
14:45
Antisense Oligodeoxynucleotide Targeting in FTD Treatment
15:05
Progranulin as a Target in FTD and other Neurodegenerative Diseases
15:25
PANEL DISCUSSION
15:45
Concluding Remarks
16:00
End of Conference

Chair/Speaker

11:00 AM
Aled Edwards, PhD - University of Toronto Read more
11:35
CHAIR: Eric Siemers, MD - Siemers Integration LLC Read more
11:35
Chair
11:40
Kaj Blennow, MD, PhD - University of Gothenburg Read more
12:05
Graham Jones, PhD - Novartis AG Read more
12:30
Agneta Nordberg, MPharm, MD, PhD - Karolinska Institutet Read more
12:55
Emily Collins, PhD - Avid Radiopharmaceuticals Read more
13:15
Chair and Panelists
13:25
Break
13:25
CHAIR: Debra Niehoff, PhD - AFTD Read more
14:30
Chair
14:55
Ellen Cahir-McFarlan, PhD - Annexon Biosciences Read more
14:20
Katerina Akassoglou, PhD - UCSF and Therini Bio Read more
14:45
Adam Boxer, MD, PhD - UCSF Read more
15:05
Lawrence Carter, PhD - Alector Read more
15:25
Chair and Panelists
15:45
Howard Fillit, MD - Alzheimer’s Drug Discovery Foundation
16:00
End of Conference

CONFERENCE INTRODUCTION

Tuesday, May 4, 2021
11:00 US Eastern Time

Howard Fillit
Alzheimer’s Drug Discovery Foundation

 

Dr. Howard Fillit is ADDF’s Founding Executive Director and Chief Science Officer. His introduction will provide a welcome to our 15th Annual Drug Discovery for Neurodegeneration Workshop, a brief update on ADDF’s recent progress and will highlight the framework for this year’s meeting.

Bio

Dr. Howard Fillit is ADDF’s Founding Executive Director and Chief Science Officer. He is a geriatrician, neuroscientist, and innovative philanthropy executive, who has led the ADDF since its founding. Dr. Fillit has held faculty positions at The Rockefeller University, the SUNY-Stony Brook School of Medicine and the Cornell University School of Medicine. In 1987, he joined the Mount Sinai School of Medicine, where he is a clinical professor of geriatric medicine and palliative care, medicine and neuroscience. Dr. Fillit also maintains a limited private practice in consultative geriatric medicine with a focus on Alzheimer’s disease and related dementias.

He has authored or co-authored more than 300 publications and is the senior editor of Brocklehurst’s Textbook of Geriatric Medicine and Gerontology. Dr. Fillit is the recipient of many awards and honors including the Rita Hayworth Award from the Alzheimer’s Association. He is a fellow of the American Geriatrics Society, the American College of Physicians, the Gerontological Society of America and the New York Academy of Medicine. Dr. Fillit earned his bachelor of arts in neurobiology cum laude from Cornell University and his medical degree from the SUNY-Upstate Medical University.

KEYNOTES

Stanley Prusiner
UCSF

Bio

Stanley B. Prusiner is Director of the Institute for Neurodegenerative Diseases and Professor of Neurology and Biochemistry at the University of California San Francisco (UCSF). He received his B.A. in Chemistry in 1964 and his M.D. in 1968 from the University of Pennsylvania. After completing his military service as a lieutenant commander in the U.S. Public Health Service at the National Institutes of Health and his neurology residency training at UCSF, he joined the UCSF faculty in 1974 and set up a laboratory to study brain diseases.

Prusiner discovered an unprecedented class of pathogens that he named prions. Prions are proteins that acquire an alternative shape that becomes self-propagating. As prions accumulate, they cause neurodegenerative diseases in animals and humans. Prusiner’s discovery led him to develop a novel disease paradigm: prions cause disorders such as Creutzfeldt-Jakob disease (CJD) in humans that manifest as (1) sporadic, (2) inherited and (3) infectious illnesses. When proposed, many scientists considered Prusiner’s concept of “infectious proteins” as well as his proposal that a single protein could possess multiple biologically active shapes or conformations to be heretical. Based on his seminal discovery that prions can assemble into amyloid fibrils, Prusiner proposed that the more common neurodegenerative diseases including Alzheimer’s and Parkinson’s diseases may be caused by prions. Remarkably, a wealth of evidence continues to accumulate arguing that prions cause not only these common degenerative diseases, but also the frontotemporal dementias (FTDs), chronic traumatic encephalopathy (CTE), multiple system atrophy (MSA) and dementia with Lewy bodies (DLB). Much of Prusiner’s current research focuses on developing therapeutics that reduce the levels of the specific prions responsible for MSA, DLB, and some FTDs as well as CTE.

Prusiner’s contributions to scientific research have been internationally recognized: He is a member of the National Academy of Sciences, the National Academy of Medicine, the American Academy of Arts and Sciences and the American Philosophical Society, and a foreign member of the Royal Society, London. He is the recipient of numerous prizes, including the Potamkin Prize for Alzheimer’s Disease Research from the American Academy of Neurology (1991); the Richard Lounsbery Award for Extraordinary Scientific Research in Biology and Medicine from the National Academy of Sciences (1993); the Gairdner Foundation International Award (1993); the Albert Lasker Award for Basic Medical Research (1994); the Wolf Prize in Medicine from the State of Israel (1996); the Nobel Prize in Physiology or Medicine (1997); and the United States Presidential National Medal of Science (2009).

Prusiner is the author of over 550 scientific research and 300 review articles, and editor of 13 books on diseases caused by prions. Prusiner’s single-author book Madness and Memory, which chronicles his discovery of prions, received wide acclaim. He holds 50 issued or allowed United States patents, all of which are assigned to the University of California. He has delivered over 150 honorary and over 750 invited lectures.

Aled Edwards
SGC/University of Toronto

Bio

Dr. Aled Edwards is the founder and Chief Executive of the Structural Genomics Consortium (SGC), a research organization celebrated for being a pioneer of open science, particularly as it applies to protein science, chemical biology and drug discovery. Aled leads the six SGC laboratories from the SGC headquarters in Canada.

Dr. Edwards is a Professor at the University of Toronto and an Adjunct Professor at McGill University. He has published over 200 papers and his teams have contributed over 4,500 structures into the Protein Data Bank, as of 2021.

Dr. Edwards has also founded many companies, including Affinium Pharmaceuticals, which developed a novel antibiotic currently in late-stage clinical trials, and M4K Pharma, the first pharmaceutical company formed explicitly to invent new, and affordably priced, medicines for pediatric cancers.

Prion Biology: Implications for Alzheimer’s Disease Therapeutics

Monday, June 6, 2022
11:20 US Eastern Time

Open Science in Biomedical Venture Philanthropies: Pros & Cons

Tuesday, June 7, 2022
11:00 US Eastern Time

SESSION I: Novel Tools and Mechanistic Insights to Embark on a Drug Discovery Campaign

Monday, June 6, 2022
12:00 – 14:05 US Eastern Time

Bio
Angela Taylor is the Interim Executive Director of the Lewy Body Dementia Association. Prior to this, she had served as Director of Programs for the LBDA, overseeing all education, advocacy and research initiatives at the association. Bringing a personal connection to LBD as a family caregiver for her father, she is regularly invited to speak to lay and professional audiences, as well as the media on the impact of LBD. A nationally-recognized LBD advocate, she serves on the federal Advisory Council on Alzheimer’s Research, Care and Services.
Additional resources:

Angela Taylor
LBDA

CHAIR
Session Overview

Bio
Douglas Galasko, MD, is a board-certified neurologist with broad expertise in assessing people with changes in memory and cognition, including Alzheimer’s disease, frontotemporal degeneration, dementia with Lewy bodies and cognitive problems associated with Parkinson’s disease.

He is a specialist with Brain Health and Memory Disorders program which offers comprehensive diagnostic testing, with access to brain imaging and comprehensive laboratory testing as needed, medical management and community resources. Patients are also offered access to research opportunities, including potential new treatments.

Dr. Galasko is associate director of the UC San Diego Shiley-Marcos Alzheimer’s Disease Research Center, and has conducted research on Alzheimer’s and related disorders for over 25 years. He has published over 400 research articles on Alzheimer’s disease and related disorders, and has received research funding from the NIH, Michael J. Fox Foundation and Alzheimer’s Drug Discovery Foundation, as well as funding as a clinical trials site.

Dr. Galasko is a member of the American Academy of Neurology and the American Neurological Association.

 

Additional resources:

Douglas Galasko
UCSD

Bio

Samuel Hasson
Rgenta

What Makes a Good Translational Drug Target?

Bio

Dr. Rhoda Au is Professor of Anatomy & Neurobiology, Neurology and Epidemiology at Boston University Schools of Medicine and Public and is a Senior Fellow at the Institute for Health Systems Innovation and Policy at the Questrom School of Business. She also serves as Director of Neuropsychology at the Framingham Heart Study.

She has been developing multi-sensory brain health monitoring platform that is customizable, technology agnostic, and scalable. Her objectives include transitioning the platform from reliance on high friction technologies to low friction ones and developing broad based data sharing resources to accelerate data science/AI driven discovery of digital biomarkers. Her research seeks to move the primary focus of health technologies from precision AD medicine to a broader emphasis on precision brain health.

Rhoda Au
Boston University

“Big Data” Analytics to Enable Precision Medicine
Bio

The driving vision of Dr. James (“J”) Hickman is fundamental to Hesperos’ technological “Human-on-a-Chip” platform.  He published the first serum-free, defined culture system for neuronal systems in 1995 (Schaffner, Barker et al. 1995) and has now extended this from rat to mouse, both embryonic and adult, as well as to human.

J has also pioneered the establishment of functional in vitro systems and was one of the first to report toxicity studies from neurons on microelectrode arrays in a defined system back in the 90’s (Jung, Cuttino et al. 1998). He has extended these defined in vitro systems now to cardiac, muscle, glia, endothelial, hepatocytes, bone marrow, immune, cancer, and epithelial cells. In most cases, the cells have been shown to survive at least 2-3 months in this system while maintaining full functionality.

His work developed a common media system, which supports a wide variety of cells, thus, establishing a common media system for multiple cell types with a high degree of test/retest reliability essential to commercial utility. J received his Ph.D. from the Massachusetts Institute of Technology (Chemistry), his M.S. and B.A. from Penn State, he was elected to the Board of Directors of the American Institute for Medical and Biological Engineers (AIMBE), the premier society for
Biomedical Engineering of which he is a Fellow.

Dr. Hickman is the sole or co-inventor on multiple pending and issued U.S. and international patents. He has presented
over 220 invited presentations with more than 275 total presentations, as well as 161 publications and 20 book chapters, in addition to 30 issued patents.


Additional resources:
hesperosinc.com/james-j-hickman-ph-d/

James Hickman
Hesperos

Multi-organ Human-on-a-chip Model to Test Alzheimer’s Disease Drugs
Bio

David A. Bennett, MD, is director of the Rush Alzheimer’s Disease Center and the Robert C. Borwell Professor of Neurological Sciences.

The Rush Alzheimer’s Disease Center is a large, free-standing multidisciplinary research and clinical center that studies a wide range of common chronic conditions of aging, including Alzheimer’s disease, stroke, Parkinson’s disease, other neurodegenerative diseases, sleep, neuro- and behavior economics, decision making and well-being. Studies range from community-based epidemiologic studies that incorporate genomics, imaging and biomedical devices, to phase I to phase IV clinical trials. Dr. Bennett is internationally known for his research and is principal investigator of several studies funded by the National Institute on Aging, including the Rush Alzheimer’s Disease Research Center, the Religious Orders Study, the Rush Memory and Aging Project, and the pathology, Alzheimer’s and related dementias study in Sao Paulo, Brazil.

Dr. Bennett leads projects designed to identify novel therapeutics for common neurologic diseases. He also directs the Regional Alzheimer’s Disease Assistance Center for Northern Illinois. He serves on numerous national and international advisory and editorial boards. He is currently on a member of the National Advisory Council on Aging for the National Institutes of Health. Dr. Bennett was the winner of the 2018 Potamkin Prize for research on dementia. He has more than 950 peer-reviewed manuscript publications, with more than 135,000 citations and an h index = 175.

Additional resources:
www.rushu.rush.edu/faculty/david-bennett-md

David A. Bennett
Rush University

AMP-AD and Multilevel Brain “omics” Resource to Feed a Computational Pipeline for Novel Drug Target Discovery

SESSION II: Accelerate Preclinical Studies

Monday, June 6, 2022
14:15 – 16:20 US Eastern Time

Bio

Kristophe Diaz, PhD, is the executive director and chief scientific officer of CurePSP. In this role, he oversees the foundation’s operations, including research grants, patient and family advocacy and support, education and awareness, its Centers of Care, partnerships, and fundraising.

Prior to being hired by CurePSP, Dr. Diaz led multidisciplinary programs at Cohen Veterans Bioscience, Cambridge, Mass., a nonprofit organization focusing on trauma-related brain disorders. He received his PhD in molecular and cellular biology at the University of Massachusetts, Boston; his master’s degree in biochemistry and molecular biology at Université du Québec, Montreal; and his Bachelor of Arts degree in molecular biology and genetics at Université de Montpellier (France), where he was an Erasmus Mundus Fellow.

Additional resources:
https://www.psp.org/

Kristophe Diaz
CurePSP

CHAIR
Session Overview

Bio
Dr. Garet Howell is Associate Professor and the Diana Davis Spencer Foundation Chair for Glaucoma Research at the Jackson Laboratories.
Additional resources:

Gareth Howell
The Jackson Laboratory

MODEL-AD: Model Organism Development and Evaluation for Late-onset AD
Bio
Dr. Isabel Gonzalez has over 30 years of research experience, resulting in more than 50 publications in peer reviewed journals and book chapters. She combines 12 years experience in University research laboratories and 20 years of experience in the pharmaceutical industry, in three different multinational companies (Parke-Davis/Pfizer, Novartis and GSK) and one biotechnology company (Proximagen, currently BenevolentAI), where she was Head of Biology for the last 9 years.

Dr. Gonzalez specialises in the areas of pain, inflammation and neurodegeneration and she has been a successful project leader, with a proven track record of achievements in several programme leadership roles across all these organisations. In her latest role at Proximagen, she was responsible for the scientific integrity of the biological work, timely delivery of objectives and development of the team, in charge of scientific direction with relation to biological sciences and expert input into all programme teams at Proximagen, covering all stages from exploratory through discovery and into clinical phases. Key contributor of scientific assessment of business development opportunities, due diligence and external presentations to potential partners of internal assets. Responsibility to identify, monitor and present studies run by CROs in a variety of different therapeutic areas. Deep knowledge of all Home Office licences and regulations.

Dr. Gonzalez is a Graduate of the Universidad Complutense, Madrid (Spain) where she studied biology and completed a PhD in Animal Physiology in 1990.

Additional resources:
3dconsultants.org.uk/dr-isabel-gonzalez

Isabel Gonzalez
3D Consultants

Challenges to Create a Drug Repurposing Library and Its Application to the Treatment of AD
Bio

Amy Ripka, PhD, is a medicinal chemist with two decades of success in drug discovery for multiple disease areas including five biotech investigational new drug applications and more than 30 issued patents.

Dr. Ripka is founder and Chief Executive Officer of Lucy Therapeutics. She has worked as a medicinal chemist to develop drugs for various therapeutic areas including the central nervous system, anti-infectives, cancer, and cardiovascular disease. Her efforts have contributed to two approved drugs (Daclastasvir, Asunaprevir) and a compound in Phase II for schizophrenia. Dr. Ripka has held roles with Bristol Myers-Squibb, biotechnology companies including Infinity, HydraBiosciences, EnVivo [now FORUM], and two contract research organizations serving the pharmaceutical community, Sai Life Sciences and WuXiAppTec. This diversity gives her a unique appreciation and perspective on the interplay between these organizations and the drug discovery model.

Dr. Ripka received a bachelor’s degree in chemistry and music at Northwestern University. She earned her doctorate degree in organic chemistry and medicinal chemistry at the University of Wisconsin in Madison. Dr. Ripka was awarded a National Institutes of Health postdoctoral fellowship and continued her studies at The Scripps Research Institute under the Nobel Laureate, K. Barry Sharpless.

Additional resources:
www.lucytherapeutics.com/

 

Amy Ripka
Lucy Therapeutics

Ask your chemist which chemical series is right for YOU!
Bio

Dr. Feldman is a Professor in the Department of Neurosciences and Dean of Alzheimer’s and Neurodegenerative Research at UC San Diego.

He directs the Alzheimer’s Disease Cooperative Study (ADCS), a national grant-funded network and coordinating center that has a current portfolio of 10
clinical trials.

Show Less
Dr. Feldman has made vital contributions in scientific discoveries and clinical research studies focused on aging, mild cognitive impairment/Alzheimer’s disease (AD), frontotemporal dementia (FTD) and diagnostic/therapeutic trials. He has led numerous international clinical trials in AD resulting in important original data and informing care across the continuum of the disease. In the field of frontotemporal dementia, he has contributed to the discoveries of the progranulin (Nature 2006) and C9ORF72 (Neuron 2011) genetic mutations as well as to the elucidation of TDP43. He has helped lead the international working group that has reframed diagnostic criteria for Alzheimer’s disease that have re-conceptualized the disease and transformed the approach to clinical trials (Lancet Neurology 2007, 2011, 2014, 2016, 2021).

His career contributions have been profiled in Lancet Neurology in 2007. He has been named by Web of Science Clarivate Analytics as a ‘highly cited’ neuroscientist and among ‘the world’s most influential scientific minds’ (2008-18). He was appointed as a Fellow of the Canadian Academy of Health Sciences and the American Academy of Neurology in 2008. In 2019, he was also ranked #1 PI in Neurosciences funding from NIH by Blueridge Institute for Medical Research.

Additional resources: profiles.ucsd.edu/howard.feldman

Howard Feldman
UCSD

Early Trial Design for Repurposed Drugs

SESSION III: Advances in Biomarkers and Disease Monitoring

Tuesday, June 7, 2022
11:35 – 13:40 US Eastern Time

Bio

Eric Siemers, MD, has over 25 years of experience in clinical trials of neurodegenerative disease. His research research focus is on the use of biomarkers in investigational drug research, the development of trial designs that fully characterize the effects of investigational drugs on chronic diseases, and more specifically, the development of strategies for treating individuals before the onset of symptoms of neurodegenerative diseases.

Dr. Siemers most recently served as a distinguished medical fellow for Eli Lilly and Company’s Alzheimer’s Disease Global Development Team, where he was responsible for the design and implementation of five large Phase III clinical studies, in addition to playing a major collaborative role in 2 public-private partnership studies.

Prior to his appointments at Lilly, Dr. Siemers founded and headed the Indiana University Movement Disorder Clinic; his research there included investigations of Parkinson’s and Huntington’s diseases, and he established one of the first centers for surgical Parkinson’s treatments in the US.

Dr. Siemers served on the Board of Directors of the American Society of Experimental Neurotherapeutics, as founding member and Chair of the Alzheimer’s Association Research Roundtable, and Steering Committee member for the Alzheimer’s Disease Neuroimaging Initiative (ADNI). Dr. Siemers earned his medical degree with highest distinction from the Indiana University School of Medicine, where he completed an internship in the Department of Internal Medicine and residency in the Department of Neurology.

Eric Siemers
Siemers Integration LLC

CHAIR
Session Overview

Bio

Dr. Kaj Blennow obtained his medical degree (MD) in 1984, his PhD in Medical Science in 1990 and holds a Specialist Competence in both General Psychiatry and in Clinical Chemistry.

Dr. Blennow holds the Torsten Söderberg Professorship in Medicine and the Royal Swedish Academy of Sciences since 2015 and was appointed University Hospital Senior Physician in 2011. He is a professor and academic chair of the Clinical Neurochemistry department at the University of Gothenburg as well as Senior Consultant and Head of the Clinical Neurochemistry Lab at Sahlgrenska University Hospital.

The major research areas are cerebrospinal fluid biochemical markers, clinical proteomics, and the neurochemical pathogenesis of Alzheimer’s disease and other brain disorders.

Blennow has published more than 1200 original research papers and 150 review articles in peer-reviewed journals, and has an H-index of 133.

He has received a number of scientific awards, such as the 2019 Bengt Winblad Award, the 2013 International Foundation for Research in Alzheimer’s Disease (IFRAD) European Grand Prix in Research, Paris, the Henry Wisniewski Lifetime Achievement Award in Alzheimer’s Disease Research (2011), the ECNP Clinical Research Award (2010), the Alois Alzheimer Research Award (2001), and many more.

Kaj Blennow
University of Gothenburg

Classification and Prediction of Clinical Alzheimer’s Diagnosis Based on Plasma Proteins
Bio

.

Graham Jones
Novartis AG

Digital Biomarkers in AD and Related Dementia
Bio

Dr. Agneta Nordberg obtained her MPharm, MD and PhD at Uppsala University, Uppsala, Sweden.  After positions as Associate Professor and Senior Lecture in Pharmacology  at Uppsala University, she has been, since 1992, Professor in Clinical Neuroscience at Karolinska Institutet and Senior Consultant at Theme Aging, Karolinska University Hospital, Stockholm.

Dr. Nordberg has pioneered in the field of cholinergic neurotransmission, nicotinic acetylcholine receptors as well as in the development of amyloid PET imaging, introducing the multi-tracer PET concept, evaluating drug mechanism of action and visualising reactive astrogliosis and tau pathology in patients with Alzheimer’s disease and dementia disorders. Her frontline research findings have been published in leading journals (500+ publications, H-index 79) and she has been PI for more than 15 clinical PET trials and received several prizes and rewards.

Dr. Nordberg is leading a research team with a major focus on developing early diagnostic biomarkers and new drug targets in Alzheimer’s disease and other neurodegenerative proteinopathies.

Additional resources:
staff.ki.se/people/agneta-k-nordberg

Agneta Nordberg
Karolinska Institutet

Tau PET Imaging in Neurodegenerative Tauopathies
Bio

Emily Collins
Avid Radiopharmaceuticals

A Path Toward Precision Medicine in Alzheimer’s Disease

SESSION IV: Case Studies: Strategies for CNS Targets and Emerging Therapeutics

Tuesday, June 7, 2022
13:250 – 15:30 US Eastern Time

Bio

Dr. Debra Niehoff is the Director of Research & Grants for the Association for Frontotemporal Degeneration (AFTD). Dr. Niehoff oversees AFTD’s research portfolio, including grant programs supported in partnership with the Alzheimer’s Drug Discovery Foundation and Target ALS as well as networking, training, and mentorship opportunities that provide additional support to the FTD research community.

Dr. Niehoff received her PhD in Pharmacology from the Johns Hopkins University. Prior to joining AFTD, she developed and served as the coordinator for one of the first two-year neuroscience programs in the U.S. She is the author of The Biology of Violence: How Understanding the Brain, Behavior, and Environment Can Break the Vicious Circle of Aggression and The Language of Life: How Cells Communicate in Health and Disease and serves on the editorial board of the journal Violence and Gender.

Additional resources:
www.theaftd.org/

Debra Niehoff
AFTD

CHAIR
Session Overview

Bio

Dr. Ellen Cahir-McFarland earned a PhD in Immunology as Washington University in St. Louis studying T cell receptor signaling. Her postdoctoral fellowship and academic research lab at Brigham and Women’s Hospital focused on how Epstein-Barr Virus co-opts cellular signaling pathways to drive B-lymphocytes transformation.

In 2011, Ellen joined Biogen Discovery Research and advanced programs in virology, immunology, neuroimmunology and multiple sclerosis. In 2020, Ellen joined Annexon be part of the Ben Barres legacy to evaluate anti-C1q and complement therapies in neurodegeneration.

 

Additional resources: https://annexonbio.com/

Ellen Cahir-McFarlan
Annexon Biosciences

Inflammation, Immune Response, and Alzheimer’s
Bio

Dr. Katerina Akassoglou has pioneered studies in the investigation of neurovascular and neuroimmune mechanisms in neurologic diseases, and in particular the role of the blood clotting factors in CNS autoimmunity, trauma, and neurodegeneration. Her aim is to understand the mechanisms that control the communication between the brain, immune and vascular systems with the ultimate goal to design novel therapies for neurologic diseases—and in particular, multiple sclerosis and neurodegenerative diseases.

Dr. Akassoglou identified blood clotting factors as major mediators of neurologic disease. She made the unanticipated discovery that the blood clotting factor fibrinogen is a key activator of microglia in the CNS. She developed novel imaging tools to study the neurovascular interface and therapeutic strategies to protect from neuroimmune diseases by blocking the damaging effects of blood factors in the brain without affecting their beneficial effects in blood clotting. Dr. Akassoglou takes a multifaceted approach to her research, incorporating animal modeling, in vivo two-photon microscopy, drug discovery, preclinical translational research, and biomarker studies. Dr. Akassoglou has published over 90 papers in peer-reviewed journals and she is active in several national and international organizations, editorial boards, and funding agencies.

Dr. Akassoglou has received several awards, including the Presidential Early Career Award for Scientists and Engineers, the highest honor bestowed by the US government on outstanding scientists and engineers beginning their independent careers. She has also been presented with the John J. Abel Award, the Dana Foundation for Brain and Immunoimaging Award, the Vilcek Prize for Creative Promise honor, The Marilyn Hilton Award for Innovation in Multiple Sclerosis Research, the EUREKA and R35 Research Program Award by NINDS, and the Barancik Prize for Innovation in MS Research.

Dr. Akassoglou earned a BSc degree in biology and a PhD in neurobiology at the University of Athens, Greece. She was trained in neuropathology at the University of Vienna before performing her postdoctoral work at the Rockefeller University, and New York University. She started her laboratory as an Assistant Professor at the Department of Pharmacology at the University of California, San Diego where she was promoted to Associate Professor with tenure. She is now a Senior Investigator at the Gladstone Institute of Neurological Disease, and a Professor in the Department of Neurology at the University of California, San Francisco. She is also the founder and Director of the Gladstone Center for In Vivo Imaging Research.

Additional resources:
profiles.ucsf.edu/katerina.akassoglou

Katerina Akassoglou
UCSF and Therini Bio

Blood-brain Barrier in Health and Disease
Bio

Adam L. Boxer, MD, PhD, is Endowed Professor in Memory and Aging in the Department of Neurology at the University of California, San Francisco (UCSF). He directs UCSF’s Neurosciences Clinical Research Unit and the Alzheimer’s Disease and Frontotemporal Degeneration (FTD) Clinical Trials Program at the UCSF Memory and Aging Center. Dr. Boxer’s research is focused on developing new treatments and biomarkers for neurodegenerative diseases, particularly those involving tau and TDP-43.

Dr. Boxer received his medical and doctorate 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, a residency in Neurology at Stanford University Medical Center, followed by a fellowship in behavioral neurology at UCSF.

He is the principal investigator (PI) of the Advancing Research and Treatment for FTLD (ARTFL) Rare Disease Clinical Research Consortium, a collaborative project funded by the National Institutes of Health to create an 18-center North American research network to support the development of new therapies for FTLD. He also leads the Four Repeat Tauopathy Neuroimaging Initiative (4RTNI), a multicenter, longitudinal tau PET and biomarker study focused on PSP and CBD. He has been the PI for a variety of multicenter, randomized, placebo controlled clinical trials in FTLD spectrum disorders, including memantine for FTLD, davunetide for PSP, TPI-287 for primary and secondary tauopathies, and salsalate for PSP. In the past he has led a variety of clinical trials in FTD and PSP including a 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.

He is lead principle investigator for an international Phase 2 clinical trial of the tau monoclonal antibody, BIIB092, for PSP. He also leads the FTD Treatment Study Group (FTSG), a group looking to speed the development of new therapies for FTD.

Additional resources:
memory.ucsf.edu/people/adam-boxer-md-phd

Adam Boxer
UCSF

Antisense Oligodeoxynucleotide Targeting in FTD Treatment
Bio

Lawrence (Larry) Carter, PhD, is Vice President Clinical Science, Neurology at Alector where he is serving as the Clinical Lead on Alector’s development programs in FTD and ALS. He has worked across academic, industry, and government settings, with approximately 15 years of industry experience.

Prior to joining Alector, Dr. Carter was VP of R&D at Alexza Pharmaceuticals where he led drug-device combination development programs in rare diseases. Prior to that he was an Executive Director of Clinical Development and a Global Development Lead at Jazz Pharmaceuticals where he led the global development program for Sunosi, resulting in successful regulatory submissions for multiple indications in the US, EU, and Canada.

Dr. Carter has also served as a Special Government Employee to FDA and as a consultant to several pharmaceutical companies. He has a PhD in Pharmacology from the University of Texas Health Sciences Center at San Antonio and completed a postdoctoral clinical research fellowship in the Department of Psychiatry at Johns Hopkins University School of Medicine. He also currently serves as an Adjunct Assistant Professor of Pharmacology and Toxicology at the University of Arkansas for Medical Sciences.

Lawrence Carter
Alector

Progranulin as a Target in FTD and other Neurodegenerative Diseases