Neuroscience 2019 Show Preview: The newest in the neurological

From neurodegenerative disorders to neurogenesis, Neuroscience 2019 showcases cutting-edge research

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49th Annual Meeting
Neuroscience 2019
October 19-23, 2019
McCormick Place Convention Center
The newest in the neurological
From neurodegenerative disorders to neurogenesis, Neuroscience 2019 showcases cutting-edge research
By Mel J. Yeates
The Society for Neuroscience (SfN) Neuroscience 2019 conference will be held in Chicago this year, offering a wide variety of sessions and events for its attendees. SfN’s meeting is, according to the society, an ideal venue for neuroscientists to present emerging science, learn from experts in the field, explore collaborations with peers, learn about new tools and technologies and advance careers—attendees should also plan to visit the exhibit hall for the huge gathering of neuroscience-focused companies that will showcase the latest tools and resources.
Neuroscientists, clinicians and advocates will be at the meeting, held Oct. 19 to 23, to be part of what is said to be the largest gathering of neuroscientists in the world. This will be SfN's 49th annual meeting, taking place at the McCormick Place convention center. SfN expects to welcome more than 30,000 attendees representing 80 countries, 536 exhibiting companies and almost 300 journalists. This year, the society has received nearly 14,000 abstracts with new research. There will be lectures by world-renowned scientists, symposia discussing advances in neuroscience and events for networkers, advocates and career-seekers.
According to Dr. Patricia Janak, Bloomberg Distinguished Professor at Johns Hopkins University and chair of the SfN Program Committee for Neuroscience 2019, “The SfN annual meeting is the biggest gathering of neuroscientists in the world. There is no better place to make serendipitous connections, get exposure to new sources of inspiration, or reveal new perspectives on your own research.”
Janak highlights in particular for our readers the Presidential Special Lecture by Dr. Adrian Krainer of Cold Springs Harbor Laboratory, noting: “An audience interested in clinical translation stories would get great value out of attending [this lecture]. He will discuss the development and clinical impact of nusinersen, the first FDA-approved drug for spinal muscular atrophy.”
Janak also notes three Basic-Translational-Clinical Roundtables entitled “Mechanisms of Drug Addiction: A Translational Perspective,” “Exoskeletons and Robotics for Neurorehabilitation” and “Gene Therapy in Neurological Diseases” that might particularly interest DDNews readers.
“We host a wide variety of professional development workshops for all career paths in the neuroscience community. Active researchers may benefit from our Short Course training opportunities, held on Friday, Oct. 18,” adds Janak.
SfN’s Pre-Conference Sessions, of which the Short Courses are a part, occur before the official start of the annual meeting. They provide an opportunity to learn from experts on emerging scientific topics and techniques, and advances in knowledge of neurobiological diseases.
 SfN has also brought back the Dual Perspectives session, which was new to the Neuroscience 2018 meeting. This year’s discussion will center around neurogenesis, and whether it happens in adult brains.
“After its standing-room only debut last year, the Dual Perspectives session is back for Neuroscience 2019,” continues Janak. “The session is a moderated discussion between two neuroscientists on opposite sides of an active question in neuroscience. This year, the lively discussion will revolve around [the question] ‘Does adult neurogenesis occur in the human brain?’”
Janak also expects the “Dialogues Between Neuroscience and Society” session to be quite popular this year.
“This year [the session] will center on the evolving role of AI in our day-to-day lives and on society as a whole. Our speaker is [Dr.] Fei-Fei Li, professor of computer science and co-director of the Stanford University Human-Centered AI Institute, and thought leader in this area since her time as vice president of AI and machine learning at Google Cloud,” Janak says.
Li plans to discuss the transformative potential that artificial intelligence and machine learning holds for society. She will use her perspective as a scientist and an ethical leader who advocates for future technologies to incorporate an understanding of how to augment, not replace, elements of the human experience.
 “Finally, this year we are celebrating SfN’s 50th anniversary,” Janak points out. “A number of events throughout the meeting will feature a past, present, and future perspective on the field of neuroscience. These events will have a special icon next to them in our online Neuroscience Meeting Planner and Neuroscience 2019 app.”
Attendees can access the Neuroscience Meeting Planner at The Neuroscience 2019 mobile app can be used to navigate the meeting’s science, exhibitors and events. It will be available in iOS and Google Play app stores in September.
As for looking forward towards next year’s meeting, Janak concludes, “Neuroscience 2020 in Washington, D.C., will be the 50th SfN annual meeting. We look forward to continuing our celebration with events and sessions that reflect on the past 50 years of progress, where the field stands today, and how our members can shape the neuroscience of tomorrow.”
Symposia at Neuroscience 2019
Theme A: Development
  • Circuit Variability and Plasticity in the Central Nervous System of Drosophila
  • From Single-Cell Profiling to Human Brain Organoids: Capturing Neural Development and Disease
Theme B: Neural Excitability, Synapses, and Glia
  • Dissecting Cerebellar Function: A Prototypical Circuit Critical for Motor Learning and Cognition
Theme C: Neurodegenerative Disorders and Injury
  • CNS Scarring, Inflammation, and Repair
  • Comparing Dopamine Metabolism in Mouse and Human Neurons: Relevance for Parkinson’s Disease
  • The Molecular and Spatial Complexity of Tau: What Forms and Loci to Target?
Theme D: Sensory Systems
  • New Approaches to Vision Restoration
Theme F: Integrative Physiology and Behavior
  • Cortical Disinhibitory Circuits: Cell Types, Connectivity, and Function
  • Neural Circuit and Plasticity Mechanisms of Cognitive Control of Feeding Behavior
Theme G: Motivation and Emotion
  • Epigenetic Mechanisms: Shared Pathology Across Brain Disorders
  • The Paraventricular Thalamus (PVT): Salience and Timing Orchestrator for Learning and Deciding
Theme H: Cognition
  • Opening the Black Box of the Hippocampus: Visualizing Memories in Distinct Cell Types, Microcircuits, and Cellular Compartments
Theme I: Techniques
  • Brain Somatic Mosaicism: Implications for Development and Disorders

Featured Panel Sessions
Dual Perspectives
Does Adult Neurogenesis Occur in the Human Brain?
Dr. Arturo Alvarez-Buylla and Dr. Maria Llorens-Martin
Whether neurogenesis continues in the adult human brain has been contested for decades. Adult neurogenesis is a fascinating phenomenon involving the birth, migration and functional integration of a new neuron into established neural networks. In this Dual Perspectives session, Llorens-Martín will present recent evidence supporting and Alvarez-Buylla will present recent evidence questioning the presence of new neurons in the adult human hippocampus.
Storytelling Session
The Storytelling Brain: How Neuroscience Stories Help Bridge the Gap Between Research and Society
Dr. Susana Martinez-Conde
This storytelling session brings together neuroscientific discovery, science reporting, and personal storytelling in a synergetic and memorable way. Broad-appeal lectures will interweave with personal stories to showcase the effective ways in which neuroscience researchers and journalists can collaborate, how storytelling provides a common ground.
Basic-Translational-Clinical Roundtables
Mechanisms of Drug Addiction: A Translational Perspective
Dr. Trevor W. Robbins
This roundtable will focus on translatability of basic research in animals to human research in addiction in order not only to understand neurobehavioral mechanisms of addiction, but also to define new strategies for discovery of clinical treatments, especially regarding the current opioid crisis. Topics to be discussed include the neural systems underlying addiction, neuronal adaptations occurring within those systems, how different drugs of abuse produce addiction, and the role of aberrant learning and vulnerabilities in the drive to addiction.
Exoskeletons and Robotics for Neurorehabilitation
Dr. Ann M. Spungen
This session will include a state-of-the-art overview of the use of robotics and exoskeletons in populations with neurological impairments. Specific presentations will include upper body robotic interventions for functional and neurological gains, robotic interventions for children with neurological impairments, and lower extremity exoskeletons for over ground ambulation. The lower extremity exoskeletal-assisted walking data will be presented from a randomized, controlled clinical trial.
Gene Therapy in Neurological Diseases
Dr. Asa Abeliovich
Gene therapy has advanced rapidly in the past five years, with technological advances and encouraging early clinical studies. This roundtable will focus on the opportunities and challenges as the field progresses, with a focus on the development of disease-modifying therapies that address urgent unmet needs of patients with neurological disorders. Discussion topics will include: technologies that are driving the field, with a focus on AAV platforms; leveraging advances in neurogenetics to identify validated therapeutic targets and patient subpopulations; rare monogenic disorders and clinical therapeutic strategies; the pursuit of gene therapy approaches for genetically complex disorders; and CNS region- and cell-selective approaches.
Featured Lectures
Albert and Ellen Grass Lecture—Neural Learning Rules in the Cerebellum
Dr. Jennifer L. Raymond, Stanford University School of Medicine
The cerebellum is known for its role in motor learning, and is increasingly implicated in cognitive functions such as navigation, reward prediction, emotion and social behavior. Its simple, repeated circuit architecture facilitates study of the functional links between events occurring at the molecular, cellular, circuit and behavioral levels as the cerebellum computes. By leveraging this analytical advantage, recent work has yielded new insight in the principles governing how neural circuits tune their performance through experience.
David Kopf Lecture On Neuroethics—The Neuroethics Frontier
Dr. Nita Farahany, Duke University
How should we think about our emerging capabilities of accessing and altering human brains, particularly in light of advances in genome-editing technologies? This lecture will focus on the ethical, legal and social issues arising from accessing and altering human brains. It will discuss consumer neuro-technologies, corporate interests in accessing and changing brains and government attempts to do the same. It will also consider the current and future potential directions of these neuroethical issues, particularly in light of recent controversies about human genome editing.
Dialogues Between Neuroscience and Society
Dr. Fei-Fei Li, Stanford Human-Centered AI Institute
Li challenges us to be the stewards of technology to serve humanity at its broadest and most diverse extent. In this session, she will discuss the transformative potential that AI and machine learning pose for society from her unique perspective as a scientist and an ethical leader who advocates for future technologies to incorporate an understanding of how to augment, not replace, elements of the human experience.
History of Neuroscience Lecture—Exocytosis of Synaptic Vesicles: From Quantal Release to Molecular Machines
Dr. Reinhard Jahn, Max Planck Institute for Biophysical Chemistry
At chemical synapses, depolarization-induced calcium influx triggers neurotransmitter release, a key step in synaptic signaling. In the 1950s, Katz found that transmitter release is quantal, and synaptic vesicles were discovered. In the following decades, recycling routes for synaptic vesicle and for neurotransmitters were worked out, but only since the mid-1980s are the molecular mechanisms governing the steps in synaptic vesicle cycling becoming known. The history of the field will be briefly reviewed, focusing on exocytosis and membrane fusion.
Peter and Patricia Gruber Lecture—Molecular Basis of the Circadian Clock in Mammals and Its Fundamental Role in Aging and Longevity
Dr. Joseph S. Takahashi, University of Texas Southwestern Medical Center, Howard Hughes Medical Institute
The molecular basis of circadian clocks involves a 24-hour autoregulatory transcriptional network that is cell-autonomous and widely expressed. The suprachiasmatic nucleus acts as master pacemaker, but peripheral oscillators can respond to proximal signals. In addition to behavior and physiology, the clock gene network interacts directly with many other pathways in the cell. With respect to metabolism, the timing of nutrient consumption is critical, and restricting the timing of feeding has many health benefits that impact aging, health span and longevity.
Presidential Special Lecture—From Base Pairs to Bedside: Antisense Modulators of RNA Splicing to Treat Neurological Diseases
Dr. Adrian R. Krainer, Cold Spring Harbor Laboratory
Nusinersen, the first FDA-approved drug for spinal muscular atrophy (SMA), exemplifies a successful path from basic studies to an effective therapy. It is an antisense oligonucleotide (ASO) that modulates alternative splicing of SMN2, increasing functional SMN protein in motor neurons. After clinical trials in SMA infants and children, nusinersen was approved in 2016. This lecture will describe the development of this drug and its clinical impact. Using a similar approach, an ASO was developed to correct defective RNA splicing of IKBKAP, which causes familial dysautonomia.
Presidential Special Lecture—The Cell Biology of the Synapse and Behavior
Dr. Daniel A. Colón-Ramos, Yale University School of Medicine
When, where, and how synapses form underpin the architecture of the nervous system and behaviors. Synapses are both precisely assembled during development and flexible during learning and memory. How can synapses be both precise and malleable to facilitate both the assembly and function of the brain? This lecture will discuss new findings that link the fundamental cell biological properties of single synapses to how they underpin the emergent property of the nervous system: behavior.
Presidential Special Lecture—Understanding Cortical Development and Disease: From Embryos to Brain Organoids
Dr. Paola Arlotta, Harvard University
Much remains unknown regarding the cellular and molecular mechanisms governing mammalian brain development. Focusing on the cerebral cortex, this lecture will present data on the mechanistic principles that control the developmental generation of cellular diversity in vivo, and consider to what extent processes of cortical development can be replicated outside the embryo, within brain organoids. This lecture will also discuss the challenges of modeling human corticogenesis in the dish, and the promise that brain organoids hold to investigate complex human neurodevelopmental disease.
Presidential Special Lecture—Wavefront Engineering: Illuminating the Neural Landscape
Dr. Valentina Emiliani, Vision Institute (CNRS, INSERM, Sorbonne University)
The revolution of optogenetics has opened perspectives in both fundamental and medical neuroscience unimaginable 10 years ago. Joint progress in the design of microbial opsins and in the shaping of wave fronts to precisely guide light through tissues is now bringing the field into a new phase that we can call circuit optogenetics, where neural circuits distributed across several brain areas can be optically interrogated and controlled with millisecond precision and single-cell resolution.
Professional Development Workshops
Saturday October 19, 2019
  • Preparing for Your Career Away From the Bench: Essential Skills for Navigating Your Career Transition
  • Reproducibility for Everyone
  • Imposter Syndrome: Confronting the Career Development Monster Hiding Under the Bed
  • Integrating Research and Teaching at Primarily Undergraduate Institutions
  • Getting Creative with Course-Based Research Experiences to Enhance Scholarship and Generate Publishable Data
  • How to Thrive as a Woman in Neuroscience
Sunday October 20, 2019
  • Bringing Genetic Diversity to Neuroscientific Research
  • Navigating Team Science
  • Becoming a Resilient Scientist
  • Science Management
  • Neuroscience Departments and Programs Workshop - Hiring and Promoting Faculty in the Era of Team Science
  • Building a Neuroscience Career at a Teaching Focused Institution
Monday October 21, 2019
  • Advancing Your Career Through Effective Science Writing for the Public and Creating Eye-Catching Research Statements
  • The Art of Building a Career
  • Optimize Your Grant Application: News You Can Use From the NIH
  • Teaching Computation in Neuroscience

SfN-sponsored Socials
Sunday Oct. 20
Brain and Retina Organoids Social
Breaking Barriers for Young Women in Science Social
Conversations on Cajal Social
Faculty for Undergraduate Neuroscience (FUN) Poster Session and Social
International Brain Bee Social
Neural Oscillations Social
Neuroethics Social
Neuroethology/Invertebrate Neurobiology Social
Neuroscience and Architecture: Measurement for Design Social
Open, FAIR, and Reproducible Neuroscience Social
Spinal Cord Injury Social
Monday Oct. 21
Behavioral Neuroendocrinology Social
Cerebellum Social
Chemical Senses Social
Epilepsy Social
Ingestive Behavior Social
Marmoset Social
Music Social
Open-Source Technology Social
Pain, Touch, and Itch Social
Psychopharmacology Social
Tuesday Oct. 22
Alzheimer's Disease and Related Dementias Social
Computational Neuroscience Social
Decision Neuroscience Social
Glia Social
Global Neuroscience Social
Hippocampus Social
Neuroendocrinology Social
Neuroscience and Writing Social
Platforms for Team Science and Data Sharing: Unlocking Data to Drive Innovation in Translational Research Social
Synapses Social
Future annual meetings
2020: Washington, D.C., Oct. 24-28
2021: Chicago, Nov. 13-17
2022: San Diego, Nov. 12-16
Alzheimer’s model reveals role of blood-brain barrier
CAMBRIDGE, Mass.—MIT engineers have developed a tissue model that mimics beta-amyloid’s effects on the blood-brain barrier, and used it to show that this damage can let molecules like thrombin into the brain, which can cause additional damage to Alzheimer’s neurons.
“We were able to show clearly in this model that the amyloid-beta secreted by Alzheimer’s disease cells can actually impair barrier function, and once that is impaired, factors are secreted into the brain tissue that can have adverse effects on neuron health,” said Roger Kamm, the Cecil and Ida Green Distinguished Professor of Mechanical and Biological Engineering at MIT.
Kamm and Rudolph Tanzi, a professor of neurology at Harvard Medical School and Massachusetts General Hospital (MGH), are the senior authors of the study, which appears in Advanced Science.
Alzheimer’s patients often experience damage to brain blood vessels caused by beta-amyloid proteins, called cerebral amyloid angiopathy (CAA). It’s believed that this damage allows harmful molecules to enter the brain more easily. Kamm decided to study this phenomenon by modeling brain and blood vessel tissue on a microfluidic chip.
“What we were trying to do from the start was generate a model that we could use to understand the interactions between Alzheimer’s disease neurons and the brain vasculature,” noted Kamm. “Given the fact that there’s been so little success in developing therapeutics that are effective against Alzheimer’s, there has been increased attention paid to CAA over the last couple of years.”
Kamm’s lab has worked on this project for several years, along with researchers at MGH who have engineered neurons to produce large amounts of beta-amyloid proteins. Led by MIT postdoc Yoojin Shin, who is the paper’s lead author, the researchers devised a way to grow neurons in a microfluidic channel. On the same chip, in a parallel channel, the researchers grew brain endothelial cells.
After 10 days of cell growth, the researchers added collagen to the central channel separating the two tissue types, allowing molecules to diffuse in between the channels. They found that within 3-6 days, beta-amyloid proteins began accumulating in the endothelial tissue, which became leakier. The endothelial cells also showed a decline in proteins that form tight junctions, and an increase in enzymes that break down the extracellular matrix that normally surrounds blood vessels. As a result, thrombin was able to pass from blood flowing through the leaky vessels into the Alzheimer’s neurons.
“We were able to demonstrate this bidirectional signaling between cell types and really solidify things that had been seen previously in animal experiments, but reproduce them in a model system that we can control with much more detail and better fidelity,” Kamm added.
The researchers also showed that a drug which restores the blood-brain barrier can slow down the cell death seen in Alzheimer’s neurons. They tested two FDA-approved drugs that have previously been shown to solidify the blood-brain barrier in simpler models of endothelial tissue. Researchers found that one of the drugs, etodolac, worked very well. In tissue treated with etodolac, the blood-brain barrier became tighter and neurons’ survival rates improved.
The MIT and MGH team is now working with a drug discovery consortium to look for other drugs that might be able to restore the blood-brain barrier in Alzheimer’s patients.
“We’re starting to use this platform to screen for drugs that have come out of very simple single cell screens that we now need to validate in a more complex system. This approach could offer a new potential form of Alzheimer’s treatment,” Kamm concluded.
Adapted from an article by Anne Trafton of the MIT News Office
A neurological research collaboration
LAUSANNE, Switzerland—AC Immune SA recently announced a research partnership with leading scientists in the Perelman School of Medicine at the University of Pennsylvania, which is focused on studying the pathological mechanisms of TDP-43 misfolding and aggregation.
TDP-43, the transactive response (TAR) DNA binding protein, is a transcription factor found in most human tissues, and a recently identified target of growing interest for neurological orphan indications such as frontotemporal lobar degeneration and amyotrophic lateral sclerosis. TDP-43 also plays an important role in other neurodegenerative diseases like Alzheimer’s disease.
The collaboration is aimed at figuring out how pathological forms of TDP-43 spread from neuron to neuron, leading to a better understanding of the TDP-43 pathologies in order to support the development of novel therapeutic and diagnostic approaches against FTLD and other neurodegenerative diseases.
AC Immune will contribute a two-year research grant to the laboratory of Dr. John Trojanowski and Dr. Virginia Man-Yee Lee. Trojanowski’s research focuses on neurodegeneration and has led to the novel view that major disease-related proteins (including Tau, alpha-synuclein and TDP-43) are co-deposited in most neurodegenerative diseases. He also studies how these copathologies impact the clinical representation of these diseases. Lee is a world-renowned leader in research on Tau, alpha-synuclein and the Abeta precursor protein, studying their pathobiological roles in neurodegenerative diseases.
Alkahest releases positive top-line data in Alzheimer’s
SAN CARLOS, Calif.—In August, Alkahest, Inc. announced top-line data from the Phase 2 clinical trial of its candidate GRF6019 in mild-to-moderate Alzheimer’s disease (AD) dementia.
The study was designed to evaluate the safety, tolerability, and potential therapeutic effects of multiple doses of GRF6019 in patients with mild-to-moderate AD over six months. Subjects were randomized and treated intravenously with 100mL or 250mL of GRF6019 for five consecutive days during week 1, and again for five consecutive days during week 13. A treatment-free interval of 12 weeks followed each dose. Dosing with GRF6019 was safe and well-tolerated in subjects with AD.
Secondary endpoints showed that subjects receiving GRF6019 had no decline in cognition, as measured by the 11-item AD Assessment Scale-cognitive subscale (ADAS-Cog11) and the Mini-Mental State Examination (MMSE). There was negligible decline in function by the AD Cooperative Study Activities of Daily Living scale 23-item version (ADCS-ADL23), and the Clinical Dementia Rating scale Sum-of-Boxes (CDR-SB) score. These data demonstrated a maintenance of cognitive and functional status over a period of 6 months in patients who would otherwise be expected to decline in this timeframe.
“These top-line results indicate a potential benefit of this plasma protein fraction in slowing the progression of cognitive decline in patients with mild to moderate Alzheimer’s disease,” said Dr. Karoly Nikolich, CEO of Alkahest. “GRF6019 represents an innovative approach to effectively treating Alzheimer’s disease by targeting multiple underlying mechanisms of disease, and these data support the continued development of GRF6019 and other plasma protein fractions in Alzheimer’s disease.”
Rewind Therapeutics gains €2.9 million VLAIO grant
LEUVEN, Belgium—Rewind Therapeutics has announced that the company will lead a new industry-academic R&D collaboration to discover and develop first-in-class inhibitors of a key G protein-coupled receptor (GPCR) target that has an influential role in myelin-related neurological diseases. The program is being supported with a €2.9 million grant from Flanders Innovation and Entrepreneurship (VLAIO).
“Therapies that promote myelin repair would represent an unprecedented approach to treating multiple progressive neurological diseases such as multiple sclerosis, and could prevent or reverse disability,” said Ian J. Reynolds, CEO of Rewind Therapeutics. “We are delighted to collaborate with world-leading teams in Flanders and further afield to discover and advance candidate molecules that may arise from the program. We are also grateful for the financial support from VLAIO and pleased to support its mission of advancing world-class innovation in the Flanders region.”
The collaboration partners include the Stem Cell Institute at KU Leuven (KUL) and Imec, a world-leading research and innovation hub in nanoelectronics and digital technologies. KUL will develop cellular screening assays based on human stem cells to assess the potential of small molecule candidates to block the target GPCR and induce nerve remyelination. Imec plans to devise a novel chip-based engineering approach to measure nerve myelination.
Added Professor Catherine Verfaillie, head of the KUL Stem Cell Institute, “We believe that our unique expertise with stem cells destined to become nerve cells such as oligodendrocytes, will be highly valuable as part of this new initiative to advance the understanding of re-myelination and the identification of new approaches to repair damaged nerves.”
Collectively, the collaboration partners will provide the expertise necessary to establish a disruptive innovation platform for studying the biology of nerve myelination and neuronal function. They will also provide unique advantages in discovering and advancing new potent and selective drugs for nerve and brain repair.
 “Imec, with a broad neurotechnology portfolio, engages in various research projects that aim to push forward the understanding of the brain and the development of therapies for neurological diseases,” noted Dries Braeken, R&D manager of Imec. “We are excited to leverage our multielectrode array chip platform and neural interfacing technology to help find new therapeutics for neurological diseases.”
Oryzon presents Phase 2a ETHERAL data
MADRID, Spain & CAMBRIDGE, Mass.—In July, Oryzon Genomics S.A. presented data from the company’s ongoing ETHERAL (Epigenetic Therapy in Alzheimer’s Disease) Phase 2a clinical trial with vafidemstat in Alzheimer’s disease (AD) at the 2019 Alzheimer's Association International Conference in Los Angeles. Dr. Roger Bullock, Oryzon’s medical director, presented a poster entitled “Safety Evaluation of Vafidemstat on Mild to Moderate Alzheimer’s Subjects.”
Vafidemstat (ORY-2001) is an oral, brain penetrant drug that selectively inhibits LSD1 and MAOB. The molecule acts on several levels by reducing cognitive impairment, including memory loss and neuroinflammation. It is also said to have neuroprotective effects.
Positive safety data from the first 104 patients in ETHERAL were presented, suggesting that vafidemstat is safe and well tolerated in AD patients. With 87.5 percent (91/104) of patients having completed at least one month of treatment, no clinically relevant effects on platelets, neutrophils and other hematological parameters have arisen, in keeping with the previous vafidemstat safety data from other clinical studies. Thirty-six patients have already passed the six-month threshold and no significant safety issues have been reported.
Bullock stated, ”We are happy with these first ETHERAL results, the drug is showing a very good safety profile in this aged and frail population often with comorbidities. This reinforces the safety data obtained in psychiatric younger patients and suggests that vafidemstat is well tolerated and with few adverse events. Although it is obviously premature to speculate about efficacy, as the study remains blind, the evolution of the functional parameters in these first 33 patients allow us to expect meaningful clinical readouts at the end of the study.”
The initial blind analysis into certain functional parameters in the first 33 individual patients who completed the first 24 weeks of treatment showed that while some patients show disease progression, in others the baseline values are maintained or even improved, in memory performance values measured by MMSE or aggressivity values measured by CMAI.
As for biomarker analysis (also blind), CSF levels of S100A9, a well know proinflammatory biomarker highly abundant in the AD brain, follow a similar pattern after the 24 first weeks of treatment. Only six patients showed a strong increase, while the others remained stable or showed a significant decrease.
The ongoing ETHERAL Phase 2a study is being conducted in 17 European hospitals in UK, France and Spain, and three additional sites in the U.S. The trial intends to evaluate the safety, tolerability and preliminary efficacy of vafidemstat in patients with mild to moderate AD. Secondary endpoints include measures of cognition, function and behavior. Several traditional and novel CSF biomarkers are also measured.
The European arm of the study plans to enroll up to 125 patients, with more than 100 patients already randomized. The U.S. arm of the trial plans to enroll up to 30 patients, to complete a minimum of 150 patients on the aggregate. The company has received a grant of $1.5 million from the Alzheimer’s Drug Discovery Foundation (ADDF) to support the U.S. arm of ETHERAL clinical trial.
A new research collaboration for chronic pain therapies
PALO ALTO, Calif.—Inscopix recently entered into a new research collaboration with Biogen to research and develop a new class of therapies for chronic pain. The collaboration builds on a study published in Science by leading pain researcher, Gregory Scherrer, Ph.D., and colleagues at Stanford University that identified the neural mechanism by which the brain encodes the affective component of pain.
In the study, the Stanford team found a group of neurons in the basolateral amygdala (BLA), a structure deep within the brain, that is responsible for the affective, i.e. unpleasant, component of pain. The BLA helps associate either positive or negative meaning to experiences encoded by brain signals, and earlier studies using functional magnetic resonance imaging have shown it to exhibit heightened activity during chronic pain in both humans and rodents.
Supported by Inscopix and Biogen, researchers in Scherrer’s laboratory will use Inscopix’s miniature microscope-based brain mapping platform to elucidate the fundamental neural circuitry underlying the unpleasantness of pain. The research will investigate how candidate pain therapeutics, such as opioid analgesics and sodium channel blockers, might modify its encoding in the BLA neuronal ensembles.
“Our growth at Inscopix has been focused on empowering a new era in neuroscience with our brain mapping solutions,” stated Dr. Kunal Ghosh, CEO of Inscopix. “We are proud to continue to support this research in chronic pain alongside Biogen, as the Stanford team builds on its breakthrough published work.”
Inscopix will support the research with scientific personnel and its nVoke product, which enables simultaneous imaging and manipulation of neural activity in living brains. Biogen will retain rights to develop therapeutic candidates based on the findings.

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