Show Preview for Neuroscience 2017: A mind-trip in D.C.
SfN marches on Washington (figuratively speaking) to expand neuroscience knowledge and networking
47th Annual Meeting
November 11-15, 2017
Walter E. Washington Convention Center
A mind-trip in D.C.
SfN marches on Washington (figuratively speaking) to expand neuroscience knowledge and networking
After hitting Chicago in 2015 and San Diego in 2016, the Society for Neuroscience (SfN) has cycled back around to the East Coast—Washington, D.C., to be exact—with its Neuroscience 2017 conference.
“SfN invites you to its renowned venue where neuroscientists collaborate and network with peers, learn from experts, explore the newest neuroscience tools and technologies, and discover great career opportunities,” the organization says, with the society’s 47th annual meeting—the world’s largest neuroscience conference for scientists and physicians devoted to understanding the brain and nervous system—to be held Nov. 11-15 at the Walter E. Washington Convention Center. Adds SfN: “Join more than 30,000 colleagues from more than 80 countries at the world’s largest marketplace of ideas and tools for global neuroscience.”
Regardless of which part of the country the annual meeting is held (and this year’s city is also home to SfN itself), one of the characteristics that remains constant is the broad variety of neuroscience and neurology topics and the depth that many of the educational sessions offer.
And perhaps one of the best ways to show that is to take a look at the myriad “featured lectures” on tap for Neuroscience 2017.
The Albert and Ellen Grass Lecture is titled “On Balance: Fine-Tuning Protein Levels for Neurological Health” and will be presented by Dr. Huda Y Zoghbi of the Baylor College of Medicine and Howard Hughes Medical Institute (HHMI). As SfN notes of the lecture, “When we think of the genetics of neurodevelopmental and neurodegenerative disorders, we tend to think about mutations that alter a protein’s function. An emerging theme among both classes of disorders, however, is the vulnerability of neurons to modest increases or decreases in protein levels—even when those proteins are wild type. This sensitivity to protein levels provides a new avenue to understanding pathogenesis and suggests we should search for regulators of disease-driving proteins that could provide therapeutic entry points for various neuropsychiatric disorders.”
Meanwhile, the David Kopf Lecture on Neuroethics, titled “The Fallacy of Fairness: Diversity in Academic Science” and the speaker for which is Dr. Jo Handelsman of the University of Wisconsin-Madison, will explore how we carry unconscious biases about other people that shape our evaluations of them and their work. “Evidence shows scientists are no exception and, despite our belief in objectivity, we apply substantial prejudice to many decisions,” SfN notes. “There are, however, proven methods and best practices that mitigate the impact of bias.”
In the featured lecture “Dialogues Between Neuroscience and Society” by Dr. Siddhartha Mukherjee of Columbia University, Mukherjee—a physician and researcher who wrote the Pulitzer Prize-winning book The Emperor of All Maladies: A Biography of Cancer—will engage in a conversation with SfN President Eric Nestler about the excitement and importance of communicating the promise of scientific inquiry to the public.
The History Of Neuroscience Lecture, titled “Neuronal Migration and Brain Map Formation During Evolution, Development and Disease” to be presented by Dr. Pasko Rakic of the Yale School of Medicine, for its part, deals with the notion that neuronal position is fundamental to a neuron’s identity, synaptic connections and, ultimately, function. “For example, cortical neurons are not generated locally and acquire their areal, laminar and columnar positions by migration from multiple distant sites of origin,” notes SfN. “Over the years, new experimental approaches have enabled identification of cellular mechanisms, genes, and molecular pathways that control neuronal production, fate, and migration to the proper position. These findings provide insight into brain evolution and development and the pathogenesis of its congenital disorders.”
Dr. Joshua R. Sanes of Harvard University will present the Peter and Patricia Gruber Lecture, “Assembling Neural Circuits: Cells and Synapses” and discuss how the retina is emerging as a leading model system for elucidating mechanisms that govern neural circuit assembly and function. This lecture will describe genetic, morphological and physiological studies that have led to identification of some molecules and mechanisms that underlie assembly of these circuits and then discuss new molecular methods that are enabling a comprehensive cataloging of neuronal cell types and the recognition molecules they use.
Four Presidential Special Lectures will be offered during Neuroscience 2017: “Illuminating Neurobiology at the Nanoscale and Systems Scale by Imaging” by Dr. Xiaowei Zhuang of Harvard University and HHMI; “Insights From Nonhuman Animals Into the Neurobiology of Language” by Dr. Erich D. Jarvis of the Rockefeller University and HHMI; “Polymorphous Polygenicity: The Story of the Genome in Schizophrenia” by Dr. Pamela Sklar of the Icahn School of Medicine at Mount Sinai; and “The Gut Microbiota and Childhood Undernutrition: Looking at Human Development From a Microbial Perspective” by Dr. Jeffrey I. Gordon of the Washington University School of Medicine.
Several featured lectures also are organized under nine themes that delineate much of the educational program: Theme A is Development; Theme B is Neural Excitability, Synapses, and Glia; Theme C is Neurodegenerative Disorders and Injury; Theme D is Sensory Systems; Theme E is Motor Systems; Theme F is Integrative Physiology and Behavior; Theme G is Motivation and Emotion; Theme H is Cognition; and Theme I is Techniques.
“Genetic Dissection of Neural Circuit Assembly and Organization” by Dr Liqun Luo of Stanford University and HHMI and “Neuroepigenetic Pathways in Learning and Memory in Mouse and Ant” by Dr. Shelley L Berger of the Perelman School of Medicine at the University of Pennsylvania both fall under Theme A.
Theme B also boasts two lectures: “Bridge Over Troubled Synapses: C1q Proteins, GluD Receptors, and Beyond” by Dr. Michisuke Yuzaki of the Keio University School of Medicine and “Spontaneous Activity in Developing Sensory Systems” by Dr. Dwight E Bergles of the Johns Hopkins University School of Medicine.
Likewise, Theme C offers a pair of featured lectures, which are the Clinical Neuroscience Lecture titled “Insights Into Neural Degeneration From Drosophila Genetics” by Dr. Nancy M Bonini of the University of Pennsylvania and “From Mechanisms of Neurogenesis to Neural Repair: Turning Scar-Forming Glia Into Neurons” by Dr. Magdalena Götz of Ludwig Maximilian University and Helmholtz Center’s Institute of Stem Cell Research.
Under Theme D are “Neural Circuits Controlling the Selection and Persistence of Sensory Information” by Dr. Tirin Moore of Stanford University and HHMI and “Processing Gustatory Information in Drosophila” by Dr. Kristin Scott of the University of California, Berkeley.
Themes E, F and G sport one featured lecture apiece which are, respectively, “Diversified Spinal and Brain Circuits for Locomotor Behavior” by Dr. Ole Kiehn of Karolinska Institutet and University of Copenhagen, “Molecular Architecture of the Circadian Clock in Mammals” by Dr. Joseph S Takahashi of the University of Texas Southwestern Medical Center and HHMI and “Carving the World Into Useful Task Representations” by Dr. Yael Niv of Princeton University.
Themes H and I, like most of the others, have two featured lectures. For the former, they are “Building Models of the World for Behavioral Control” by Dr. Timothy E. Behrens of the University of Oxford and “Using Memory to Guide Decisions” by Dr. Daphna Shohamy of Columbia University. For the latter, “Artificial Intelligence and Imagination: Exploring the Frontiers of Knowledge” by Dr. Demis Hassabis of DeepMind and “Tools for Optically Monitoring Neural Activity and Signaling Pathways” by Dr. Loren Looger of HHMI’s Janelia Research Campus.
For additional material the meeting not included here, of course, visit SfN’s website at www.sfn.org.
Symposia at Neuroscience 2017
Theme A: Development
- Developmental Origins of Neuronal Diversity in the Cerebral Cortex
- Impact of Zika Virus Infection in the Nervous System and Its Underlying Mechanisms
- Social Origins of Developmental Risk for Mental and Physical Illnesses
Theme B: Neural Excitability, Synapses, and Glia
- Assembly and Maintenance of the Peripheral Nerve Node of Ranvier in Development, Health, and Disease
- Unconventional NMDA Receptor Signalling
Theme C: Neurodegenerative Disorders and Injury
- Experimental Models Versus Reality of Neurological Disease
- Illuminating Neural Circuits: From Molecules to MRI
- Neuroimmune Interactions: A Status Change
- Tau Homeostasis and Toxicity in Neurodegeneration
- The Role of RNA Biology in Neurological Disease
Theme D: Sensory Systems
- Cortical Plasticity Following Sensory Loss and Restoration
Theme E: Motor Systems
- Neural Mechanisms of Voluntary Action Control: From Habits to Intentionality in Animals and Humans
Theme F: Integrative Physiology and Behavior
- Central Network Dynamics Regulating Visceral And Humoral Functions
- The Role of Extra-Suprachiasmatic Nucleus Brain Clocks in Circadian Regulation of Brain Function: Time Matters!
Theme G: Motivation and Emotion
- Circuit and Synaptic Plasticity Mechanisms of Drug Relapse
- From Salient Experience to Learning and Memory: Instructive Signals for Aversion and Reward
Theme H: Cognition
- Neural Correlates of Consciousness: Progress and Problems
- Neuronal Adaptation and Behavioral Performance in Perceptual and Economic Decisions
Theme I: Techniques
- Exciting New Tools and Technologies Emerging From the BRAIN Initiative
Future annual meetings
2018 San Diego Nov. 3-7
2019 Chicago Oct. 19-23
2020 Washington, D.C. Oct. 24-28
2021 Chicago Nov. 13-17
Professional Development Workshops
Professional Development Workshops at the SfN annual meeting are categorized by tracks to help attendees quickly identify the workshops that are of the greatest interest to them. This year’s tracks are Career Paths, Career Skills, Funding, and Teaching and Program Development.
Saturday, Nov. 11
- Careers in Translational Drug Discovery (Career Paths)
- Global Approaches for Collaboration & Networking (Career Skills)
- Incorporating Public Engagement Into Your Professional Portfolio: A Practical Guide (Career Skills)
- News You Can Use in Writing Grant Applications: Updates From NIH (Funding)
- How to be Successful in a Career in Academia (Career Paths)
- Research Mentor Training for Neuroscience Faculty (Career Skills)
Sunday, Nov. 12
- FAIR Data, Metadata, and Data Sharing in Neurotrauma (Career Skills)
- Navigating Career Transitions in Neuroscience (Career Paths)
- A Practical Guide to Science Communication (Career Skills)
- Funding Opportunities to Build Interdisciplinary Neuroscience Research for the Future (Funding)
- Addressing Issues Facing Women in the Early Stages of Their Scientific Career (Career Paths)
- Neuroscience Departments and Programs Workshop: Trends in Neuroscience Training—A Discussion of the SfN NDP Survey Results (Teaching and Program Development)
Monday, Nov. 13
- Evidence-Based Approaches to Teaching Neuroscience (Teaching and Program Development)
- The Power of Effective Storytelling: Communicating the Value of Brain Research (Career Skills)
- Improving Your Science: Sample-Size Planning, Pre-Registration, and Reproducible Data Analysis (Career Skills)
Monday, Nov. 13
Hearing Loss, Brain Function, and Healthy Aging
Dr. Frank R Lin
Strategies to optimize healthy aging are imperative. From 2000 to 2050, the proportion of the world’s population over age 60 will double from 11 percent to 22 percent, making this population larger than that of children under 14 for the first time in history. This session will discuss what constitutes healthy aging, explore hearing loss as an exemplar of a potential intervention target, and discuss the current and future role of neuroprostheses for hearing.
Tuesday, Nov. 14
Advances and Challenges in Deep Brain Stimulation
Dr. Andres M Lozano
More than 160,000 patients have received deep brain stimulation (DBS), mostly for Parkinson’s disease. This session will provide an overview of new DBS applications and discuss some of the emerging mechanisms and biological effects being discovered. The overall aim is to identify areas that require further exploration to optimize DBS therapy and to develop novel directions for this technology.
Wednesday, Nov. 15
Emerging Neuroimaging Biomarkers for Schizophrenia
Dr. John Krystal
This session will introduce the development of neuroimaging biomarkers for schizophrenia. It will cover diagnostic and subtyping biomarkers, biomarkers of genetic risk for schizophrenia, neurochemical and molecular markers of pathophysiology, and pathophysiological biomarkers related to illness progression and treatment.
Lab tour showcases value of research
In August, SfN member Christopher Cowan and his colleagues at the Medical University of South Carolina (MUSC) hosted an interactive lab tour for Daniel Head, a senior staffer for U.S. Sen. Lindsey Graham (R-S.C.). Cowan is a William E. Murray Endowed Chair in Neuroscience and the vice chair of the Department of Neuroscience at MUSC. Cowan’s research there explores the mechanisms that control proper brain wiring during development.
During the four-hour tour, Head visited MUSC’s neuroscience research facilities, including the Human Brain Recovery and Rehabilitation Labs and the Alcohol Research Center and learned about the sweeping impact of NIH funding on research at the institution.
Using Head as their subject, lab members demonstrated transcranial magnetic stimulation, which uses magnetic fields to stimulate nerve cells in the brain, triggering movement in his arm. This technology will help scientists to map brain function and has the potential to treat disorders such as Parkinson’s disease, chronic pain, and depression.
The tour also showcased cutting-edge neuroscience research made possible through NIH support, including the behavioral testing equipment used for studies related to autism, intellectual disability, and drug addiction, highlighting the critical role NIH funding plays in advancing scientific understanding on public health issues. Cowan also emphasized to Head the value of animal models for learning about human disease. The tour concluded with a roundtable discussion where Head and MUSC researchers discussed the value of federally supported research.
The trials of the mind
A roundup of recent clinical study news related to neurodegeneration and dementia
SAN DIEGO—On Oct. 4, ACADIA Pharmaceuticals Inc. announced the initiation of HARMONY, a Phase 3 study to evaluate pimavanserin for the treatment of hallucinations and delusions associated with dementia-related psychosis, a serious medical condition for which there is no therapy approved by the U.S. Food and Drug Administration (FDA).
The company also announced that the FDA has granted Breakthrough Therapy designation to pimavanserin for dementia-related psychosis. Dementia-related psychosis includes psychosis in patients with Alzheimer’s disease, dementia with Lewy bodies, Parkinson’s disease dementia, vascular dementia and frontotemporal dementia.
If the clinical development program is successful, and pimavanserin is ultimately approved by the FDA for the treatment of dementia-related psychosis, it would represent a significant expansion of the approved use of pimavanserin. Currently, pimavanserin is the only drug approved by the FDA for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis, and it is marketed under the trade name Nuplazid.
“We are pleased the FDA has agreed to an efficient development path for pimavanserin in this broad indication and granted Breakthrough Therapy designation in recognition of this serious unmet need,” said Dr. Serge Stankovic, executive vice president and head of research and development for ACADIA. “Initiation of our Phase 3 study is supported by clinical and preclinical evidence of pimavanserin’s antipsychotic activity without detrimental effects on cognitive function or other side effects associated with antipsychotics currently used off-label for this indication.”
Around 8 million people in the United States are living with dementia and approximately half are diagnosed with the disease. Studies suggest that approximately 30 percent of patients with dementia have psychosis, commonly consisting of hallucinations and delusions. Serious consequences have been associated with severe or persistent psychosis in patients with dementia such as repeated hospital admissions, earlier progression to nursing home care, more rapid progression of dementia and increased risk of morbidity and mortality.
“With receipt of FDA’s Breakthrough Therapy designation for pimavanserin, we are able to accelerate this important program,” said Steve Davis, president and CEO of ACADIA. “Pimavanserin has a unique biological mechanism that distinguishes it from any other antipsychotic. We believe the profile we observed in our [Phase 2 study] in Alzheimer’s disease psychosis could be particularly beneficial in this elderly underserved population. In that study, pimavanserin demonstrated antipsychotic effect without impairing cognition and we also observed a very favorable tolerability profile. We were very excited to be the first and only FDA-approved drug for the treatment of Parkinson’s disease psychosis and are equally excited about the potential to help many more patients suffering from dementia-related psychosis.”
Trial commences for ITI-214 in Parkinson’s disease
NEW YORK—Intra-Cellular Therapies Inc., a biopharmaceutical company focused on the development of therapeutics for central nervous system (CNS) disorders, announced in early October that it had begun enrollment in the ITI-214-105 Phase 1/2 clinical trial in patients with Parkinson’s disease (PD).
“ITI-214 is a potent and selective phosphodiesterase 1 (PDE1) inhibitor. Following the favorable safety and tolerability results in our Phase 1 program, we are pleased to announce the advancement of ITI-214 for the treatment of patients with Parkinson’s disease,” said Dr. Sharon Mates, chairman and CEO of Intra-Cellular Therapies. “This study is designed to evaluate the safety and tolerability of ITI-214 in this patient population, as well as evaluate the ability of ITI-214 to treat both motor and nonmotor symptoms associated with Parkinson’s disease and to provide a framework for future trials to assess disease modification.”
As the clinical lead compound in the company’s PDE1 portfolio, ITI-214 has been found to be generally well tolerated with a favorable safety profile in four Phase 1 clinical trials in healthy volunteers as well as patients with schizophrenia. Inhibitors of PDE1 block the breakdown of cyclic nucleotides (cAMP, cGMP), potentiating downstream intracellular signaling. The PDE1 enzyme is highly active in pathological or disease states, and the company's PDE1 inhibitors are designed to reestablish normal function in these disease states.
PDE1 inhibitors have minimal effect on normal function, only acting when cells in the nervous system are stimulated. These “on-demand” effects make this a promising and novel approach for the treatment of disease. In animal models, inhibition of PDE1 has been shown to reduce neuroinflammation and to reduce neurodegeneration. The mechanism of action of PDE1 inhibitors suggests therapeutic potential across a variety of neurological and cardiovascular diseases.
Preclinical studies suggest that PDE1 inhibitors potentiate L-DOPA and other dopamine replacement therapies for motor symptom control at lower doses of dopamine replacement therapies while inhibiting the adverse dyskinesias induced by these treatments. Preclinical models have also shown the potential for PDE-1 inhibitors to address non-motor symptoms: excessive daytime sleepiness, cognitive impairment and other symptoms.
Phase 1b study begins for NP001 in Alzheimer’s Disease
SAN BRUNO, Calif. & HONOLULU—Neuraltus Pharmaceuticals Inc., a privately held biopharmaceutical company dedicated to developing innovative therapeutics for neurodegenerative diseases, and the John A. Burns School of Medicine at the University of Hawaii at Manoa recently announced the initiation of a Phase 1b placebo-controlled biomarker study of the company’s investigational therapy, NP001, in individuals with Alzheimer’s disease. This study (NCT03179501) is being conducted to determine the effect of a single dose of NP001 on blood markers of inflammation in individuals with mild-to-moderate Alzheimer’s disease.
“Although Alzheimer’s disease is the most common cause of dementia, there is presently no cure for the more than five million Americans living with the disease,” said Dr. Beau Nakamoto, who is an associate professor in the Department of Medicine at the University of Hawaii at Manoa and at the John A. Burns School of Medicine, as well as principal investigator. “Given the key role inflammatory monocytes and macrophages may play in the progression of Alzheimer’s disease, researching immune regulators—like NP001—is critical to understanding more about this serious and complex disease.”
AI drives discovery of novel predictors of Parkinson’s disease progression
CAMBRIDGE, Mass.—In late September, GNS Healthcare (GNS), a precision medicine company, announced the discovery of genetic and molecular markers of faster motor progression of Parkinson’s disease patients—the LINGO2 gene together with a second genetic variant, along with demographic factors.
The publication describing the discovery, titled “Large-scale identification of clinical and genetic predictors of Parkinson’s disease motor progression in newly-diagnosed patients: a longitudinal cohort study and validation,” appears in the journal The Lancet Neurology. The discovery was powered by patient data from the Parkinson’s Progression Markers Initiative sponsored by the Michael J. Fox Foundation for Parkinson’s Research.
“Being able to use these predictors in the clinical setting will lead to faster and significantly cheaper clinical trials and accelerate the availability of new Parkinson’s disease drugs for patients in need,” said Colin Hill, chairman, CEO and co-founder of GNS. “A major hurdle in Parkinson’s research is that rates of progression are extremely varied. Some patients progress very quickly while others do not. With accurate predictors of rates of progression, we will be able to remove uncertainties from drug development and patient response, reduce the number of clinical trial enrollees required by as much as 20 percent, and speed up the development of effective new drugs.”
REFS, the GNS causal machine learning and simulation platform, was used to transform the longitudinal genetic and clinical patient data from 429 individuals (312 Parkinson’s patients and 117 controls) into computer models that connect the genetic and molecular variation of patients to motor progression rates. These computer models were used to simulate the future effects of the genetic and prognostic variables on motor outcomes, essentially predicting the motor progression rate for each patient. The models were validated in an independent longitudinal study, and clearly demonstrated the ability to prospectively differentiate between patient progression rates.
“There is still so much to understand about the progression of chronic, debilitating illnesses like Parkinson’s disease,” said Dr. Jeanne C. Latourelle, a co-author of the study and director of precision medicine at GNS Healthcare. “The validation of our models in this study underscores the power of our REFS technology and its ability to accelerate the development of effective therapies for patients in need.”
AC Immune gets continuation on 2015 Michael J. Fox grant
LAUSANNE, Switzerland—AC Immune SA, a Swiss-based, clinical stage biopharmaceutical company with a broad pipeline focused on neurodegenerative diseases,on Oct. 3 announced it had been awarded a continuation of a February 2015 research grant from the Michael J. Fox Foundation for Parkinson’s Research (MJFF). This provides funds for the development of Positron Emission Tomography (PET) tracers for the alpha-synuclein protein to support the early diagnosis and clinical management of Parkinson’s disease.
AC Immune has been collaborating on this biomarker program with Biogen, since April 2016. AC Immune expects to move the program into clinical development in 2018.
“We are honored to receive continued funding from The Michael J. Fox Foundation for this important diagnostic program in Parkinson’s disease, which we are working on in collaboration with Biogen,” said Prof. Andrea Pfeifer, CEO of AC Immune. “The support of MJFF further validates AC Immune’s leading expertise in the pathology of misfolded proteins. We continue to leverage our proprietary technology platforms, developing both diagnostic and therapeutic agents for multiple neurodegenerative diseases.”
“The development of an alpha-synuclein imaging agent would be transformative for Parkinson’s disease patient care and drug development. We are pleased with the progress of the AC Immune program toward this critical goal,” commented Dr. Jamie Eberling, director of research programs at MJFF.
Such alpha-synuclein-PET tracers would help to diagnose Parkinson’s disease earlier and more accurately. This technology has multiple advantages including direct detection of alpha-synuclein pathology in patients and the capacity to monitor the efficacy of therapeutics reducing alpha-synuclein aggregates in clinical trials. AC Immune’s proprietary Morphomer chemistry technology platform is designed to interact with misfolded and aggregated proteins. Promising small molecules have been identified with good selectivity for alpha-synuclein and suitable properties for the development as PET ligands.
A pair of nods from FDA
On Sept. 6, NeuroRx, a clinical stage biopharma company developing the first oral therapy for acute suicidal ideation and behavior (ASIB), reported that the U.S. Food and Drug Administration (FDA) had granted Fast Track status for its sequential therapy of NRX-100 (ketamine HCl) followed by NRX-101 (D-cycloserine+lurasidone).
The company will shortly begin enrolling patients in a pivotal trial of this sequential therapy targeting patients who are admitted to emergency departments with ASIB in bipolar depression.
Fast Track Designation is awarded by the FDA to investigational drugs that are deemed by FDA to treat a serious medical condition and for which there is preclinical and/or clinical data to demonstrate that the drug has the potential to address an unmet medical need. Suicidality in bipolar depression is a condition for which there is currently no approved drug therapy and for which the only FDA-approved treatment is electroconvulsive therapy.
Nearly a week later came news from global biotherapeutics company CSL Behring that the FDA has approved Privigen for the treatment of adults with chronic inflammatory demyelinating polyneuropathy (CIDP) to improve neuromuscular disability. CIDP is a rare autoimmune disorder that affects the peripheral nerves and may cause permanent nerve damage.
“The FDA approval of Privigen for CIDP represents a significant milestone for individuals with this debilitating and progressive disease. It is a testament to our commitment to meet the needs of patients with disabling neurologic conditions, including CIDP,” said Dr. Andrew Cuthbertson, chief scientific officer and director of research and development for CSL Limited. “As we focus on building a leading neurology franchise, we continue to advance clinical research to determine innovative and improved uses of immunoglobulin therapy that can benefit patients and improve their quality of life.”
“It is a priority in the care of CIDP patients to provide therapies that improve and maintain strength and function while at the same preventing relapses and minimizing side effects. However, current treatments do not work for all CIDP patients,” added Dr. Mazen M. Dimachkie, executive vice chairman of the Department of Neurology at the University of Kansas Medical Center and an investigator in the PATH study that helped lead to the approval. “Privigen’s approval by the FDA for the treatment of CIDP means that people with CIDP and their treating physicians have gained another treatment option that is safe and effective in helping improve strength and motor function, while potentially delaying disease relapse.”
ProQR spins out Amylon to focus CNS therapeutics
LEIDEN, the Netherlands—In mid-September, ProQR Therapeutics N.V. announced that it had spun out Amylon Therapeutics, a privately held company focused on the development of therapies for central nervous system (CNS) disorders, with seed funding from a group of institutional and private investors.
As part of the transaction, ProQR has granted an exclusive license to Amylon to develop therapeutics for beta amyloid related disorders. Amylon will use the license and proceeds to continue research and development of therapies for CNS disorders, with an initial focus on a RNA-based treatment for a rare genetic disease that leads to strokes at mid-adulthood, called hereditary cerebral hemorrhage with amyloidosis of the Dutch type. A future beta amyloid related indication could include cerebral amyloid angiopathy. ProQR retains majority ownership in Amylon and is entitled to future milestones and royalties from the products developed by Amylon on the terms and conditions set forth in the license.
Amylon will be led by Thomas de Vlaam as founding CEO; Thomas joined ProQR in 2015 to incubate the CNS activities, with the goal of spinning it out into a new company.
Since its inception in 2012, ProQR has invested significantly in discovering and developing innovative RNA therapies for severe genetic rare diseases. This has led to an extensive pipeline of discovery development programs that are harbored in separate business units with dedicated teams of specialists focused on cystic fibrosis, eye diseases, skin diseases and CNS diseases.
“As part of our corporate strategy to bring as many of our programs to patients as possible, we built ProQR as a group of focused business units that enable us to attract external funding directly into the individual development programs or ultimately spin programs out as we have now done with Amylon,” said Daniel de Boer, CEO of ProQR. “Through spinning out Amylon, we position the beta amyloid program for optimal success with a dedicated team and the funding to develop treatments for patients suffering from these devastating brain diseases.”
Collaborating on Brain Commons
WASHINGTON, D.C.—Cohen Veterans Bioscience recently announced its partnership with the Open Commons Consortium (OCC) to establish the Brain Commons, a one-of-a-kind cloud-hosted site for combining Big Data that will be critical for the understanding of brain conditions. The OCC platform is uniquely positioned to manage large-scale imaging data, genomic data, wearables, and clinical data, as well as enable machine learning and analytics at state-of-the-art computing speeds to accelerate our understanding of brain conditions and brain health.
The Brain Commons database will be hosted at the University of Chicago’s OCC—its director of the Center for Data Intensive Science, Robert Grossman, said: “We are thrilled to collaborate with a progressive organization like Cohen Veterans Bioscience to develop an entirely new way to bring together brain data from across the community. This has already been a powerful approach in cancer genomics, and we are excited ... to see how our experience can now be extended to impact brain health.”
The Brain Commons will also leverage the partnership between Cohen Veterans Bioscience and Exaptive Inc. to build a Cognitive City, a user interface to the Commons. The Cognitive City brings data, software tools, and people together in an ecosystem where interoperability, modularity, and community are core features and boundary-crossing through repurposing data and code is actively facilitated.
“Just like physical cities are built where there are ample natural resources, a Cognitive City must be built atop an easily accessible supply of data,” said Dave King, founder and CEO of Exaptive. “The Brain Commons offers just such an abundance – a flexible repository that can scale in both breadth and depth, allowing for new types of analyses to be performed across diverse datasets.”