Welcome!

News Feed Item

The ALS Association Announces New Grants to Investigate the Causes of and Treatments for Lou Gehrig's Disease

WASHINGTON, Feb. 4, 2014 /PRNewswire-USNewswire/ -- The ALS Association is pleased to announce the awarding of five new grants to investigate the causes and treatment of ALS. The ALS Association identified the focus of each of these grants as areas with high potential for progress.

(Logo: http://photos.prnewswire.com/prnh/20131211/MM32178LOGO)

ALS, which is also known as Lou Gehrig's Disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. There is no cure and only one drug approved by the U.S. Food and Drug Administration (FDA) that modestly extends survival.

"Through our TREAT ALS™ Portfolio, we are able to accelerate research in the most promising areas across the spectrum of ALS," said Lucie Bruijn, Ph.D., Chief Scientist for The ALS Association.

The TREAT ALS (Translational Research Advancing Therapies for ALS) Portfolio is a research endeavor enabling important global research to progress from the laboratory to the bedside.  The focus of the program is to support novel ideas, build tools, partner with academia and industry to identify new potential therapies and support the infrastructure for clinical trials with the goal to find meaningful treatments for ALS and a cure.

The funded grants are:

Palmitoylation of SOD1 and the pathogenesis of ALS
Principal Investigators: Raymond Roos, M.D. and William Green, M.D., University of Chicago, Chicago, Ill.
Funded by The ALS Association, Greater Chicago Chapter, through the State of Illinois

The investigators will determine whether a specific chemical change in the mutant SOD1 protein contributes to the misfolding of the protein that occurs in the disease. Mutations in SOD1 are one cause of familial ALS. Mutant protein misfolds, which is thought to contribute to the degeneration of motor neurons containing it. The investigators have discovered that mutant SOD1 undergoes a chemical change, called palmitoylation, more often than normal SOD1. They will examine the effect of this change on misfolding to determine if and how it contributes to the disease process and whether altering palmitoylation can be therapeutic.

Corticospinal motor neuron developmental control genes as candidates for human ALS susceptibility
Principal Investigators: Jeffrey Macklis, Ph.D. (Harvard University, Cambridge, Mass.), and Ammar Al Chalabi, M.D. (Kings College, London, UK)
Additional funds have been provided toward this study by MNDA UK

The development of motor neurons is under the control of a group of genes, each of which can exist in a variety of forms, or alleles. The specific set of alleles a person has may influence not only the development of their motor neurons, but also the susceptibility of those neurons to disease later in life, including ALS. The investigators will determine whether the alleles for these genes differ between people with ALS and those without it. Understanding whether and how developmental genes influence disease susceptibility will allow identification of many new targets for therapy.

Cognitive change and ALS staging: The relationship between cognitive and motor system involvement in ALS
Principal Investigators: Sharon Abrahams, Ph.D. (University of Edinburgh, Scotland); Ammar Al Chalabi, M.D. (Kings College, London, UK), and Orla Hardiman, M.D. (Trinity College, Dublin, Ireland)

People with ALS may experience cognitive changes as part of their disease, and these changes may influence their physical abilities. In this study, researchers will develop a better understanding of those cognitive changes and their relationship to physical functioning during the disease course. The aim is to both better understand this interaction and to develop a new disease staging system incorporating both cognitive and physical changes that can be used in clinical trials to track response to therapy.

AAV therapeutic agents for ALS
Principal Investigator: Zuoshang Xu, M.D. Ph.D., University of Massachusetts, Worcester, Mass.

In this study, researchers will further develop a gene therapy delivery vehicle, or vector, using a new type of adeno-associated virus (AAV). AAV has been shown to be a versatile vector for reaching neurons in the central nervous system with a good safety record. Here, the scientists will develop the ability to pack a so-called "interfering RNA" into the vector and test its therapeutic ability in rats carrying a disease-causing SOD1 mutation. Success in this study will increase the rationale for development of gene therapy in people with ALS.

Familial ALS exome capture and genomic sequencing collaboration
Principal Investigators: Benjamin Neale, Ph.D. (Massachusetts General Hospital, Boston, Mass.) and Christopher Shaw, M.D. (Kings College London, UK)

Several large-scale studies have produced volumes of data on gene variants associated with an increased risk for ALS. In this project, researchers will combine the data from those studies to better determine which of these gene variants is most strongly associated with disease. A key aspect of the research will be to stringently remove from consideration those variants that, despite some hint of association, are unlikely to be actually involved in ALS, thus streamlining the search for true risk genes. This project represents a major collaboration among multiple research groups, and its results will likely set the stage for developing much deeper insights into the genetics of ALS.

About The ALS Association
The ALS Association is the only national non-profit organization fighting Lou Gehrig's Disease on every front.  By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure.  For more information about The ALS Association, visit our website at www.alsa.org.

SOURCE The ALS Association

More Stories By PR Newswire

Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
While presenting own advanced Robo-Advisory Platform, Michał Różański, Managing Partner at EARP and CEO at Empirica, will illustrate the most important issues of building tailored FinTech software in his session at 20th Cloud Expo. He will share experiences we have gained for over 6 years of developing solutions for financial institutions and FinTech companies, including robo-advisors. We welcome all FinTech innovators interested in how properly implemented technology can move their businesses f...
A strange thing is happening along the way to the Internet of Things, namely far too many devices to work with and manage. It has become clear that we'll need much higher efficiency user experiences that can allow us to more easily and scalably work with the thousands of devices that will soon be in each of our lives. Enter the conversational interface revolution, combining bots we can literally talk with, gesture to, and even direct with our thoughts, with embedded artificial intelligence, whic...
Detecting internal user threats in the Big Data eco-system is challenging and cumbersome. Many organizations monitor internal usage of the Big Data eco-system using a set of alerts. This is not a scalable process given the increase in the number of alerts with the accelerating growth in data volume and user base. Organizations are increasingly leveraging machine learning to monitor only those data elements that are sensitive and critical, autonomously establish monitoring policies, and to detect...
Internet of @ThingsExpo, taking place October 31 - November 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA, is co-located with the 21st International Cloud Expo and will feature technical sessions from a rock star conference faculty and the leading industry players in the world. @ThingsExpo Silicon Valley Call for Papers is now open.
DevOps is often described as a combination of technology and culture. Without both, DevOps isn't complete. However, applying the culture to outdated technology is a recipe for disaster; as response times grow and connections between teams are delayed by technology, the culture will die. A Nutanix Enterprise Cloud has many benefits that provide the needed base for a true DevOps paradigm.
Most DevOps journeys involve several phases of maturity. Research shows that the inflection point where organizations begin to see maximum value is when they implement tight integration deploying their code to their infrastructure. Success at this level is the last barrier to at-will deployment. Storage, for instance, is more capable than where we read and write data. In his session at @DevOpsSummit at 20th Cloud Expo, Josh Atwell, a Developer Advocate for NetApp, will discuss the role and valu...
With major technology companies and startups seriously embracing Cloud strategies, now is the perfect time to attend @CloudExpo | @ThingsExpo, June 6-8, 2017, at the Javits Center in New York City, NY and October 31 - November 2, 2017, Santa Clara Convention Center, CA. Learn what is going on, contribute to the discussions, and ensure that your enterprise is on the right path to Digital Transformation.
DevOps is often described as a combination of technology and culture. Without both, DevOps isn't complete. However, applying the culture to outdated technology is a recipe for disaster; as response times grow and connections between teams are delayed by technology, the culture will die. A Nutanix Enterprise Cloud has many benefits that provide the needed base for a true DevOps paradigm. In his Day 3 Keynote at 20th Cloud Expo, Chris Brown, a Solutions Marketing Manager at Nutanix, will explore t...
Automation is enabling enterprises to design, deploy, and manage more complex, hybrid cloud environments. Yet the people who manage these environments must be trained in and understanding these environments better than ever before. A new era of analytics and cognitive computing is adding intelligence, but also more complexity, to these cloud environments. How smart is your cloud? How smart should it be? In this power panel at 20th Cloud Expo, moderated by Conference Chair Roger Strukhoff, pane...
SYS-CON Events announced today that SoftLayer, an IBM Company, has been named “Gold Sponsor” of SYS-CON's 18th Cloud Expo, which will take place on June 7-9, 2016, at the Javits Center in New York, New York. SoftLayer, an IBM Company, provides cloud infrastructure as a service from a growing number of data centers and network points of presence around the world. SoftLayer’s customers range from Web startups to global enterprises.
SYS-CON Events announced today that delaPlex will exhibit at SYS-CON's @ThingsExpo, which will take place on June 6-8, 2017, at the Javits Center in New York City, NY. delaPlex pioneered Software Development as a Service (SDaaS), which provides scalable resources to build, test, and deploy software. It’s a fast and more reliable way to develop a new product or expand your in-house team.
In his keynote at @ThingsExpo, Chris Matthieu, Director of IoT Engineering at Citrix and co-founder and CTO of Octoblu, focused on building an IoT platform and company. He provided a behind-the-scenes look at Octoblu’s platform, business, and pivots along the way (including the Citrix acquisition of Octoblu).
SYS-CON Events announced today that Tintri, Inc, a leading provider of enterprise cloud infrastructure, will exhibit at SYS-CON's 20th International Cloud Expo®, which will take place on June 6-8, 2017, at the Javits Center in New York City, NY. Tintri offers an enterprise cloud platform built with public cloud-like web services and RESTful APIs. Organizations use Tintri all-flash storage with scale-out and automation as a foundation for their own clouds – to build agile development environments...
SYS-CON Events announced today that DivvyCloud will exhibit at SYS-CON's 20th International Cloud Expo®, which will take place on June 6-8, 2017, at the Javits Center in New York City, NY. DivvyCloud software enables organizations to achieve their cloud computing goals by simplifying and automating security, compliance and cost optimization of public and private cloud infrastructure. Using DivvyCloud, customers can leverage programmatic Bots to identify and remediate common cloud problems in rea...
SYS-CON Events announced today that Carbonite will exhibit at SYS-CON's 20th International Cloud Expo®, which will take place on June 6-8, 2017, at the Javits Center in New York City, NY. Carbonite protects your entire IT footprint with the right level of protection for each workload, ensuring lower costs and dependable solutions with DoubleTake and Evault.