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
The speed of software changes in growing and large scale rapid-paced DevOps environments presents a challenge for continuous testing. Many organizations struggle to get this right. Practices that work for small scale continuous testing may not be sufficient as the requirements grow. In his session at DevOps Summit, Marc Hornbeek, Sr. Solutions Architect of DevOps continuous test solutions at Spirent Communications, explained the best practices of continuous testing at high scale, which is rele...
Web Real-Time Communication APIs have quickly revolutionized what browsers are capable of. In addition to video and audio streams, we can now bi-directionally send arbitrary data over WebRTC's PeerConnection Data Channels. With the advent of Progressive Web Apps and new hardware APIs such as WebBluetooh and WebUSB, we can finally enable users to stitch together the Internet of Things directly from their browsers while communicating privately and securely in a decentralized way.
Hardware virtualization and cloud computing allowed us to increase resource utilization and increase our flexibility to respond to business demand. Docker Containers are the next quantum leap - Are they?! Databases always represented an additional set of challenges unique to running workloads requiring a maximum of I/O, network, CPU resources combined with data locality.
"Matrix is an ambitious open standard and implementation that's set up to break down the fragmentation problems that exist in IP messaging and VoIP communication," explained John Woolf, Technical Evangelist at Matrix, in this SYS-CON.tv interview at @ThingsExpo, held Nov 4–6, 2014, at the Santa Clara Convention Center in Santa Clara, CA.
"A lot of times people will come to us and have a very diverse set of requirements or very customized need and we'll help them to implement it in a fashion that you can't just buy off of the shelf," explained Nick Rose, CTO of Enzu, in this SYS-CON.tv interview at 18th Cloud Expo, held June 7-9, 2016, at the Javits Center in New York City, NY.
Web Real-Time Communication APIs have quickly revolutionized what browsers are capable of. In addition to video and audio streams, we can now bi-directionally send arbitrary data over WebRTC's PeerConnection Data Channels. With the advent of Progressive Web Apps and new hardware APIs such as WebBluetooh and WebUSB, we can finally enable users to stitch together the Internet of Things directly from their browsers while communicating privately and securely in a decentralized way.
As software becomes more and more complex, we, as software developers, have been splitting up our code into smaller and smaller components. This is also true for the environment in which we run our code: going from bare metal, to VMs to the modern-day Cloud Native world of containers, schedulers and micro services. While we have figured out how to run containerized applications in the cloud using schedulers, we've yet to come up with a good solution to bridge the gap between getting your contain...
"We host and fully manage cloud data services, whether we store, the data, move the data, or run analytics on the data," stated Kamal Shannak, Senior Development Manager, Cloud Data Services, IBM, in this SYS-CON.tv interview at 18th Cloud Expo, held June 7-9, 2016, at the Javits Center in New York City, NY.
Who are you? How do you introduce yourself? Do you use a name, or do you greet a friend by the last four digits of his social security number? Assuming you don’t, why are we content to associate our identity with 10 random digits assigned by our phone company? Identity is an issue that affects everyone, but as individuals we don’t spend a lot of time thinking about it. In his session at @ThingsExpo, Ben Klang, Founder & President of Mojo Lingo, discussed the impact of technology on identity. Sho...
Information technology (IT) advances are transforming the way we innovate in business, thereby disrupting the old guard and their predictable status-quo. It’s creating global market turbulence. Industries are converging, and new opportunities and threats are emerging, like never before. So, how are savvy chief information officers (CIOs) leading this transition? Back in 2015, the IBM Institute for Business Value conducted a market study that included the findings from over 1,800 CIO interviews ...
"Operations is sort of the maturation of cloud utilization and the move to the cloud," explained Steve Anderson, Product Manager for BMC’s Cloud Lifecycle Management, in this SYS-CON.tv interview at 18th Cloud Expo, held June 7-9, 2016, at the Javits Center in New York City, NY.
Due of the rise of Hadoop, many enterprises are now deploying their first small clusters of 10 to 20 servers. At this small scale, the complexity of operating the cluster looks and feels like general data center servers. It is not until the clusters scale, as they inevitably do, when the pain caused by the exponential complexity becomes apparent. We've seen this problem occur time and time again. In his session at Big Data Expo, Greg Bruno, Vice President of Engineering and co-founder of StackIQ...
"I think that everyone recognizes that for IoT to really realize its full potential and value that it is about creating ecosystems and marketplaces and that no single vendor is able to support what is required," explained Esmeralda Swartz, VP, Marketing Enterprise and Cloud at Ericsson, in this SYS-CON.tv interview at @ThingsExpo, held June 7-9, 2016, at the Javits Center in New York City, NY.
The buzz continues for cloud, data analytics and the Internet of Things (IoT) and their collective impact across all industries. But a new conversation is emerging - how do companies use industry disruption and technology enablers to lead in markets undergoing change, uncertainty and ambiguity? Organizations of all sizes need to evolve and transform, often under massive pressure, as industry lines blur and merge and traditional business models are assaulted and turned upside down. In this new da...
"We got started as search consultants. On the services side of the business we have help organizations save time and save money when they hit issues that everyone more or less hits when their data grows," noted Otis Gospodnetić, Founder of Sematext, in this SYS-CON.tv interview at @DevOpsSummit, held June 9-11, 2015, at the Javits Center in New York City.