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
You think you know what’s in your data. But do you? Most organizations are now aware of the business intelligence represented by their data. Data science stands to take this to a level you never thought of – literally. The techniques of data science, when used with the capabilities of Big Data technologies, can make connections you had not yet imagined, helping you discover new insights and ask new questions of your data. In his session at @ThingsExpo, Sarbjit Sarkaria, data science team lead ...
Struggling to keep up with increasing application demand? Learn how Platform as a Service (PaaS) can streamline application development processes and make resource management easy.
Much of the value of DevOps comes from a (renewed) focus on measurement, sharing, and continuous feedback loops. In increasingly complex DevOps workflows and environments, and especially in larger, regulated, or more crystallized organizations, these core concepts become even more critical. In his session at @DevOpsSummit at 18th Cloud Expo, Andi Mann, Chief Technology Advocate at Splunk, will show how, by focusing on 'metrics that matter,' you can provide objective, transparent, and meaningfu...
If there is anything we have learned by now, is that every business paves their own unique path for releasing software- every pipeline, implementation and practices are a bit different, and DevOps comes in all shapes and sizes. Software delivery practices are often comprised of set of several complementing (or even competing) methodologies – such as leveraging Agile, DevOps and even a mix of ITIL, to create the combination that’s most suitable for your organization and that maximize your busines...
In his session at @ThingsExpo, Chris Klein, CEO and Co-founder of Rachio, will discuss next generation communities that are using IoT to create more sustainable, intelligent communities. One example is Sterling Ranch, a 10,000 home development that – with the help of Siemens – will integrate IoT technology into the community to provide residents with energy and water savings as well as intelligent security. Everything from stop lights to sprinkler systems to building infrastructures will run ef...
SYS-CON Events announced today that Peak 10, Inc., a national IT infrastructure and cloud services provider, will exhibit at SYS-CON's 18th International Cloud Expo®, which will take place on June 7-9, 2016, at the Javits Center in New York City, NY. Peak 10 provides reliable, tailored data center and network services, cloud and managed services. Its solutions are designed to scale and adapt to customers’ changing business needs, enabling them to lower costs, improve performance and focus inter...
In the world of DevOps there are ‘known good practices’ – aka ‘patterns’ – and ‘known bad practices’ – aka ‘anti-patterns.' Many of these patterns and anti-patterns have been developed from real world experience, especially by the early adopters of DevOps theory; but many are more feasible in theory than in practice, especially for more recent entrants to the DevOps scene. In this power panel at @DevOpsSummit at 18th Cloud Expo, moderated by DevOps Conference Chair Andi Mann, panelists will dis...
Up until last year, enterprises that were looking into cloud services usually undertook a long-term pilot with one of the large cloud providers, running test and dev workloads in the cloud. With cloud’s transition to mainstream adoption in 2015, and with enterprises migrating more and more workloads into the cloud and in between public and private environments, the single-provider approach must be revisited. In his session at 18th Cloud Expo, Yoav Mor, multi-cloud solution evangelist at Cloudy...
Artificial Intelligence has the potential to massively disrupt IoT. In his session at 18th Cloud Expo, AJ Abdallat, CEO of Beyond AI, will discuss what the five main drivers are in Artificial Intelligence that could shape the future of the Internet of Things. AJ Abdallat is CEO of Beyond AI. He has over 20 years of management experience in the fields of artificial intelligence, sensors, instruments, devices and software for telecommunications, life sciences, environmental monitoring, process...
Increasing IoT connectivity is forcing enterprises to find elegant solutions to organize and visualize all incoming data from these connected devices with re-configurable dashboard widgets to effectively allow rapid decision-making for everything from immediate actions in tactical situations to strategic analysis and reporting. In his session at 18th Cloud Expo, Shikhir Singh, Senior Developer Relations Manager at Sencha, will discuss how to create HTML5 dashboards that interact with IoT devic...
See storage differently! Storage performance problems have only gotten worse and harder to solve as applications have become largely virtualized and moved to a cloud-based infrastructure. Storage performance in a virtualized environment is not just about IOPS, it is about how well that potential performance is guaranteed to individual VMs for these apps as the number of VMs keep going up real time. In his session at 18th Cloud Expo, Dhiraj Sehgal, in product and marketing at Tintri, will discu...
So, you bought into the current machine learning craze and went on to collect millions/billions of records from this promising new data source. Now, what do you do with them? Too often, the abundance of data quickly turns into an abundance of problems. How do you extract that "magic essence" from your data without falling into the common pitfalls? In her session at @ThingsExpo, Natalia Ponomareva, Software Engineer at Google, will provide tips on how to be successful in large scale machine lear...
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.
The IoTs will challenge the status quo of how IT and development organizations operate. Or will it? Certainly the fog layer of IoT requires special insights about data ontology, security and transactional integrity. But the developmental challenges are the same: People, Process and Platform. In his session at @ThingsExpo, Craig Sproule, CEO of Metavine, will demonstrate how to move beyond today's coding paradigm and share the must-have mindsets for removing complexity from the development proc...
SYS-CON Events announced today that Ericsson has been named “Gold Sponsor” of SYS-CON's @ThingsExpo, which will take place on June 7-9, 2016, at the Javits Center in New York, New York. Ericsson is a world leader in the rapidly changing environment of communications technology – providing equipment, software and services to enable transformation through mobility. Some 40 percent of global mobile traffic runs through networks we have supplied. More than 1 billion subscribers around the world re...