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2013
Unexpected Discovery Could Indicate New Target for ALS Therapy
Researchers funded by The ALS Association have discovered evidence of an unexpected cellular process in some people with amyotrophic lateral sclerosis (ALS). The results should allow researchers to better track the disease in these people and may offer a new target for developing therapy.
Study Funded by The ALS Association Finds That Neurons Can Be Reprogrammed to Take on New Identities
In work supported by The ALS Association and funded through The Milton Safenowitz Post-Doctoral Fellowship Program, researchers have for the first time reprogrammed a neuron from one type into another and have done so in a living organism. The finding will help scientists better understand how to control neuronal development and may one day aid in treating diseases in which neurons die, such as amyotrophic lateral sclerosis (ALS). The study was published in the journal Nature Cell Biology.
Biogen Announces It Will Discontinue Development of Dexpramipexole Based on Phase 3 Trial Results
Today, Biogen Idec reported top-line results of EMPOWER, a Phase 3 trial investigating dexpramipexole in people with amyotrophic lateral sclerosis (ALS). According to the company’s website.
2012
Scientists Discover Gene Appears to Influence How Long People Live with ALS
According to a new study published on August 26 in Nature Medicine, scientists have discovered a gene that influences how long people live with ALS. The international team that made the discovery was led by Wim Robberecht, M.D., of University Hospital in Leuven, Belgium.
Researchers Find Genetic Region Influencing Age at Which People Develop ALS
The ALS Association announced today that an international consortium of researchers it convened and funded has identified a region on chromosome 1 that strongly influences the age at which an individual develops amyotrophic lateral sclerosis (ALS). For the first time, scientists have found that people with ALS, who have a specific genetic signature within this region on chromosome 1, had an age of onset that is approximately two-and-a-half years earlier than those without it. The study was funded in part by The ALS Association and published today in Neurobiology of Aging.
ALS Association-funded Study Identifies Potential Therapeutic Target in RNA Enzyme
A new study funded in part by The ALS Association has revealed a potential new target for therapy to treat amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s Disease. This study, published in Nature Genetics on October 28, shows that lowering the level of a cellular enzyme reduces the disease-causing activity of a major ALS-related protein called TDP-43
The ALS Association Announces New Research Awards to Focus on Therapies for Lou Gehrig's Disease
The ALS Association will add ten awards totaling $2.9 million to its Translational Research Advancing Therapies for ALS (TREAT ALS™) portfolio, funding two clinical management grants, three drug discovery contracts, two clinical pilot studies and three biomarker studies—all focused on finding improved treatments for people with amyotrophic lateral sclerosis (ALS), otherwise known as Lou Gehrig’s disease. The ALS Association’s TREAT ALS ™ portfolio presently has 80 active research projects with a total award value of $12 million.
ALS Association-funded Study Identifies Potential Therapeutic Target in RNA Enzyme
A new study funded in part by The ALS Association has revealed a potential new target for therapy to treat amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s Disease. This study, published in Nature Genetics on October 28, shows that lowering the level of a cellular enzyme reduces the disease-causing activity of a major ALS-related protein called TDP-43.
Newest ALS Gene Impairs Axon Growth
A newly discovered ALS gene points to defects in axon growth as one cause of the disease. Mutations in the gene, called profilin 1 (PFN1), are responsible for 1% to 2% of familial ALS, according to the new study.
Anti-SOD1 Immunization Delays Onset, Increases Lifespan in ALS Mice
Immunizing ALS mice against the mutant SOD1 protein delays disease onset and increases lifespan, according to research funded by The ALS Association and published in Journal of Neuroscience. “This study supports previous data demonstrating the potential of immunization as a treatment strategy,” said ALS Association Chief Scientist Lucie Bruijn, Ph.D., “and furthermore describes the exact binding domain of the antibody on the mutant protein.” The study, led by Janice Robertson, Ph.D. at the University of Toronto, Canada, provides crucial insight into the mechanism through which mutant SOD1 causes disease.
New Study Suggests that ALS Progression May Be Tracked With Blood Test
According to a new study, funded in part by The ALS Association, published today in the Journal of Clinical Investigation, scientists have identified a biomarker in the blood that signals the earliest stages of the disease.
The ALS Association Announces $4 Million in Global Research Grants
The ALS Association announced today its latest research awards that include funding commitments of $4 million to researchers in 31 laboratories in the United States, United Kingdom, Belgium, Germany and Canada. These awards are part of its Translational Research Advancing Therapies for ALS (TREAT ALS™) program, through which The Association funds a diverse portfolio of research to find treatments and a cure for Lou Gehrig’s Disease.
Fly Model of ALS Suggested That Nuclear Localization of Mutant Protein Is Important for Toxicity
ALS Association-funded Haining Zhu, Ph.D., published together with Jianhang Jia, Ph.D., and colleagues in the publication Molecular Neurodegeneration demonstrating that over-expression of normal (Fused in Sarcoma) FUS protein and FUS protein carrying ALS-linked mutations led to motor degeneration and damage at the neuromuscular junction in a fly model.
Quest Diagnostics Announces C9orf72 Genetic Test for ALS
The company's Athena Diagnostics business unit unveiled the new test at the American Academy of Neurology Annual Meeting this week in New Orleans.
Quest Diagnostics, the world's leading provider of diagnostic testing, information and services, today announced a new genetic testing service from its Athena Diagnostics business unit, a leader in neurology diagnostics, for amyotrophic lateral sclerosis (ALS).
New ALS Research Involves TDP-43, Motor Neurons, Other Findings
Reducing expression of TDP-43 increases the level of SOD1, according to new research funded by The ALS Association. The finding highlights a previously unknown molecular link between two genetic causes of ALS and may lead to a better understanding of disease pathogenesis. Mutations in the TDP-43 gene are a rare cause of familial ALS, and the TDP-43 protein is found in protein aggregates in most forms of ALS, both familial and sporadic. The normal TDP-43 protein helps process RNA in the cell. It is not yet known how mutations in TDP-43 cause ALS.
Stem Cell Study Aids Quest for ALS Therapies
An article published in the journal Proceedings of the National Academy of Sciences reports a breakthrough using cutting-edge stem cell research, which could speed up the discovery of new treatments for amyotrophic lateral sclerosis (ALS) also known as motor neuron disease (MND) outside the United States.
Over-oxidized Form of Superoxide Dismutase in Sporadic Bulbar ALS Shares Toxic Mechanism with Mutant SOD1
A recent study published in the Proceedings of the National Academy of Sciences (PNAS) explored the role of Cu/Zn Superoxide Dismutase 1 (SOD1) found in sporadic ALS with bulbar onset. The team led by ALS Association-funded investigator Piera Pasinelli, Ph.D., Thomas Jefferson University, Pennsylvania, asked whether post-translational modifications to SOD1 could lead to disease in sporadic ALS cases.
The ALS Association and the Robert Packard Center for ALS Research at Johns Hopkins have entered into a partnership to expedite the development of animal model systems to expand the knowledge about the C9ORF72 gene, which has been identified as the most common cause of inherited amyotrophic lateral sclerosis (ALS or Lou Gehrig’s Disease) and Frontotemporal dementia (FTD).
MODDERN Cures Act to Spur ALS Research
At a press conference last month in Westfield, NJ, U.S. Representative Leonard Lance (7th District) announced the introduction of the Modernizing Our Drug and Diagnostics Evaluation and Regulatory Network (MODDERN) Cures Act. This bill would remove barriers to scientific research, provide incentives to develop new diagnostics, and foster medical innovations in order to ultimately accelerate the search for an effective cure and treatment for rare diseases such as ALS.
Northeast ALS Consortium Launches A New Website
The Northeast ALS Consortium (NEALS) has launched their new website devoted to supporting clinical research of Amyotrophic Lateral Sclerosis (ALS) and other motor neuron disease (MND). The website, supported by the ALS Therapy Alliance and The ALS Association, increases the availability, clarity, and accuracy of clinical research information for the ALS and MND community. To connect directly to the Basic Trial Search page click here.
2011
FDA Approves NeuRx Diaphragm Pacing System (DPS)®
Synapse Biomedical, Inc. announces that the U.S. Food and Drug Administration (FDA) has approved its NeuRx Diaphragm Pacing System (DPS)® for treating amyotrophic lateral sclerosis (ALS) patients who have stimulatable diaphragms and are experiencing chronic hypoventilation.
Interim Data on Neuralstem ALS Phase I Trial Announced
Eva Feldman, M.D., Ph.D., Principal Investigator of the Phase I safety trial of Neuralstem Inc's human spinal cord stem cells in ALS, presented primary and secondary endpoint data on the first 12 patients at the American Neurological Association's annual meeting. The report indicates the procedure to be safe and some improvement in motor function.
Linkage Found Between Familial ALS and Frontotemporal Dementia (FTD)
ALS Association-Funded Research Identifies New Genetic Mutation: the Most Common Cause of FTD and ALS Accounting for as Much as One Third of All Familial ALS
The ALS Association Greater New York Chapter is excited to announce a new major milestone in the battle against ALS. A recent study funded by The Abendroth ALS Genetic Discovery Fund has identified genetic abnormalities linking familial ALS and frontotemporal dementia (FTD). This finding provides the most significant clues to-date, as it accounts for greater than one-third of the inherited cases of ALS and FTD.
Funding for the Abendroth Fund was made possible by significant contributions from The ALS Association Greater New York Chapter and the Chapter Research Council. To become a member of the Research Council and help fund groundbreaking research such as this, please visit our website at www.als-ny.org to make a donation. Be sure to mark your gift’s designation as “Research”. Through the generous support of the Greater New York Research Council, members have contributed nearly $7 million to fund unique and diverse studies throughout the world.
Two independent studies, both funded by The ALS Association, have found a genetic abnormality that, according to researchers, is the most common cause of Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). As reported in the recent online issue of the scientific journal Neuron, an unusual mutation was discovered, where a short DNA sequence is repeated many more times as compared to healthy individuals.
Research Indicates TDP-43 and FUS/TLS Work Together
Although several new genes have now been linked to ALS including the recent exciting finding of mutations in the gene Ubiquilin-2 linked to familial ALS, exactly how these mutations cause disease remains unclear and is the focus of research in labs world-wide. Two proteins with similar structure and function, TDP43 and FUS, linked to familial ALS with and without frontotemporal dementia are thought to be involved in the disease, either by causing some new toxic property or by a loss of their normal function.
Two Genes that Cause Familial ALS Shown to Work Together
Although several genes have been linked to amyotrophic lateral sclerosis (ALS), it is still unknown how they cause this progressive neurodegenerative disease. In a new study, Columbia University Medical Center (CUMC) researchers have demonstrated that two ALS-associated genes work in tandem to support the long-term survival of motor neurons. The findings were published in the September 1 online edition of the Journal of Clinical Investigation.
New Research Points to Common Cause of ALS
According to an article in the journal Nature, investigators from Northwestern University Feinberg School of Medicine have identified a new gene linked to familial ALS involved in the processing of accumulated proteins. This provides further support for abnormal protein handling as an underlying cause of ALS.
2010
ALS Association and NINDS Sponsor Disease Modeling Workshop
The ALS Association and the National Institute of Neurological Disorders and Stroke (NINDS) co-sponsored a workshop that focused on the strategies to generate Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal lobar degeneration (FTLD) disease models.
ALS Association partners with ATA to match Tow Foundation challenge grant to establish ALS mouse model repository at The Jackson Laboratory
Researchers seeking ways to treat and cure amyotrophic lateral sclerosis (ALS) will soon have better access to mouse models of the disease.
Update on Stem Cell Research
On October 18, Neuralstem, Inc. updated the progress of its ongoing Phase I human clinical trial of the company’s spinal cord stem cells in the treatment of ALS at Emory University in Atlanta, Georgia.
Study Identifies New Genetic Risk Factor for Lou Gehrig’s Disease
An international study led by biologists and neuroscientists from the University of Pennsylvania, published this week in Nature, has identified a new genetic risk factor for amyotrophic lateral sclerosis, which is commonly known as ALS or Lou Gehrig’s disease.
Additional Information on New Study that Suggests a Potential Relationship Between Head Trauma and Motor Neuron Diseases
The study published in the Journal of Neuropathology and Experimental Neurology states that epidemiological evidence suggests that the incidence of an ALS-like motor neuron disease may be increased in association with repeated head injury.
New Study on Head Trauma And ALS
TDP-43 Proteinopathy and Motor Neuron Disease in Chronic Traumatic Encephalopathy. Researchers from Boston University are publishing a new study in the Journal of Neuropathology and Experimental Neurology on the relationship of head trauma and amyotrophic lateral sclerosis (ALS).
ALS Biomarkers Clinical Research Study Call for Participants
Researchers are looking for 250 people to participate in an ALS biomarker study.
Download The ALS Association's Spring 2010 Research ALS Today
Read all the latest research news from The ALS Associaiton's official publication.
Click here to download publication.
First U.S. Stem Cells Transplanted Into Spinal Cord
ALS Association Chief Scientist Lucie Bruijn, Ph.D., was interviewed by CNN regarding Thursday’s announcement about the first ALS patient to receive transplanted stem cells.
2009
$7.5 Million for the ALS Research Program at DOD $2.5 Million for Respite Care
This weekend, the U.S. Senate appropriated an additional $7.5 million for the ALS Research Program at the Department of Defense (DOD), a 50% increase over last year! The funding is in addition to the $6 million (20% increase) Congress appropriated for the National ALS Registry last week and also comes as Congress added another $2.5 million to support the Lifespan Respite Care Act, which helps to fund respite care programs in the states.
New “Antisense” Experimental Therapy Clinical Trial Announced
A new experimental therapy using an approach known as antisense, in which a drug is designed to shut down the RNA (Ribonucleic acid) that is responsible for the production of disease-causing proteins, is being prepared for a clinical trial in people with a familial form of ALS later this year. The clinical trial follows research funded by The ALS Association through TREAT ALS (Translational Research Advancing Therapy for ALS), our research pipeline that funds and facilitates the development of treatments for ALS based on important laboratory findings.
CDC/ATSDR Launch National ALS Registry Website
TodThe Centers for Disease Control and Prevention/Agency for Toxic Substances and Disease Registry (CDC/ATSDR) has launched the National ALS Registry website. This is the first phase of building a website that later will include an online portal where people with ALS can enroll in the ALS Registry. The website is available at www.cdc.gov/als. The ALS Association also will host a link to the Registry website on our homepage at www.als-ny.org.
A Randomized Clinical Trial of Lithium Carbonate with Riluzole versus Placebo with Riluzole in ALS Shows No Benefit.
In February 2008, Dr. Francesco Fornai and colleagues at the University of Pisa, Italy, reported in a pilot study that lithium carbonate at dosages of 300-450 mg daily (titrated to a plasma level of 0.4-0.8 mEq/liter) combined with riluzole showed a large positive effect in people with ALS (Fornai, F., et al., Lithium delays progression of amyotrophic lateral sclerosis. PNAS, 2008.105(6): p. 2052-2057).
Neuralstem Receives FDA Approval To Begin First ALS Stem Cell Trial
Neuralstem, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application to commence a Phase I trial to treat Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s disease) with its spinal cord stem cells.
Government Partnerships and Stem Cell Research Provide the Roadmap to a Cure
Leading scientists and government officials addressed a packed room at the Science Update and Stem Cell Update sessions held during The ALS Association’s 2009 National ALS Advocacy Day and Public Policy Conference, which took place in Washington, D.C., May 10-12. The speakers delivered the latest exciting information about the state of ALS research, educating attendees about new strategies for developing treatments and providing new reasons for hope in the fight against ALS.
International Group of Scientists and Drug Company Representatives Meet to Discuss Treatments for ALS
In April, The ALS Association convened a meeting of its Drug Company Working Group, bringing together more than 50 ALS researchers, clinicians, representatives of the pharmaceutical and biotech industries and the federal government, all of whom are working to create treatments for ALS.
The group discussed advances in four paths to treatment of ALS. Members exchanged ideas, asked each other hard questions, and worked together as they refined their approaches to their shared goal, helping to treat ALS.
Protective Gene Enables People with ALS to Live Longer, Study Finds
A new genetic discovery may help researchers understand factors that improve survival in people who have amyotrophic lateral sclerosis (ALS). The discovery, made as a result of a study funded in part by The ALS Association, also strengthens the theory that changes in cellular transport contribute to the death of motor neurons, the cells that die in ALS.
Clinician Scientist Receives Award
The AAN/TREAT ALS Clinician Scientist Development Award is funded through The Neil Brourman, M.D. ALS Research Fund.
The ALS Association and the American Academy of Neurology (AAN) are pleased to announce that Swati Aggarwal, M.D. from Massachusetts General Hospital, Massachusetts, is this year’s recipient for the 2009 AAN/ALS Association Clinician Scientist Development Award as part of TREAT ALS (Translational Research Advancing Therapies for ALS). The purpose of the award is to recruit talented and promising young clinicians to the ALS research field, and to foster their development to make significant contributions to ALS clinical research. Dr. Aggarwal’s study will focus on selection trial design for promising therapeutics in ALS.
ALS Association and American Academy of Neurology Give Top Scientists Sheila Essey Award for ALS Research
The ALS Association joins the American Academy of Neurology in presenting The 2009 Sheila Essey Award for ALS Research to two clinician scientists who have significantly impacted clinical trials, epidemiology and genetics in ALS.
The ALS Association Certifies ALS Clinic on Long Island as the 34th Center of Excellence in the United States
The ALS Association has certified the ALS clinic at Stony Brook University Hospital in East Setauket, N.Y., as its 34th Center of Excellence in the nation.
After a comprehensive review of its medical treatment, rehabilitation techniques and psychological support, The Association concluded that the Stony Brook ALS Center, which opened in 2002 and is located on Long Island, is providing the best care possible to those fighting Lou Gehrig’s Disease.
President Lifts Restrictions on Stem Cell Research
President Obama signed an Executive Order which lifted restrictions that limited federal funding for embryonic stem cell research. Under the previous policy, implemented by President Bush, federal funds could not be used to support embryonic stem cell research involving stem cell lines derived after August 9, 2001.
New Gene Mutation Discovery by ALS Association Consortium is Major Breakthrough in Lou Gehrig’s Disease Research
In one of the most significant breakthroughs in the recent history of ALS research, a consortium of scientists organized and funded by The ALS Association has discovered a new gene, ALS6 (Fused in Sarcoma), responsible for about 5 percent of the cases of inherited ALS. The discovery will provide important clues to the causes of inherited ALS, which accounts for 10 percent of all cases, and sporadic ALS, which occurs in individuals with no family history of the disease and accounts for the other 90 percent of cases diagnosed.
“This is a momentous discovery in furthering our understanding of ALS,” said Lucie Bruijn, Ph.D., senior vice president of Research and Development at The ALS Association. “A new gene provides a new piece of the puzzle we can use to shed light on why ALS develops, and where to focus our efforts on creating new treatments and finding a cure.”
2008
ALS Association Co-Funds Study About Nerve Cell Loss
ALS Association Co-Funds Study Showing Boosting the Body’s Detoxifying System Counteracts Nerve Cell Loss in ALS Mice; New Therapies Suggested
In a study that demonstrates a much-anticipated proof of principle, scientists report that raising activity of a natural detoxification system in the body can counteract the progressive loss of nerve cells that characterizes amyotrophic lateral sclerosis (ALS), significantly delaying the onset of disease and extending life.
The research, led by ALS Association-funded scientist Jeffrey Johnson at the University of Wisconsin, was carried out on two different mouse models carrying the human gene for a familial (inherited) type of ALS (Lou Gehrig’s Disease) and on cultures of motor neurons at risk of death from the gene.
Disappointing Results from IGF-1 Clinical Trial
By Richard Robinson, Science Writer
Subcutaneous (under the skin) delivery of insulin-like growth factor 1 (IGF-1), known as the drug Myotrophin, does not benefit people with ALS at a dose of 0.5 milligrams per kilogram of body weight, according to a large clinical trial whose results were announced.
IGF-1 is a substance the body produces to sustain motor neurons, the nerve cells that die in ALS. Experiments in animal models of the disease suggested IGF-1 treatment may delay death of motor neurons. IGF-1 was tested in ALS a decade ago in two trials, but the results of the two were inconsistent, with one suggesting treatment was beneficial, and the other showing no benefit.