“This is the largest collection of familial Alzheimer’s whole-genome sequences in the world,” Dr. Tanzi says, comprising half a petabyte of data, equivalent to the entire contents of the Library of Congress. “This is as big as big data gets.”
Neurofibrillary tangles - largely composed of tau protein- are one of the two pathological hallmarks of Alzheimer’s disease.
12/10/2013: Alzheimer's Early Treatment
Can anti-amyloid antibody treatment reverse Alzheimer’s disease pathology before memory loss sets in?
12/10/2013: Alzheimer’s Preclinical Disease Biomarker
Mark Albers, MD, PhD, is developing a set of simple but powerful tools to screen for the very earliest stages of preclinical Alzheimer’s Disease.
A study led by MGH investigators shows that even low levels of the Alzheimer's-associated APOE4 protein can increase toxic amyloid beta brain plaques and the characteristic neuronal damage in mouse models of the disease. Introducing APOE2, a rare, potentially protective variant, reduced amyloid deposits and associated damage.
MGH researchers have identified and validated two rare gene mutations that appear to cause the common form of Alzheimer's disease (AD) that strikes after the age of 60. The two mutations occur in a gene called ADAM10, which now becomes the second pathologically-confirmed gene for late-onset AD and the fifth AD gene overall.
Understanding the molecular pathogenesis of Huntington’s disease.
An assay designed to measure normal and abnormal forms of the huntingtin protein – the mutated form of which causes Huntington's disease – was successful in detecting levels of the mutant protein in a large multicenter study of individuals at risk for the devastating neurological disorder.
Applications are being sought for a joint MGH/Biogen Idec two-year training program in translational neuroscience.
The second study and first clinical trial conducted by the Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT) will focus on Progressive MS.
07/22/2013: E Pluribus Unum for Parkinson Disease - Researchers Draw on Sources to Improve Treatment of PD
What do Gaucher’s disease, gout, and amyloid plaques have in common? For researchers at the MGH, each of them may shed light on the causes and treatment of Parkinson’s disease.
07/22/2013: NeuroBlast e-Newsletter
NeuroBlast: the newsletter of translational neuroscience and clinical care advances in neurology, neurosurgery, and neuroscience from Massachusetts General Hospital.
06/18/2013: Project A.L.S. Internship 2013
The ALS Internship Program teaches up-and-coming future scientists about clinical care and ongoing research in Amyotrophic Lateral Sclerosis (ALS).
06/17/2013: Rare genomic mutations found in 10 families with early-onset, familial Alzheimer's disease
MGH researchers have discovered a type of mutation known as copy-number variants – deletions, duplications, or rearrangements of human genomic DNA – in affected members of 10 families with early-onset Alzheimer's. These are the first new early-onset familial Alzheimer’s disease gene mutations to be reported since 1995.
06/13/2013: Prize4Life and Knopp Neurosciences Announce Donation of Empower Phase 3 Data to PRO-ACT Database
Data from one of the largest clinical trials ever conducted in ALS to be part of PRO-ACT database.
MGH investigators have determined that one of the recently identified genes contributing to the risk of late-onset Alzheimer's disease regulates the clearance of the toxic amyloid beta (A-beta) protein that accumulates in the brains of patients with the disease.
04/12/2013: Mass. General Neurological Clinical Research Institute and Prize4Life receive Bio-IT World Award for creation of ALS data platform
The MGH Neurological Clinical Research Institute and Prize4Life, an organization dedicated to accelerating discovery of treatments and a cure for ALS, received a Best Practices Award at the 2013 Bio-IT World Conference & Expo for their creation of PRO-ACT ,the largest database of information from ALS clinical trials and patient care.
The initial clinical trial of a novel approach to treating amyotrophic lateral sclerosis – blocking production of a mutant protein that causes an inherited form of the progressive neurodegenerative disease – may be a first step towards a new era in the treatment of such disorders.
James D. Berry, MD, MPH has received the Richard Olney, MD, Clinician-Scientist Development Award for his investigation of cells in the immune system.
03/18/2013: ALS Clinical Trial Pipeline Webinar Spring 2013
Join us on Wednesday March 27th for an informational webinar for PALS and their families.
The Informatics Core at MIND offers state-of-the-art bioinformatics services, connecting researchers with tools, each other, and expertise to manage their clinical and research databases.
02/26/2013: US Brainstorm Trial Statement
Formal statement on the forthcoming US Brainstorm trial
02/11/2013: ALS Drug Development Gets FDA Hearing
PALS, caregivers, ALS advocates and researchers will gather on Monday February 25th to talk to the FDA about ALS, from the patient perspective.
01/03/2013: Biogen Announces It Will Discontinue Development of Dexpramipexole Based on Phase 3 Trial Results
Biogen Idec has announced that the EMPOWER trial has failed to demonstrate efficacy in primary and secondary endpoints.
Join us on Friday January 11th 2013 as Dr. Michael Weiss (University of Washington) will provide study rationale and background for Mexiletine in patients with sporadic ALS. He will also be available for Q&A.
12/17/2012: Genetic manipulation of urate alters neurodegeneration in mouse model of Parkinson's disease
A study by MGH researchers adds further support to the possibility that increasing levels of urate may protect against Parkinson's disease. The investigators report that mice with a genetic mutation increasing urate levels were protected against Parkinson's-like neurodegeneration, while the damage was worse in animals with abnormally low urate.
12/03/2012: Mass General Hospital ALS Internship Program for College and Medical School Students 2013
The Mass General Hospital ALS Internship Program offers both college and medical school students an exceptional opportunity to gain first-hand experience in the clinical research and care of ALS.
Treatment with a novel agent that inhibits the activity of SIRT2, an enzyme that regulates many important cellular functions, reduced neurological damage, slowed the loss of motor function and extended survival in two animal models of Huntington's disease.
The Department of Neurology at Massachusetts General Hospital is pleased to announce that the Neurology Clinical Trials Unit has changed its name to the Neurological Clinical Research Institute (NCRI).
BENEFIT-ALS Will Evaluate Longer Term Effects of Novel Skeletal Muscle Activator and Represents a Key Step Forward Towards Potential Registration
Understanding who is most susceptible to Alzheimer's disease and developing early detection models, effective therapies and possibly a cure, is the goal of the largest single private scientific grant ever invested in Alzheimer's Whole Genome Sequencing focused on families afflicted with the disease.
Cytokinetics plans to initiate this clinical trial, designed to evaluate the safety, tolerability and efficacy of tirasemtiv (formerly CK-2017357) in patients with ALS, in the fourth quarter of 2012.
This study will assess the ability of the NeuRx Diaphragm Pacing System (DPS) to improve respiratory function and quality of life in people with ALS.
08/08/2012: Statement on the Clinical Trial of Ceftriaxone
The DSMB for the NINDS-sponsored clinical trial of ceftriaxone in ALS recommended that based on existing data the trial be stopped because the study was unlikely to reach the pre-determined efficacy criteria.
Published results demonstrated that single oral doses of 250 mg and 500 mg of CK-2017357 appeared safe and well-tolerated in patients with ALS.
Results indicate that ISIS 333611 was well-tolerated.
Use of the antioxidant urate to protect against the neurodegeneration caused by Parkinson's disease appears to rely on more than urate's ability to protect against oxidative damage.
04/19/2012: Cytokinetics Announces Fast Track Designation
The FDA has granted Cytokinetic's drug candidate CK-2017357 a fast track designation for the potential treatment of ALS.
Published safety data shows that spinal cord stem cells can be delivered safely into the spines of patients with ALS.
03/26/2012: Stem Cell Study Aids Quest for ALS Therapies
An international research team has created motor neurons using skin cells from a person with an inherited form of ALS/MND.
02/14/2012: Phase II TDI 132 Trial Announced
The ALS Therapy Development Institute (ALS TDI) has announced they will launch a Phase II clinical trial investigating TDI 132 as a potential treatment for ALS.
Press release states no significant side effects noted in initial Brainstorm participants.
01/16/2012: Phytopharm Releases Positive Preliminary Results
Phytopharm lead drug candidate Cogane has demonstrated efficacy in genetic preclinical model of ALS.
12/21/2011: Veterans with ALS - New Disability Rating Rule
As of January 19th, 2012, veterans with service connected ALS will be eligible for a 100% disability rating.
12/18/2011: Increased expression of regulatory enzyme may protect against neurodegeneration in Huntington's disease
Treatment that increases brain levels of an important regulatory enzyme may slow the loss of brain cells that characterizes Huntington's disease and other neurodegenerative disorders.
12/14/2011: Trophos Phase III Trial Results Announced
Trophos, a French pharmaceutical company, announced results from its phase III trial of Olesoxime.
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).
11/24/2011: Rebuilding the Brain’s Circuitry
Neuron transplants have repaired brain circuitry and substantially normalized function in mice with a brain disorder, an advance indicating that key areas of the mammalian brain are more reparable than was widely believed.
11/21/2011: Knopp Announces Positive Phase II Results
Phase II Dexpramipexole, funded by Knopp Biosciences, shown to be safe and tolerable in patients with ALS.
The phase III trial of Ceftriaxone; Neuraltus, the Phase II study of NP001 in ALS; and EMPOWER, the Phase III study of Dexpramipexole in ALS, are no longer recruiting participants.
10/03/2011: Biomarker for Huntington's disease identified
In a new research paper BWH and MGH researchers identify a transcriptional biomarker that may assist in the monitoring of Huntington's disease activity and in the evaluation of new medications.
MGH investigators may have found the mechanism behind a previously reported link between the rare genetic condition Gaucher disease and the common neurodegenerative disorder Parkinson's disease.
Patients with amyotrophic lateral sclerosis (ALS) may be an exception to the rule that being overweight is a health hazard. In a retrospective study of over 400 ALS patients, MGH researchers found that those who were mildly obese survived longer than patients who were normal weight, underweight or even overweight.
ANNE YOUNG, MD, PHD, chief of Neurology, made history for women in academic medicine by making a $1 million gift to the Department of Neurology through a deferred charitable gift annuity.
05/17/2010: New study characterizes cognitive and anatomic differences in Alzheimer’s disease gene carriers
In the most comprehensive study to date, neurologists have clearly identified significant differences in the ways that Alzheimer’s disease (AD) affects patients with and without the apolipoprotein E ε4 gene, a known genetic risk factor for the neurodegenerative disease.
Amyloid-beta protein – the primary constituent of the plaques found in the brains of Alzheimer's disease patients – may be part of the body's first-line system to defend against infection. In their report in the March 3 issue of PLoS One, a team led by MGH researchers describe their evidence that amyloid-beta protein is an antimicrobial peptide.
Researchers at Massachusetts General Hospital are seeking recently diagnosed Parkinson's disease (PD) patients to participate in a clinical trial investigating whether inosine taken to raise the body’s level of urate — a naturally occurring antioxidant — can be used to slow the progress of PD.
Therapeutic drug trial is a definitive test of whether high-dose creatine can slow the progression of HD.
01/01/2010: Testing new drugs for ALS
Merit Cudkowicz, MD, director of the Mass General Neurological Clinical Research Institute, talks about clinical trials for Amyotrophic Lateral Sclerosis, also known as ALS, or Lou Gehrig's disease.
By examining data from a 20-year-old clinical trial, a research team based at the MassGeneral Institute for Neurodegenerative Diseases and Harvard School of Public Health, has found evidence supporting the findings of their 2008 study – that elevated levels of the antioxidant urate may slow the progression of Parkinson’s disease.
Alzheimer’s disease researcher Rudolph Tanzi, PhD, of Massachusetts General Hospital adds another distinction to his scientific career when he joins Aerosmith’s Joe Perry and other rock celebrities in a designer menswear photo shoot as a “Rock Star of Science” in the June issue of GQ Magazine.
A new study has identified a potential strategy for removing the abnormal protein that causes Huntington’s disease from brain cells, which could slow the progression of the devastating neurological disorder.
The impact of the amyloid plaques that appear in the brains of patients with Alzheimer’s disease may extend beyond the deposits’ effects on neurons– the cells that transmit electrochemical signals throughout the nervous system.
A collaborative research effort spanning nearly a decade between researchers at Massachusetts General Hospital and King’s College London has identified a novel gene for inherited amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease).
01/01/2009: Gender differences in Parkinson’s disease
Drs. Anne B. Young, Ippolita Cantuti-Castelvetri and Michael Schwarzschild study gender differences in Parkinson’s disease from three different investigative approaches.
Leveraging Alzheimer’s Genome Project™ data, geneticist Rudy Tanzi, PhD, completes research to discover all gene variants that increase a person’s risk of Alzheimer’s disease.
09/23/2013: The Lancet: Neurology
Dexpramipexole vs placebo for patients with ALS (EMPOWER): a randomised, double-blind, phase 3 trial
In a phase III trial, dexpramipexole was generally well tolerated but did not differ from placebo on any pre-specified efficacy endpoint measurement. The trial can inform the design of future clinical research strategies in amyotrophic lateral sclerosis.
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