The HEALEY ALS Platform Trial has evaluated four treatment regimens for Amyotrophic Lateral Sclerosis (ALS), a fatal neurodegenerative disease, using a common research setup. The trial showed promising trends in other outcome measures and biomarkers, informing the design of future phase 3 trials.
A recent study from IR Sant Pau investigates the peripheral immune system's role in amyotrophic lateral sclerosis (ALS) at the single-cell level. The research found altered NK cell subpopulations associated with ALS progression, suggesting a potential target for new therapies.
A Michigan Medicine-led study suggests that living in disadvantaged communities may decrease the length of time a person can survive with amyotrophic lateral sclerosis (ALS) by up to 37%. The study found that patients from more resourced areas tended to live longer, possibly due to better access to care and reduced caregiver burnout. T...
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The project's recordings help improve voice recognition tools by providing diverse speech patterns to train artificial intelligence models. Microsoft has seen significant improvements in recognizing non-standard English speech, with accuracy gains ranging from 18% to 60%, depending on the speaker's disability.
Organoids derived from patient iPSCs accurately replicate ALS phenotypes, supporting drug development. However, challenges remain in utilizing these models for comprehensive ALS research and other neurodegenerative diseases.
A recent study suggests that tiny antenna-like structures on cells called primary cilia could be a potential therapeutic target for Amyotrophic Lateral Sclerosis (ALS). Researchers have identified mutations in the C21orf2 gene, which impair primary cilia formation and structure, leading to motor neuron death.
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A new method has been developed to create motor neurons from stem cells taken directly from ALS patients, allowing for fast and individualized drug testing. The cultured ALS motor neurons had increased susceptibility to cell death, underscoring the utility of this system to identify potential drugs.
The partnership integrates Answer ALS’s Neuromine Data Portal with the AD Data Initiative's AD Workbench, providing a unified platform for data sharing and analysis. This collaboration enables integrative analysis of ALS and Alzheimer’s data, streamlining efforts and enhancing research impact.
Recent studies suggest a connection between the protein G3BP1 and ALS disease, with toxic dipeptide repeats impairing normal cellular functions. The findings provide valuable insights into the underlying mechanisms of neurodegenerative disorders.
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The largest ALS patient-based iPSC and bio data repository was released by Answer ALS, offering an unprecedented resource for researchers studying ALS. The repository encompasses biological and clinical data from nearly 1,000 ALS patients and provides a comprehensive model for disease research and drug discovery.
A recent study has identified 51 amyotrophic lateral sclerosis (ALS)-associated mutations in mitochondrial DNA that could help diagnose the disease. The mutations, which include 13 that increase the risk of ALS and 38 protective ones, were found to be significantly associated with an increased or decreased risk of developing the disease.
Researchers have unveiled a complex interplay between liver X and thyroid hormone receptors that regulates critical brain functions and protects against neurodegeneration. LXRs play a crucial role in regulating thyroid hormone function in the brain, while both receptors are essential for normal brain development and maintenance.
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Researchers have identified specific human endogenous retrovirus sequences associated with increased risk of neurodegenerative diseases, including multiple sclerosis and amyotrophic lateral sclerosis. The study suggests that these viral elements contribute to disease susceptibility by influencing brain function.
A new study found that toxic SOD1 protein trimers interact with various proteins in different tissues, contributing to cellular dysfunction and degeneration in ALS. Septin-7 is identified as a potential therapeutic target, potentially slowing or disrupting ALS progression.
Scientists have developed new therapies that selectively remove aggregated tau proteins associated with Alzheimer's disease in mice. The approach utilises TRIM21 to target tau aggregates, leaving healthy tau intact, and demonstrates potential for other brain disorders driven by protein aggregation.
A highly accurate diagnostic blood test has been developed for amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease that affects neurons in the brain and spinal cord. The test requires only a simple blood draw and can distinguish between ALS patients and healthy controls with an overall accuracy of up to 98%.
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New research reveals that downregulating the enzyme EGLN2 can protect motor neurons and mitigate ALS symptoms in animal models. This discovery brings hope to understanding how to slow or prevent this devastating disease.
A clinical trial led by UC San Francisco aims to develop new therapies for progressive supranuclear palsy, with a focus on reducing time to find effective treatments and increasing diverse participant enrollment. The five-year grant could lead to the first effective drugs for this incurable neurodegenerative disorder.
Researchers have identified two proteins, PARP1 and histone H1.2, that interact with an ALS-causing mutant FUS protein, leading to pathological changes. Inhibiting these proteins may be a possible therapeutic target for familial ALS cases caused by mutations in the FUS gene.
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Researchers discovered that Huntington's disease protein aggregates cause breaks in the nuclear envelope, leading to DNA damage and misregulation of neuronal genes. The study suggests a common mechanism for neurodegenerative diseases involving nuclear aggregate-induced ruptures.
Researchers developed a new brain-computer interface that translates brain signals into speech with up to 97% accuracy, enabling a man with amyotrophic lateral sclerosis (ALS) to communicate with friends and family. The system was tested in real-time conversations with continuous updates, achieving high word accuracy rates.
Researchers have identified a metal deficiency in SOD1 protein associated with motor neurone disease using native ambient mass spectrometry imaging. This breakthrough could lead to new insights and treatments for the disease, which affects around 5,000 people in the UK.
A University of Michigan-led study found that higher levels of metals in blood and urine are associated with a greater risk for ALS and shorter survival. Occupational exposure to metals was also linked to increased metal levels, emphasizing the need for accounting for environmental factors when evaluating overall exposure risk.
A new immune cell therapy has been shown to delay ALS onset and extend survival in mice, as well as reduce inflammation in an individual with the disease. In a phase I clinical trial, the therapy is now set to be tested on humans.
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A new study published in Neurology suggests that moderate levels of physical activity and fitness may be linked to a reduced risk of amyotrophic lateral sclerosis (ALS) later in life. Male participants who engaged in higher levels of physical activity had a lower risk of developing ALS compared to those with lower activity levels.
A study by TUM researchers discovered four subtypes of Amyotrophic Lateral Sclerosis (ALS) with different molecular processes, including sex differences. The findings suggest repurposing an approved cancer drug targeting the MAPK pathway as a promising therapeutic approach for ALS.
Researchers have developed a blood test that can detect rare forms of dementia, such as frontotemporal dementia (FTD) and progressive supranuclear palsy (PSP), as well as amyotrophic lateral sclerosis (ALS). The test uses biomarkers, including tau and TDP-43 proteins, to identify underlying pathology.
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A new study reveals how the FUS protein behaves in ALS and FTD, mirroring what is observed in human diseases where protein aggregates spread and contribute to neurodegeneration. The research supports the broader hypothesis that many neurodegenerative diseases involve prion-like mechanisms.
Dr. Jeetain Mittal's NIH grant will support multiscale computational models investigating phase separation in biology, particularly heterochromatin formation and its role in neurodegenerative diseases. The research aims to elucidate the molecular origins of phase separation using innovative models and methods.
A new study published in PLOS One found that ALS patients treated by neurologists received interventions endorsed by the American Academy of Neurology, including non-invasive ventilation and feeding tubes. Disparities were found in treatment among Black and disadvantaged patients, highlighting the need for equitable care.
Researchers at Ben-Gurion University of the Negev and international colleagues found that augmenting MIF protein levels delays motor function decline, modulates critical pathways, and extends lifespan in a SOD1 mouse model of ALS. Diminished MIF levels are also observed in familial and sporadic ALS cases.
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FutureNeuro, a leading SFI Research Centre, is expanding its research programme with a focus on diagnostics, therapeutics, and digital health. The centre aims to develop precision diagnostics, future treatments, and systems using real-time health data.
A team of Western University researchers has discovered a potential path toward a cure for amyotrophic lateral sclerosis (ALS) by targeting an interaction between two proteins. The breakthrough, fueled by the Temerty Foundation's $10-million investment, could lead to treatment in human clinical trials within five years.
Researchers found a significant association between storing chemicals in attached garages and increased ALS risk. The study suggests that volatile chemicals released into the living space may contribute to this link.
A traumatic brain injury quadruples the risk of developing dementia and neurodegenerative diseases like ALS. USC scientists used lab-grown human brain structures called organoids to study TBI's effects. They identified a gene, KCNJ2, that helps protect nerve cells against injury.
Columbia University has received a $15 million grant to develop tailor-made gene-based therapies for nine people with rare forms of amyotrophic lateral sclerosis (ALS). The Silence ALS program will use antisense oligonucleotide (ASO) technology to silence genes that produce toxic proteins in motor neurons. The project aims to create pe...
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A recent study published in Cell reveals significant similarities between amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD), two distinct neurological disorders. The research found shared gene expression patterns across various brain regions, suggesting potential therapeutic targets for both conditions.
Researchers identify 'junk proteins' as possible cause of ALS, a degenerative disease characterized by motor neuron death. The accumulation of these proteins is linked to the aging process, suggesting a new hypothesis for understanding ALS.
A study by Michigan Medicine found that recreational activities such as golfing, gardening, and woodworking may increase the risk of developing amyotrophic lateral sclerosis (ALS) in men. The study surveyed over 700 individuals with and without ALS to assess their hobbies and non-work related activities.
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Scientists developed an innovative neural cell culture model that replicates the aberrant behavior of TDP-43 in neurons. The model identified NPTX2 as a toxic protein that accumulates in cells containing abnormal TDP-43, leading to neurodegeneration in ALS and FTD.
Scientists have mapped out the proteins involved in motor neurone disease (MND) across its trajectory, identifying potential therapeutic pathways for further investigation. The study found that a protein-folding factor called DNAJB5 is elevated early on in MND, sparking curiosity about its role in disease progression.
ATH-1105, a small molecule positive modulator of the neurotrophic HGF system, demonstrates significant neuroprotective effects and extends survival in preclinical models of ALS. The study highlights the therapeutic potential of ATH-1105 in slowing or stopping neurodegeneration.
A novel drug principle has been successfully tested in a mouse model and brain organoids of ALS patients, preventing cell death and improving motor abilities. The discovery of the TwinF interface inhibitor FP802 offers a promising path for fighting ALS and could lead to the development of effective treatments.
A phase II clinical trial found Muse cell-based product CL2020 to be highly tolerated and improved ALS symptoms, but may not halt disease progression. The treatment's efficacy depends on combining it with other drugs for future treatments.
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A novel cyclic thiosulfinate cross-linker has favorable drug-like properties and can stabilize the SOD1 dimer in vivo, indicating potential therapeutic benefits for fALS. This study uses a mouse model to evaluate protein cross-linking as a strategy to treat SOD1 variants associated with familial ALS.
A new study in the Canadian Medical Association Journal found that organ donations after medical assistance in dying (MAiD) represented 14% of all deceased organ donations in Quebec in 2022. The majority of donors had neurodegenerative diseases, with amyotrophic lateral sclerosis being the most common condition.
Researchers at Nagoya University have discovered a relationship between ALS progression and the disruption of mitochondria-associated membranes (MAM) and TBK1 activity. Decreased activation of TBK1 is linked to motor neuron death in ALS patients and mice with disrupted MAM.
Research suggests that structural changes in upper motor neurons send a signal to immune cells, leading to toxic effects on neurons and reduced synaptic connections. Blocking inflammation with a semi-synthetic drug can restore synaptic connections and improve ALS symptoms.
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A team of scientists identified VAP as a molecular anchor that stabilizes mitochondria near synapses in dendrites, supporting memory formation and plasticity. The discovery links VAP to ALS-linked protein and suggests that mitochondrial stabilization is critical for neuronal function and health.
A study found increased brain inflammation and structural changes in special operations personnel exposed to blasts, potentially increasing the risk of long-term brain-related disease. The research suggests that repetitive exposure to low-level blasts may cause brain injuries and highlights the need for better protective measures.
Researchers found that misfolded prion proteins can trigger the clumping of TDP-43 in nerve cells, leading to reduced splicing activity and altered protein expression. This study reveals a new mechanism of how disease-associated prion proteins affect physiological signaling pathways through cross-seeding.
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A molecular trap prevents toxic Poly-GR dipeptide compounds from causing harm to neurons, reducing death in ALS patients and animal models. The study demonstrates promising results for protecting neurons from abnormal proteins generated in other neurodegenerative diseases.
Researchers have identified BMAA, a chronic neurotoxin linked to ALS and Alzheimer's, in dust particles from the Great Salt Lake. This dust poses an environmental health risk due to its inhalation potential.
Dr. Vidhya Rangaraju has received a $1.2 million grant from the Chan Zuckerberg Initiative to investigate disrupted energy supply in neurons causing cognitive decline in ALS. Her lab will use super-resolution microscopy and biosensors to study metabolic disruptions in ALS.
Researchers developed a drug that triggers the destruction of damaged mitochondria, restored motor function in aging mice with ALS-like symptoms. The results show promising promise for treating neurodegenerative diseases like Parkinson's and Alzheimer's disease.
Researchers developed an environmental risk score to assess ALS risk and survival using a blood sample, associating several individual pollutants with disease risk. A mixture of pesticides in the blood significantly increased ALS risk, with those in the highest group having twice the risk as those in the lowest.
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A new national ALS research consortium, Access for All in ALS, has been launched with a $16.7 million NIH award. The consortium will include 34 clinical sites nationwide and generate a longitudinal biorepository linked to detailed clinical information.
Dr. Qihui Zhou aims to investigate the role of immune cells in ALS and FTD with a focus on the C9orf72 gene mutation, potentially leading to better treatments.
A promising drug candidate CDNF has been found to prolong the lifespan of ALS patients and alleviate symptoms in rats and mice. The study suggests that CDNF may rescue motoneurons by reducing ER stress response and cell death.
A recent study found that over 70% of ALS patients and their caregivers are not disaster-prepared, highlighting the need for improved measures. The research also revealed significant gaps in equipment and knowledge among respondents, including ventilator users.
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