Lou Gehrig's Disease News and Research

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Lou Gehrig's Disease or Amyotrophic Lateral Sclerosis (ALS) is a neurological disorder characterized by progressive degeneration of motor neuron cells in the spinal cord and brain, which ultimately results in paralysis and death. The disease takes its less-scientific name from Lou Gehrig, a baseball player with the New York Yankees in the late 1920s and 1930s, who was forced to retire in 1939 as a result of the loss of motor control caused by the disease.

In 1991, a team of researchers linked familial ALS to chromosome 21. Two years later, the SOD1 gene was identified as being associated with many cases of familial ALS. The enzyme coded for by SOD1 carries out a very important function in cells: it removes dangerous superoxide radicals by converting them into non-harmful substances. Defects in the action of this enzyme mean that the superoxide radicals attack cells from the inside, causing their death. Several different mutations in this enzyme all result in ALS, making the exact molecular cause of the disease difficult to ascertain.

Recent research has suggested that treatment with drugs called antioxidants may benefit ALS patients. However, since the molecular genetics of the disease are still unclear, a significant amount of research is still required to design other promising treatments for ALS.

Eikonoklastes completes a license to add novel gene therapy for treating neurodegenerative diseases

Eikonoklastes Therapeutics, a preclinical stage biopharmaceutical company, today announced it has completed a license with the University of California San Diego to add a novel gene therapy for the treatment of neurodegenerative diseases.

20 Jan 2022

Model of neuromuscular junction could accelerate new treatments for neuromuscular diseases

The neuromuscular junction-;where nerves and muscle fibers meet-;is an essential synapse for muscle contraction and movement.

6 Jan 2022

Study offers new insight into the role of tau proteins in ALS pathogenesis

A team led by investigators at Massachusetts General Hospital has shown that people living with amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, who carry a mutation in the C9orf72 gene exhibit elevated levels of tau and phosphorylated tau protein in the motor cortex region of the brain.

15 Nov 2021

Inflammatory chemicals involved in fat metabolism play key role in ALS, study shows

A new study using genetically engineered mice and human cell and tissue samples has added to evidence that higher levels of inflammatory chemicals involved in fat metabolism occur in people with amyotrophic lateral sclerosis (ALS), the neuromuscular disorder, also known as Lou Gehrig's disease.

15 Nov 2021

Atomic-level imaging of infectious prions reveals new therapeutic targets for untreatable diseases

The highest-ever resolution imaging of an infectious prion provides the first atomic-level data of how these abnormal proteins are assembled to cause fatal neurodegenerative diseases in people and animals-; and how they can be potentially targeted by new therapies.

24 Aug 2021

Scientists identify the main components driving amyloid beta-associated synapse degeneration

Healthy adult brains are endowed with a vast number of synapses, structures that relay signals across nerve cells to enable communications, information processing and storage throughout the nervous system.

18 Aug 2021

Dying patients with rare diseases struggle to get experimental therapies

At 15, Autumn Fuernisen is dying. She was diagnosed at age 11 with a rare degenerative brain disorder that has no known cure or way to slow it down: juvenile-onset Huntington's disease.

14 Jul 2021

Cedars-Sinai receives $11.99 million from CIRM to launch ALS clinical trial

Cedars-Sinai has been awarded $11.99 million by California's stem cell agency to launch a clinical trial testing a potential gene and stem cell therapy for amyotrophic lateral sclerosis (ALS).

23 Jun 2021

Research identifies distinctive inflammatory signature in a genetic form of ALS

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a neurodegenerative disease that strikes nearly 5,000 people in the U.S. every year. About 10% of ALS cases are inherited or familial, often caused by an error in the C9orf72 gene.

16 Jun 2021

Researchers use gene therapy to prevent learning, memory loss in Alzheimer's mouse model

Researchers at University of California San Diego School of Medicine, with colleagues elsewhere, have used gene therapy to prevent learning and memory loss in a mouse model of Alzheimer's disease (AD), a key step toward eventually testing the approach in humans with the neurodegenerative disease.

5 May 2021

Targeting brain protein may lead to new therapeutic approaches for neurodegenerative disorders

Neurological disorders are the number one cause of disability in the world, leading to seven million deaths each year. Yet few treatments exist for these diseases, which progressively diminish a person's ability to move and think.

16 Apr 2021

Researchers identify nonelectrical cells that transition the brain from plasticity to stability

Researchers exploring the developing central nervous system of fruit flies have identified nonelectrical cells that transition the brain from highly plastic into a less moldable, mature state.

8 Apr 2021

Researchers unveil a neuroprotective pathway that suppresses ALS

In general, the RAN proteins are more abundant in the cell nucleus, but in patients with Lou Gehrig's disease, they begin to leak out into the cytoplasm, thereby resulting in abnormal concentration differences.

19 Mar 2021

After a decade of lobbying, ALS patients gain faster access to disability payments

Anita Baron first noticed something was wrong in August 2018, when she began to drool. Her dentist chalked it up to a problem with her jaw.

21 Jan 2021

Antiepileptic drug reduces early signs of clinical dysfunction in people with ALS

The antiepileptic drug ezogabine reduced pathologic excitability of cortical and spinal motor neuron cells that are early signs of clinical dysfunction in people with amyotrophic lateral sclerosis (ALS), according to a study conducted by the Neurological Clinical Research Institute of Massachusetts General Hospital.

10 Dec 2020

New drug lures stem cells to damaged tissue, improves treatment efficacy

Scientists at Sanford Burnham Prebys Medical Discovery Institute have created a drug that can lure stem cells to damaged tissue and improve treatment efficacy--a scientific first and major advance for the field of regenerative medicine.

25 Nov 2020

Research on genetic heart disease uncovers new mechanism for heart failure

Research on genetic heart disease has uncovered a new and unexpected mechanism for heart failure. This landmark discovery found a correlation between the clumping of RNA-binding proteins long linked to neurodegenerative disease and the aggregates of protein found in the heart tissue of patients with RBM20 dilated cardiomyopathy.

19 Nov 2020