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.

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

Mayo clinic researchers develop a potential test for Machado-Joseph disease

Mayo Clinic researchers, along with national and global collaborators, have developed a potential test for Machado-Joseph disease, or spinocerebellar ataxia type 3 (SCA3) ? a disease that has no cure.

22 Oct 2020

Experimental ALS drug shows potential to prolong patient survival

An experimental medication that was recently shown to slow the progression of the neurodegenerative disease amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, has now demonstrated the potential to also prolong patient survival.

16 Oct 2020

First pathoconnectome shows how eye disease alters retinal circuitry

Scientists from the John A. Moran Eye Center at the University of Utah have achieved another first in the field of connectomics, which studies the synaptic connections between neurons.

30 Sep 2020

ALS and FTLD result from reduced transportation of messenger RNA

The lack of protein TDP-43 in two neurodegenerative diseases is shown to prevent neuron growth via failed transport of RNA and subsequent absence of protein synthesis in axons

23 Sep 2020

Study implicates new gene as driver of motor neuron diseases

Failures in a quality control system that protects protein-building fidelity in cells can lead to motor neuron degeneration and related diseases, according to a new study from an international team co-directed by Scripps Research molecular biologist Claudio Joazeiro, PhD.

16 Sep 2020

Investigational drug may reduce protein buildup and neurological decline linked to ALS, FTD

An investigational drug that targets an instigator of the TDP-43 protein, a well-known hallmark of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), may reduce the protein's buildup and neurological decline associated with these disorders, suggests a pre-clinical study from researchers at Penn Medicine and Mayo Clinic.

8 Sep 2020

Experimental drug for ALS shows promising results in clinical trial

An experimental medication slows the progression of the neurodegenerative disease called Amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, according to recently released results from a clinical trial run by investigators at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and Amylyx Pharmaceuticals, Inc., the company that manufactures the medication.

2 Sep 2020

Salmonella biofilm protein can cause autoimmune responses, arthritis in animals

Scientists from the Vaccine and Infectious Disease Organization-International Vaccine Centre at the University of Saskatchewan and Temple University have demonstrated that a Salmonella biofilm protein can cause autoimmune responses and arthritis in animals.

10 Jul 2020

Link between ALS and Alzheimer's disease can help advance the search for new therapies

On the surface, amyotrophic lateral sclerosis (ALS) and Alzheimer's disease share two commonalities: Both are progressively debilitating neurodegenerative conditions--meaning symptoms get worse--and, at least for now, neither has an effective treatment, let alone a cure.

16 Jun 2020