First Drug Candidate from ALS Therapy Development Institute Starts Clinical Trial

First Drug Candidate from ALS Therapy Development Institute Starts Clinical Trial

The ALS Therapy Development Institute (ALS TDI) is proud to announce that the first participant was successfully given a dose of AT-1501 in the first clinical trial of the experimental treatment for amyotrophic lateral sclerosis (ALS). The phase 1 safety trial is enrolling healthy volunteers and people living with ALS, following the acceptance of an Investigational New Drug (IND) application by the Food & Drug Administration (FDA). A total of 8 people living with ALS will receive AT-1501 in this study, with the aim of determining safety and tolerability of the experimental antibody. The trial, sponsored by Anelixis Therapeutics, LLC, will also determine the pharmacokinetics of AT-1501, i.e. how the drug acts within the body.

Westport, Connecticut family spearheads ALS Pepper Challenge

Westport, Connecticut family spearheads ALS Pepper Challenge

On Oct. 18, Patty Haberstroh got news no person hopes to receive in her lifetime. She was diagnosed with amyotrophic lateral sclerosis, colloquially known as Lou Gehrig’s disease, a neuromuscular degenerative illness that causes muscle atrophy and the eventual loss of all voluntary muscle control.

When Haberstroh, a Westport resident since 1990, told her husband, Charlie, and their four children of the diagnosis, she said they were understandably upset, but vowed do something and felt they could make a difference in fighting the disease.

Researchers show how Lou Gehrig’s disease progression could be delayed

Researchers show how Lou Gehrig’s disease progression could be delayed

A team of biomedical scientists has identified a molecule that targets a gene known to play a critical role in the rapid progression of amyotrophic lateral sclerosis (ALS), sometimes known as Lou Gehrig’s disease, the neurodegenerative disease that affects motor neurons — nerve cells in the brain and spinal cord that link the nervous system to the voluntary muscles of the body.

Eye muscles resist ALS progression, but more research needed to understand why

Patients with amyotrophic lateral sclerosis (ALS) have intact eye muscle movement, even at more advanced stages of the disease, according to new research. However, the reason remains unknown. Researchers say that discovering how this happens may help in the design of novel treatments to fight the loss of muscle activity in ALS patients.

Protein seen to have ‘housekeeping’ talents that protect neurons in diseases like ALS

A protein called Nrf2 could clear the harmful, misfolded proteins that cause Parkinson’s and other neurodegenerative diseases — including amyotrophic lateral sclerosis (ALS) — by activating “housekeeping” mechanisms within cells, according to a new study in Parkinson’s models.

Neuron Death in ALS May Be Triggered by Increased Toxic Excitatory Nerve Signaling

Increased excitatory toxic signaling in neurons in the part of the brain controlling movement triggers the breakdown of cells long before any symptoms of amyotrophic lateral sclerosis (ALS) are noticed — at least in mice with ALS.

Scientists find stem cells in brain’s meninges, possibly leading to future ALS therapies

Researchers have found neuronal progenitor cells (immature cells that can become neurons) in the meninges, a three-layer structure enclosing the brain that protects the nervous system, according to a new study. This finding may lead to the development of new stem cell therapies for brain damage and neurodegenerative disorders such as amyotrophic lateral sclerosis (ALS)

Repeated head injury causes neurodegeneration, but link to ALS less clear

The study, “Microglial neuroinflammation contributes to tau accumulation in chronic traumatic encephalopathy,” was published in the journal Acta NeuropathologicaCommunications.

Does Smoking Lead to Worse Outcomes in People with ALS?

People who are smokers at the time of their ALS diagnosis are unlikely to survive as long as people who have already quit smoking or never started, according to a report in the September 21 Journal of Neurology, Neurosurgery, and Psychiatry. The correlation between between smoking and disease prognosis, which is largely independent of respiratory status at diagnosis, suggests that smoking plays a role in ALS beyond increasing the risk of developing the disease.

Antibodies Circulating in Blood May Serve as Biomarker for ALS, Disease Severity

The presence of a certain type of proteins of the immune system in the blood may serve as a reliable biomarker for the diagnosis of amyotrophic lateral sclerosis (ALS), as well as the severity of the disease, according to a new study conducted by Japanese and American researchers

Early Signs of Bulbar Disease in ALS May Be Evident in Tongue’s Movement While Talking

Researchers report that evaluating a person’s control of tongue movement during speech can help to diagnose bulbar disease, especially in its early stages, in patients with amyotrophic lateral sclerosis (ALS).


New strategy targets range of toxic effects of ALS gene

New strategy targets range of toxic effects of ALS gene

Work from yeast, flies, worms, and cultured neurons from patients reveal a novel way to target the most common genetic cause of amyotrophic lateral sclerosis (ALS). Instead of directly targeting the genetic mutation in the C9orf72 gene, an international team of researchers, including Packard scientists Aaron Gitler of Stanford University, Fen-Biao Gao of the University of Massachusetts Medical School, and Leonard Petrucelli of the Mayo Clinic, developed a method to interrupt the process that turns the gene into protein. The work, published in Science, introduces a new strategy to potentially slow the progress of ALS.

ALS patients may have problems recognizing emotions in others, study finds

Patients with amyotrophic lateral sclerosis (ALS) may have problems recognizing emotions in other people, even though they do not have other cognitive or behavioral problems. This inability is linked to microscopic changes in brain anatomy in regions linked to emotional processing, and resembles changes seen in patients with some types of frontotemporal dementia

Brain imaging may detect early biomarkers for ALS, review suggests

Magnetic resonance imaging (MRI) techniques may detect biomarkers for early diagnosis in patients with amyotrophic lateral sclerosis (ALS), according to a review study from the University of Lille in France, titled “The value of magnetic resonance imaging as a biomarker for amyotrophic lateral sclerosis: a systematic review” and published in BMC Neurology.