Author Archives: Ana Pena, PhD

Human cells produce a type of proteins, termed “Heroes,” that protect against the formation of toxic protein aggregates involved in several neurological conditions, including amyotrophic lateral sclerosis (ALS), and are extremely resistant to harsh conditions, early study results show. Organisms naturally use Hero proteins as molecular shields to stabilize…

A new spinal cell delivery method may reduce the risks and boost the effectiveness of stem cell therapy designed to regenerate the nervous system in neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), an early study in rats has found. The study, “Spinal parenchymal occupation by…

A single spinal injection of an investigational RNA therapy blocked motor neuron degeneration and saved motor function in animal models of familial amyotrophic lateral sclerosis (ALS) linked to SOD1 mutations, a study  reports. Giving the treatment — a virus-delivered gene-silencer targeting the SOD1 gene —  before the onset of…

Treatment with oral reldesemtiv appears to slow the deterioration of function — including breathing capacity and muscle strength — in amyotrophic lateral sclerosis (ALS) patients, regardless of whether or not they were taking additional medications such as Radicava (edaravone) or Rilutek (riluzole), a new…

A new study identified a group of sporadic amyotrophic lateral sclerosis (ALS) patients whose disease may arise from a group of “jumping genes” unleashed when TDP-43 protein accumulates in clumps in the brain and spinal cord. These “jumping genes” — which can randomly hop from one location on…

People with amyotrophic lateral sclerosis with a common mutation in the interleukin 6 receptor (IL6R) gene appear likely to have more severe disease symptoms and faster progression than ALS patients without this inherited gene variant, a study reports. This mutation, its researchers reported, is found with a frequency of…

Mexiletine, an oral medicine to treat irregular heartbeats, is safe and effective for reducing muscle cramps in patients with amyotrophic lateral sclerosis (ALS), results from a Phase 4 clinical trial show. To date, this is the only treatment shown to reduce both the number and intensity of cramps…

Experimental treatment reldesemtiv appears to reduce functional deterioration in patients with fast progressing amyotrophic lateral sclerosis (ALS), a new analysis of the FORTITUDE-ALS trial shows. The additional analyses were presented by the trial’s principal investigator, Jeremy M. Shefner, MD, PhD, a professor at the Barrow Neurological Institute…

Excessive energy demands to overcome gravity in nerve cells directed downward — such as those involved in the control of limb movements — may explain why amyotrophic lateral sclerosis (ALS) affects certain motor neurons while leaving others untouched, a study has proposed. As people age, nerve fibers disposed vertically…

Stem cell-derived motor neurons from people with familial amyotrophic lateral sclerosis (ALS) have mutation-specific alterations in glutamate receptors and calcium signals, which may alter nerve signaling and play a role in the disease, a study found. These findings may lead to future…