Patricia Inácio, PhD,  science writer—

A traffic jam inside nerve cells is a feature of most forms of amyotrophic lateral sclerosis and frontotemporal dementia, a study shows. It suggests that easing the jam is a potential strategy for treating ALS. Therapies to do this are moving into clinical trials, the researchers said. The study, “…

The U.S. Food and Drug Administration (FDA) has issued a warning to the American CryoStem Corporation of Monmouth Junction, New Jersey, for marketing Atcell, the company’s adipose-derived stem cell product, without FDA approval. In its warning letter, the FDA noted “significant deviations from current good manufacturing practice requirements, including some…

Researchers at the University of Calgary in Canada are enrolling amyotrophic lateral sclerosis (ALS) patients for a Phase 2 clinical trial to evaluate the potential therapeutics effects of a well-established antipsychotic drug called Orap (pimozide). The research team, led by Dr. Lawrence Korngut, associate professor at the…

BrainStorm Cell Therapeutics released new data on potential biomarkers from its Phase 2 trial investigating NurOwn, the company’s cell therapy for amyotrophic lateral sclerosis (ALS), at the International Symposium on ALS/MND meeting held in Boston last week. Both these findings and an ongoing Phase 3 trial, now recruiting patients in…

The rate of ALS patients’ lung-function decline can predict their risk of breathing failure, their need for assisted ventilation, and their risk of death, according to Cytokinetics, a company that develops muscle-disease therapies. “These results suggest that respiratory function is an important prognostic indicator of clinical progression in people with…

The schizophrenia therapy Orap (pimozide) may be able to prevent the progression of ALS, Canadian researchers report. The team confirmed in a clinical trial of 25 ALS patients the results it saw in studies with worms, fish and mice. An antipsychotic drug, Orap is used to treat other psychiatric conditions…

Boston-based Flex Pharma has announced positive topline results from its exploratory Phase 2 trial testing investigational therapy FLX-787 in Australian amyotrophic lateral sclerosis (ALS) patients with frequent muscle cramps. The data showed that FLX-787 significantly reduced the intensity of cramp-associated pain and stiffness compared to placebo. The positive results support the…

Findings from a Phase 3 study of tirasemtiv to improve muscle strength — particularly of respiratory muscles — in amyotrophic lateral sclerosis (ALS) patients will likely be announced for a first time on Dec. 8 at the 28th ALS/MND meeting in Boston, a vice president of Cytokinetics, its developer, said…

Researchers at Germany’s University of Würzburg have discovered how a malfunctioning autophagy – a cell’s natural self-degradation and cleanup system – plays a central role in the development of amyotrophic lateral sclerosis (ALS) and other motor neuron diseases. The study, “Plekhg5-regulated autophagy of synaptic vesicles reveals a pathogenic mechanism…

Voyager Therapeutics has recently presented data of its leading adeno-associated virus (AAV) gene therapy for several neurological diseases, including amyotrophic lateral sclerosis (ALS), at the Congress of the European Society of Gene and Cell Therapy (ESGCT), Oct. 17-20 in Berlin, Germany. In their poster, “Translation of Intrathecal Delivery of an AAV Gene…