FDA Staff Voice Serious Concerns About Investigational ALS Cell Therapy
FDA reviewers voiced serious concerns about the investigational amyotrophic lateral sclerosis (ALS) treatment debamestrocel — mesenchymal stromal cells secreting neurotrophic factors, also known as MSC-NTF, or NurOwn — from BrainStorm Cell Therapeutics ahead of an advisory committee meeting this week. When the FDA first received the drug’s application for approval, it determined the submission was […]
Omega-3 fatty acids linked to slower decline in ALS
People with amyotrophic lateral sclerosis (ALS) who eat more foods high in certain omega-3 fatty acids like flaxseed oil, walnuts, canola oil and pumpkin seeds may have a slower physical decline from the disease and may have a slightly extended survival. The study, which looked at the survival of people with ALS over the course […]
Drug for Rare Form of ALS Earns FDA Nod
The FDA granted accelerated approval to tofersen (Qalsody) for amyotrophic lateral sclerosis (ALS) patients with mutations in the SOD1 gene, the agency announced on Tuesday. The drug was cleared under the accelerated approval pathway, a program that uses a surrogate endpoint to approve drugs for serious conditions with an unmet medical need. For tofersen, the […]
Investigational ALS Drug May Have Clinical Benefit, FDA Staff Says
Tofersen, an investigational antisense drug developed to treat amyotrophic lateral sclerosis (ALS) associated with a mutation in the SOD1 gene (SOD1-ALS), may have clinical benefit, FDA reviewers indicated in briefing documents ahead of an advisory committee meeting. On Wednesday, the Peripheral and Central Nervous System Drugs Advisory Committee will vote on whether evidence supporting a […]
Investigational ALS Drug Betters Biomarkers, but Can It Improve Motor Functions?
Tofersen, an investigational antisense oligonucleotide developed to treat an inherited form of amyotrophic lateral sclerosis (ALS), changed biomarkers and curbed neurodegeneration but did not improve motor control and muscle strength at 6 months, the phase III VALOR trial showed. In a subgroup of patients with fast-progressing SOD1 ALS, the 28-week change in scores on the […]
Muscle biopsy test for biomarker could lead to earlier diagnosis of ALS
Amyotrophic lateral sclerosis (ALS) is a progressive disease of the nervous system. It affects nerve cells in the brain and spinal cord called motor neurons. Motor neurons control muscle movement and ALS causes them to deteriorate and eventually die. The motor neurons lose the ability to send messages to the muscles in the body, affecting […]
Lawmakers Press FDA on Getting ALS Therapies to Patients
House lawmakers urged the FDA to be more flexible in reviewing potential therapies for amyotrophic lateral sclerosis (ALS), and other neurodegenerative diseases, during a hearing of the House Energy & Commerce Health Subcommittee on Thursday. Members also stressed the need to increase diversity in and improve access to clinical trials. Each year, 50 million people […]