Patricia Inácio, PhD, science writer —

The ALS Association and I AM ALS have filed a petition with the U.S. Food and Drug Administration (FDA), calling on the regulatory agency and Amylyx Pharmaceuticals to make AMX0035 available to people with amyotrophic lateral sclerosis (ALS) as quickly as possible. Signed by more than 50,000 patients, their caregivers and family, the…

Scribe Therapeutics and Biogen, a biotechnology company focused on neurological diseases, have established a research partnership to develop new gene therapies for amyotrophic lateral sclerosis (ALS) using the gene-editing technology CRISPR-Cas9. The collaboration will focus initially on the development and commercialization of CRISPR-based therapies that address some…

The first patient has been dosed in a Phase 2a clinical trial evaluating the safety and effectiveness of ALZT-OP1a, a dry-powder inhaled formulation of cromolyn, in people with mild-to-moderate amyotrophic lateral sclerosis (ALS). The trial (NCT04428775) is recruiting up to 80 patients, ages 18–75, who have been…

The ALS Association and I AM ALS have opened a petition calling for the earliest possible approval of Amylyx’s experimental oral treatment AMX0035, after a Phase 2/3 trial found the therapy to be safe and to slow functional decline in amyotrophic lateral sclerosis (ALS) patients with rapidly progressing disease. Addressing the…

The Phase 3 clinical trial testing an oral suspension formulation of edaravone for amyotrophic lateral sclerosis (ALS) has resumed dosing and patient recruitment, its developer, Mitsubishi Tanabe Pharma America (MTPA), announced. The trial (NCT04165824) was launched in November, but was paused in March due to the COVID-19 pandemic. The study,…

Researchers report having created a new human-on-a-chip model, one able to more accurately simulate the clinical features of amyotrophic lateral sclerosis (ALS) in people. The chip uses motor neurons — the nerve cells responsible for controlling voluntary muscles — derived from stem cells isolated from ALS patients. This approach…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Clinigen’s aldesleukin for the treatment of amyotrophic lateral sclerosis (ALS). Aldesleukin is a lab-made version of the interleukin 2 (IL-2), an immune signalling molecule known to play a key role in the maintenance of a…

The levels of certain indicators of iron status, namely ferritin and transferrin, are altered in patients with amyotrophic lateral sclerosis (ALS) relative to healthy controls, suggesting a possible link between iron metabolism and ALS, a study has found. The results, “Abnormal Serum Iron-Status Indicator Changes in…

The pivotal Phase 3 clinical trial testing BrainStorm Cell Therapeutic’s cell-based therapy NurOwn in amyotrophic lateral sclerosis (ALS) patients continues with only occasional scheduling changes to treatment due to the COVID-19 pandemic. Patient dosing is expected to be complete by July, the company announced. “BrainStorm made…

Tikomed has secured $5.5 million in financing from investors to support the ongoing clinical development of ILB, its lead therapy candidate for degenerative neurological diseases, including amyotrophic lateral sclerosis (ALS). The funding also will help develop Ibsolvmir, a therapy to increase the effectiveness of transplanting cells to treat patients…