Thanks to the implementation of proactive safety measures for the COVID-19 pandemic, Mitsubishi Tanabe Pharma America (MTPA) has enrolled at least 33 adults with amyotrophic lateral sclerosis (ALS) in its ongoing REFINE-ALS biomarker study. “ALS patient needs remain MTPA’s primary focus, so it was a priority to address the…

Most of the initial amyotrophic lateral sclerosis (ALS) patients enrolled in Cytokinetics’ COURAGE-ALS Phase 3 trial testing its experimental therapy reldesemtiv have moderate-to-fast progressing disease at study entry. An analysis shows that, as intended, COURAGE-ALS is increasing the proportion of patients with rapidly progressing disease compared with the…

An oral suspension formulation of edaravone (MT-1186) is safe and well-tolerated in adults with amyotrophic lateral sclerosis (ALS), according to interim, six-month data from a Phase 3 clinical trial. In addition, exploratory efficacy results suggest that edaravone’s experimental oral formulation may slow patients’ functional decline to a similar level…

While it’s long been believed that the death of motor neurons in the spinal cord is the main cause of amyotrophic lateral sclerosis (ALS), new research suggests that degeneration of motor neurons in the brain takes place independently and may also contribute to the disease. The study’s work in…

A Phase 2 trial of ZZ Biotech’s investigational therapy 3K3A-APC in people with amyotrophic lateral sclerosis (ALS) has started dosing patients at its single site in Australia, the company announced in a press release. The open-label trial (NCT05039268), underway at the Macquarie University in Sydney, aims to evaluate…

CNM-Au8, an investigational disease-modifying therapy for people with early amyotrophic lateral sclerosis (ALS), showed consistent clinical benefits in patients with both limb and bulbar onset disease, according to new analyses of RESCUE-ALS Phase 2 clinical trial data. In particular, the treatment significantly extended the time to disease progression —…

When Leah Stavenhagen was 27, she was living in Paris and traveling through Europe. But an amyotrophic lateral sclerosis (ALS) diagnosis forced her to give up her job as a consultant with Ernst & Young and dashed her dreams of continuing to work abroad. At the time, she seemed…

Repeat injections of mesenchymal stem cells (MSCs) into the spinal canal were safe and well tolerated in patients with amyotrophic lateral sclerosis (ALS), and showed potential to significantly slow the rate of disease progression, a small Phase 2 clinical trial found. “Larger studies are warranted to confirm our observations…

Brainstorm Cell Therapeutics‘ investigational cell-based therapy, NurOwn, might be able to slow disease progression and be of “meaningful” benefit to  people with less severe amyotrophic lateral sclerosis (ALS), according to new analyses of Phase 3 trial data. The company announced last year that its Phase 3 trial…

A combined gene therapy that delivered two nerve growth factors — NRG1-I and NRG1-III — to muscle and nerve cells improved motor function and delayed disease onset in a mouse model of amyotrophic lateral sclerosis (ALS), a study demonstrated. However, the combo therapy did not show a synergistic effect,…