Latest ALS Clinical Trial Information

Autologous bone marrow mesenchymal stem cell therapy

Action: Cell therapy in which mesenchymal stem cells harvested from bone marrow fluid. Stem cells are expected to protect neurons and suppress neuro-inflammation.

Clinical trial status: Double-blind, randomized, controlled trial of intravenous administration of autologous bone marrow mesenchymal stem cells in patients with amyotrophic lateral sclerosis (ALS).

jRCT2013190010

Key Selection Criteria: ALS severity classification 1–3 within 3 years of onset.

Administration: Autologous mesenchymal stem cells harvested from bone marrow fluid, cultured and expanded in vitro, and administered intravenously to patients.

Participants wanted: Available in Japan (Sapporo Medical University Hospital)

Scheduled completion: July 2024

NurOwn® (Autologous bone marrow-derived neurotrophic factor-secreting mesenchymal stromal cells)

Action: A cell therapy in which autologous bone marrow mesenchymal stem cells harvested from bone marrow are cultured and grown ex vivo and administered intrathecally to patients.

Current Status of Clinical Trials: A phase III trial failed to demonstrate efficacy on the primary endpoint in all patient groups. However, subgroup analysis showed statistically significant efficacy in the patient group with an ALSFRS-R of at 27-35 points at baseline.

Details

Action: A cell therapy in which autologous bone marrow mesenchymal stem cells harvested from the patient’s bone marrow are cultured and expanded ex vivo and administered intrathecally. The cells produce neurotrophic factors and are expected to have neuroprotective effects.

Dosing: Three bi-monthly intrathecal injections

The preceding phase III trial of BrainStorm’s NurOwn cells, an autologous mesenchymal stem cell transplant, failed to demonstrate efficacy. (Percentage of ALSFRS-R change showing improvement of 1.25 points/month or more before and after treatment: 32.6% in the NurOwn group and 27.7% in the placebo group; no statistically significant difference) (Cudkowicz et al., 2022).

However, after correction of the statistical methods, the subgroup analysis showed that the subgroups of patients with ALSFRS-R of 27-35 points at baseline had significantly lower ALSFRS-R decline in Erratum.

（https://onlinelibrary.wiley.com/doi/epdf/10.1002/mus.27472） ClinicalTrials.gov Identifier: NCT03280056