Brainstorm Cell Therapeutics

NurOwn is a stem cell-based treatment that focuses on the cell support system in a person with motor neurons damaged by ALS. It aims to slow the progression of the disease by replacing the damaged system with an enhanced one.

Autologous MSC-NTF cells are produced from the patient's own bone marrow-derived MSCs that have been differentiated in culture.

A patient's own MSCs are harvested and differentiated to secrete high levels of NTFs using a proprietary technology. The differentiated MSCs, known as MSC-NTF cells, are then harvested and prepared for injection into the patient. The MSC-NTF cells are not genetically modified.

Autologous MSC-NTF cells represent an innovative cellular therapy approach that can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

Autologous MSC-NTF cells secrete a unique profile of bioactive molecules, including NTFs, microRNA and cytokines.

Following intrathecal administration, the autologous MSC-NTF cells may activate neuroprotective and immunomodulatory pathways.

Participants undergoing NurOwn administration provided stem cells derived from their own bone marrow aspirate. These stem cells are then programmed to secrete neurotrophic factors (NTFs), aimed at promoting the growth and survival of nerve cells after being released into the patient's spinal cord. The hypothesis is that these cells act by releasing protective factors capable of promoting the survival of neurons and in this way are able to slow down the progression of the disease by alleviating its symptoms.

BrainStorm conducted a Phase 3 randomized, double-blind, placebo-controlled, repeat-dose MSC-NTF autologous cell study in 200 ALS patients at six US sites.

The study ended in November 2020, and at the end of 2021 an article was also published in the journal Nerve and Muscle for peer review by the scientific community.

Currently access to NurOwn is allowed only and exclusively to some patients who have completed the phase III clinical study in the United States, thanks to the Expanded Access Program.

Developments are expected soon.