NurOwn, an investigational therapy developed by Israel’s BrainStorm Cell Therapeutics, uses a mesenchymal stem cell (MSC) platform to treat patients with amyotrophic lateral sclerosis (ALS).

NurOwn can be injected into muscle (the intramuscular, or IM, method) or into the spinal canal (the intrathecal, or IT, method).

How NurOwn works

ALS is a progressive neurodegenerative disease. People with ALS suffer a gradual decline in muscle function and strength as motor neurons — or nerve cells that control muscle movement — die off.

NurOwn aims to help these nerve cells survive by fighting the neurodegeneration process that causes ALS symptoms.

NurOwn uses MSCs harvested from the patient. MSCs are precursor cells that have the ability to differentiate into various cell types. NurOwn’s technology encourages MSCs to develop into cells that secrete neurotrophic factors (NTFs) that promote the growth of nervous tissue. NTFs can also help nerve cells survive through their own neuroprotective function.

NurOwn in clinical trials

BrainStorm has successfully completed three clinical trials across Israel and the United States.

The first two, a Phase 1/2 trial (NCT01051882) of 12 patients and a Phase 2a trial (NCT01777646) of 14 participants took place in Israel. The trials tested the safety of both IM and IT administration of NurOwn as well as any clinical effects. The studies’ results appeared in the scientific journal JAMA Neurology.

The report suggested that the treatment is safe and well-tolerated, with only mild and temporary side effects observed. Furthermore, researchers noted an improvement in the ALS Functional Rating Scale-Revised (ALSFRS-R) and forced vital capacity (FVC) in patients following treatment. These assess progression of ALS in terms of patients’ ability to carry out activities of daily living and lung function.

The third trial, conducted in the United States, was a randomized, double-blind, placebo-controlled Phase 2 study (NCT02017912) assessing the safety of NurOwn in 48 ALS patients. Patients were assessed for three months prior to treatment and then received a combined IM-IT injection of either NurOwn cells or a placebo. Patients were then monitored for another six months to see if the treatment was safe, and whether produced a clinical response. Doctors monitored the patients using several measures including ALSFRS-R and slow vital capacity (SVC), which assesses breathing capacity to check lung function. According to BrainStorm Cell, the results of the study were positive.

BrainStorm Cell are awaiting FDA approval to carry out Phase 3 clinical trial, planned for later this year. Clinical sites confirmed for the planned trial include Massachusetts General Hospital in Boston and California Pacific Medical Center in San Francisco.

Other information

BrainStorm Cell has recently validated the cryopreservation process for NurOwn cells, which preserves MSCs harvested from patients by freezing them in liquid nitrogen. This prevents patients from having to undergo repeated harvesting procedures to produce further doses of NurOwn cells.

 

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