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

NurOwn can be injected into a muscle, called 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 experience a gradual decline in muscle function and strength as motor neurons — the 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), or factors 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 U.S. 

The first two, a Phase 1/2 trial (NCT01051882) in 12 patients, and a Phase 2a trial (NCT01777646) in 14 participants, took place in Israel. They tested the safety of both IM and IT administration of NurOwn, as well as any clinical effects.

The results appeared in the scientific journal JAMA Neurology, and suggested that the treatment is safe and well-tolerated, with only mild and temporary side effects observed. Further, researchers noted an improvement in the ALS functional rating scale-revised (ALSFRS-R) and forced vital capacity (FVC) in patients following treatment. These measures assess progression of ALS in terms of patients’ ability to carry out daily living activities, and lung function.

The third trial, conducted in the U.S., was a randomized, double-blind, placebo-controlled Phase 2 study (NCT02017912) assessing the safety of NurOwn in 48 ALS patients. Participants were followed for three months prior to treatment and then received a combined IM-IT injection of either NurOwn or a placebo. They were then monitored for another six months to see whether the treatment was safe, and whether it 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.

The results of the Phase 2 trial were presented at the 2018 American Association of Neurological Surgeons (AANS) conference and 2018 Northeast amyotrophic lateral sclerosis consortium (NEALS) meeting. The presentations showed improvement in ALSFRS-R scores as well as biomarker changes associated with neuroprotection and immunomodulation (changes in the immune system response).

A Phase 3, randomized, double-blind, placebo-controlled clinical trial (NCT03280056) is currently recruiting participants at the UC Irvine Alpha Stem Cell Clinic, Cedars-Sinai Medical Center, Pacific Medical Center Research Institute, Massachusetts General Hospital, Mayo Clinic, and University of Massachusetts. Participants in the study will receive repeated IT doses of either NurOwn or a placebo over a 28-week period, and their ALSFRS-R scores and biomarkers will be compared.

Other information

BrainStorm Cell has 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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