NurOwn is an investigational therapy by BrainStorm Cell Therapeutics. It uses a mesenchymal stem cell (MSC) platform to treat patients with amyotrophic lateral sclerosis (ALS). It can be injected into a muscle (intramuscular, IM), or the spinal canal (intrathecal, IT).
How does NurOwn work?
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.
NurOwn aims to help these nerve cells survive by fighting the neurodegeneration process. It uses MSCs harvested from the patients themselves. 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). NTFs are factors that promote the growth of nervous tissue. They also can help nerve cells survive through their own neuroprotective function.
NurOwn in clinical trials
BrainStorm has successfully completed three clinical trials in 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 patients, 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. They suggested that the treatment is safe and well-tolerated, with only mild and temporary side effects. Researchers further noted an improvement in the ALS functional rating scale-revised (ALSFRS-R) and forced vital capacity (FVC) following treatment. These measures assess the 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. Researchers followed participants for three months prior to treatment. They then treated them with a combined IM-IT injection of either NurOwn or a placebo. Investigators monitored participants for another six months to see whether the treatment was safe and whether it produced a clinical response. They used several measures, including ALSFRS-R and slow vital capacity (SVC), which assesses breathing capacity to check lung function.
Researchers presented the results of the trial 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) investigated NurOwn in 189 ALS patients 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 received three IT doses of either NurOwn or a placebo three weeks apart. Researchers then monitored them over a 28-week period and compared their ALSFRS-R scores and biomarkers.
They announced topline results from the trial in November 2020. Those showed that the treatment did not lead to a significant decrease in the deterioration compared to the placebo. Researchers attributed the failure to having more severely affected patients in the study than in the previous Phase 2 trial. In a prespecified group of patients in the early phases of ALS, however, there was the expected level of difference between patients receiving NurOwn and the placebo. Around a third (34.6%) of patients receiving NurOwn showed a slowing of disease progression compared to 15.6% of those receiving the placebo.
BrainStorm is in discussions with the U.S. Food and Drug Administration about the future of the treatment.
Ongoing clinical trials
The company initiated an expanded access program (NCT04681118) for patients with early-stage ALS who participated in the Phase 3 trial. Patients will receive up to three additional IT treatments. Researchers then will follow them for 28 weeks to monitor changes in the ALSFRS-R score.
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.
Last updated: Jan. 7, 2020
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