November 2020: Update from the Scientfiic Advisory Council (SAC)
In November 2020, the results from the Phase 3 trial of NurOwn were made available. The trial unfortunately did not meet statistical significance in any of the reported data. Based on the Phase 3 NurOwn data released by BrainStorm, the SAC recommends that there is no conclusive evidence at this time to suggest that NurOwn provides benefit to people with MND. Please click here to read the full briefing note from the Scientific Advisory Council. 

What is BrainStorm’s NurOwn?

NurOwn and the treatment approach were developed by BrainStorm Cell Therapeutics, a biotechnology company based in New York. The company research and develop autologous stem cell therapies for debilitating neurodegenerative diseases, including motor neurone disease (MND), Multiple Sclerosis (MS) and Parkinson’s Disease (PD).

NurOwn is a process that is specifically targeted to grow a person’s own stem cells into a form that can then help stimulate growth of brain cells (MSC-NTF cells). The NurOwn process is part of a treatment involving the stem cells being removed from a person’s bone marrow, and then grown outside their body in the Brainstorm laboratories.

The treatment aims to increase the stem cells’ ability to make and secrete substances called “neurotropic growth factors” (NTF), that specifically stimulate the growth of brain cells. These specially treated stem cells are then injected into muscle or the fluid that bathes the brain and spinal cord (called cerebrospinal fluid or CSF) with a needle (called intrathecal or IT injection), multiple times during the treatment period.

The hope is that these stem cells with boosted abilities will be able to slow motor neuron degeneration, and therefore MND progression.

A brief history of Nurown’s clinical trials for NurOwn development
In 2016, the first peer reviewed study on the safety data of the NurOwn treatment in MND was published following a combined Phase 1/2 and Phase 2a MND clinical trial at the Hadassah Medical Center in Jerusalem, Israel. The trial produced promising safety data and cautious optimism that there was some treatment effect, though the trial was not large enough to confirm this.

A subsequent Phase 2 clinical trial at three renowned centres in the US finished in 2016. This trial has shown some effectiveness in a sub-group of faster-progressing patients and also provided important information about biomarkers and also how long a single dose may last for.

Completion of these clinical trials has come after several years of related and unrelated media that increased the popular belief that stem cells are all-encompassing agents of healing. Media and popular belief contributed to the clinical trials of NurOwn becoming one of the most publicly discussed experimental therapeutics in the field. Brainstorm has continually been in MND related news since 2012.

In 2017, a 200 participant Phase 3 clinical trial was started at six sites in the United States. Recruitment for the trial is complete, and results will hopefully become available in 2020. The Phase 3 trial should be sufficiently large enough to provide a clear indication of the potential effectiveness of Nurown as a MND treatment.
Phase 2 Clinical Trial Details:
  • It was conducted at Massachusetts General Hospital and UMass Medical School (Massachusetts, United States), and Mayo Clinic (Minnesota, United States) and commenced in 2014 and was completed in 2016.
  • 48 patients were enrolled who had been diagnosed with MND, who had limb weakness, and had not previously received stem cell therapy of any kind (more information about eligibility)
  • Patients were treated with single autologous dose of MSC-NTF cells by combined intramuscular and intrathecal administration – a transplantation of autologous bone marrow derived mesenchymal stromal cells (MSC), which are enriched from the patients' own bone marrow, grown in the laboratory and induced to secrete NTFs. The autologous MSC-NTF cells are back-transplanted into the ALS patient into the sites of damage, the spinal cord and the muscles.

Phase 2 Clinical Trial Results:
Results from the Phase 2 trial were recently published in November 2019. The results showed promising effectiveness in a sub-group of faster progressing patients (a “responder analysis”).
  • The rate of disease progression (Revised ALS Functional Rating Scale [ALSFRS-R] slope change) in the overall study population was similar in treated and placebo participants.
  • Treatment stabilised the rate of ALS disease progression (ALSFRS-R slope) for up to 12-16 weeks in a pre-specified group of participants with rapid progression following a single transplantation.
  • Treatment increased CSF neurotrophic factors and decreased inflammatory biomarkers 2 weeks post-transplantation.
  • CSF MCP-1 levels (a marker of microglial activation and neuroinflammation) significantly decreased post-transplantation and correlated with ALSFRS-R slope improvement at all time points.
  • In the responder analyses, a higher proportion of MSC-NTF-treated participants achieved a ≥1.5-point improvement per month, which may reflect disease stabilization. This improvement was most prominent immediately following transplantation and gradually decreased toward the end of the study, suggesting the need for repeated treatments to maintain a sustained therapeutic effect.
Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer of BrainStorm stated "We met our primary endpoint and demonstrated that a single dose of NurOwn was safe and well-tolerated while supporting NurOwn's mechanism of action on neuroprotection and neuroinflammation pathways in ALS. We look forward to completing the current Phase 3 study to confirm the promising Phase 2 findings and expand our understanding of the potential of MSC-NTF cell therapy in ALS."

At this stage, evidence suggests the NurOwn treatment is safe, and has potential benefits for slowing the progression of MND – although the clinical data is currently insufficient to know with certainty.

If the Phase 3 trial goes well, it will inform the field about whether NurOwn will be able to significantly slow MND. The Phase 3 trial was reported to be fully enrolled with 200 patients in October 2019. The trial is expected to be completed in October 2020 and we look forward to seeing the results sometime after that date.

Content and information included here was adapted and/or drawn from documents provided by the ALS/MND Alliance Scientific Advisory Council, and published research and clinical trial reporting.

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