NurOwn

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 also been reported to show promising safety data and some limited clinical efficacy. However, the trial data has not yet been published and subjected to robust external analysis to confirm this.

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 more than halfway done, 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 1/2 and 2a Clinical Trial: Details

  • It was conducted at the Hadassah Medical Center in Jerusalem, Israel and commenced in 2010 and completed in 2014
  • 26 patients were enrolled
  • It recruited people diagnosed with MND, who had limb weakness, and had not previously received stem cell therapy of any kind (more information about eligibility)
  • The primary end points of the studies were safety and tolerability of this cell therapy.
  • Secondary end points included the effects of the treatment on various clinical parameters, such as the ALS Functional Rating Scale-Revised score and the respiratory function.
  • During the phase 1/2 part of the trial, 6 patients with early-stage MND were injected intramuscularly and 6 patients with more advanced disease were transplanted intrathecally.
  • In the second stage, 14 patients with early-stage MND received a combined intramuscular and intrathecal transplantation of autologous MSC-NTF cells (More information about trial procedures).
Phase 1/2 and 2a Clinical Trial: Assessment of safety and tolerability
The results of the trial suggested that the NurOwn treatment was safe and indicated possible clinical benefits, which need to be confirmed in upcoming clinical trials.

Phase 1/2 and 2a Clinical Trial: Assessment of effects on disease
To determine the effect of NurOwn on the disease, the treatment was measured by: the amyotrophic lateral sclerosis functional rating scale; and, biomarkers (e.g. cell-secreted neurotrophic factors, inflammatory factors and cytokines).
Among other results, the trial found a reduction in the rate of forced vital capacity progression during the 6 months following treatment, compared with the pre-treatment phase.
More information about trial results
 
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.
  • BrainStorm reports that findings of the trial are promising with regards to safety and treatment effects, although the results have not yet been published.

summary

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.

 

acknowledgements

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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