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BrainStorm Cell Therapeutics – NurOwn

BrainStorm Cell Therapeutics – NurOwn

Background

The BrainStorm treatment regimen consists of a person’s own stem cells (called autologous) being removed from bone marrow and then grown outside of the body in the presence of a chemical owned by the company called NurOwn, which aims to increase the stem cells’ ability to make and secrete protective substances called growth factors. The stem cells are then injected into the fluid that bathes the brain and spinal cord (called cerebrospinal fluid or CSF) with a needle (called intrathecal or IT injection) at multiple intervals. The hope is that these treated stem cells will be able to slow the progression of motor neuron degeneration and hence, the progression of ALS symptoms.

In 2016, the first peer reviewed publication appeared, demonstrating preliminary, positive safety data in combination with BrainStorm completing a phase 2 clinical trial at three renowned US clinical sites.

The phase 2 trial data involved 48 ALS patients (36 treated and 12 placebo) and was published in December 2019 in the journal Neurology, titled “A single-dose transplantation of MSC-NTF cells is safe and demonstrated early promising signs of efficacy.”

A phase 3 clinical trial testing multiple doses of NurOwn in 200 participants at six sites in the United States was started in 2017. The trial was double-blinded, meaning neither researchers nor participants knew if they were on active treatment or placebo. The primary measurements were to examine safety of repeated intrathecal injections of NurOwn and the ability of NurOwn to slow progression of ALS/MND using a scale called the ALSFRS-R. To determine if NurOwn-treated stem cells were providing the intended biological effect, the CSF was measured for biomarkers including neurotrophic factors (aiming for increased levels) and neurodegenerative/neuroinflammatory factors (aiming for reduced levels).

On November 17, 2020, a press release was issued describing the initial data from the phase 3 clinical trial. The trial did not meet statistical significance in any of the reported data; notably in the primary measurement of disease progression using the ALSFRS-R. A pre-specified subgroup of those with early disease was highlighted in the press release as showing clinically meaningful slowing of ALS progression, however these results were also not statistically significant. Further, it was reported that that NurOwn treatment resulted in an increase of neurotrophic biomarkers and reduction in neurodegenerative and neuroinflammatory biomarkers when compared to the placebo group, which is aligned with what the trial hoped to achieve. Follow up analysis will investigate whether these biomarker results, in addition to further analysis of the pre-specified subgroup of those with early disease, can reveal any additional information.

Recommendation

Based on the data released by BrainStorm, the SAC recommends that there is no conclusive evidence at this time to suggest NurOwn provides benefit to people with ALS. When additional trial data becomes available and further analysis is performed, the SAC will continue to provide updates as to what the scientific and medical community learns that can support development of effective treatments for ALS/MND.

 

International Alliance of ALS/MND Associations
November 2020

 


The original language of communication is English and any translation cannot be guaranteed for accuracy of messaging.

Primary Sidebar

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

  • David Watson, MND Scotland, Diagnosed 2018, Scotland

    David Watson, MND Scotland, Diagnosed 2018, Scotland

  • Joanne Pratt, Diagnosed 2011 - MND Australia

    Joanne Pratt, Diagnosed 2011 – MND Australia

  • Angie Bordaen, Diagnosed 2014 - ALS Liga België, Belgium

    Angie Bordaen, Diagnosed 2014 – ALS Liga België, Belgium

  • Joyce Rusinak, Forbes Norris ALS Center, USA

    Joyce Rusinak, Forbes Norris ALS Center, USA

  • Sally Pauls, Diagnosed 2006 - Les Turner ALS Foundation

    Sally Pauls, Diagnosed 2006 – Les Turner ALS Foundation

  • Susan Anderson, Diagnosed 2014 - Hope Loves Company, USA

    Susan Anderson, Diagnosed 2014 – Hope Loves Company, USA

  • Osiel Mendoza, Diagnosed 2016 - ALS Therapy Development Institute, USA

    Osiel Mendoza, Diagnosed 2016 – ALS Therapy Development Institute, USA

  • Steven Spencer, Diagnosed 2014 - MND New Zealand

    Steven Spencer, Diagnosed 2014 – MND New Zealand

  • Conny van der Meijden, Diagnosed 2001 - ALS Netherlands

    Conny van der Meijden, Diagnosed 2001 – ALS Netherlands

  • Irene McCaughey, Diagnosed 2011 - MND Australia

    Irene McCaughey, Diagnosed 2011 – MND Australia

  • Eddy LeFrançois, Diagnosed 1992 - ALS Canada

    Eddy LeFrançois, Diagnosed 1992 – ALS Canada

  • Jon Newsome, Les Turner ALS Foundation, USA

    Jon Newsome, Les Turner ALS Foundation, USA

  • Monica Soriano, Diagnosed 2011 - Asociación ELA Argentina

    Monica Soriano, Diagnosed 2011 – Asociación ELA Argentina

  • Roxana Canova, Diagnosed 2012 - Asociación ELA Argentina

    Roxana Canova, Diagnosed 2012 – Asociación ELA Argentina

  • Mary Thomas, Diagnosed 2013 - MND Australia

    Mary Thomas, Diagnosed 2013 – MND Australia

  • Len Johnrose, MND Association, Diagnosed 2017, England

    Len Johnrose, MND Association, Diagnosed 2017, England

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Liong Ting Ngu, MND Malaysia, Diagnosed 2014, Malaysia

    Liong Ting Ngu, MND Malaysia, Diagnosed 2014, Malaysia

  • Maurice LeClerc, ALS Canada

    Maurice LeClerc, ALS Canada

  • Shay Rishoni, Diagnosed 2011 - Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 – Prize4Life, Israel

  • Ailsa Malcolm-Hutton, Diagnosed 2013 - MND Association of England, Wales and N Ireland

    Ailsa Malcolm-Hutton, Diagnosed 2013 – MND Association of England, Wales and N Ireland

  • Dorette Lüdi, Diagnosed 2014 - ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 – ALS Schweiz, Switzerland

  • Chris McCauley, Diagnosed 2015 - ALS Canada

    Chris McCauley, Diagnosed 2015 – ALS Canada

  • Margreth Burger-Saile, Diagnosed 2011 - ALS Schweiz, Switzerland

    Margreth Burger-Saile, Diagnosed 2011 – ALS Schweiz, Switzerland

  • Zabun Nassar, MND Association, Diagnosed 2016, England

    Zabun Nassar, MND Association, Diagnosed 2016, England

  • Wilfried Leusing, Diagnosed 2010 - DGM, Germany

    Wilfried Leusing, Diagnosed 2010 – DGM, Germany

  • Karl Hughes, Diagnosed 2010 - IMNDA, Ireland

    Karl Hughes, Diagnosed 2010 – IMNDA, Ireland

  • Marcel R. Wernard, Diagnosed 2016 - ALS Patients Connected, The Netherlands

    Marcel R. Wernard, Diagnosed 2016 – ALS Patients Connected, The Netherlands

  • Zelina-Brito-Diagnosed-2018-Brazil

    Zelina-Brito-Diagnosed-2018-Brazil

  • Joy Blakeley, Diagnosed 2017 - MND Australia

    Joy Blakeley, Diagnosed 2017 – MND Australia

  • Philip Brindle, MND Association, Diagnosed 2015, England

    Philip Brindle, MND Association, Diagnosed 2015, England

  • Oliver Juenke, DGM, Germany

    Oliver Juenke, DGM, Germany

  • Denis Blais, Diagnosed 2015 - ALS Canada

    Denis Blais, Diagnosed 2015 – ALS Canada

  • Richard Clark, MND New Zealand, Diagnosed 2011, New Zealand

    Richard Clark, MND New Zealand, Diagnosed 2011, New Zealand

  • Erwin Coppejans, Diagnosed 2007 - ALS Liga België, Belgium

    Erwin Coppejans, Diagnosed 2007 – ALS Liga België, Belgium

  • Nicholas (Nic) Bowman, MND Association of South Africa, Diagnosed 2016, Australia

    Nicholas (Nic) Bowman, MND Association of South Africa, Diagnosed 2016, Australia

  • Brian Lovell, Diagnosed 2011 - MND Australia

    Brian Lovell, Diagnosed 2011 – MND Australia

  • Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013, Belgium

    Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013, Belgium

  • Duncan Bayly - MND Australia

    Duncan Bayly – MND Australia

  • Carlos Gomez Matallanas, Diagnosed 2014 - FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 – FUNDELA, Spain

  • Mike Rannie, ALS Canada, Diagnosed 2017, Canada

    Mike Rannie, ALS Canada, Diagnosed 2017, Canada

  • Inta Grubb, Diagnosed 2014 - MND Australia

    Inta Grubb, Diagnosed 2014 – MND Australia

  • Josée Kolijn-de Man, Diagnosed 2015 - ALS Patients Connected, The Netherlands

    Josée Kolijn-de Man, Diagnosed 2015 – ALS Patients Connected, The Netherlands

  • Sanjay Kumar Srivastava, Asha Ek Hope Foundation for ALS/MND, Diagnosed 2018, India

    Sanjay Kumar Srivastava, Asha Ek Hope Foundation for ALS/MND, Diagnosed 2018, India

  • Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Rolf Mauch, Association ALS Switzerland, Diagnosed 2015, Switzerland

    Rolf Mauch, Association ALS Switzerland, Diagnosed 2015, Switzerland

  • Norm MacIsaac, ALS Society of Canada, ALS Society of Quebec, Diagnosed 2014, Canada

    Norm MacIsaac, ALS Society of Canada, ALS Society of Quebec, Diagnosed 2014, Canada

  • Francisco Perez Palop, Diagnosed 2013 - FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 – FUNDELA, Spain

  • Kirsten Harley, Diagnosed 2013 - Australia

    Kirsten Harley, Diagnosed 2013 – Australia

Learn more about the March of Faces

Advocacy

  • Advocacy Toolkit
  • Rights of People with ALS/MND
  • Biogen – Tofersen
  • BrainStorm Cell Therapeutics – NurOwn
  • Collaborative Medicinal Development – CuATSM
  • Cytokinetics – Reldasemtiv
  • Right to Try
  • Unproven (Off-Label) Treatments
  • Amylyx – AMX0035
  • Kadimastem – AstroRx

Allied Professionals Forum Registration

The International Alliance of ALS/MND Associations will host a virtual Allied Professionals Forum spread over two days to accommodate time zones on 3 – 4 December 2020.

Click here to register for the Allied Professionals Forum

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