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International Alliance of ALS/MND Associations

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Kadimastem – AstroRx

Background

Kadimastem is a company that is currently testing an “off-the-shelf”, clinical grade  astrocyte cell product called AstroRx®. This investigational treatment consists of  astrocytes in suspension, derived from human embryonic stem cells. Astrocytes are  neighbouring (glial) cells that provide support to motor neurons when healthy. As  of July 2023, there has been a single, phase1/2a, open label trial in 10 participants  for AstroRx®. Kadimastem announced in March 2023 that it has received IND  approval to commence a phase 2a trial for AstroRx® and that this trial will evaluate  multiple doses at 3-month intervals.

Trial Design & Results

In February 2023, the results of a phase 1/2a study for AstroRx® were published.  The open label trial involved a 3-month monitoring period followed by a single  intrathecal injection of AstroRx®. Prior to dosing, all participants began a regimen  of immunosuppressant mycophenolate mofetil (MMF) because AstroRx® is not  autologous (i.e. not generated from the patient receiving the treatment).  Participants were monitored for 6 months after dosing and then could enter a  second protocol for an additional 6 months of evaluation. Three cohorts were  planned. Cohort B received a dose with a higher concentration of AstroRx® cells  than Cohort A. The last group, Cohort C, was intended to receive multiple doses,  but was discontinued because of COVID-19 pandemic challenges.  

The primary study outcomes were safety and tolerability. Central Nervous System  (CNS) imaging was conducted at specified intervals. Secondary outcomes evaluated  efficacy, with ALSFRS-R collection as well as measures of strength. Serum  biomarkers were analyzed including creatinine, creatine, and neurofilament light  (NfL).  

During the study, 3 of the 10 participants died, although not attributed by  investigators to AstroRx®. Of documented adverse events, the most common was  post lumbar puncture headache in 50% of participants. There were also a few mild  to moderate adverse events potentially related to the immunosuppressant. 

Lab values, ECG, and physical exams showed no clinically significant changes. MRI  imaging at 6 months did not show tumours, which had been a concern because  embryonic derived cells may contribute to teratomas (a type of tumour).  

In both cohorts, at 3-months after administration of AstroRx® there was a  statistically significant slowing in the rate of ALSFRS-R decline that reverted to the  pre-treatment rate at the 6 and 12 month assessments. However, the rate of SVC  decline (a marker of breathing functionality) had a statistically significant  acceleration across both cohorts at the 3 and 12-month timepoints which is  concerning and should be explored in future studies. Hand-held dynamometry, a  measure of strength, trended towards improvement but was not statistically  significant. There was no significant effect on NfL within this study.

Summary

Kadimastem has conducted a single dose open label study of AstroRx® in 10  people, of which only 6 were followed up at the one-year timepoint. Within this  trial there were multiple protocol modifications due to the COVID-19 pandemic.  

The primary focus of the trial was safety and tolerability, although measures of  efficacy and biomarkers were evaluated as well. While there were statistically  significant reductions in the rate of decline of ALSFRS-R in both cohorts of 5 people  at 3 months post treatment, for those participants that followed up at 6 and 12  months, ALSFRS-R, those changes were lost. Additionally, the statistically significant  increase in rate of SVC decline seen post treatment is concerning. There is also the  question of whether the immunosuppressant, MMT, which was administered in  addition to AstroRx® may have effects on the disease course and may be  contributing to outcomes either positively or negatively.  

The SAC acknowledges the potential ability of healthy astrocytes to provide support  to motor neuron health. Given the very small size of the trial and the single-dose  treatment of participants with no placebo there is insufficient evidence to  determine efficacy of AstroRx® in ALS/MND currently. 

A number of messages provided by Kadimastem could raise questions from  people affected by ALS/MND. Kadimastem uses the slogan “cells to cure diseases”  and their website explains that the goals of AstroRx® treatment include “replacing  malfunctioning cells” and “restoring functionality”. Evidence has not been presented  showing that AstroRx® could replace malfunctioning cells or restore functionality in  humans. 

Caution should be taken when interpreting the results so far and should continue  until larger, properly controlled clinical trials have been completed.  

International Alliance of ALS/MND Associations
March 2024


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

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Drugs in Development

  • AB Science – Masitinib
  • BrainStorm Cell Therapeutics – NurOwn
  • Clene Nanomedicine – CNM-Au8
  • Collaborative Medicinal Development – CuATSM
  • ILB – Tikomed
  • Kadimastem – AstroRx
  • Methylcobalamin
  • Mitsubishi Tanabe Pharma America – Oral Edaravone
  • Neuronata-R/Lenzumestrocel
  • NeuroSense – PrimeC
  • Neuvivo – NP001
  • Prilenia Therapeutics – Pridopidine
  • SOD1 Therapies & Trials
  • T Regulatory Cell Therapies
  • Ulefnersen – Ionis Pharmaceuticals

  • Xian-Zhang Niu, Diagnosed 2006 , Shaanxi ALS Association, China

    Xian-Zhang Niu, Diagnosed 2006 , Shaanxi ALS Association, China

  • Kirsten Harley,  Diagnosed 2013,  Australia

    Kirsten Harley, Diagnosed 2013, Australia

  • Bayley, Australia

    Bayley, Australia

  • Daniel Hare

    Daniel Hare

  • Enzo Maccarrone, AISLA ONLUS, Italy

    Enzo Maccarrone, AISLA ONLUS, Italy

  • Claudette Sturk, ALS Society of Canada

    Claudette Sturk, ALS Society of Canada
    Picture2

  • Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

    Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

  • Alfredo Santos, Diagnosed 2013 , ACELA, Colombia

    Alfredo Santos, Diagnosed 2013 , ACELA, Colombia

  • Fabio Carvalho

    Fabio Carvalho

  • Ian and Teresa Roberts

    Ian and Teresa Roberts

  • Catherine Pearce, Australia

    Catherine Pearce, Australia

  • Angela Jansen, Deutsche Gesellschaft für Muskelkranke e.V.-DGM, Diagnosed 1995, Germany

    Angela Jansen, Deutsche Gesellschaft für Muskelkranke e.V.-DGM, Diagnosed 1995, Germany

  • Yessenia Hernandez Mendoza, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Yessenia Hernandez Mendoza, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

    João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

  • Willi Klein

    Willi Klein

  • Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

    Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

  • Lucy Lintott, Diagnosed 2013 , MND Scotland, UK

    Lucy Lintott, Diagnosed 2013 , MND Scotland, UK

  • Duncan Bayly , MND Australia

    Duncan Bayly , MND Australia

  • Maurice Leclerc, Canada

    Maurice Leclerc, Canada

  • Ana Lilia RodriguezApoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Ana Lilia RodriguezApoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • Joyce Rusinak, Forbes Norris ALS Center, USA

    Joyce Rusinak, Forbes Norris ALS Center, USA

  • Lachlan Terry,  MND Australia,  Diagnosed 2015

    Lachlan Terry, MND Australia, Diagnosed 2015

  • Sébastien Batiot, Diagnosed 2012 , ARSLA, France

    Sébastien Batiot, Diagnosed 2012 , ARSLA, France

  • Roy

    Roy
    roy

  • Timothy Holman, Switzerland

    Timothy Holman, Switzerland

  • Philip Brindle,  MND Association,  Diagnosed 2015,  England

    Philip Brindle, MND Association, Diagnosed 2015, England

  • Monica Soriano, Diagnosed 2011 ,  Asociación ELA , Argentina

    Monica Soriano, Diagnosed 2011 , Asociación ELA , Argentina

  • Lombana, Spain

    Lombana, Spain

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Chris McCauley, Diagnosed 2015 , ALS Canada

    Chris McCauley, Diagnosed 2015 , ALS Canada

  • Greg Heydet, ALS Hope Foundation, USA

    Greg Heydet, ALS Hope Foundation, USA

  • Chun Ju Xiao, China

    Chun Ju Xiao, China

  • Steve Gallagher, ALS Society of Canada

    Steve Gallagher, ALS Society of Canada
    Picture1

  • Natalya Rybakova, Russian Charity ALS Foundation

    Natalya Rybakova, Russian Charity ALS Foundation

  • Andrietta

    Andrietta

  • Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

    Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

  • Andrea Zicchieri, Associazione conSLAncio Onlus, Italy

    Andrea Zicchieri, Associazione conSLAncio Onlus, Italy
    AndreaZicchieri_conSLAncioItaly

  • Ian Roberts

    Ian Roberts

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • Shera Mukherjee, Diagnosed 2013,  Asha Ek Hope Foundation, India

    Shera Mukherjee, Diagnosed 2013, Asha Ek Hope Foundation, India

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

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Liong Ting Ngu, MND Malaysia, Diagnosed 2014

    Liong Ting Ngu, MND Malaysia, Diagnosed 2014

  • Fabio Carvalho, Associação Pró-Cura da ELA, Brazil

    Fabio Carvalho, Associação Pró-Cura da ELA, Brazil

  • Susan Keldani, Les Turner ALS Foundation, USA

    Susan Keldani, Les Turner ALS Foundation, USA

  • Dad

    Dad

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
    final3878

  • Aida Trzmiel de Guterman, Asociacion ELA Argentina, Diagnosed 2007, Argentina

    Aida Trzmiel de Guterman, Asociacion ELA Argentina, Diagnosed 2007, Argentina

  • David Bishop

    David Bishop

  • unnamed

    unnamed

  • 83

    83

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