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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 and 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.

Primary Sidebar

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
  • T Regulatory Cell Therapies
  • Prilenia Therapeutics – Pridopidine
  • SOD1 Therapies & Trials

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

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

    Erwin Coppejans, Diagnosed 2007 , ALS Liga België, Belgium

  • Kirsty Gerlach, MND New Zealand, Diagnosed 2017

    Kirsty Gerlach, MND New Zealand, Diagnosed 2017

  • Roxana Canova, Diagnosed 2012 ,  Asociación ELA Argentina

    Roxana Canova, Diagnosed 2012 , Asociación ELA Argentina

  • Elisabeth Zahnd, Switzerland

    Elisabeth Zahnd, Switzerland

  • Philip Brindle,  MND Association,  Diagnosed 2015,  England

    Philip Brindle, MND Association, Diagnosed 2015, England

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • Jeff Sutherland

    Jeff Sutherland
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  • Steve

    Steve

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

  • Shay Rishoni, Diagnosed 2011 , Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 , Prize4Life, Israel

  • David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

    David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
    final3878

  • Debbie Craghill, USA

    Debbie Craghill, USA

  • Jon Newsome, USA

    Jon Newsome, USA

  • Wilfried Leusing, Diagnosed 2010 , DGM, Germany

    Wilfried Leusing, Diagnosed 2010 , DGM, Germany

  • Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

    Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

  • Joy Blakeley, Diagnosed 2017 , MND Australia

    Joy Blakeley, Diagnosed 2017 , MND Australia

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • Monica Soriano, Diagnosed 2011 ,  Asociación ELA , Argentina

    Monica Soriano, Diagnosed 2011 , Asociación ELA , Argentina

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

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

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

    Susan Anderson, Diagnosed 2014 , Hope Loves Company, USA

  • Calum Ferguson, Diagnosed 2010 , MND Scotland, UK

    Calum Ferguson, Diagnosed 2010 , MND Scotland, UK

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

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

  • Mauril Belanger

    Mauril Belanger

  • Irene McCaughey, Diagnosed 2011,  MND Australia

    Irene McCaughey, Diagnosed 2011, MND Australia

  • Zabun Nassar, MND Association, Diagnosed 2016, England

    Zabun Nassar, MND Association, Diagnosed 2016, England

  • Maurice LeClerc, ALS Canada

    Maurice LeClerc, ALS Canada

  • Robbie Caliste, UK

    Robbie Caliste, UK

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

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

  • unnamed

    unnamed

  • Den Haag, Diagnosed 2016 , The Netherlands

    Den Haag, Diagnosed 2016 , The Netherlands

  • Imelda Arenas, ACELA, Colombia

    Imelda Arenas, ACELA, Colombia

  • Valdomiro Xavier Honório, Brazil

    Valdomiro Xavier Honório, Brazil

  • Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

    Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

  • Shay Rishoni, Diagnosed 2011 - Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 – Prize4Life, Israel

  • Michael Lee, Australia

    Michael Lee, Australia

  • Steven Gallagher, Canada

    Steven Gallagher, Canada

  • Jorge Melo, ABrELA, Brazil

    Jorge Melo, ABrELA, Brazil

  • Cassio Fernando da Silva, Diagnosed 2013 , ABrELA, Brazil

    Cassio Fernando da Silva, Diagnosed 2013 , ABrELA, Brazil

  • David Bishop

    David Bishop

  • Colm Francis Davis, Ireland

    Colm Francis Davis, Ireland

  • Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

  • Jean Waters, Diagnosed 2004, MND Association of England, Wales and N Ireland

    Jean Waters, Diagnosed 2004, MND Association of England, Wales and N Ireland

  • Greg Heydet, ALS Hope Foundation, USA

    Greg Heydet, ALS Hope Foundation, USA

  • Christian Bär, Germany

    Christian Bär, Germany

  • Shay Rishoni

    Shay Rishoni

  • Mark Miller

    Mark Miller

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

    Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

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