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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.

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

  • Calum Ferguson, Diagnosed 2010 , MND Scotland, UK

    Calum Ferguson, Diagnosed 2010 , MND Scotland, UK

  • Amparo Muriel Engativa, Colombia

    Amparo Muriel Engativa, Colombia

  • Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

  • Alan Liz Ogg 29042016 000799 lo res

    Alan Liz Ogg 29042016 000799 lo res

  • Timmy, ALS Liga

    Timmy, ALS Liga

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

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

  • Joy Blakeley, Diagnosed 2017 , MND Australia

    Joy Blakeley, Diagnosed 2017 , MND Australia

  • Horacio Fritzer, Argentina

    Horacio Fritzer, Argentina

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

    Susan Anderson, Diagnosed 2014 , Hope Loves Company, USA

  • Zabun Nassar, MND Association, Diagnosed 2016, England

    Zabun Nassar, MND Association, Diagnosed 2016, England

  • Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

    Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

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

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • 83

    83

  • Conny van der Meijden, Diagnosed 2001,  ALS Netherlands

    Conny van der Meijden, Diagnosed 2001, ALS Netherlands

  • Claudia Cominetti, Associazione conSLAncio Onlus,  Italy

    Claudia Cominetti, Associazione conSLAncio Onlus, Italy

  • Mike Rannie,  ALS Canada,  Diagnosed 2017

    Mike Rannie, ALS Canada, Diagnosed 2017

  • Ann Nicol

    Ann Nicol

  • Chun Ju Xiao, China

    Chun Ju Xiao, China

  • Guoqiang Xu, Diagnosed 2016 , Shaanxi ALS Association, China

    Guoqiang Xu, Diagnosed 2016 , Shaanxi ALS Association, China

  • Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

  • 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

  • Inta Grubb, Diagnosed 2014,  MND Australia

    Inta Grubb, Diagnosed 2014, MND Australia

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

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

  • Cath Muir

    Cath Muir
    Cath

  • Colm Francis Davis, Ireland

    Colm Francis Davis, Ireland

  • Bob Simonds and Drew O'Neill , Les Turner ALS Foundation, USA

    Bob Simonds and Drew O’Neill , Les Turner ALS Foundation, USA

  • Michel Perrozzo, ARSLA, Diagnosed 2015, France

    Michel Perrozzo, ARSLA, Diagnosed 2015, France

  • Camilla Heiberg Freiberg, Muskelsvindfonden, Denmark

    Camilla Heiberg Freiberg, Muskelsvindfonden, Denmark

  • Steve Gallagher, ALS Society of Canada

    Steve Gallagher, ALS Society of Canada
    Picture1

  • Andrietta

    Andrietta

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
    final3878

  • Dawn Morton, Diagnosed 2014 , MND Scotland, UK

    Dawn Morton, Diagnosed 2014 , MND Scotland, UK

  • Natalya Rybakova, Russian Charity ALS Foundation

    Natalya Rybakova, Russian Charity ALS Foundation

  • Liam Dwyer, England

    Liam Dwyer, England

  • Fabio Correia

    Fabio Correia

  • Steve Lufkin, USA

    Steve Lufkin, USA
    IMG_3993

  • Steve

    Steve

  • Brigitte Wernli,  Association ALS Switzerland,  Diagnosed 2014

    Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014

  • Roy

    Roy
    roy

  • Karl Hughes, Diagnosed 2010 , IMNDA,  Ireland

    Karl Hughes, Diagnosed 2010 , IMNDA, Ireland

  • Bob Simonds and Drew O'Neil, USA

    Bob Simonds and Drew O’Neil, USA

  • John Dinon, MND Australia

    John Dinon, MND Australia

  • England-Lee-Millard, UK

    England-Lee-Millard, UK

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Den Haag, Diagnosed 2016 , The Netherlands

    Den Haag, Diagnosed 2016 , The Netherlands

  • Shay Rishoni, Diagnosed 2011 - Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 – Prize4Life, Israel

  • Mahmood Anwar, UK

    Mahmood Anwar, UK

  • Joanne Pratt, Diagnosed 2011 , MND Australia

    Joanne Pratt, Diagnosed 2011 , MND Australia

  • Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • Jason Goodman, Les Turner ALS Foundation, USA

    Jason Goodman, Les Turner ALS Foundation, USA

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