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

  • Karl Hughes, Diagnosed 2010 , IMNDA,  Ireland

    Karl Hughes, Diagnosed 2010 , IMNDA, Ireland

  • Chen Yin Xue, Taiwan MND Association, Diagnosed 1995, Taiwan

    Chen Yin Xue, Taiwan MND Association, Diagnosed 1995, Taiwan

  • Daniel Hare

    Daniel Hare

  • Margarita Pizarro, Asociacion ELA Argentina, Diagnosed 2017, Argentina

    Margarita Pizarro, Asociacion ELA Argentina, Diagnosed 2017, Argentina

  • Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

    Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

  • Calum Ferguson, Diagnosed 2010 , MND Scotland, UK

    Calum Ferguson, Diagnosed 2010 , MND Scotland, UK

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

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

  • Mike Small, Motor Neurone Disease (MND) Association, UK

    Mike Small, Motor Neurone Disease (MND) Association, UK

  • Steven Spencer, Diagnosed 2014 , MND New Zealand

    Steven Spencer, Diagnosed 2014 , MND New Zealand

  • Enzo Maccarrone, AISLA ONLUS, Italy

    Enzo Maccarrone, AISLA ONLUS, Italy

  • Sam Hayden-Harler, Motor Neurone Disease (MND) Association, UK

    Sam Hayden-Harler, Motor Neurone Disease (MND) Association, UK

  • Lin Yong Yi, Taiwan MND Association, Diagnosed 2004

    Lin Yong Yi, Taiwan MND Association, Diagnosed 2004

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • Gudjon Sigurdsson, Diagnosed 2004 , MND Association of Iceland

    Gudjon Sigurdsson, Diagnosed 2004 , MND Association of Iceland

  • Eddy LeFrançois, Diagnosed 1992,  ALS Canada

    Eddy LeFrançois, Diagnosed 1992, ALS Canada

  • Jean

    Jean
    jean

  • Colm Francis Davis, Ireland

    Colm Francis Davis, Ireland

  • Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

  • Richard Clark, MND New Zealand,  Diagnosed 2011

    Richard Clark, MND New Zealand, Diagnosed 2011

  • Horacio Fritzer, Argentina

    Horacio Fritzer, Argentina

  • Andrietta

    Andrietta

  • Alan Liz Ogg 29042016 000799 lo res

    Alan Liz Ogg 29042016 000799 lo res

  • Shay Rishoni, Diagnosed 2011 , Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 , Prize4Life, Israel

  • Fabio Carvalho

    Fabio Carvalho

  • Steven Gallagher, Canada

    Steven Gallagher, Canada

  • Roy

    Roy
    roy

  • Jack Buzby, USA

    Jack Buzby, USA

  • Liam Dwyer, England

    Liam Dwyer, England

  • Orlando Ruiz, Diagnosed 2001,  ACELA, Colombia

    Orlando Ruiz, Diagnosed 2001, ACELA, Colombia

  • Ian Gale, MND Australia

    Ian Gale, MND Australia

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

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

  • Jan Zuring, Diagnosed 2010 , The Netherlands

    Jan Zuring, Diagnosed 2010 , The Netherlands

  • Inta Grubb, Diagnosed 2014,  MND Australia

    Inta Grubb, Diagnosed 2014, MND Australia

  • Ada Garrido Benavidez, Diagnosed 2016,  FYADENMAC, Mexico

    Ada Garrido Benavidez, Diagnosed 2016, FYADENMAC, Mexico

  • Ian Roberts

    Ian Roberts

  • Amparo Muriel Engativa, Colombia

    Amparo Muriel Engativa, Colombia

  • Malcolm Buck, Australia

    Malcolm Buck, Australia

  • JP

    JP

  • Michel Perrozzo, ARSLA, Diagnosed 2015, France

    Michel Perrozzo, ARSLA, Diagnosed 2015, France

  • Maria Lucia Wood Saldanha, Associação Pró-Cura da ELA, Brazil

    Maria Lucia Wood Saldanha, Associação Pró-Cura da ELA, Brazil

  • Timmy, ALS Liga

    Timmy, ALS Liga

  • Carlos Alberto Báez Murillo, ACELA, Colombia

    Carlos Alberto Báez Murillo, ACELA, Colombia

  • Olga Cosentino, Diagnosed 2013,  Asociación ELA Argentina

    Olga Cosentino, Diagnosed 2013, Asociación ELA Argentina

  • Christian Bär, Germany

    Christian Bär, Germany

  • Natalya Rybakova, Russian Charity ALS Foundation

    Natalya Rybakova, Russian Charity ALS Foundation

  • Leon Ryba, Argentina

    Leon Ryba, Argentina

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Den Haag, Diagnosed 2016 , The Netherlands

    Den Haag, Diagnosed 2016 , The Netherlands

  • Bruno Leanza Mantegna, Diagnosed 1999 , AISLA Onlus, Italy

    Bruno Leanza Mantegna, Diagnosed 1999 , AISLA Onlus, Italy

  • Claudia Cominetti, Associazione conSLAncio Onlus,  Italy

    Claudia Cominetti, Associazione conSLAncio Onlus, Italy

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