AstroRx Given at Higher Dose Still Safely Slowing ALS Progression, Trial Reports

AstroRx Given at Higher Dose Still Safely Slowing ALS Progression, Trial Reports
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A single infusion of AstroRx, an off-the-shelf, investigational cell therapy for amyotrophic lateral sclerosis (ALS), safely and significantly slows disease progression in the first three months following treatment, according to updated data from an ongoing Phase 1/2 trial.

These benefits waned after six months, indicating that repeat dosing may be needed to prolong AstroRx’s effectiveness.

“The results after 6 months of follow up are encouraging, as they suggest a clinically meaningful signal of effect for a period of 3 months by a single administration of AstroRx and confirm the safety of AstroRx,” Marc Gotkine, MD, head of the ALS Clinic at the Department of Neurology at Hadassah Medical Center in Israel, and the trial’s principal investigator, said in a press release.

“These results support a further, randomized-controlled, clinical trial with repeated doses of AstroRx in patients with ALS, in order to prolong the clinical effect observed by a single dose,” Gotkine added.

AstroRx, being developed by Kadimastem, is made up of astrocytes, star-shaped nerve cells that are needed to maintain a healthy brain environment. In people with ALS, these cells don’t work as they should, and are thought to contribute to disease progression.

The healthy astrocytes that make up AstroRx are derived from human embryonic stem cells. They are expected to make up for the lack of functional astrocytes in ALS patients and prevent disease worsening once injected into their spinal canal.

A Phase 1/2 trial (NCT03482050), ongoing in Israel, is investigating the safety and preliminary efficacy of increasing doses of AstroRx in adults, ages 18–70, with early stage ALS.

In addition to treatment safety and tolerability, the study is assessing AstroRx’s effectiveness at slowing disease progression, as well as its effects on muscle strength and quality of life.

Measures of disease worsening are based on changes on the ALS Functional Rating Scale revised (ALSFRS-R), a scale that evaluates patients’ ability to perform daily tasks and is considered the gold standard for such assessments.

Previous data from the first five patients included in the trial (group A), all of whom received a single infusion of 100 million cells, showed that AstroRx slows the rate of disease worsening over the first three to four months following dosing.

More recently, the company announced data from a second group of five patients who received a single infusion of AstroRx at a dose of 250 million cells (group B). In this group, the treatment also lowered the rate of ALS progression over the first few months after treatment, based on changes in the decline of participants’ ALSFRS-R scores.

In the three to four months leading up to treatment, their ALSFRS-R scores were dropping at a rate of 1.43 points per month, the company reported. This reduction shifted to a drop of 0.78 points per month in the three months following the therapy’s administration, corresponding to a reduction of 45% in the rate of disease worsening.

New data from this second group of patients showed that their ALSFRS-R decline after six months was similar to the decline observed before treatment, suggesting that repeated dosing may be needed to prolong the therapy’s beneficial effects.

These findings are in agreement with those seen in group A patients, whose ALSFRS-R scores also returned to values identical to those obtained before treatment within roughly the same period of time (five to six months post-dosing).

AstroRx was found to be safe, with no serious side effects and no dose-limiting toxicities in the six months following its use in group B patients. This group’s favorable safety profile was consistent with that seen earlier in group A.

“The 6-month follow up data demonstrates the safety of AstroRx as a treatment for ALS.  The results also support the strategy of a repeated administration of the AstroRx cells using the intrathecal [spinal canal] route. As such, we are planning to continue with this strategy during the next phase of the clinical development program,” said Michel Revel, MD, PhD, founder and chief scientific officer of Kadimastem.

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência. Inês currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.
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Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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