AstroRx is a stem cell therapy by Kadimastem in testing to possibly treat amyotrophic lateral sclerosis (ALS).

The therapy comprises fully matured human astrocytes derived from human embryonic stem cells, given by intrathecal (into the fluid surrounding the spinal cord) injection to support damaged motor neurons. Motor neurons are nerve cells controlling muscle movement, whose loss is the hallmark of ALS.

How does AstroRx work?

Astrocytes are star-shaped cells of the central nervous system (brain and spinal cord) and play a major role in maintaining a healthy brain environment. Research has shown that problems with the workings of astrocytes contribute to disease progression in ALS.

AstroRx is derived from human embryonic stem cells that are coaxed into becoming specialized cells called astrocyte progenitor cells (APCs). These APCs then further mature into astrocytes.

These healthy astrocytes can be directly injected, along with a variety of neurogenic (nerve-cell forming) and neuroprotective factors (nerve-cell protecting), into the spinal fluid of ALS patients. Treatment aims to compensate for the malfunctioning astrocytes and help to prevent the loss of motor neurons, slowing ALS progression.

AstroRx in clinical trials

Researchers first tested the effectiveness of AstroRx in preclinical studies in mouse models of ALS. Treated animals showed improvements in voluntary movement, delayed disease onset, and they lived longer than mice left untreated. The cell therapy’s safety in the animals was also supported over nine months of observation.

A Phase 1/2a dose-escalating clinical trial (NCT03482050) is testing the safety and efficacy of a single infusion AstroRx in 10 ALS patients enrolled at a single site in Israel. 

A first set of five patients was given AstroRx at low dose (100 million cells), and experienced a significant decrease in the rate of ALS progression that lasted three to four months after the treatment. Over the previous three months, these same patients lost an average of 0.87 points on the ALS functional rating scale revised (ALSFRS-R) each month. That decline fell to an average of 0.26 points per month over the three months post-treatment.

A second group of five patients given a higher dose of AstroRx (250 million cells) also showed a statistically significant decrease in disease progression. Here, average ALSFRS-R score declines of 1.43 points per month prior to treatment moved to an average 0.78 monthly decline in the three months after treatment. At six months post-treatment, these patients’ rate of decline had returned to pretreatment levels, indicating repeat dosing may be needed to maintain benefits.

Researchers reported no serious adverse events in any of these 10 low- or high-dose patients.

Enrollment of two additional patient groups was planned for this study, to test a repeat of the cell treatment at both doses. But those plans changed with COVID-19 pandemic and infection concerns. Kadimastem announced plans to make repeat patient dosing part of future studies.

Additional information

The U.S. Food and Drug Administration designated AstroRx an orphan drug as a potential ALS treatment in November 2018, providing financial and other incentives for its development.

 

Last updated: Jan. 12, 2021

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