AstroRx is a stem cell therapy developed by Kadimastem for the treatment of amyotrophic lateral sclerosis (ALS). It is comprised of fully matured human astrocytes derived from human embryonic stem cells that are injected into the fluid surrounding the spinal cord of the patient to support damaged motor neurons.
The U.S. Food and Drug Administration granted AstroRx orphan drug status for the treatment of ALS in November 2018.
How AstroRx works
Astrocytes are star-shaped cells of the central nervous system that play a major role in maintaining a healthy brain environment. Although the loss of motor neuron function is a major hallmark of ALS, it has been shown that malfunctioning astrocytes also play a major role in disease progression.
AstroRx is derived from human embryonic stem cells that are made to develop into specialized cells called astrocyte progenitor cells (APCs). These APCs are then further allowed to mature into astrocytes.
This preparation of healthy astrocytes can be directly injected, along with a variety of neurogenic and neuroprotective factors, into the spinal fluid of ALS patients. These healthy astrocytes can compensate for the malfunctioning astrocytes and help in preventing the loss of motor neurons, slowing the progression of ALS.
AstroRx in clinical trials
The effectiveness of AstroRx was first successfully demonstrated in preclinical studies with animal models of ALS. In these studies, a mouse model of ALS injected with AstroRx showed improvements in voluntary movement, delayed disease onset, and prolonged survival. Moreover, the astrocytes in AstroRx did not become cancerous within a nine-month observation period.
A Phase 1/2a clinical trial (NCT03482050) is currently underway at the Department of Neurology at the Hadassah Ein-Kerem Medical Center in Israel to demonstrate the safety and efficacy of AstroRx in ALS patients. The trial aims to recruit a total of 21 participants and is expected to be completed by 2020.
In this trial, the first set of five patients have already received AstroRx treatment, and the next set of patients are expected to receive the therapy in April 2019. Results for the first set of patients are expected to be available in the second half of 2019.
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