Israeli biotech firm's ALS treatment shows safety of use in trials
The objective of this trial was to evaluate the safety of their treatment, with a secondary objective of the trial of estimating its preliminary efficacy.
By Jerusalem Post Staff, December 16, 2020
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AstroRx cells (photo credit: Courtesy) |
Ness Ziona-based biotech firm Kadimastem has shown encouraging results
of Cohort B of its Phase 1/2a clinical trial for AstroRx, its
Amyotrophic Lateral Sclerosis (ALS) treatment trial.
The
objective of this trial was to evaluate the safety of their treatment,
with a secondary objective of the trial of estimating its preliminary
efficacy.
The treatment was developed by Kadimastem and contains functional,
healthy astrocytes (nervous system support cells) derived from Human
Embryonic Stem Cells (hESC) that aim to protect diseased motor neurons.
The company's technology allows injecting AstroRx into the spine of the patient, to slow down the progression of the disease. The treatment has been granted orphan drug designation by the FDA for the treatment of ALS.
The five patients included in this part of the trial showed no serious adverse effects during the half a year follow up after the treatment was given. The rate in which it slows down the disease was also tested for, using the ALS Functional Rating Scale-Revised (ALSFRS-R), which tracks ALS progression, and has shown that after the treatment was given, there was a 45% decline in the disease's progression rate.
At
the end of the 6-month post-treatment period, the rate of ALSFRS-R
progression was similar to the rate that was measured before treatment.
"The
results after 6 months of follow up are encouraging, as they suggest a
clinically meaningful signal of effect for 3 months by a single
administration of AstroRx and confirm the safety of AstroRx," said Dr.
Marc Gotkine, Head of the ALS Clinic at the Department of Neurology at
Hadassah Medical Center in Jerusalem, and the Principal Investigator of
the trial.
"These
results support a further, randomized-controlled, clinical trial with
repeated doses of AstroRx in patients with ALS, to prolong the clinical
effect observed by a single dose,"
“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 route," Professor Michel Revel, Founder and CSO of the Company said. "As such, we are planning to continue with this strategy during the next phase of the clinical development program.”
Earlier this year, encouraging results were also reported for Cohort A, in which five patients were treated with a lower dose of the treatment, which presented similar safety and clinical benefit.
Also
known as Lou Gehrig's Disease, ALS is a rapidly progressive, fatal
neurodegenerative disease-causing dysfunction in the upper and lower
motor nerves that control muscle function. A release shared by the
company described the disease’s symptoms as including muscle weakness,
loss of motor function, paralysis, breathing problems, and eventually
death.
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