Israeli biopharmaceutical company Kadimastem (KDST), which is developing treatment for ALS (Lou Gehrig's Diease) has announced additional results ofor the first group of patients treated with its drug in a Phase I/IIa (initial effectiveness and safety) cell replacement trial.
After six months of monitoring, no severe side effects were reported, and no toxicity limiting the dosage in treatment was found. The company previously reported that after three months of treatment, the condition of patients improved by 0.26 points on the motor function scale for ALS patients, compared with a deterioration of 0.87 points in the same patients in the three months preceding the beginning of treatment. The statistical significance of this result was very high. In the new report, deterioration in patients resumed, but after four months, was still slower than expected without treatment. After 5-6 months, the patients again deteriorated at the same pace expected without treatment.
Kadimastem said that the trial had been designed so that the first group would begin receiving a low dosage in order to test for side effects; if the low dosage proved to be safe, the next patients would receive higher dosages. Two more groups of patients are due to participate in the trial and receive two dosages higher than the one received by the first five patients. The company is now considering a change in the trial protocol to eventually include four groups. Instead of merely increasing the dosage, one of the groups will receive two injections at a 2-3-month interval.
With such a deadly disease, for which there are currently no treatment options, even a slight improvement is sometimes enough to bring a drug to market. The drug is a shelf product produced from stem cells that support the nervous system.
Kadimastem CEO Rami Epstein said, "We are satisfied with the results of Group A, which demonstrate clinically important effectiveness. We are looking forward to continuation of the trial and an assessment of the potential long-term advantages of administering a higher dosage and repeating our groundbreaking stem cell treatment."
Kadimastem founder and chief scientist Prof. Michel Revel said, "Since the clinical benefit for ALS patients treated in Group A continued significantly for 3-4 months after treatment was administered, the second stage of the trial, which includes an enlarged dosage of cells, and the third trial, which includes double treatment with two repeated injections at a three-month interval, has the potential to achieve a prolonged positive response. A good safety profile also makes it possible to test the AstroRx product for other degenerative diseases of the nervous system."
Published by Globes, Israel business news - en.globes.co.il - on January 1, 2020
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