TEL AVIV – Israel's BrainStorm Cell Therapeutics said final results from a clinical trial of its adult stem cell treatment in amyotrophic lateral sclerosis (ALS) were positive, with most patients showing a slowing in the disease's progression.
According to the ALS Association, 5,600 people in the United States are diagnosed each year with the neurodegenerative disease, which has severely disabled British physicist Stephen Hawking.
A single dose of the stem cell treatment called NurOwn was administered in a mid-stage phase 2a trial in 14 patients with ALS, also known as Lou Gehrig's Disease, at Hadassah Medical Center in Jerusalem.
"Nearly all subjects in this study experienced clinical benefit from treatment with NurOwn," the company said on Monday.
Of the 12 patients with three or more months of follow-up, 92 percent experienced an improvement in disease progression. NurOwn slowed the progression of ALS using two different parameters and had a strong effect on the rate of decline in lung function, BrainStorm said.
Shares in the company, which is also developing stem cell treatments for multiple sclerosis and Parkinson's disease, have nearly doubled in the past two sessions on Nasdaq in anticipation of the trial's results. The stock has nearly tripled in the past year, closing at $7.50 on Friday.
In October the U.S. Food and Drug Administration designated NurOwn as a "fast-track" product to treat ALS.
"We observed not just a highly meaningful slowing of ALS progression on two different parameters ... but subjects with prolonged stabilization and even improvements in function, and this was achieved with just a single dose of NurOwn," said BrainStorm Chief Executive Tony Fiorino.
BrainStorm, which is also conducting clinical trials at three sites in the United States, plans to move to a study in the next few months to see if the results can be amplified with repeated doses.