BrainStorm Cell Therapeutics Inc (BCLI)

[sokol]

Dr. Jeremy Shefner, senior vice president of Barrow Neurological Institute, believes that while it may be frustrating for patients waiting for an ALS drug to be approved, the FDA process is not for nothing.

Dr. Shefner says the trouble is that if you have a disease, which on the average, leads to death in somewhere between three and five years, a drug can’t be looked at for only a couple of months.“You need a significant amount of time,” he said. “Another issue is that ALS patients are relatively rare so you need to recruit in the hundreds of patients to definitively determine if the drug works or not.

Since there are no BIOMARKERS with ALS, the only endpoint or measure is whether the patient administered the real drug is getting worse or not. We have anecdotal evidence that several patients have improved; however, the FDA will be looking at how the patients receiving the drug compared to patients receiving the placebo. Additionally, according to Dr. Shefner, the FDA will need to be convinced that patients receiving the drug did better for a "significant amount of time."

https://yourvalley.net/stories/facing-devastating-diagnosis-als-community-remains-dedicated-to-finding-new-treatments,163938

So, it may be that Brainstorm (for some unknown reason) believes that NurOwn patients in the phase 3 trial performed better for a significant period of time than those administered the placebo, which is why it has gone to the trouble and some expense to get production facilities in place. However, what will the data demonstrate to the FDA? What does the data actually show regarding NurOwn patients? No one knows which trial participants received NurOwn versus the placebo. I suppose that if we received reports that a number of patients in the phase 3 trial did not get worse or improved, than we may speculate that those patients received NurOwn, but we do not know that.