The FDA has granted approval after reviewing safety data from the 1st 12 patients for the trial to advance to transplanting patients in the cervical (upper back) region. Until now, patients have received injections in the lumbar (lower back) region only.
- The trial will now advance to the final 2 cohorts of patients with ALS, all of whom will be transplanted in the cervical region of the spine.
The Bottom Line: The goal of this cell therapy program is to create therapies that will slow down, stabilize, or reverse, functional deficits in central nervous system (NYSE:CNS) diseases. Prolongation of life for patients with ALS will require therapeutic intervention at the level of the cervical spinal motor neurons affecting respiratory function. By moving the cell delivery to cervical spinal cord—the 1st time the FDA has approved intra-spinal injections in this region; CUR hopes to demonstrate delivery of cells safely and routinely to all parts of the spinal cord. CUR has successfully treated 12 ALS patients with unilateral or bilateral intra-spinal injections of neural stem cells. All of the patients tolerated the procedure without major surgical complications, and there are no indications to date that the stem cells themselves are either toxic or injurious to the spinal cord.