Neuralstem has obtained FDA approval for a Phase II stem cell trial to treat amytrophic lateral sclerosis (ALS or Lou Gehrig’s disease).
The trial, which will use NSI-566 spinal cord-derived human neural stem cells, will expand to two centres Emory University Hospital in Atlanta and the University of Michigan Health System, in Ann Arbor, subject to approval by the Institutional Review Board at each institution.
Neuralstem chairman and chief scientific officer Karl Johe said Phase II trial aims to obtain the maximum tolerated dose using the same route of administration as in Phase I that is through direct injections into the gray matter of the spinal cord.
"As a result of the excellent safety and tolerability demonstrated in Phase I, we will be able to proceed more aggressively in Phase II," Johe said.
"In Phase I, we started with just five injection sites per patient, and advanced to a maximum of 15 injections of 100,000 cells each. In Phase II, we will advance up to a maximum of 40 injections, and 400,000 cells per injection based on safety."
Around 15 patients will be treated in five different dosing cohorts of the Phase II dose escalation and safety trial.
The first 12 patients will be administered with injections in the cervical region of the spinal cord only, where the stem cells could help preserve breathing function.
Last three patients will receive both cervical and lumbar injections.
The Emory ALS Center director Jonathan Glass said: "We are looking forward to progressing the dosage to the maximum safe tolerated dose, where we could also hope to see meaningful effects for the patients."
Image: MRI (parasagittal FLAIR) demonstrates increased T2 signal within the posterior part of the internal capsule and can be tracked to the subcortical white matter of the motor cortex, outlining the corticospinal tract), consistent with the clinical diagnosis of ALS. Photo: courtesy of Frank Gaillard.