Neuralstem’s ALS Treatment Receives FDA Orphan Drug Designation
The FDA’s Office of Orphan Products Development has granted it orphan drug designation for Neuralstem’s treatment of Amyotrophic Lateral Sclerosis (ALS) with its human spinal cord-derived neural stem cells (NSI-566RSC), currently in a Phase I safety study to evaluate the safety of the product and the surgical route of administration in a wide range of ALS patients.
Rockville, Md — Neuralstem, Inc, has updated the progress of its ongoing Phase I human clinical trial of the company’s spinal cord stem cells in the treatment of amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) at Emory University in Atlanta, Ga. The company announced that, after reviewing the safety data from the first nine patients, the trial’s Safety Monitoring Board has unanimously approved moving to the last group of ALS patients in this part of the safety trial. These next three patients, all of whom are ambulatory, will each receive 10 injections, bilaterally, in the lumbar spinal cord. After this cohort, the FDA will review the trial data to date before approving it to move into the final cohort of patients, who will receive injections in the cervical region of the spinal cord.
"We are delighted that we’ve advanced to this final cohort of patients in the first part of the safety trial," commented Eva Feldman, PhD, MD, principal investigator of the trial and a consultant to Neuralstem. Feldman is director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System. "We have been pleased with the smooth progress of the trial thus far, from non-ambulatory to ambulatory patients who have an earlier stage of the disease."
"We are greatly encouraged by the SMB’s approval to advance the trial to the final three patients to receive full lumbar injections," said Karl Johe, PhD, chief scientific officer and chairman of Neuralstem’s Board of Directors. "The trial has progressed as expected, and we are deeply indebted to the patients and families who have taken part."
The Phase I trial to evaluate the safety of Neuralstem’s spinal cord stem cells in the treatment of ALS, the first FDA-approved ALS stem cell trial, has been underway since January 2010. The trial plans to enroll up to 18 ALS patients, who will be examined at regular intervals post-surgery.
All of the first six patients treated in the trial were non-ambulatory. Of these, the first three received five injections each, unilaterally, in the lumbar region of the spinal cord. The next three patients received ten injections each, bilaterally in the lumbar region. All remaining trial patients are ambulatory, and therefore represent earlier stages of disease progression. Of the ambulatory group, the first three patients received five injections each, unilaterally, in the lumbar region. The next three, which have just been approved, will receive ten injections each, bilaterally, in the lumbar region. After the required FDA approval, the final six patients in the trial will receive injections in the cervical region.
While the trial is evaluating only the safety of the cells and procedure, it includes exploratory endpoints including attenuation of motor function loss, maintenance of respiratory capacity, and stabilization of patients along the ALS functional rating scale.
The Emory ALS Center has [removed]posted the relevant trial information for patients on its website at www.neurology.emory.edu[/removed]