Neuralstem updated the progress of its ongoing phase I human clinical trial of the company’s spinal cord stem cells in the treatment of ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease) at Emory University in Atlanta, Georgia. The company announced that, after reviewing the safety data from the first twelve patients, all of whom received injections in the lumbar region of the spine only, the trial’s Safety Monitoring Board has unanimously approved advancing the trial to transplantions in cervical region.
The next three patients, all of whom are ambulatory, will each receive five injections, unilaterally, in the cervical spinal cord. In addition to the Safety Monitoring Board approval, the FDA must also approve the trial’s moving to the next level of transplantations. The company expects to present the data from the first twelve patients to the FDA in the near future.
“We are delighted to have completed the first part of the safety trial,” commented Eva Feldman, principal investigator of the trial and an unpaid 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 particularly excited to begin the cervical injections, once we have received FDA approval. While this is still primarily a safety trial, it is our hope that cervical injections can help the patient’s breathing and swallowing, the areas which most often lead to loss of life in ALS.”
“We are greatly encouraged by the Safety Monitoring Board’s approval to advance the trial to the next level,” said Karl Johe, chief scientific officer and chairman of Neuralstem’s board of directors. “We believe that cervical injections are critical to actually being able to improve the quality of life—and ultimately extend the life—of ALS patients. The trial has progressed as expected, and we are deeply indebted to the patients and families who have taken part.”
About the trial
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 enrol 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 last three received 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 stabilisation of patients along the ALS functional rating scale.
The Emory ALS Center has posted the relevant trial information for patients on its website at http://www.neurology.emory.edu/ALS/Stem%20Cell.html