Trial of ALS Stem Cell Treatment Reports First Results

(National Institute on Aging, NIH)

Researchers from the biotechnology company Neuralstem in Rockville, Maryland and three universities report that an early clinical trial of transplanted spinal cord stem cells to treat amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) shows the 12 patients were able to tolerate the treatment without long-term complications. The team reports its findings online in the journal Stem Cells (paid subscription required).

ALS is a debilitating disease characterized by the progressive degeneration of motor neurons, resulting in paralysis of a patient’s limbs and organs. The clinical trial involves transplanting neural stem cells into the spinal cords of 12 ALS patients, who received five injections — or in some cases pairs of injections — into the lumbar spinal cord at a dose of 100,000 cells per injection.

The trial, conducted at Emory University in Atlanta, is designed to test the safety of the procedure. The findings show all of the 12 ALS patients tolerated the treatment without any long-term complications from either the surgical procedure or the implantation of stem cells. “For these first 12 patients,” says the study’s lead author and director of the Emory ALS Center Jonathan Glass, “we have met the objective of the phase 1 trial, demonstrating safety for both the procedure of intraspinal injection and the presence of the neural stem cells in the spinal cords of ALS patients.”

The authors also report that based on assessments ranging from 6 to 18 months after transplantation, the patients demonstrated no evidence of acceleration of the disease progression due to the intervention. One patient has as well shown improvement in his clinical status. The authors caution that these results must be interpreted with caution since the trial was not designed or conducted to measure treatment effectiveness.

In October 2011, FDA approved advancing the trial to allow for transplants into a second area, the cervical region of the spinal cord. Based on these early results, the researchers have started to inject the next group of patients in the cervical region, with the goal of protecting motor neuron pools affecting respiratory functions.

“We have already transplanted two patients in the cervical spinal cord, where we believe we can affect patients’ lives the most by improving their breathing,” says Neuralstem’s chairman Karl Johe. “We are in active discussions with the FDA to increase the number of cells and the number of injections as well.”

Read more: FDA Approves Advance of ALS Stem Cell Trial

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