Early clinical trial for ALS looks promising, points to ways to do even better

ALS patient and world famous physicist Stephen Hawking talked with CIRM's ALS team at Cedars-Sinai last year

The stem cell company Neuralstem had earlier reported promising data from part of the patients enrolled in their phase 1 clinical trial for patients with amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease. Now they have published data for all the patients in the study including the last six who were the first to receive the stem cell injections in the neck instead of the back. More important, three of those last patients received the injections in the full length of their spine, both the neck and back.

The number of patients is small and this is very early data, but the results seem to confirm what seems intuitively obvious: delivering more cells over more area of the spine increases the beneficial effect of the stem cells. Like all phase 1 studies, the primary goal of this trial was to test the safety of the cells and the procedure for injecting them. Both appeared to be uniformly safe. But the team did build measures into the trial that could begin to detect benefit.

The team, split between the University of Michigan and Emory in Atlanta, carefully measured the rate of disease progression prior to the surgery and plotted out an expected rate of decline. They measured several indicators such as grip strength six, nine, 12 and 15 months after the surgery. In half the patients they detected improvement in some of those measures. In the three patients that had stem cells injected in their neck and spine, they detected improvements in several of the measures.

The San Francisco Business Times picked up the company’s press release that quoted the lead researcher from the Emory arm of the study Jonathan Glass on the importance of this first phase:

"We have now shown that the procedure is safe for both lumbar and cervical injections, allowing us to move forward with an aggressive program to test whether this treatment will improve the course of disease for patients with ALS. They have already begun a phase two trial that includes the first two sites plus Massachusetts General Hospital."

The release also suggests that while the main benefit of the stem cells may be a protective effect preventing further disease damage to the motor nerves, the results showed some level of cell replacement and improvement of the environment in which the nerves are living. These measures will need confirmation in a larger trial.The current work was published in the Annals of Neurology this week.

However, CIRM is funding an effort aimed at upping the ante on the protective benefit. A team at Cedars-Sinai in Los Angeles plans on conducting a trial with stem cells that have been genetically modified to secrete a hormone that has been shown to be protective. They will be using the same surgical equipment and protocol as the current teams, so that aspect of the work should pass the safety test. You can read about that and related CIRM projects on our stem cells for ALS fact sheet.

Don Gibbons