Experimental Stem Cell Treatment Shows Promise Halting ALS

Some patients with amyotrophic lateral sclerosis who received the stem cell therapy showed less progression and improved respiratory and motor functions.

Data from a recent clinical trial revealed promising results from a stem cell treatment that inhibits the progression of amyotrophic lateral sclerosis (ALS).

ALS is a neurodegenerative disease that affects the nerve cells in the brain and spinal cord. The motor neurons begin to die off, resulting in the atrophy of muscles. Patients lose their ability to walk, speak, swallow, and breathe. There is currently no known cure for the disease.

The study, which was performed at Hadassah Medical Organization (HMO) and developed by BrainStorm Cell Therapeutics (BCLI), was published recently in JAMA Neurology.

The clinical trials, which started in 2011, evaluated 26 patients administered an adult stem cell self-transplantation treatment that involved the harvesting of stem cells from the patient’s own bone marrow. Researchers used Brainstorm’s NurOwn, which they describe as “an autologous, adult stem cell therapy technology that differentiates bone marrow derived from mesenchymal stem cells (MSC) into specialized, neuron-supporting cells.”

It was then injected into the patient’s cerebrospinal fluid.

“The results are very encouraging,” lead investigator Dimitrios Karussis, MD, PhD, said in a press release. “Close to 90% of patients who were injected intrathecally through the spinal cord fluid were regarded as responders to the treatment either in terms of their respiratory function or their motor disability. Almost all of the patients injected in this way showed less progression and some even improved in their respiratory functions or their motor functions.”

In one 22-year-old ALS patient who received the treatment, the disease completely stopped progressing. There was also significant improvement in the patient’s speaking ability, and motor functions in his hands, according to ABC 7 Chicago.

“I am optimistic that within the foreseeable future, we may provide a treatment to ALS patients that can slow down or stop the progression,” Dr. Karussis said. “I believe we are in the early stages of something new and revolutionary with this harvested stem cell infusion therapy. While this is absolutely by no means a cure, it is the first step in a long process in that direction. I see this treatment as being potentially one of the major future tools to treat degenerative diseases of the brain and spinal cord, in general.”