Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease and Lou Gehrig’s disease, is a rare condition characterized by the degeneration of nerve cells that control voluntary muscles. The disease leads to gradually worsening symptoms that include muscle weakness, twitching, and stiffness. As more of these nerve cells, or motor neurons, are lost, muscles decrease in size and continue to weaken, eventually resulting in problems with speaking, swallowing, and breathing.

ALS currently has no cure and few effective treatments exist, but scientists are working on new ways of treating the disease. 

What is stem cell therapy and it can help ALS patients

Stem cell therapy is emerging as a potential new approach to treating ALS. Researchers use many types of stem cells in their work, including induced pluripotent stem cells (iPSCs), which may be among the most promising of cells with a potential for treatment. iPSCs are derived from adult human tissues, including those of patients, and have shown an ability to develop into astrocytes, cells that support nerve cells.

Mesenchymal stem cells, or adult stem cells, are also under investigation. Here, cells taken from a patient’s own bone marrow or fat tissue are then modified to boost their ability to produce neurotrophic factors. Neurotrophic factors are molecules known to help support and protect nerve cells.

Other forms of pluripotent stem cells, which can be coaxed into specialized cells, are also of interest in ALS research.

Stem cell therapy in clinical trials for ALS

BrainStorm Cell Therapeutics is developing NurOwn, an approach that uses mesenchymal stem cells that develop into cells that secrete neurotrophic factors. The cells can be injected into the muscle or the spinal canal.

A Phase 1/2 open-label clinical trial (NCT01051882) evaluated the safety, tolerability, and therapeutic effects of NurOwn in ALS patients with early disease. The cells were directly injected into the muscle tissue of 12 patients.

Another, Phase 2a prospective study (NCT01777646) enrolled 14 patients to check the effects of combined multiple injections of NurOwn into the fluid surrounding the brain and spinal cord. (This process is called intrathecal administration.)

Results of the trials, reported in 2016 in the journalJAMA Neurology, demonstrated that the treatment was safe and well-tolerated, and slowed the rate of disease progression during six months after the injections as compared to six months before treatment.

The safety of intrathecal treatment with mesenchymal stromal cells derived from fat tissue was also investigated and appeared to be safe at the tested doses.

Another Phase 3 clinical trial (NCT03280056) assessing the safety and efficacy of repeated NurOwn injections is currently recruiting patients in US California, Massachusetts and Minnesota. ALS patients will receive three intrathecal NurOwn or placebo injections at bi-monthly intervals.

A Phase 1/2 clinical trial (NCT03482050), investigating the use of astrocytes derived from human embryonic stem cells (called AstroRx), is currently recruiting about 21 patients at a single site in Israel. AstroRx will be injected into the spinal cord of ALS patients with early stage disease.