Stem Cells in ALS Clinical Trial

In a rare move, the Food and Drug Administration has awarded “fast-track” status to a stem cell therapy for amyotrophic lateral sclerosis (ALS). According to Rhys Baker, writing for BioNews, the treatment uses modified stem calls taken from patients’ bone marrow. The treatment is being developed by an Israeli biotechnology company called BrainStorm Cell Therapeutics Ltd. and is currently in a mid-stage clinical trial.

Tony Fiorrino, M.D., chief executive of BrainStorm, told Reuters, “the receipt of fast-track designation from the FDA is an acknowledgement of the unmet medical need in ALS. The Motor Neurone Disease Association in the UK estimates that seven out of every 100, 000 people in the general population are living with the disease.”

As Baker explained, ALS is a degenerative condition in which neurons in the spinal cord and brain die. Patients in later stages of the disease may become totally paralyzed. Among victims of ALS is British physicist Professor Stephen Hawking.

The technique developed by BrainStorm, called NurOwn, involves taking stem cells from patients’ bone marrow and treating the cells so they will secrete neurotrophic factors. These factors are defined as proteins that are essential to the growth and survival of nerve cells. Doctors then inject the cells back into the patients in areas of their body which have experienced the most nerve damage.

Baker quoted Professor Karussis Dimitrios, of the Hadassah Medical Centre in Jerusalem, who led the trial, as saying: “I have now treated over 30 ALS patients with NurOwn in two clinical trials, as well as in several compassionate use treatments, and I am enthusiastic about its emerging safety and efficacy profile.” Reuters reports that NurOwn is also being studied in phase 2 clinical trials at three U.S. hospitals.