Encouraging results from a clinical study were reported recently in Science & Enterprise describing a treatment for ALS using stem cells extracted from a patient’s bone marrow.
The clinical trial was conducted by New York-based Brainstorm Cell Therapeutics. The results, which appear in Neurology, confirmed that the treatment promoted improvement in patients and that it showed significant evidence of safety.
About Amyotrophic Lateral Sclerosis (ALS)
ALS is a motor neuron disease of the nerves. As the disease progresses, the nerves eventually cease to function, causing a weakening of muscles including those muscles that control breathing.
About NurOwn Technology by BrainStorm
The procedure introduced by NurOwn technology converts the stem cells into cells that stimulate nerve cell development.
The newly transformed cells release neurotrophic factors (proteins) that are protective of nerve cells. These proteins also promote their growth and interaction with muscles.
The transformed cells have another advantage in that they originate from the patient (autologous). This means that they carry a minimal risk that the patient’s immune system will reject the cells.
About the Clinical Trial
Forty-eight ALS patients were assigned randomly to either receive a placebo or the NurOwn stem cell treatment. The trial took place at three locations within the U.S. with tolerability and safety as dual objectives.
Prior to the treatments, various measures were recorded such as efficacy and the decline of certain communication and muscle functions. The team also conducted an analysis of cerebrospinal fluid and inflammatory biomarkers. The preparation period took three months.
Twelve of the forty-eight ALS patients received a placebo while thirty-six patients received the NurOwn stem cell therapy.
During the trial, severe adverse events were experienced by nine participants plus an additional two patients from the placebo group. The team concluded that these reactions were a result of ALS progression and not the treatments. It was the opinion of the researchers that NurOwn treatments were safe and could be administered to ALS patients.
Results of the Study
In the final analysis, the researchers found that NurOwn patients exhibited a 1.5 improvement each month over the placebo group. The team also rated communication and muscle function.
They used the same rating for ALS participants who were experiencing a rapid progression of the disease. Upon examination post-treatment, the researchers were able to determine that improvements with these patients were also fairly significant.
Samples of cerebrospinal fluid were analyzed two weeks after treatment. The patients who received NurOwn stem cells had fewer inflammatory biomarkers and more proteins that supported the growth of neurons than patients in the placebo group.
The results were satisfactory and a Phase III trial involving a larger number of ALS participants was recommended.
Brainstorm’s CEO recently announced that their Phase III team has already recruited two hundred ALS patients. The new trial will be underway in the very near future.
