The California Institute of Regenerative Medicine (CIRM) has given a $16 million grant to Brainstorm Cell Therapeutics, Inc., a stem cell developer in Israel. The grant will support a phase III trial for amyotrophic lateral sclerosis (ALS).
ALS: An Overview
ALS is a progressive disorder of painless muscle weakness. It is also known as Lou Gehrig’s disease. The disorder is of gradual onset and manifests in different people with different symptoms, such as muscle cramps, tripping, slurring of speech, dropping objects from the hands, uncontrolled crying/laughter, and/or abnormal fatigue. In advanced stages of ALS, the breathing muscles are affected and patients require ventilatory support. The disease attacks motor neurons and sensory functions, such as hearing, taste, smell, sight, and touch are unaffected. Many people are able to retain bladder muscle control.
ALS is diagnosed with electromyography and nerve conduction studies as well as blood tests and nerve/muscle biopsies. As yet, there is no cure for ALS, which is a devastating disease. The average lifespan following diagnosis is less than five years and there is quick and severe deterioration in the quality of life of people afflicted by the condition.
NurOwn: A New Treatment for ALS with Stem Cells
The CIRM grant will help Brainstorm develop and test NurOwn, a novel treatment for ALS that utilizes stem cells. Chaim Lebovits, the CEO and President of the company, explains that the grant is a shot in the arm to ALS research. Brainstorm plans to use the substantial award to further develop technology and new treatments for people afflicted with ALS.
NurOwn is a platform for cell therapy which utilizes mesenchymal stem cells (MSCs) to create customized therapies for neurodegenerative diseases. Stem cells are grown under proprietary conditions to convert them into biological factories producing a number of NTFs (neurotrophic factors) that support neuron growth and survival.
Phase III Clinical Trial for ALS
The Brainstorm trial is in the advanced stages of planning. This phase III study will investigate the efficacy of NurOwn in treating ALS. Approximately 200 patients will be enrolled in the study which will be conducted at six institutions in the United States. The ALSFR-S score will be used to assess outcome. Patients who demonstrated good results and fast progress in phase II of the study will be preferentially selected for phase III.