BrainStorm Cell Therapeutics Will Conduct Phase 2 Clinical Trial on ALS Patients with Their NurOwn® Cells


The biotechnology company BrainStorm Cell Therapeutics Inc. has developed an autologous stem cell therapy for several neurodegenerative diseases including Amyotrophic Lateral Sclerosis (ALS, also known as Lou Gehrig’s disease), Multiple Sclerosis (MS) and Parkinson’s Disease (PD). BrainStorm has designed a proprietary product called NurOwn™ that is made from the patient’s own bone marrow mesenchymal stem cells (BM-MSCs). Essentially, the patient’s BM-MSCs are isolated, purified, and cultured in a specialized culture system that drives the BM-MSCs to differentiate into nerve-like cells that Neurotrophic Factors (NTF). These NTFs have the capacity to keep nerve cells alive and prevent moribund cells from dying.

Figure taken from http://www.brainstorm-cell.com/index.php/science-technology/nurown%E2%84%A2
Figure taken from http://www.brainstorm-cell.com/index.php/science-technology/nurown%E2%84%A2. 

By transplanting NurOwn cells back into the patient at or near the site of neural damage, in the spine and/or muscles, it could potentially delay or even roll back damage from neurodegeneration. NurOwn cells have proven their efficacy in animal experiments (e.g., STEM CELLS 2008;26:2542–2551), and in a few small clinical trials.

In one case, a 75-year-old man who suffered from ALS and myasthenia gravis (the immune system attacks your own receptors for acetylcholine at the neuromuscular junction, which prevents the muscle contraction), was treated with NurOwn cells, and experienced the following improvement 1 month later.

Figure copying (a test of visuospatial function) of the patient, before and 1 month after the first enhanced (neurotrophic factors producing) mesenchymal stem cell (MSC‐NTF) transplantation.
Figure copying (a test of visuospatial function) of the patient, before and 1 month after the first enhanced (neurotrophic factors producing) mesenchymal stem cell (MSC‐NTF) transplantation.

This is only a case study and involves only one patient, which is the absolute lowest-quality evidence you can have in medicine.  Therefore, this study is suggestive that NurOwn cells can help ALS patients improve.

Now BrainStorm Cell Therapeutics has entered into a collaborative agreement with Hadassah Medical Center in Jerusalem, Israel, to conduct a Phase 2 clinical trial to test the ability of NurOwn cells to treatment patients with Amyotrophic Lateral Sclerosis (ALS).

This clinical trial is not BrainStorm’s first rodeo, since they have conducted two other clinical trials in collaboration with Hadassah Medical Center. BrainStorm hopes that the results of this clinical trial will provide guidance in preparing a Phase 3 clinical trial that will test their NurOwn® stem cell based therapy in patients suffering from ALS.

In this trial, BrainStorm plans to enroll up to 24 ALS patients, all of whom will receive three consecutive stem cell transplantations of their own BM-MSCs that have been genetically engineered to secrete NFTs. The goal of this trial is to establish the safety and efficacy of a treatment regimen that includes multiple doses of stem cells. Because this trial includes human subjects, it must be approved by Hadassah’s Helsinki Committee and the Israeli Ministry of Health before the study can commence.

Professor Dimitrios Karussis, MD, PhD, Head of the Unit of Neuroimmunology and Cell Therapies at Hadassah’s Department of Neurology, who served as Principal Investigator in Brainstorm’s prior ALS studies, will serve as the Principal Investigator for this trial.

“NurOwn has generated promising clinical data in ALS and has the potential to offer a new approach for the treatment of patients afflicted with this disease,” stated Professor Karussis. “We are excited to be collaborating with BrainStorm to advance this product to the next phase of development and the application of stem cell therapies in similar neurological diseases in general.”

“Evaluating multiple doses with NurOwn is an important next step in our efforts to understand the treatment effect of this investigative medicine,” stated Chaim Lebovits, CEO of Brainstorm. “We are pleased to continue our partnership with Hadassah Medical Center, which has long maintained a reputation for excellence in the treatment of neurological disorders.”

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mburatov

Professor of Biochemistry at Spring Arbor University (SAU) in Spring Arbor, MI. Have been at SAU since 1999. Author of The Stem Cell Epistles. Before that I was a postdoctoral research fellow at the University of Pennsylvania in Philadelphia, PA (1997-1999), and Sussex University, Falmer, UK (1994-1997). I studied Cell and Developmental Biology at UC Irvine (PhD 1994), and Microbiology at UC Davis (MA 1986, BS 1984).