January 6, 2014
Significant Clinical Findings in ALS Patient Treated with BrainStorm's NurOwn Featured in “Muscle and Nerve”
NEW YORK & PETACH TIKVAH, (Israel) – January 6, 2014 – BrainStorm Cell Therapeutics Inc. (OTC.QB: BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, today announced the upcoming publication of a case report on an individual patient treated with the company’s NurOwn™ cell therapy in "Muscle & Nerve" (John Wiley & Sons). The report, which looked at one patient diagnosed with both Myasthenia Gravis (MG) and Amyotrophic Lateral Sclerosis (ALS) showed significant improvements in both cognitive and motor function following initial and repeat treatments with NurOwn.
The paper, entitled "Rare Combination of Myasthenia and ALS, Responsive to MSC-NTF Stem Cell Therapy" by Professors D. Karussis, Z. Argov, O. Abramsky and their colleagues at Hadassah University Medical Center in Jerusalem, as well as Professor V. A. Lennon from Mayo Clinic in Rochester MN, describes the rare case of a 75-year old man diagnosed with MG and ALS who was treated with NurOwn cells on a compassionate basis. One month after receiving both intrathecal and intramuscular injections of the cells, the patient demonstrated significant improvement in cognition, speech, and muscle power. Initial therapy led to improvement in the ALS Functional Rating Scale (ALSFRS-R), respiratory forced vital capacity (FVC) and cognitive function. Following therapy, the patient’s ability to speak improved from dysarthria to being able to deliver a speech clearly to an audience.
At 6 months post-transplantation, due to progression of weakness and deterioration in cognition, a repeat injection of Brainstorm's NurOwn, was performed. Again, improvements in ALSFRS-R and respiratory forced vital capacity (FVC) were observed following second treatment. Treatment with NurOwn appeared safe and well tolerated; the patient did not experience any serious adverse events after either course of treatment.
The results suggest that repeated therapy may maintain and enhance the clinical benefits of NurOwn cell therapy in neurological diseases. The authors stated that “the outcome of the second transplantation was even more remarkable.” They noted that laboratory and clinical improvements of the magnitude observed are “highly unusual in the natural course of ALS.” They speculated that "repeated transplantation might be needed to maintain and enhance the clinical benefits of stem cell therapies," and that "the improvement in many functional parameters suggest that the motor nerve function may have been more significantly affected by the treatment than MG."
Chaim Lebovits, BrainStorm's President, commented on the results, “This report is very promising and this unique case provides insights into the possible effects of repeat treatments of MSC-NTF cell transplantation on ALS. This publication is an important milestone for BrainStorm as this is the first human data on NurOwn to be published in a highly respected scientific journal. While our PI already reported positive and promising saftey and efficacy data from our Phase I/II clinical trial, we look forward to finalizing our ongoing Phase IIa clinical trial data and thus anounce the combined data. Together, these data will include a total of 26 patients with ALS.”
A preview of the paper is available at this link.
NurOwn™ is a proprietary, first-of-its-kind technology that induces autologous bone marrow-derived Mesenchymal Stem Cells (MSCs) to secrete Neurotropic Factors (NTF). The NurOwn treatment approach is to transplant these “MSC-NTF” cells back into the patient at or near the site of damage, in the spine and/or muscles.
Brainstorm Cell Therapeutics is currently conducting a Phase IIa ALS clinical trial with NurOwn at the Hadassah University Medical Center in Jerusalem. In this safety and preliminary efficacy trial, 12 patients will receive combined intramuscular and intrathecal administration of NurOwn cells in three cohorts with increasing doses. The study participants are being followed for six months after transplantation.
The Company also plans to begin a Phase II clinical trial in the US in early 2014, pending FDA approval.
NurOwn was granted Orphan Drug designation by the US FDA and the European Commission (EC). Over 20 publications in leading scientific journals demonstrating NurOwn's mechanism of action, pharmacology and in vivo efficacy have been published by the Company’s scientific team.
About BrainStorm Cell Therapeutics, Inc.
BrainStorm Cell Therapeutics Inc. is a biotechnology company engaged in the development of first-of-its-kind adult stem cell therapies derived from autologous bone marrow cells for the treatment of neurodegenerative diseases. The Company holds the rights to develop and commercialize its NurOwn technology through an exclusive, worldwide licensing agreement with Ramot, the technology transfer company of Tel Aviv University. For more information, visit the company’s website at www.brainstorm-cell.com.
Safe Harbor Statement
Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as “may”, “should”, “would”, “could”, “will”, “expect”, “likely”, “believe”, “plan”, “estimate”, “predict”, “potential”, and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm’s forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management’s beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.
BrainStorm Cell Therapeutics Inc. (OTC.QB: BCLI)
Mr. Chaim Lebovits, President