Results of NurOwn(TM) Clinical Trial Suggest Efficacy in ALS Patients
Data Indicate Initial Clinical Benefit in Overall Clinical and Respiratory Function
BrainStorm Cell Therapeutics ( OTCQB : BCLI ), a leading developer of adult stem cell technologies for neurodegenerative diseases, today reported some of the final results from a clinical study evaluating the company's NurOwn™ technology in 12 ALS patients. NurOwn is a proprietary, first-of-its-kind technology for the propagation and differentiation of autologous Mesenchymal Stem Cells (MSCs) into NeuroTrophic Factor (NTF)-secreting cells. The data were presented yesterday, Wednesday, March 20, 2013 during the 65th Annual Meeting of the American Academy of Neurology (AAN) in San Diego, California.An oral and poster presentation were made in the Emerging Science Session by Principal Investigator Dimitrios Karussis, M.D., Ph.D., entitled, "Analysis of 12 Patients with Amyotrophic Lateral Sclerosis (ALS) Treated with Autologous Differentiated Mesenchymal Stem Cells: a Phase I/II Clinical Trial." Karussis reported a significantly slower decline in overall clinical and respiratory function, as measured by the ALS Functional Rating Score (ALSFRS-R) and Forced Vital Capacity (FVC) score respectively, in the six patients that received an intrathecal (IT) injection of the cells in the six months following treatment, as compared to the three months preceding treatment. The study concluded that in addition to establishing the safety of the treatment protocol, initial indications of clinical benefit were observed, which require further confirmation in additional trials. The company is currently conducting a Phase IIa dose-escalating trial pursuant to recent acceleration by the Israeli Ministry of Health.
"These encouraging results confirm the importance and therapeutic potential of NurOwn as a breakthrough treatment for patients with ALS," said Prof. Karussis, Head of the Neuroimmunology Laboratory, Department of Neurology, Hadassah Medical Center, Jerusalem. "Additionally, beyond its benefit in treating patients with ALS, NurOwn may have utility in the treatment of other severe neurodegenerative and neuroimmunological conditions including multiple sclerosis and Parkinson's disease."
"We are excited by these data and the potential of NurOwn to positively impact the lives of patients with ALS," said Alon Natanson, Chief Executive Officer of BrainStorm. "We look forward to continuing to advance this potentially important therapy. To that end, we have begun a Phase IIa dose-escalating trial at Hadassah and plan to launch a multi-center Phase II trial in the USA later this year in order to further validate the results that were presented today."
About NurOwn™NurOwn is an autologous, adult stem cell therapy technology that differentiates bone marrow-derived mesenchymal stem cells (MSC) into specialized, neuron-supporting cells. These neuron-supporting cells (known as "MSC-NTF" cells) secrete neurotrophic, or nerve-growth, factors for PROTECTION of existing motor neurons, PROMOTION of motor neuron growth, and RE-ESTABLISHMENT of nerve-muscle interaction. The ability to differentiate mesenchymal stem cells into MSC-NTF cells, and confirmation of their activity and potency before transplantation, makes NurOwn a first-of-its-kind approach for treating neurodegenerative diseases. More information about NurOwn™ can be found at http://brainstorm-cell.com/index.php/science-a-technology/-nurown.
About ALSOne of the most common neuromuscular diseases worldwide, Amyotrophic lateral sclerosis (ALS), sometimes called Lou Gehrig's disease, is a rapidly progressive, invariably fatal neurological disease that attacks the nerve cells responsible for controlling voluntary muscles. As many as 30,000 people in the United States have ALS and an estimated 5,000 Americans are newly diagnosed each year. The disease belongs to a group of disorders known as motor neuron diseases, which are characterized by the gradual degeneration and death of motor neurons. There is currently no cure available for ALS.
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 athttp://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.
IntroductionInvestor Presentation – March 2013
BrainStorm Cell Therapeutics (OTC.QB: BCLI) is a biotechnology company developing innovative, autologous stem cell therapies for highly debilitating neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS, also known as Lou Gehrig's disease), Multiple Sclerosis (MS) and Parkinson’s Disease (PD).
NurOwn™, our proprietary, first-of-its-kind technology for the propagation and differentiation of autologous Mesenchymal Stem Cells (MSCs) into NeuroTrophic Factor (NTF)-secreting cells and their transplantation at or near the site of damage, offers the hope of conquering neurodegenerative diseases.
We are 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 will be followed for three to six months after transplantation.
The company also plans to begin a Phase II clinical trial in the USA in 2013, pending FDA approval.
In February 2011, NurOwn was granted Orphan Drug designation by the FDA.
Over 20 publications in leading scientific journals demonstrating NurOwn's mechanism of action, pharmacology and in vivo efficacy have been published by the scientific team.
MissionBrainStorm is developing autologous, adult stem cell therapies to treat a variety of debilitating neurodegenerative diseases, such as Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig's Disease), Parkinson's Disease (PD), and Multiple Sclerosis (MS).
Our approach uses the patient's own bone marrow to generate specialized NeuroTrophic Factor (NTF)-secreting cells, which are then transplanted into the spine or muscles, to protect existing motor neurons, promote new motor neuron growth, and re-establish nerve-muscle interaction.
We are committed to developing an effective treatment that will overcome the devastating effects of neurodegenerative disease.
Stem Cell Therapy
Stem Cell therapies are one of the most promising areas of medicine today.Management Team
The aim of cell therapy is to support and repair damaged tissues and organs by providing healthy stem cell transplants. Bone marrow stem cell transplants for replacement and restoration of the haemopoietic system (blood and lymph) of cancer patients have been successfully used for the past 20 years.
Stem cells are generally described as cells that are capable of both self-renewal and differentiation. Stem cells have the ability to undergo asymmetric division such that one of the two daughter cells retains the properties of the stem cell, while the other begins to differentiate into a more specialized cell type. Stem cells are therefore central to normal human growth and development and a potential source of new cells for the regeneration of diseased and damaged tissue.
BrainStorm is developing cellular therapeutics based on stem cell technologies. The medical benefits afforded by stem cell therapies offer hope to millions of patients suffering from many types of neuro-degenerative diseases such as Amyotrophic Lateral Sclerosis (ALS, often referred to as Lou Gehrig's Disease), Parkinson’s disease (PD), and Multiple Sclerosis (MS).
Alon Natanson, MBA
Adrian Harel, Ph.D.
Director of R&D
Liat Sossover, CPA, MBA
Prof. Eldad Melamed, M.D.
Chief Medical Advisor
Prof. Daniel Offen, Ph.D.
Chief Scientific Advisor
Our PartnersAt BrainStorm we’re interested in exploring strategic partnerships that will further our clinical and technological goals. These academic and clinical collaborations with leading scientific and medical investigators, as well as technological alliances with biotechnology and pharmaceutical companies.
We're proud to be partnering today with a leading university medical center and an innovative technology company in the areas of clinical and technology development:
James S. Painter III
Emerging Markets Consulting LLC
10724 High Crest Court
Howey In The Hills Florida 34737
Thursday, March 21, 2013
Posted by Ryan K at 12:12 PM