BrainStorm Cell Therapeutics Inc., a leading developer of adult stem cell technologies and therapeutics, announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to the company’s NurOwn autologous adult stem cell product candidate for the treatment of amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease.

“We are extremely pleased to have been granted the benefits of orphan drug designation as we continue to advance NurOwn as a potential new treatment for ALS, for which we expect to begin human clinical trials during the first half of 2011,” said Chaim Lebovits, president of BrainStorm. “Because of the safety profile with autologous adult stem cells, potential for intramuscular or intrathecal administration, and multiple mechanisms of action, we feel this product candidate could help effectively address the needs of patients with ALS and other neurological disorders.”

The United States Orphan Drug Act of 1983 was created to promote the development of new drug therapies for the treatment of diseases that affect fewer than 200,000 individuals in the United States. Orphan status entitles BrainStorm to seven years of marketing exclusivity for NurOwn upon regulatory approval, as well as the opportunity to apply for grant funding from the U.S. government to defray costs of clinical trial expenses, tax credits for clinical research expenses and potential exemption from the FDA's application user fee.

Amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. According to the ALS Association, approximately 5,600 people in the US are diagnosed with ALS each year and it is estimated that as many as 30,000 Americans may have the disease at any given time. The financial cost to families of patients is exceedingly high, and it is estimated that in the advanced stage, care can cost an average of $200,000 per year, which represents more than a $6 billion cost to the healthcare system.

BrainStorm’s core technology, NurOwn, is based on the scientific achievements of Professor Eldad Melamed, former Head of Neurology, Rabin Medical Center, and Tel-Aviv University, and a member of the Scientific Committee of the Michael J. Fox Foundation for Parkinson's Research, and Professor Daniel Offen, Head of the Neuroscience Laboratory, Felsenstein Medical Research Center (FMRC) at the Tel-Aviv University.

The NurOwn technology processes adult human mesenchymal stem cells that are present in bone marrow and are capable of self-renewal as well as differentiation into many other cell types. The research team is among the first to have successfully achieved the in vitro differentiation of adult bone marrow cells (animal and human) into astrocyte-like cells capable of releasing neurotrophic factors, including glial-derived neurotrophic factor (GDNF). The ability to induce differentiation into astrocyte-like cells along with intramuscular or intrathecal (or other) delivery makes NurOwn technology highly attractive for treating ALS and Parkinson’s disease as well as MS and spinal cord injury.

BrainStorm’s stem cell therapy contains human mesenchymal stromal cells induced to differentiate into astrocyte-like cells secreting neurotrophic factors by means of a specific differentiation-inducing culture medium.

BrainStorm Cell Therapeutics Inc. is an emerging company developing adult stem cell therapeutic products, derived from autologous (self) bone marrow cells, for the treatment of neurodegenerative diseases. The company holds rights to develop and commercialize the technology through an exclusive, worldwide licensing agreement with Ramot at Tel Aviv University Ltd., the technology transfer company of Tel-Aviv University.