The Israel Innovation Authority (IIA) has awarded BrainStorm Cell Therapeutics a $2.1 million grant to support the development of NurOwn, an innovative mesenchymal stem cell-based platform to treat neurodegenerative diseases including amyotrophic lateral sclerosis (ALS).
NurOwn was developed by Tel Aviv University professors Dani Offen and the late Eldad Melamed.
NurOwn builds on research developing mesenchymal stem cells (MSCs) as a treatment for human diseases. MSCs are multipotent precursor cells that exist in many tissues in the body. Most commonly, they are obtained from bone marrow, adipocytes (fat cells), or placenta cells.
MSCs were first used during bone marrow transplants, but researchers realized they could be used to halt the immune system in various ways. Researchers began studying MSCs as potential treatments for immune and inflammatory diseases.
NurOwn’s MSCs are also secretors of neurotrophic factors (NTF), which are involved in the growth, survival, and differentiation of neurons. These cells, called MSC-NTF, are designed to treat neurodegenerative diseases such as ALS, Parkinson’s, and multiple sclerosis, among others, because they allow NTFs to be delivered at, or close to, an injury site.
MSC-NTF cells secrete several NTFs, including brain–derived neurotrophic factor (BDNF), glial-derived neurotrophic factor (GDNF), and hepatocyte growth factor (HGF). MSC-NTF cells protect neurons from toxins and have been effective in ALS mouse models and clinical trials.
To date, over 60 patients with ALS have received experimental treatment with NurOwn in trials in the United States and Israel. These trials showed NurOwn to be clinically safe and tolerable, and potentially an effective treatment.
“The Israel Innovation Authority’s support of our NurOwn program provides further validation for the potential of this treatment to help patients suffering from amyotrophic lateral sclerosis (ALS),” Chaim Lebovits, BrainStorm’s CEO, said in a press release. “The continued financial support for our research and development is an important contribution to our ability to execute on our strategic plans, as we commence a Phase 3 pivotal trial with NurOwn.”
Last April, BrainStorm announced positive top-line Phase 2 data on NurOwn. The company said the U.S. Food and Drug Administration had accepted all key elements to continue a Phase 3 program to support a Biologic License Application (BLA) for NurOwn, a necessary step for commercializing the therapy.
The IIA has supported BrainStorm Cell Therapeutics since 2007 with several research grants. That funding has totaled approximate $7.5 million for the development of NurOwn. In exchange, the IIA will receive royalties based on product sales if NurOwn is approved and marketed.