BrainStorm Cell Therapeutics, an Israeli company formed around technology developed at Tel Aviv University, has entered into an agreement with Massachusetts General Hospital (MGH) and the University of Massachusetts Medical School (UMMS) to test their NurOwn product in clinical trials for the treatment of Amyotrophic Lateral Sclerosis (ALS).
NurOwn, which was granted FDA Orphan Drug status, involves putting patients’ own bone marrow through a proprietary process that drives the differentiation of the marrow cells into unique nerve-supporting cells that secrete factors responsible for neural system growth, repair, and survival. One of those factors, Glial-Derived Neurotrophic Factor (GDNF), is thought to be extremely important in the pathophysiology and potential treatment of ALS, also known as Lou Gehrig’s disease.
ALS is a progressive, fatal disease characterized by motor neuron loss without an identifiable cause. It is hypothesized that a lack of neuronal support factors leads to the “dying back” of neurons and subsequent loss of motor function for patients. BrainStorm believes that once marrow mesenchymal cells complete the NurOwn processing, the now-differentiated glial cells will secrete protective factors such as GDNF and Brain-Derived Neurotrophic Factor (BDNF) that could ameliorate or even cure the disease.
Dr. Robert Brown, the professor of neurology known for discovering the first gene linked to familial ALS who will be leading the trials at UMMS, stated: “Professor Cudkowicz (MGH) and I are delighted to join forces with BrainStorm to prepare for BrainStorm’s first human clinical trials in the United States. We are hopeful that together we may achieve a breakthrough for the treatment of people suffering from ALS. This trial will be conducted after receiving all necessary FDA approvals and in parallel with the human clinical trials that are being conducted by BrainStorm in Israel in collaboration with the Hadassah Medical Center.”
Despite several companies attempting to create nerve-supporting cells that secrete GDNF and other neurotrophic compounds, BrainStorm is among the first to do so without the need for fetal or embryonic stem cells and without including genetic engineering steps that may increase the risk of cancer in treated patients because of an unstable genome.
The NurOwn process may also be leveraged towards treatment of other debilitating and fatal neurologic diseases such as Parkinson ’s disease, Huntington ’s disease, and Alzheimer’s Dementia. You can learn more by viewing Rasheda Ali, an advisor to BrainStorm and daughter of Muhammed Ali, speaking about the potential of BrainStorm’s technology here.
UMMS Press Release: UMMS, BrainStorm Cell to Collaborate on ALS Trial
BrainStorm Press Release: BrainStorm Signs MOU for Collaboration