 Robert H. Brown Jr., DPhil, MD |
BrainStorm Cell Therapeutics Inc., a biotech company developing stem cell technologies to provide treatments for currently incurable neurodegenerative diseases, has announced that it will partner with UMass Medical School and its chair of neurology, Robert H. Brown Jr., DPhil, MD, in anticipation of applying for FDA approval to begin amyotrophic lateral sclerosis (ALS) human clinical trials in the United States. The trial, which will be conducted at Massachusetts General Hospital, will investigate the use of the company’s NurOwn treatment for ALS.
ALS, also known as Lou Gehrig’s disease, is a progressive, neurodegenerative disorder affecting the motor neurons in the central nervous system. It is estimated that 5,000 people in the United States are newly diagnosed with the disease each year. As motor neurons die, the brain’s ability to send signals to the body’s muscles is compromised, leading to the loss of voluntary muscle movement, paralysis and eventually death from respiratory failure. The average survival rate for patients with ALS is three to five years.
In 1993, a team of researchers led by Dr. Brown, professor of neurology, discovered the first gene linked to familial ALS, a protein anti-oxidant known as superoxide dismutase, or SOD1. Only 10 percent of ALS cases are familial, while roughly 90 percent are sporadic in nature—meaning there is no identifiable familial risk or family history.
“I am delighted to join forces with BrainStorm to prepare for its 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," said Brown.
BrainStorm was granted FDA Orphan Drug Designation for its NurOwn treatment for ALS. NurOwn technology, based on discoveries at Tel Aviv University, processes adult human stem cells in bone marrow that can self-renew and differentiate into many cell types. BrainStorm believes the adult stem cells can differentiate into cells that can release neurotrophic factors, including glial-derived neurotrophic factor, a small protein that may promote survival of many types of neurons.
