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New hope for a cruel disease
PETACH TIKVAH, Israel—Biotechnology firm BrainStorm Cell Therapeutics has entered into an agreement with the Dana-Farber Cancer Institute to provide cGMP-compliant clean room facilities for production of BrainStorm's NurOwn stem cell technology during an upcoming clinical trial for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease.
Named for the popular Yankees slugger who retired from baseball in 1939 at age 36 after losing the endurance, strength and speed he played the game with for 17 years, ALS is described by the Amyotrophic Lateral Sclerosis Association as a "progressive neurodegenerative disease … a particularly cruel disease that destroys a person's ability to control all muscle movement, typically striking adults in the prime of their lives."
Gehrig was diagnosed with ALS at the Mayo Clinic and died of the disease in 1941.
More than 70 years later, there is still no known cause or cure for ALS, which affects an estimated 350,000 worldwide, including 25,000 in the United States. An estimated 5,000 Americans are diagnosed with the disease each year, according to the U.S. National Institutes of Health. The disease is 100-percent fatal, as patients die within an average of two to five years following diagnosis.
Israel-based BrainStorm's NurOwn technology propagates and differentiates autologous mesenchymal stem cells into neurotrophic factor-secreting cells and their transplantation at or near the site of damage, and offers the hope of conquering neurodegenerative diseases. BrainStorm is preparing for a Phase II ALS trial at Massachusetts General Hospital (MGH) in Boston. The trial will be launched in the second half of 2013, pending U.S. Food and Drug Administration approval, and will be conducted not only at MGH, but also at the University of Massachusetts (UMass) Hospital and the Mayo Clinic. The Connell and O'Reilly Cell Manipulation Core Facility at Dana-Farber will produce NurOwn for the MGH and UMass Hospital clinical sites.
"We are pleased to begin this work that could be of great value to ALS patients," says Dr. Jerome Ritz, director of the Connell and O'Reilly Cell Manipulation Core Facility at Dana-Farber and professor of medicine at Harvard Medical School. "This is exactly the kind of service that the facility was set up to provide. Although we have previously supported a large number of clinical trials in stem cell transplantation and cancer immunotherapy, this will be our first opportunity to manufacture therapeutic cells for patients with ALS."
Ritz says Dana-Farber's role in the collaboration will be to provide therapeutic cells that meet very precise phenotypic and functional specifications.
"If these unique cells are found to be safe and effective, this will greatly expand the therapeutic options for patients with ALS, where conventional treatments have not been effective, and may also lead to potential applications of cell therapy for other neurologic diseases," he says.
The mission of Ritz's facility is to provide a variety of therapeutic cells that can be evaluated in carefully designed clinical research studies, he explains.
"This agreement with BrainStorm will expand our ability to manufacture a unique type of mesenchymal stromal cell designed to support nerve cell function in vivo," he says. "This will be the first time we will be manufacturing cells with this specific function, and we will undoubtedly learn a great deal from this process."
BrainStorm will provide funding for manufacturing cellular products for patients enrolled on the clinical research protocol. Alon Natanson, BrainStorm's CEO, says the agreement with top-notch facilities "brings us another step closer to developing a potentially effective treatment option for patients with ALS."
"Several months ago, BrainStorm initiated a search for a cGMP cleanroom production facility in the Boston area and identified Dana-Farber as having the best facility and greatest expertise to meet our standards," Natanson tells ddn. "Given the high unmet need of the ALS market, we are hopeful that our clinical trials program will progress quickly."
ALS is a multisystem disorder characterized by disruption of multiple mechanisms resulting in a toxic microenvironment for motor neurons, Natanson says. Drug treatment candidates for ALS, thus far, have not been effective largely due to their single-mechanism approach. The only currently approved inhibitory drug for ALS is riluzole, he notes, "which merely defers the development of breathing problems and loss of voluntary movement—and can extend life for about three months for some patients. In contrast, stem cell therapy provides a more systemic, multifactorial approach, while providing overall protection and stimulation of growth to dying motor neurons."