New hope for a cruel disease

PETACH TIKVAH, Israel—Biotechnology firm BrainStorm CellTherapeutics has entered into an agreement with the Dana-Farber CancerInstitute to provide cGMP-compliant clean room facilities for production ofBrainStorm's NurOwn stem cell technology during an upcoming clinical trial foramyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease.

Named for the popular Yankees slugger who retired frombaseball in 1939 at age 36 after losing the endurance, strength and speed heplayed the game with for 17 years, ALS is described by the Amyotrophic LateralSclerosis Association as a "progressive neurodegenerative disease … aparticularly cruel disease that destroys a person's ability to control allmuscle movement, typically striking adults in the prime of their lives."

Gehrig was diagnosed with ALS at the Mayo Clinic and died ofthe disease in 1941.

More than 70 years later, there is still no known cause orcure for ALS, which affects an estimated 350,000 worldwide, including 25,000 inthe United States. An estimated 5,000 Americans are diagnosed with the diseaseeach year, according to the U.S. National Institutes of Health. The disease is100-percent fatal, as patients die within an average of two to five yearsfollowing diagnosis.

Israel-based BrainStorm's NurOwn technology propagates anddifferentiates autologous mesenchymal stem cells into neurotrophicfactor-secreting cells and their transplantation at or near the site of damage,and offers the hope of conquering neurodegenerative diseases. BrainStorm ispreparing for a Phase II ALS trial at Massachusetts General Hospital (MGH) inBoston. 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 MayoClinic. The Connell and O'Reilly Cell Manipulation Core Facility at Dana-Farberwill produce NurOwn for the MGH and UMass Hospital clinical sites.

"We are pleased to begin this work that could be of greatvalue to ALS patients," says Dr. Jerome Ritz, director of the Connell andO'Reilly Cell Manipulation Core Facility at Dana-Farber and professor ofmedicine at Harvard Medical School. "This is exactly the kind of service thatthe facility was set up to provide. Although we have previously supported alarge number of clinical trials in stem cell transplantation and cancerimmunotherapy, this will be our first opportunity to manufacture therapeuticcells for patients with ALS."

Ritz says Dana-Farber's role in the collaboration will be toprovide therapeutic cells that meet very precise phenotypic and functionalspecifications.

"If these unique cells are found to be safe and effective,this will greatly expand the therapeutic options for patients with ALS, whereconventional treatments have not been effective, and may also lead to potentialapplications of cell therapy for other neurologic diseases," he says.

The mission of Ritz's facility is to provide a variety oftherapeutic cells that can be evaluated in carefully designed clinical researchstudies, he explains.

"This agreement with BrainStorm will expand our ability tomanufacture a unique type of mesenchymal stromal cell designed to support nervecell function in vivo," he says."This will be the first time we will be manufacturing cells with this specificfunction, and we will undoubtedly learn a great deal from this process."

BrainStorm will provide funding for manufacturing cellularproducts for patients enrolled on the clinical research protocol. AlonNatanson, BrainStorm's CEO, says the agreement with top-notch facilities"brings us another step closer to developing a potentially effective treatmentoption for patients with ALS."

"Several months ago, BrainStorm initiated a search for acGMP cleanroom production facility in the Boston area and identifiedDana-Farber as having the best facility and greatest expertise to meet ourstandards," Natanson tells ddn. "Giventhe high unmet need of the ALS market, we are hopeful that our clinical trialsprogram will progress quickly."

ALS is a multisystem disorder characterized by disruption ofmultiple mechanisms resulting in a toxic microenvironment for motor neurons,Natanson says. Drug treatment candidates for ALS, thus far, have not beeneffective largely due to their single-mechanism approach. The only currentlyapproved inhibitory drug for ALS is riluzole, he notes, "which merely defersthe development of breathing problems and loss of voluntary movement—and canextend life for about three months for some patients. In contrast, stem celltherapy provides a more systemic, multifactorial approach, while providingoverall protection and stimulation of growth to dying motor neurons."