Technicians help develop a stem cell treatment for nerve-degenerative disease ALS in the laboratory..
(photo credit: BRAINSTORM)
You may recall friends and family dumping buckets of ice on their head to raise funds for treating amyotrophic lateral sclerosis (ALS), a debilitating neuro-degenerative disease.
Current treatments are able to slow ALS’ progression but fail to maintain or restore motor movement. Now, multiple clinical trials conducted by an Israeli firm, Brainstorm Cell Therapeutics, show a first-ever reversal in expected decline for patients, likely to transform how we treat ALS.
“We showed a very strong improvement when we compare pre-treatment to post-treatment in the patients that were treated,” said Chaim Lebovits, CEO of Brainstorm, referring to the company’s advanced stem cell therapy treatment. “It means that it’s not even a slowdown of the disease, or a halt, but it’s a reversal of it. It’s unheard of in ALS, if we replicate this in a third trial.”
BrainStorm announced on Monday that its first patients had enrolled in a phase three clinical trial for its treatment of ALS at two American hospitals. The research will occur at Massachusetts General Hospital, UC Irvine Medical Center in California and the Mayo Clinic in Minnesota — and it will include some 200 patients, half of whom will be given a placebo.
The company expects that if the stage three trial goes well, that the US Food and Drug Administration may give regulatory approval for mass use among doctors and patients. The trial will be measured by the ALSFR-S score responder analysis and final data is expected by 2019.
BrainStorm’s first two clinical trials of its stem cell therapy treatment, called NurOwn, were conducted at Jerusalem’s Hadassah hospital and also in the United States.
During the treatment process, doctors extract cells from the bone marrow of from patients with ALS, and the cells are multiplied and matured, then prepared for injection back into the patient. During the maturation process, the cells from the bone marrow are manipulated into behaving like brain cells, in that they produce materials that are the building blocks of the brain. When these modified cells are put back in patients, the building blocks circulate in the spinal fluid and help repair the damaged brain.
Since the cells are taken directly from the patient, that avoids the need for patients to be immunosuppressed so their bodies don’t attack a foreign substance.
“This is unprecedented, I’m not aware of another treatment program where the potential to alter or improve ALS is as great. We’re very encouraged by the promising results seen so far, and the changes in biomarkers that measure the building blocks of the brain — they suggest that the delivery of these substances is having an effect… we’re confident that we’re on the right track,” said Ralph Kern, MD, Brainstorm’s Chief Operating Officer and Chief Medical Officer.
For patients suffering from ALS, the disease affects the brain’s motor system, or what controls muscle movement and muscle contraction. Patients who are diagnosed with ALS have, on average, some 30 months to live, and famed British astrophysicist Stephen Hawking who suffers from the disease has been an outlier when it comes to longevity.
Because the motor system controls all of our muscles, it can affect our arms and legs, it can also affect our swallowing and our breathing. And it’s the loss of our ability to swallow and breathe that is the most dangerous,” said Kern.
Founded in 2006, the Israeli start-up is based in the central town of Petah Tikva, employs some 22 people, and it has raised some $70 million. The phase three clinical trial received a $16 million grant from the California Institute for Regenerative Medicine, which specializes in stem cell therapy. The company also previously benefited from funding provided by the Israel Innovation Authority.
The stem cell therapy was developed at Tel Aviv University and licensed through Ramot, the university’s technology transfer arm. The university will receive some royalties from the treatment.
Commonly known as Lou Gehrig’s Disease in the United States, ALS is diagnosed in thousands of people worldwide each year.
“We’re trying to change the course of ALS,” Lebovits said. "Brainstorm plans to quickly advance the phase 3 trial to… bring much needed hope to ALS patients and their families.”