Prof. Dimitrios Karussis.
(photo credit: HADASSAH MEDICAL ORGANIZASTION)
After a four-year study, a pioneering treatment for amyotrophic lateral sclerosis (Lou Gehrig’s disease) using stem cells and growth factors has been shown to inhibit the progression of the fatal neurological disease in 87 percent of 26 patients.
The results of the treatment by neurologist Prof. Dimitrios Karussis of Hadassah-University Medical Center in Jerusalem’s Ein Kerem and the Israeli-American BrainStorm Cell Therapeutics company were just published in JAMA Neurology (The Journal of the American Medical Association).
“I am optimistic that within the foreseeable future, we may provide a treatment to ALS patients that can slow down or stop the progression of the disease,” Karussis said. “I see this treatment as being potentially one of the major tools to treat degenerative diseases of the brain and spinal cord in the future,” including multiple sclerosis and others.
As measured by the patients’ respiratory function or general motor disability, the treatment not only halted deterioration in patients but also caused their neurological function to improve, said Karussis on Wednesday.
The impressive results were announced at a San Francisco press conference as well.
ALS is a devastating and incurable neurodegenerative disease that robs patients of the ability to walk, to speak, to swallow and, ultimately, to breathe. Besides the late New York Yankees baseball player Gehrig, who died of ALS at 1941 at the age of 37, it also affects British theoretical physicist Prof. Stephen Hawking, who is one of the handful of ALS patients who has survived for many decades, even though he can only move his pupils to communicate with the world.
The study, published in the January issue of the journal, summarized results from Phase IIa of clinical studies, which began in 2011.
The patient’s own adult stem cells are removed from the bone marrow, enriched by a unique process developed at Hadassah and returned as NurOWN cells by injection into the ALS sufferer.
Karussis explained that he was “positively impressed by the results.
In the clinical trial, in which the stem cells were injected into the brain and spinal fluid, there were no negative influences and almost 90% of all the patients showed a slowdown in the pace of the disease’s development when judged by respiratory and motor function.
After initial studies at Hadassah, the treatment was tested in an identical way at the Mayo Clinic, Massachusetts General Hospital of the Massachusetts Memorial Medical Center. In 2011, BrainStorm initiated Phase I/IIa trials of NurOWN in ALS patients at Hadassah. The injections were done either into the spinal cord or into the muscle, and both resulted in very positive results in slowing the disease.
It does not cure the disease, but it does slow its advancement and has been proven safe.
There are some 30,000 ALS patients in the US and 450,000 in the whole world, including Israel, said Hadassah Women’s Zionist Organization of America national president Ellen Hershkin.
BrainStorm president Haim Leibovitz said he is proud that his company is a pioneer in the development of technologies to treat neurodegenerative diseases using stem cells and looks forward to double-blinded Phase IIa trials in the US and continuing testing at Hadassah.