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An Israeli biotech company has claimed strong progress in the results of mid-stage clinical trials to test a treatment for motor neurone disease, in a fresh boost to the fledgling stem cell sector.
BrainStorm’s NurOwn autologous cells – transplanted from patients’ bone marrow – showed its therapy was well-tolerated and “appears to be safe for use, and did not present any undue risks to the study participants,” the company said.
Although this was not the main aim of the test, some of the 12 patients in the phase 1/2 trial with Amyotrophic Lateral Sclerosis (ALS), the muscle-wasting disease, as well as three further patients provided with the experimental treatment, also showed highly unusual signs of improvement, the company said.
Professor Dimitrios Karussis of the Neurology Department at Hadassah Medical Center, Jerusalem, and principal investigator of the trial, said: “We cannot ignore some possible promising indications of clinical efficacy observed in single patients, such as a tendency towards improvement in some of the major ALS Functional Rating Scale variables.”
The trial was conducted at Hadassah Medical Center in Jerusalem, and the results have been reported to the Israeli Ministry of Health.
“We are very excited at these strong preliminary efficacy results. They demonstrate that BrainStorm’s NurOwn stem cells have the potential not only to stop the deterioration but perhaps even cure this terrible thus far incurable disease,” said Chaim Lebovits, the company’s president.
“The coming phases in the trial will have to prove this but these results also reaffirm our belief that we have an enormous potential of being successful with our less severe indications such as MS and Parkinson’s.”
BrainStorm recently raised $5.8m with institutional investors in New York, providing it with financing for the next 18-24 months for further planned trials in Israel and Boston to examine different dosages and preliminary efficacy data.
Professor Robert Brown, chair of neurology at the University of Massachusetts Medical School, said: “This interim report clearly documents that BrainStorm’s NurOwn stem cell therapy is safe. We are absolutely delighted that, with these data in hand, Brainstorm is now moving forward into expanded studies involving trials here in Boston, Massachusetts.”