New ALS study succeeds in Phase II

ADSC, SVF, Stem Cell Bank

Brainstorm Cell Therapeutics stock jumped by just over 17 percent this week and has outperformed the Standar & Poor’s 500 index by close to 21 percent over the last month. Why? Because it recently stated that it’s met its Phase II objectives for a new Lou Gehrig’s disease treatment, NurOwn cell therapy.

Lou Gehrig’s disease, also known as amyothrophic lateral sclerosis, is a progressive neurogenerative condition according to Kids Health. It continues to worsen to the point that typical survival time is no more than five years, although some people with the disease may live much longer than that.

“Eventually those with ALS will lose all movement in their legs, feet, arms and hands.”

Those who have Lou Gehrig’s disease may initially have problems with general movement and find themselves less likely to walk and speak, according to the ALS Association. Eventually they’ll lose all movement in their legs, feet, arms and hands, and the disease will take hold of their lungs. At that point, they may need to be placed on a ventilator.

This new study is groundbreaking in that it has been shown – so far – to slow ALS’ progression. In a news release, Dr. Robert Brown, investigator, professor and chair of neurology at the University of Massachusetts Medical Center and Medical School, said that his team was thrilled about what they found and feel confident about conducting more studies with the NurOwn cell therapy.

“We are very encouraged by these Phase 2 data that show that NurOwn cell therapy is safe and that a single administration produces a transient and clinically meaningful beneficial response in terms of both the ALS-FRS-R rating scale and CSF biomarkers,” said Dr. Brown, according to a press release. “These exciting findings clearly indicate that it is appropriate to conduct a longer study with repetitive dosing.”

Conducting the study
To conduct the study, researchers treated a random selection of 48 patients with ALS with NurOwn cells and a placebo. Scientists administered these treatment sessions at the Mayo Clinic, Massachusetts General Hospital and UMass Medical School. Over the course of 2, 4, 8, 12, 16 and 24 weeks, researchers analyzed the patient’s health. From there they analyzed the results, specifically looking at change in Slow Vital Capacity and muscle strength, the slope of Amyotrophic Lateral Sclerosis Functional Rating Scale, how patients responded compared to pretreatment health and those the researchers excluded because these patients were very unlikely to benefit from NurOwn due to their state of health.

“After 12 weeks, 40 percent of patients who had received NurOwn had experienced a marked improvement.”

Researchers analyzed the study by looking at patients who had improved by 25, 50, 75 and 100 percent. At the 12-week mark 40 percent of patients who had received NurOwn had experienced a marked improvement of at least 50 percent compared to placebo subjects (17 percent).

Carlayne Jackson, MD, and chair of the Data Safety Monitoring Board for this study, said that along with positive test results, side effects were minimal.

“Patients in the Brainstorm study tolerated treatment extremely well and there were no serious adverse events related to therapy. The safety profile certainly provides the opportunity to continue to study this approach to ALS treatment,” stated Jackson.

Anthony Windebank, professor of neurology and director for Discovery, the Mayo Clinic for Regenerative Medicine, said, “After almost 30 years of conducting clinical studies in ALS I am personally very encouraged and excited by the trial results.” He also noted that his colleagues at Mayo clinic “will be eager to move ahead with that as soon as possible.”

While a full cure for ALS is still a ways off, NurOwn has the potential to both help people with the condition and be a link to further even more advanced studies.

A Phase III study will look to expand on this research.