New York, NY – Hadassah Medical Organization announces the first objective evidence that stem cells injected into the central nervous system can improve life for people living with multiple sclerosis and other neurodegenerative diseases. The results of a Phase II trial demonstrate that repeated NG-01 stem cell treatments for these conditions are not only safe in the short to intermediate term, but also have clinical benefits that last up to four years. The results were recently published in the journal Stem Cells Translational Medicine.
The trial was led by Prof. Dimitrios Karussis, MD, Director of the Multiple Sclerosis Center at Hadassah Medical Organization, working with Petrou Panayiota, MD, and Ibrahim Kassis, MD.
Hadassah’s Phase II, randomized, double-blind, placebo-controlled clinical trial assessed the safety, tolerability and efficacy of transplanting NG-01 in people with primary-progressive MS. Forty-eight participants with the condition were enrolled in the study and randomized into three groups. Each received either an intrathecal (IT) or an intravenous (IV) NG-01 injection or a placebo injection. Cerebrospinal fluid samples were obtained from the study participants at the start of the trial and again after six months following treatment with NG-01 or a placebo.
For each sample, the investigators assessed the level of NF-L, a protein released into cerebrospinal fluid by damaged neurons and the most reliable and accepted biomarker of neurodegeneration in patients with MS and other neurodegenerative diseases. High levels of NF-L are consistently found in the cerebrospinal fluid of MS patients and can be used as a biomarker of MS disease activity and of the response to various MS treatments.
Prior to treatment, the concentrations of NF-L were exceptionally high in almost all of the study participants’ cerebrospinal fluid, indicating an active and aggressive disease. After six months of treatment, the NF-L levels were significantly reduced, the reduction correlated with significant functional improvement. Eight of the nine patients who received an intrathecal injection of NG-01 experienced a more than 50 percent reduction in their NF-L levels and showed a decrease in disability that remained steady after 12 months, when the study ended.
The results of the Phase II trial are in line with those of two previous Hadassah studies, long-term trials into primary-progressive multiple sclerosis (PPMS), MS and ALS, published in Frontiers of Neurology and Frontiers in Bioscience, respectively. The MS trial showed that repeated NG-01 treatments are safe in the short to intermediate term and have clinical benefits that last for up to four years, especially in patients treated with more than two NG-01 injections. (More information on the long-term trials can be found at www.ClinicalTrials.gov. The identifier of the MS trial is NCT04823000; the identifier of the ALS trial is NCT04821479.)
Said Dr. Karussis: “Progressive MS is a debilitating disease without effective treatment options that can substantially suppress the progression of disability. This novel trial evaluated not only the clinical effects of stem cell therapy, but also the effects of NF-L, the most reliable and accepted biomarker of neurodegeneration.” He continued, “Taken together with the long-term clinical results, which showed stabilization and even functional improvement in 22 out of the 24 participants up to four years after the initiation of treatment, the results make us optimistic for the future management of progressive MS and neurodegenerative diseases.”
About Hadassah Medical Organization
For more than a century, Hadassah Medical Organization has set the standard of excellence for medical care and research in Israel. Its doctors and scientists are on the front lines, uniquely positioned to pinpoint ever-evolving medical needs. Their experience and ingenuity have yielded new ideas with huge potential in all areas of medicine, including therapeutics, diagnostic medical devices and digital health.