NeuroGenesis and Hadassah Medical Center announced highly positive results from a placebo-controlled Phase 2 clinical trial assessing the impact of NG-01 autologous proprietary subpopulation of mesenchymal stem cells (MSCs) on patients with progressive multiple sclerosis (MS). The results have been published in Brain, a prestigious peer-reviewed journal published by Oxford University, and highlighted in the “Editor’s Choice” section.
The randomized, double-blind, placebo-controlled, clinical trial assessed the safety, tolerability and efficacy of transplantation of NG-01 in people with progressive MS. The study enrolled 48 patients who were randomized into 3 groups, receiving either an intrathecal or intravenous NG-01 injection, or a placebo injection. The two primary endpoints of the trial were: (i) the safety of the intrathecal and intravenous NG-01 treatments assessed by incidence of adverse events versus those in the placebo-treated group; and (ii) the differences among the three groups in the Expanded Disability Status Scale (EDSS) score changes and the proportion of patients with treatment failure, as evidenced by an increase in EDSS (disease progression) score, at 6 and 12 months. Overall, the study duration was 14 months.
Significantly fewer patients experienced treatment failure (disease progression) in the intrathecal (IT) and intravenous (IV) NG-01 treatment groups compared with those in the placebo group (6.7%, 9.7%, and 41.9%, respectively). Of the patients treated intrathecally with NG-01, 58% did not show any evidence of disease activity during the entire the treatment period, versus 9.7% in the placebo treated group. NG-01 treatment groups demonstrated a significant improvement in walking ability as measured by 25-foot walking time. Intrathecal administration of NG-01 was more efficacious than intravenous in several key parameters of the disease: relapse rate (89% decrease in the relapse rate), functional MRI (improvement of motor networks), monthly changes of the MRI T2 lesion load and the 9-hole peg test, as compared to the control (placebo-treated) group. No serious, treatment-related safety issues were detected.
The NG-01 technology is being developed by NeuroGenesis, following a license from Hadasit, Hadassah Medical Center Technology Transfer Company.
Neurogenesis’ technology entails collecting bone marrow from the patient. Then by utilizing a proprietary process, a unique subpopulation of bone marrow cells is identified, cultured and enhanced towards remyelinating biofactory cells (NG-01) that also possess neurotrophic immunolatory and neuroprotective properties. The NG-01 cell population is injected directly into the central nervous system (through the cerebrospinal fluid), where the cells home-in on the damaged area, take up residence and produce significant amounts of neurotrophic factors.
About Multiple Sclerosis
Multiple sclerosis (MS) is an autoimmune disease that causes damage in the myelin and the nerve cells of the central nervous system (demyelinating plaques in brain and spinal cord), resulting in cumulating neurological disability. The destruction of the myelin (the covering that protects nerves and promotes the efficient transmission of nerve impulses) causes secondary damage to the nerve cells and progressive atrophy. MS often causes sensory disturbances in the limbs, as well as motor problems. Affected individuals may have tremors, muscle stiffness (spasticity), exaggerated reflexes (hyperreflexia), weakness or paralysis of the muscles of the limbs, difficulty in walking, and poor sphincter control. MS is also associated with visual problems. There is no known cure for multiple sclerosis. The existing treatments are mostly aimed to reduce the incidence of relapses of the disease and slow down the rate of neurological deterioration.