NG-01 Cell Therapy Reduces NfL, GFAP in Patients With Progressive MS
Data from the OLE of a phase 2 clinical trial at Hadassah Medical Center were presented at the 2024 ACTRIMS meeting.
NG-01 cell therapy decreased neurofilament light (NfL) and glial fibrillary acid protein (GFAP) in patients with progressive multiple sclerosis (MS).1
The updated data, from an interim analysis of the open-label extension period of a randomized, double-blind, placebo-controlled, Phase 2 clinical trial (NCT02166021), were presented by the MS team at Hadassah Medical Center, Jerusalem, at the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum, held in West Palm Beach, Florida on March 2nd, 2024.1
"There is a significant unmet medical need for progressive MS, which is a debilitating disease without effective treatment options," lead principal investigator Dimitrios Karussis, MD, PhD, head of Hadassah’s MS team, said in a statement.1 "We are therefore excited to see that NG-01 not only prevented the progression of the disease for a rather long period of up to two years, but also led to demonstrable improvement in patient condition measured by multiple parameters— clinical assessments, cognitive tests, and objective biomarkers. Serum levels of NfL and GFAP were evaluated at repeated time points after each NG-01 treatment as the key biomarkers that correlate with neuroinflammation and neurodegeneration in progressive MS. We are encouraged to see that NG-01 treatment consistently reduced the levels of both biomarkers.”
NG-01 consists of autologous bone marrow-derived stem cells designed to secrete high levels of remyelinating and neurotrophic factors at the central nervous system site of injury. The therapy is administered by direct injection into the central nervous system through the cerebrospinal fluid. The cells are designed to settle close to the damaged areas or lesions in the brain and spinal cord, and exert neuroprotective, remyelinating, and anti-inflammatory effects.
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The interim analysis included data from 23 of 40 patients given at least 2 administrations of NG-01 3-6 months apart in addition to standard of care treatments, with follow-up of 12-18 months. Twenty participants exhibited a 33.2% mean reduction in NfL from baseline to last follow-up (P <.0008); data from the remaining 3 participants were not provided. Looking at all patients included in the analysis, there was a 22% mean reduction in GFDAP frmo baseline to last follow-up (P <.0008).
“Additionally, the positive effect on cognitive function after NG-01 treatment may point to a potential role of these cells in targeting compartmentalized CNS lesions, such as PRLs (paramagnetic lesions), that is to be tested in subsequent clinical trials. The extension study results underscore the possibility of a novel, safe, and long-acting therapy for stabilizing and/or reversing the progression of MS, especially in its progressive form, known as PIRA (Progression Independent of Relapse Activity), that advances "silently" and is very resistant to the existing conventional medical treatments,” Karussis continued.
Hadassah also reported significant improvements in 25-foot walking time (T25FW) in 16 patients after 12 months (P=.031), Symbol Digit Modalities Test (P =.0008), and patient reported outcomes (=mental and fatigue questionaries; P <.05) from baseline to the last follow-up.1
Adverse events (AEs) included headache (n = 9) and backache (n = 2), which were considered related to the procedure and resolved within 48 hours after the lumbar puncture. No serious AEs were reported.1
"We are extremely pleased with these promising results of our NG-01 cells in patients with progressive MS," Tal Gilat, CEO,NeuroGenesis, added.1 "Following the observations on the longitudinal decrease in NfL and GFAP, the FDA has cleared our Phase 2b, global, multi-center study with a double-blind, randomized, and placebo-controlled design to primarily test the efficacy and safety of different doses of intrathecal administration of NG-01 in progressive MS patients. We are now preparing for the Phase 2 double-blinded study and have already initiated work with multiple leading hospitals around the world. We look forward to the continued development of this cutting-edge treatment that has shown promise not only in MS but also in ALS, and thus may offer hope for hundreds of thousands of patients worldwide who currently have no alternative medical solution."
MS has proven to be a difficult field for cell therapy to get a foothold in, with Atara Biotherapeutics recently announcing that it would discontinue the phase 2 EMBOLD trial (NCT03283826) of ATA188, its allogeneic, Epstein-Barr Virus T-cell immunotherapy clinical-stage candidate, after it failed its primary endpoint of showing superiority over placebo in patients with MS.2
