Cytora, a clinical stage company developing unique stem cell treatments based on human Oral Mucosa Stem Cells (hOMSC), reported today data of an ongoing Phase 1 clinical study for treating moderate and advanced Multiple System Atrophy (MSA) with hOMSC300, its investigational, allogeneic, off-the-shelf, cell therapy product. The safety data collected to date demonstrate that intrathecal administration of hOMSC is safe. In addition, preliminary efficacy data suggest that hOMSC may be efficient as a disease modifying therapy in moderate stages of MSA. The interim results of the clinical trial as well as preclinical results from a mouse model of MSA were presented at the International MSA CONGRESS, BOSTON 2025.
“MSA is a debilitating, progressive neurodegenerative disease, which currently has no treatment,” said Yona Geffen, PhD, CEO of Cytora. “We are therefore very encouraged by these preliminary safety and efficacy data, demonstrating that intrathecal administration of hOMSC is safe, and may be efficient in attenuating disease progression in moderate stages of MSA. We have previously reported the successful results of a Phase 1/2a clinical study for treating chronic hard to heal diabetic foot ulcers using hOMSC200, based on our proprietary stem cell platform, and we are looking forward to further advancing both of these promising indications, for the benefit of patients around the world.”
The ongoing first-in-human, open label, single center Phase 1 study is aimed at testing the safety of hOMSC300 following intrathecal administration in patients with moderate or advanced stages of MSA with subsequent 18 months follow-up. For the analysis, the eight patients receiving the high dose were allocated to two groups according to their disease stage. Four patients with Unified Multiple System Atrophy Rating Scale (UMSARS) ≤20 points at baseline were allocated to the moderate stage group. Four patients with UMSARS > 20 points at baseline were allocated to the advanced stage group. Recruited subjects were administered intrathecally with either a low or a high single dose of hOMSC300. The first two patients in advanced stages of the disease were treated with the low dose. UMSARS scores were assessed.
To date, 3-18 months after hOMSC administration, no serious adverse events related to the hOMSC300 administration were recorded during this period. Treatment with hOMSC300 showed potential efficacy in patients with moderate disease, whose disease did not significantly progress at the 3, 6 and 9 months post injection period, as assessed by the UMSARS scale, with a mean change of 1.5, 1.8 and 2 points at 3, 6 and 9 months follow-up, respectively. For comparison, a multicenter cohort study of MSA from The Japan MSA registry study from 2023 shows that after 12 months there is a decline of 6.4 in UMSARS of moderate MSA patientsi.
Comparison of the mean change from baseline in UMSARS scores between the patients in the moderate group and those in the advanced group indicates a statistically significant lower increase in UMSARS score (2 points) in the moderate group vs. the advanced group (14.5 points) (p = 0.0345 by Linear Model for Repeated Measures). MRI volumetry data indicates no significant changes from baseline in the combined volume of gray and white matter in the cerebellum and cerebrum. More on the study design at NCT05698017.
In addition to the clinical study, hOMSC300 cells were also shown to be effective in treating a mouse model of MSA. In these preclinical studies, a single injection of either 2.5×105 or 5×105 hOMSC into the cerebrospinal fluid of 30 mice acts as a disease modifier by exerting neuroprotection on dopaminergic neurons and by dampening neuroinflammation
