Adipose-Derived Mesenchymal Stem Cell Therapy Meets Primary End Point in Phase 2 Trial of Relapsing-Remitting MS

Late-breaking data from a phase 2 trial (NCT05116540) showed that Hope Biosciences’ autologous adipose-derived mesenchymal stem cell (HB-adMSC) therapy met its primary end point in improving quality of life among patients with relapsing-remitting multiple sclerosis (RRMS).¹

Among 24 patients with RRMS randomized to receive either autologous HB-adMSCs or placebo, those treated in the active treated group showed significant improvements in both the Physical Health Composite Score (PHCS) and Mental Health Composite Score (MHCS) of the MSQOL-54, meeting the primary efficacy end point, at 52 weeks. Notably, the least square mean change from baseline in PHCS was 19.87 (SE, 2.98), with a difference from placebo of 22.12 (95% CI, 12.28-31.96; P = .0002; effect size, 1.23). For MHCS, the mean change was 13.36 (SE, 3.96), with a difference from placebo of 15.76 (95% CI, 3.31-28.22; P = .0166; effect size, 0.85).

Presented at the 2025 European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress, held September 24-26, in Barcelona, Spain, the phase 2, randomized, double-blind, placebo-controlled, single-center trial was conducted between November 2021 and December 2024. Participants, aged 18 to 75 years and with an Expanded Disability Status Scale (EDSS) score between 3.0 and 6.5, were enrolled and received 6 intravenous infusions of either 200 million HB-adMSCs or placebo at 0, 4, 8, 16, 24, and 32 weeks. Researchers performed follow-up assessments at week 42 and the end-of-study visit occurred at week 52.

Presented by lead author Ridhima Vij, PhD, clinical research scientist and medical writer at Hope Biosciences Research Foundation, the findings showed that secondary outcomes also favored the HB-adMSC -treated group. Patients demonstrated significant reductions in EDSS (−1.50 ; difference from placebo, −1.55; 95% CI, −2.91 to −0.19; P = .0278; effect size, 1.35) and in PHQ-9 scores (−4.33; difference from placebo, −4.59; 95% CI, −6.77 to −2.41; P = .0004; effect size, 0.84). The treatment demonstrated a favorable safety profile, with mostly mild adverse events and no severe treatment-related events or deaths.

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“The trial successfully met its primary endpoint, and the results clearly demonstrate that HB-adMSCs can be effective in a complex, variable condition like RRMS,” Vij, said in a statement.² “There were statistically significant improvements in both physical and mental health for the treatment group compared to the placebo group as determined by the MS Quality of Life-54 Instrument. This tool is a multidimensional quality of life measure that combines general and MS-specific points related to fatigue, physical and cognitive function, sexual impairment, pain, energy, mobility, level of disability, and other considerations – all contributors to quality of life we keep front of mind, when we think about how a therapeutic might revolutionize how we approach chronic degenerative disease.”

In this current phase 2 trial, findings revealed that HB-adMSC therapy was associated with improvements in quality-of-life measures for patients with RRMS and was generally well tolerated. Authors noted that these findings suggest that further research, including larger and longer-term studies, may be needed to evaluate the therapy’s sustained efficacy and safety. In August 2025, the FDA granted regenerative medicine advanced therapy designation for HB-adMSCsfor treatment of patients living with RRMS.²

“The results of this trial are groundbreaking for multiple sclerosis. They clearly demonstrate that high doses of fresh HB-adMSCs delivered on a regular schedule can result in consistent efficacy in a highly complex and variable condition like MS,” Donna Chang, president at Hope Biosciences Research Foundation, said in a statement.³ “We believe that this positive response will translate in other autoimmune diseases in the near future.”

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REFERENCES
1. Vij R, Kim H, Park H, et al. Adipose-Derived Mesenchymal Stem Cells Improve Quality of Life in Patients with Relapsing-Remitting MS: Results from a Phase 2 Randomized Placebo-Controlled Trial. Presented at ECTRIMS Congress; September 24-26, 2025; Barcelona, Spain. Late-Breaking Abstract P915.
2. Hope Biosciences Secures FDA RMAT Designation for Stem Cell Therapy in Relapsing-Remitting Multiple Sclerosis. News Release. Hope Biosciences Research Foundation. Published August 27, 2025. Accessed September 26, 2025. https://www.businesswire.com/news/home/20250827264017/en/Hope-Biosciences-Secures-FDA-RMAT-Designation-for-Stem-Cell-Therapy-in-Relapsing-Remitting-Multiple-Sclerosis
3. Hope Biosciences Research Foundation Reports Promising Phase II Trial Results for Stem Cell Therapy in Multiple Sclerosis. News Release. Hope Biosciences Research Foundation. Published October 23, 2024. Accessed September 26, 2025. https://www.businesswire.com/news/home/20241031106825/en/Hope-Biosciences-Research-Foundation-Reports-Promising-Phase-II-Trial-Results-for-Stem-Cell-Therapy-in-Multiple-Sclerosis