Mesenchymal Stem Cell Therapy Improves Joint Function in Rheumatoid Arthritis
Significant decreases were seen in swollen and tender joint measures.
Hope Biosciences’ adipose-derived mesenchymal stem cell therapy, HB-adMSCs, improved joint function in patients with rheumatoid arthritis (AR), according to results from a phase 1/2a trial recently published in Stem Cell Research and Therapy.1
“A small, open label pilot study was necessary not only to measure safety but also to gauge whether cell therapy could be effective in people who have been sick and suffering for a long time,” Donna Chang, chief executive officer, Hope Biosciences, said in a statement.2 “We also wanted to demonstrate that our investigational drug can produce consistent results in patients with diverse medical backgrounds.
Chang, who served as senior author for the paper, and colleagues enrolled 15 patients with RA who received a single dose of 2 × 108 adMSCs and were followed up with at 4, 12, 26, and 52 weekspost-treatment. Participants were mostly female (93.3%) and had a median age of 52 years (IQR, 38-61).
Efficacy was examined using American College of Rheumatology (ACR66/68 score) criteria for swollen and tender joint counts (S/TJC), and serum TNF-α, IL-6, CRP, and ESR levels. Safety endpoints included measures of hematologic, hepatic, and renal function.
Investigators found that participants’ swollen (effect size [ES], 0.83; P <.01) and tender joint counts (ES, 0/32; P <.001) improved on ACR66/68 score criteria at 52 weeks compared with baseline with large ES. Median scores decreased from 12.0 (IQR, 8.0-19.0) to 1 (IQR, 0.0-3.0) for swollen joints and from 20.0 (IQR, 11.0–36.0) to 1.0 (IQR, 0.0–4.0) for tender joint counts. Similar improvements with medium to large ES were observed at other timepoints during the study.
Inflammatory markers such as tumor necrosis factor-α, interleukin-6, and erythrocyte sedimentation rate remained unchanged from baseline but C-reactive protein levels did slightly decrease with a small effect size at week 4 (ES, 0.33; P =.229) and week 52 (ES, 0.37; P =.183). Hematologic, hepatic, and renal function remained largely unchanged (P >.05).
“The industry has historically faced challenges with autologous therapies - that is, using a patient’s own stem cells - causing variable results. Our patented technology, however, delivers a high volume of pure, fresh, undifferentiated mesenchymal stem cells, and results from studies like this one show that consistent manufacturing can yield consistent patient results. The effect size is unprecedented, and we believe a strong testament to the consistency and quality of our product,” Chang said.2
The treatment had a tolerable safety profile, with a majority (n = 15; 55.6%) of adverse events (AEs) classified as mild, 8 (29.6%) as moderate, and 4 (14.8%) as severe. Mild, treatment-related AEs included hematuria, right eyelid pruritis, anemia, and thrombocytopenia. Almost all participants (n = 14) experienced at least 1 AE. There were no AEs due to drug interactions.
“Ultimately, the purpose of this study is to inform,” Chang added.2 “The results of this pilot study show us that investment in larger trials is warranted, and that endpoints can be consistently achievable. We hope the proof of concept opens the door to hope for patients, and that those suffering from RA and other autoimmune conditions are as encouraged by the results as we are.”
