ABSTRACT
Objective: Using mesenchymal stem cells [MSCs] is regarded as a new therapeutic approach for improving fibrotic diseases.The aim of this study to evaluate the feasibility and safety of systemic infusion of autologous adipose tissue-derived MSCs [AD-MSCs] in peritoneal dialysis [PD] patients with expected peritoneal fibrosis
Materials and Methods: This study was a prospective, open-label, non-randomized, placebo-free, phase I clinical trial. Case group consisted of nine eligible renal failure patients with more than two years of history of being on PD. Autologous AD-MSCs were obtained through lipoaspiration and expanded under good manufacturing practice conditions. Patients received 1.2 +/- 0.1×106 cell/kg of AD-MSCs via cubital vein and then were followed for six months at time points of baseline, and then 3 weeks, 6 weeks, 12 weeks, 16 weeks and 24 weeks after infusion. Clinical, biochemical and peritoneal equilibration test [PET] were performed to assess the safety and probable change in peritoneal solute transport parameters
Results: No serious adverse events and no catheter-related complications were found in the participants. 14 minor reported adverse events were self-limited or subsided after supportive treatment. One patient developed an episode of peritonitis and another patient experienced exit site infection, which did not appear to be related to the procedure. A significant decrease in the rate of solute transport across peritoneal membrane was detected by PET [D/P cr=0.77 vs. 0.73, P=0.02]
Conclusion: This study, for the first time, showed the feasibility and safety of AD-MSCs in PD patients and the potentials for positive changes in solute transport. Further studies with larger samples, longer follow-up, and randomized blind control groups to elucidate the most effective route, frequency and dose of MSCs administration, are necessary [Registration Number: IRCT2015052415841N2]
ABSTRACT
Objective: Amyotrophic lateral sclerosis [ALS] is the most severe disorder within the spectrum of motor neuron diseases [MND] that has no effective treatment and a progressively fatal outcome. We have conducted two clinical trials to assess the safety and feasibility of intravenous [IV] and intrathecal [IT] injections of bone marrow derived mesenchymal stromal cells [BM-MSCs] in patients with ALS
Materials and Methods: This is an interventional/experimental study. We enrolled 14 patients that met the following inclusion criteria: definitive diagnosis of sporadic ALS, ALS Functional Rating Scale [ALS-FRS] .24, and .40% predicted forced vital capacity [FVC]. All patients underwent bone marrow [BM] aspiration to obtain an adequate sample for cell isolation and culture. Patients in group 1 [n=6] received an IV and patients in group 2 [n=8] received an IT injection of the cell suspension. All patients in both groups were followed at 24 hours and 2, 4, 6, and 12 months after the injection with ALS-FRS, FVC, laboratory tests, check list of side effects and brain/spinal cord magnetic resonance imaging [MRI]. In each group, one patient was lost to follow up one month after cell injection and one patient from IV group died due to severe respiratory insufficiency and infection
Results: During the follow up there were no reports of adverse events in terms of clinical and laboratory assessments. In MRI, there was not any new abnormal finding. The ALS-FRS score and FVC percentage significantly reduced in all patients from both groups
Conclusion: This study has shown that IV and IT transplantation of BM-derived stromal cells is safe and feasible [Registration numbers: NCT01759797 and NCT01771640]