The intrathecal transplantation of human fetal neural precursor cells (hfNPCs) is feasible, safe, and tolerable in patients with progressive multiple sclerosis (MS), according to the results of an open-label, phase 1 clinical trial published in the journal Nature Medicine.
“Although preliminary, these results support the rationale and value of future clinical studies with the highest dose of hfNPCs in a larger cohort of patients,” the study authors wrote.
Read more about the different types of MS
Previous research in animal models of MS has shown that neural precursor cell transplantation can promote neuroprotection and remyelination.
The feasibility, safety, and tolerability of intrathecal transplantation of hfNPCs were tested in a prospective, therapeutic exploratory, nonrandomized, not controlled, open-label, single-dose-finding phase 1 clinical trial.
The trial that took place at San Raffaele Hospital in Milan, Italy, enrolled 12 patients, 18 to 55 years of age, with progressive MS with a disease duration of 2 to 20 years and who received no other therapy.
During the trial, patients received 1 of 4 doses of treatment (0.7 × 106, 1.4 × 106 , 2.8 × 106, or 5.7 × 106 ± 10% cells per kg).
Primary outcome measures were overall survival, overall safety and tolerability measured by adverse events, changes in neurological conditions not related to disease, and feasibility measured by the proportion of successful intrathecal administration procedures.
The researchers also recorded a lower rate of brain atrophy in patients who received the highest dose of treatment (106 ± 10% cells per kg) as well as higher levels of cerebrospinal fluid and neuroprotective molecules.
MS is an autoimmune disease that affects the brain and spinal cord. It is characterized by immune-mediated demyelination and axonal damage. Progressive MS is a clinical form of MS in which disabilities increases gradually independent of relapses over time and for which there are currently no effective treatments.
Genchi A, Brambilla E, Sangalli F, et al. Neural stem cell transplantation in patients with progressive multiple sclerosis: an open-label, phase 1 study. Nat Med. Published online January 9, 2023. doi:10.1038/s41591-022-02097-3
Neural stem cell transplantation in multiple sclerosis patients (STEMS). US National Library of Medicine. Updated September 5, 2021. Accessed January 18, 2023.