Multiple sclerosis (MS), a chronic neuroinflammatory and neurodegenerative disease, is often disabling and poses substantial physical, psychological, and social burden on society and patients. The disease affects nearly 2.8 million subjects worldwide with an average lifespan ranging from 25 to 35 years following diagnosis. Although, several treatments are available to reduce the symptoms, there is no effective cure. Now, a group of researchers from San Raffaele Hospital in Milan, Italy, has shown that intrathecal transplanting fetal stem cells in MS patients may help in increasing the levels of anti-inflammatory and neuroprotective molecules.
The open-label, single-dose phase 1 clinical trial investigated the safety, feasibility, and tolerability of intrathecally transplanted human fetal NPCs (hfNPCs) in 12 MS patients with evidence of disease progression (Expanded Disability Status Scale ≥6.5) aged 18-55 years. No severe adverse reactions related to hfNPCs was noted upon 2-year follow-up and the results of exploratory secondary analyses showed reduced brain atrophy in patients who received the highest dosage of hfNPCs and improved cerebrospinal fluid levels of anti-inflammatory and neuroprotective molecules.
In recent years, autologous hematopoietic stem cell transplantation (AHSCT) has gained significant interest considering its improved efficacy and durable outcome. The National Multiple Sclerosis Society has recommended it as a useful therapeutic option in patients with relapsing MS, despite receiving treatment with high-efficacy disease-modifying therapy. The society has identified subjects <50 years of age with shorter durations of disease (<10 years) as ideal candidates for transplantation and the need to conduct the procedure at centers with substantial experience and expertise.
Although the preliminary study was promising, the researchers warranted further clinical studies with the highest dose of hfNPCs in large patient cohorts to corroborate the findings.
- 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. 2023 Jan 9. doi: 10.1038/s41591-022-02097-3.
- Miller AE, Chitnis T, Cohen BA, et al. National Medical Advisory Committee of the National Multiple Sclerosis Society. Autologous Hematopoietic Stem Cell Transplant in Multiple Sclerosis: Recommendations of the National Multiple Sclerosis Society. JAMA Neurol. 2021 Feb 1;78(2):241-246.