Stem-Cell Therapy Reverses Disability in Multiple Sclerosis

Findings from an ongoing, phase 3 clinical trial involving patients with relapsing-remitting multiple sclerosis (MS) reveal that hematopoietic stem-cell transplantation (HSCT) may be a feasible treatment option for reversing disability associated with MS (Burt RK, et al. JAMA. 2015;313:275-284).

This randomized, open-label clinical trial investigated whether nonmyeloablative HSCT improved neurologic disability and other clinical outcomes in patients with relapsing-remitting MS, because current therapy for MS has not been able to do so.

The clinical trial is comparing the efficacy of conventional MS drugs with hematopoietic stem-cell therapy for reversing MS-related disability. Unlike standard immune-based drugs, autologous hematopoietic stem-cell therapy is designed to reset rather than suppress the immune system.

In the clinical trial, peripheral blood stem cells were collected 10 days after patients received cyclophosphamide and filgrastim. The chemotherapy conditioning regimen consisted of cyclophosphamide 5 to 2 days before stem-cell infusion plus alemtuzumab or thymoglobulin administered before stem-cell infusion.

“There has been resistance to this in the pharma and academic world,” said the trial’s coordinator, Richard K. Burt, MD, Chief, Division of Medicine-Immunotherapy and Autoimmune Diseases, Northwestern University Feinberg School of Medicine, Chicago, IL, in an interview with BBC News. “This is not a technology you can patent and we have achieved this without industry backing.”

The preliminary study results were encouraging. Dr Burt and colleagues reported that nonmyeloablative HSCT helped stop disease progression, as measured by the Expanded Disability Status Scale score, and reversed neurologic disability for many patients with relapsing-remitting MS.

Personal accounts of remarkable improvement after undergoing stem-cell transplantation for relapsing-remitting MS have recently surfaced from patients participating in this clinical trial, including Steven Storey and Holly Drewry, both of whom could walk again after being wheelchair-bound.

These preliminary findings require confirmation in randomized clinical trials, and the study is estimated to be completed by 2018.