A recent prospective phase 2 study
sought to investigate autologous hematopoietic stem cell transplantation (AHSCT) as a therapeutic intervention in multiple sclerosis (MS).
The trial was designed as a single-center phase 2 study of AHSCT in patients with active relapsing remitting (RRMS) and secondary progression MS to assess the efficacy and safety of the treatment. AHSCT has been identified in prior research as being able to suppress clinical elapses and inflammation, as well as reverse disability in patients with RRMS and potentially secondary progressive MS.
AHSCT is performed by harvesting the patients hematopoietic stem cells and reinfusing them after a high dose of chemotherapy is used to destroy the immune cells reacting against the nervous system. The aim is to recreate a tolerant immune system in MS patients.
The trial enrolled 35 patients, of which 20 were diagnosed with RRMS and 15 with secondary progressive MS. The majority of patients (69%) underwent prior treatment of at least 4 or more disease-modifying therapies but had eventually relapsed.
On average, patients received a median of 7.41 million hematopoietic stem cells per kg of body weight and were followed for a median of 36 months. Prior to AHSCT, patients were administered a chemotherapy combination (carmustine, etoposide, cytarabine, melphalan), BEAM, delivered into the bloodstream to destroy the immune system before the transplant.
The primary endpoint was measured as event-free survival (EFS). At the median follow up of 36 months, EFS was 60%. Patients with RRMS demonstrated particular response to the therapy with EFS of 90% for the first year, and 70% for the following 2 years. Additionally, patients with RRMS were also found to have relapse-free survival of 97% at 1 year, and 90% at 2 and 3 years.
Overall, the scores on the Expanded Disability Status Scale (EDSS), which evaluates the severity of MS, showed a sustained significant decrease, particularly for patients with RRMS. The median EDSS score was 6 (range, 2-7).
“In conclusion, this study provides further evidence that AHSCT using BEAM conditioning is a highly efficacious therapy for patients with MS, even after failing multiple therapies.” However, due to the more beneficial results seen in patients with RRMS, researchers modified the clinical trial criteria to only include patients with RRMS.
Moore J, Massey J, Ford C, et al. Prospective phase 2 clinical trial of autologous hematopoietic stem cell transplant for treatment of refractory multiple sclerosis [published online December 11, 2018]. J Neurol Neurosurg Psychiatry.