A Phase III clinical trial comparing hematopoietic stem cell transplants to immunomodulators is underway.
Multiple sclerosis (MS) is an autoimmune disease characterized by the destruction of the myelin sheath of nerve fibers. There is no cure for the condition, but there are a number of FDA-approved immunomodulators or disease-modifying therapies to reduce relapses and slow disease progression. Despite the availability of these drugs, their varying efficacy, adverse effects, and expense are significant concerns. Therefore, a means to safely and effectively control MS symptoms and progression is still under investigation.
Cell-based immunotherapeutic strategies are showing promise as a viable treatment alternativeto other drug approaches. A Phase III clinical trial called Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS) is underway to compare the use of hematopoietic stem cells to treatment with select disease-modifying therapies. Researchers are testing the safety and efficacy of one-time autologous stem-cell transplantation in comparison to immunomodulators, such as rituximab and ocrelizumab, used to treat relapsing-remitting MS. Results of previous studies have shown that transplantation of hematopoietic stem cells alone may provide long-lasting and significant control of disease symptoms and progression.
Hematopoietic stem cell transplantation is not without risk of serious side effects. However, this risk is primarily at the time of transplantation and decreases thereafter in contrast to the risks with continued drug treatment. The clinical trial will involve collecting a patient’s own stem cells, performing myelo- and immunoablation with chemotherapy, then returning the hematopoietic stem cells to the patient to re-establish immune function. The hope is that the patient will then be free of the cells involved in the disease progression. The other patient group will receive one of the disease-modifying therapies, and all patients will be followed for 72 months.
The goal will be to determine the difference between the two groups in the time for a relapse to occur from the start of treatment, the immune status, disease state and severity, patient quality of life, and cost-effectiveness. The hope is that the data obtained will inform thebest treatment approach for patients with MS. The new information has the potential to improve the standard of care for this patient population.
HemaCare offers a wide-selection of high-quality human tissues and cellsfor leading cell-therapy research around the world.
First Direct Comparison of Stem Cells vs Drugs in MS Underway. (2020). Retrieved 14 April 2020, from https://www.medscape.com/viewarticle/923636