Hematopoietic Stem Cell Transplant (HSCT) for Multiple Sclerosis?

Hematopoietic Stem Cell Transplant (HSCT) for Multiple Sclerosis?

What You Need to Know:

Multiple sclerosis (MS) is an autoimmune disease of the central nervous system that is initially characterized by demyelination and progresses to neuronal loss and axonal destruction. More than 2.3 million people worldwide suffer from MS. It affects two to three times more women than men and diagnosis is usually made between the ages of 20 and 50.

Common presenting symptoms include weakness or numbness in one or two extremities, constricting tightness around the limbs or trunk, heavy legs that are difficult to control, an electric shocking sensation with flexion of the neck (Lhermitte sign), vision loss or optic neuritis, diplopia, and vertigo. Traditional treatments for relapsing-remitting multiple sclerosis (RRMS) include disease-modifying therapies (DMT) administered orally, via injection, or intravenously.

Researchers have been experimenting with hematopoietic stem cell transplantation (HSCT) as a treatment for RRMS. In this process autologous bone marrow are collected and stored. High-dose chemotherapy is then administered over a five-day period to destroy the defective immune system. Following chemotherapy, the stored stem cells are infused back into the body where they travel to the bone marrow and gradually rebuild the immune system, including damaged nerve cells.

Findings from this preliminary trial are encouraging as patients randomized to the HSCT treatment group reported a delay or cessation in disease symptoms. Less than 10% of patients who underwent HSCT experienced disease progression compared with more than 75% of patients who were randomized to the traditional DMT group and reported worsening symptoms. The HSCT process appears to be effective because once the immune system is rebooted in an environment free from inflammation, the body is better able to mend itself to some degree. Serious risks associated with HSCT include infection, infertility, and possible death.

Read more about multiple sclerosis:

Harrison’s Principles of Internal Medicine, 20e: Chapter 436. Multiple Sclerosis

Principles and Practice of Hospital Medicine, 2e: Chapter 212. Multiple Sclerosis

Adams and Victor's Principles of Neurology, 10e: Chapter 36. Multiple Sclerosis and Other Inflammatory Demyelinating Diseases