The Latest on HSCT Research

About 1 million people in the United States live with multiple sclerosis (MS). The disease attacks the central nervous system, leading to numbness and tingling, blindness, and paralysis. There is no cure, but 1 treatment for relapsing MS is showing promise.1

Hematopoietic stem cell transplantation (HSCT) is undergoing clinical trials in the United States and Europe to test its effectiveness in treating relapsing MS. Smaller trials have already shown that HSCT can reduce symptoms, but larger-scale studies are needed. The trials underway seek to address this.2

What is HSCT?

When a person has MS, their immune system attacks the brain and spinal cord, causing neurological damage. HSCT reboots the immune system by depleting it using drugs created for chemotherapy and regenerating it with stem cells.3

When a person undergoes HSCT, they are given a drug that stimulates bone marrow production. Then, blood is drawn, and stem cells are removed from the blood and stored away.3

In a hospital or clinical setting, the person is given chemotherapy drugs that work to kill off their natural immune system. The stored stem cells are then returned to their body through a vein.3

The person will need to be isolated, often in the hospital, for some time while their immune system rebuilds. This may take weeks. The immune system will take 3 to 6 months to fully rebuild.3

Recommendations from the National Multiple Sclerosis Society

The treatment holds enough promise that the National Multiple Sclerosis Society released recommendations for HSCT treatment in October 2020. Their guidelines are that the best candidates for HSCT:1

  • Are less than 50 years of age
  • Have had MS for less than 10 years
  • Have relapsing MS and have had breakthrough disease activity, despite treatment with a highly effective disease-modifying therapy

They recommend that people who meet these criteria who would like to try HSCT enroll in a clinical trial, if at all possible. The committee also recommended that a single registry be set up to track people’s long-term progress following HSCT to develop treatment in the safest and most effective way possible.1

Clinical trials underway

As of late 2021, there are a few clinical trials currently taking place for those interested in HSCT.

BEAT-MS is a multicenter, phase 3 trial being conducted at Duke University through the Immune Tolerance Network. It is a randomized, single-blinded trial that compares HSCT with 1 of 4 current MS treatments: natalizumab, alemtuzumab, ocrelizumab, or rituximab.4

BEAT-MS will enroll 156 people with relapse-remitting MS who have had at least 2 inflammatory flares in the last 2 years or for whom disease-modifying therapy has been unsuccessful.4

In Europe, a clinical trial testing HSCT against alemtuzumab for treatment of MS is underway with 100 people. The trial, which is being done at Norway’s Haukeland University Hospital, requires people be between the ages of 18 and 50 and have experienced significant inflammatory disease activity in the past year despite treatment with disease-modifying therapy.5

Published research on HSCT

In December 2020, a team in Lithuania published results after studying the effects of HSCT on cognition and physical disabilities. Thirteen people underwent HSCT and were followed for 24 months. The extent of disability was measured using the Expanded Disability Status Scale (EDSS). EDSS improved for 11 people in the study. However, 2 people had a relapse in the first year, and 3 had a relapse in the second year.3

In Italy, a study with 26 people showed that 42 percent of participants treated with HSCT had no progression of their disability 5 years after treatment. However, that number fell to 30 percent when participants were followed-up 10 years after treatment.3

A 2018 study in Australia reported results of a clinical trial of 35 people treated with HSCT. Twelve months after treatment, 82 percent had no relapses, new or enlarging lesions, or progression of their MS. That number fell to 65 percent 2 years after HSCT, and 60 percent after 3 years.3

To search for a clinical trial, visit ClinicalTrials.gov.

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