National MS Society Supports Stem Cell Transplant for Select, Aggressive RRMS

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by Marta Figueiredo, PhD |

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The National Multiple Sclerosis Society supports the use of a patient-derived bone marrow transplant to treat people with very aggressive relapsing-remitting multiple sclerosis (RRMS) who responded poorly to disease-modifying therapies (DMTs).

This position in line with a recent set of society recommendations on how and in whom autologous hematopoietic stem cell transplant (AHSCT) should be performed. Published last year, they were based on a systematic review of recent clinical evidence related to AHSCT use in MS patients.

“Because of effective clinical research, we now have strong evidence that autologous hematopoietic stem cell transplantation is an important treatment option for some people with MS under specific circumstance,” Cyndi Zagieboylo, the National MS Society’s president and CEO, said in the announcement.

Still, the society recommends that patients discuss treatment options with their MS specialist or physician to determine the best approach for their disease.

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The role of the National MS Society “is to communicate what is known so that effective treatments can be accessed,” Zagieboylo said, adding that “this includes supporting people with MS and their healthcare providers in considering options, ensuring that scientifically rigorous research explores unanswered questions, and advocating for affordable access and equity of healthcare.”

“We are committed to our mission: We will cure MS while empowering people affected by MS to live their best lives,” Zagieboylo added.

AHSCT is an intensive, experimental treatment approach that uses a person’s own, or autologous, healthy blood cell progenitors — also known as hematopoietic stem cells — to stop MS-related immune attacks on the brain and spinal cord.

Hematopoietic stem cells are first collected from a patient’s bone marrow, and then infused back to the patient after a fairly aggressive chemotherapy regimen is given to kill their immune cells.

In aiming for a total reset of a person’s immune system, this one-time treatment has the potential to significantly slow or halt MS progression.

Several studies of AHSCT report that it can prevent disability progression and relapses over the long term in people with relapsing MS forms who have not benefited from DMTs. Data from clinical trials and real-life studies also support its superiority over DMTs, including high-efficacy therapies such as Lemtrada (alemtuzumab).

In addition, the estimated total cost of a one-time stem cell transplant is roughly $150,000, while DMTs are associated with a mean wholesale cost of at least $80,000 each year.

After reviewing AHSCT’s clinical evidence in MS patients, the society concluded that the approach may be a safe and effective option for RRMS patients younger than age 50, who either show new brain lesions and/or clinical relapses despite treatment with a high-efficacy DMT or are unable to use such therapies.

Ideal candidates also have a disease duration of less than 10 years.

The approach does not come without risks, the society noted, and older age, greater  disability,  and certain health conditions or comorbidities, such as heart or lung disease, are associated with a greater risk of serious complications, including infections.

As such, patients undergoing the stem cell transplant need to be closely followed and given proper care after the procedure.

According to the National MS Society, it is crucial that AHSCT is performed at specialized centers certified either by the Foundation for Accreditation of Cellular Therapy in the U.S., or by the European Society for Blood and Marrow Transplantation for Europe.

Currently, a Phase 3 trial called BEAT-MS (NCT04047628) is assessing AHSCT’s safety, effectiveness, and cost-effectiveness against “best available” DMTs in up to 156 adults. Eligible patients need to have severe relapsing MS, and failed to respond to two therapy lines in the three previous years.

Patients are being recruited at sites across the U.S., with a single U.K. site expected to open; more information is available here. The trial’s top-line data are expected by October 2026.

Recent evidence also suggests that AHSCT is more effective than DMTs at slowing disease progression in people with active secondary progressive MS. But well-controlled clinical trials are needed to confirm its efficacy in this patient group.

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