Stem cell transplant outperforms Lemtrada in MS treatment: Study

A stem cell transplant was found to be better than Lemtrada (alemtuzumab) for lowering disease activity and slowing disability worsening in people with highly active relapsing forms of multiple sclerosis (MS), a small study in Lithuania showed.

While both treatments were similarly effective in the first couple of years, patients given the transplant began to see disability improvements later on; in turn, disability showed signs of accumulating in those who received Lemtrada.

“Given these findings, clinical trials evaluating efficacy of [stem cell transplant] in severe disability in MS patients should be considered,” the researchers wrote.

The study, “Autologous Hematopoietic Stem Cell Transplantation Is Superior To Alemtuzumab In Patients With Highly Active Relapsing Multiple Sclerosis And Severe Disability,” was published in Multiple Sclerosis and Related Disorders.

Some relapsing MS patients have what’s considered highly active disease, characterized by more frequent and severe relapses with incomplete recovery, as well as rapidly accumulating disability and MRI lesions.

These patients may require more aggressive treatment approaches. This may include the use of highly effective disease-modifying therapies (DMTs) such as Lemtrada, which works to deplete populations of immune cells implicated in driving inflammation in MS.

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Accumulating evidence indicates that an autologous hematopoietic stem cell transplant (aHSCT), or simply referred to as a stem cell transplant, may be able to lower disease activity and slow disability progression for patients with highly active disease.

Essentially, this approach aims to reset the immune system and repopulate it with healthy cells that won’t drive inflammatory attacks on the body’s own brain and spinal cord, as is seen in MS.

Briefly, a person’s own blood cell precursors (hematopoietic stem cells) are collected from the blood then infused back into the patient after a round of chemotherapy to wipe out existing cells and make room for new ones not primed to attack the body’s tissues.

Although the individual procedures and medications used in a stem cell transplant are approved in the U.S., aHSCT is not specifically approved for MS in the country. However, the National MS Society has supported it for patients with highly active disease who haven’t responded well to other therapies.

While a few small studies indicated that aHSCT may be superior to Lemtrada for treating patients with very active disease, evidence is still generally lacking about how the two approaches compare for treating this population in the long term.

Real-world outcomes examined in 50 MS patients with highly active disease

In this study, the researchers examined real-world outcomes from 50 relapsing MS patients with highly active disease after starting treatment with aHSCT (31 people) or Lemtrada (19 people) at a clinic in Lithuania between 2014 and 2021.

Those undergoing stem cell transplant had higher disability levels and more often had a history of high-efficacy DMT use than those in the Lemtrada group when starting treatment (baseline). Patients were followed for a median of 4.3 years (aHSCT) and 4.9 years (Lemtrada).

Over the period, individuals on Lemtrada had an overall higher annual relapse rate and faster disability progression, as assessed by the Expanded Disability Status Scale (EDSS), than those given the stem cell transplant.

The proportion of patients on Lemtrada achieving no evidence of disease activity (NEDA-3) — defined as the absence of disease relapses, disability progression, and MRI activity — in the first two years was 44%, significantly lower than the stem cell therapy group (79%).

No significant differences in NEDA-3 rates were observed beyond the second year.

More detailed analyses showed that that each individual component of NEDA did not significantly differ between the two groups in the first two years after treatment onset. However, from years three to five, aHSCT was found to outperform Lemtrada in all three of these aspects.

As a result, at five years after treatment onset, significantly more patients given aHSCT were free of MRI activity (69.6% versus 95.7%), relapses (54.5% versus 75.1%), and disability worsening (57.1% versus 90.9%) than with Lemtrada.

EDSS scores improved in the stem cell transplant group in the first two years and continued to do so in years three to five, whereas disability tended to stabilize then start accumulating after year two in the Lemtrada group.

“Importantly, our data indicate that HSCT not only halts disability worsening but might also improve disability measures,” the researchers wrote. “[Lemtrada] may halt disability progression early in the course of highly active RMS, but disability starts accumulating in later stages.”

PIRA rates low, comparable in both groups

Rates of disability progression independent of relapses, also known as PIRA, were low and comparable between the groups.

Overall, the researchers believe stem cell transplant, “might offer a broader ‘treatment window’ with beneficial effects on disease progression and disability reversal in persons with both active and progressive forms of disease and a variable degree of accumulated disability.”

Still, they noted that due to variations in national policies about the use of aHSCT and its availability, it is currently not feasible as a treatment early in the disease course.

Nevertheless, the findings establish “grounds for more clinical trials of HSCT on disease stabilization and progression reversal in patients with severe disability.”