Stem Cell Transplant More Effective for SPMS Than Medications: Study
More transplant recipients experience clinical improvements long term
A stem cell transplant more effectively slowed disability worsening in people with active secondary progressive multiple sclerosis (SPMS) than anti-inflammatory treatments and disease-modifying therapies (DMTs), a study has found.
More transplant recipients also experienced clinical improvements that were sustained after three and five years. After 10 years, disability scores improved slightly for transplanted patients compared with worse scores for patients on medications.
“Our results are encouraging, because while current treatments for secondary progressive MS have modest or small benefits, our study found stem cell transplants may not only delay disability longer than many other MS medications, they may also provide a slight improvement in symptoms,” Matilde Inglese, MD, PhD, study author and professor of neurology at the University of Genoa, in Italy, said in a press release from the American Academy of Neurology.
The findings were published in the journal Neurology, in the study “Hematopoietic Stem Cell Transplantation in People With Active Secondary Progressive Multiple Sclerosis.”
Stem cell transplant emerging as MS therapeutic approach
Most MS patients are first diagnosed with relapsing-remitting MS (RRMS), characterized by relapses, or worsening of new symptoms for at least 24 hours, followed by periods of remission, in which symptoms ease or go away entirely. Over time, some RRMS patients transition to SPMS, where symptoms steadily worsen, with or without relapses.
Autologous hematopoietic stem cell transplant (aHSCT) is an emerging therapeutic approach for MS that aims to reset the immune system to a healthy state without the autoimmune-related immune cells that drive MS.
The procedure involves harvesting hematopoietic stem cells, also known as blood stem cells, from the patient before their immune system is significantly eradicated by medications. Cells are then returned to the same patient — hence the name autologous — in the form of a stem cell transplant to repopulate the immune system with healthy cells.
“Hematopoietic stem cell transplants have been previously found to delay disability in people with relapsing-remitting MS, but less is known about whether such transplants could help delay disability during the more advanced stage of the disease,” Inglese said.
To find out, Inglese and colleagues at sites across Italy compared the effect of aHSCT with anti-inflammatory medications and DMTs on disability worsening in people with active SPMS.
our study found stem cell transplants may not only delay disability longer than many other MS medications, they may also provide a slight improvement in symptoms
The researchers collected data from the Italian Bone Marrow Transplantation Study Group and the Italian MS Registry. Their analyses included 79 people with active SPMS who received a stem cell transplant after their SPMS diagnosis and 1,975 SPMS patients who were treated with medications, matched in age, sex, and disability level to the transplant patients.
The medication group received either anti-inflammatory therapies, such as azathioprine, azathioprine, and cyclophosphamide, or approved MS DMTs, including interferon-based medications, glatiramer acetate (sold as Copaxone, among others), mitoxantrone, Gilenya (fingolimod), Tysabri (natalizumab), Aubagio (teriflunomide), Tecfidera (dimethyl fumarate), and Lemtrada (alemtuzumab).
Participants were assessed using Expanded Disability Status Scale (EDSS) scores over 10 years. Disability worsening was defined as a sustained increase in EDSS scores lasting at least six months.
In SPMS patients who underwent aHSCT, the time to a first disability worsening event was twice as long as those taking DMTs, with 62% of transplanted patients versus 46% of the medication group being free from sustained disability worsening at five years, a statistically significant difference.
Disability improvements found in more transplant patients vs medications group
Notably, significantly more patients in the aHSCT group experienced disability improvements, or an easing in symptoms, that were sustained for three years — 34.7% vs. 4.6% in the medications group. After five years, the proportion of transplant patients who maintained improvements was still greater (19% vs. 4%).
Over 10 years, EDSS scores dropped by 0.013 points per year (indicating improvement) after stem cell transplant compared to an increase of 0.16 points per year (worsening) for those on anti-inflammatory medications and DMTs.
“The use of AHSCT in people with active SPMS is associated with a slowing of disability progression and a higher likelihood of disability improvement compared to standard immunotherapy,” the researchers concluded.
“Our study shows that hematopoietic stem cell transplants were associated with a slowing of disability progression and a higher likelihood of disability improvement compared to other therapies,” said Inglese. “While these results are encouraging, they are not applicable to patients with secondary progressive MS who do not have signs of inflammatory disease activity [relapses].”
“More research is needed in larger groups of people to confirm our findings,” Inglese added.