Research News: Stem Cell Transplantation
In an online report from BBC News appearing in March 2018, results of an international trial using hematopoietic stem cell transplantation (HSCT) were given. They state that more than 100 individuals with relapsing-remitting MS (RRMS) participated in this trial, which took place in hospitals located in the United States, England, Sweden, and Brazil. HSCT involves collecting stem stems from a patient’s own blood, “wiping out” (or “resetting”) the individual’s immune system with high-dose chemotherapy, and then returning the preserved stem cells back to the patient.
According to the report, only one relapse occurred among the stem cell group after one year, compared with 39 in the group given a “drug treatment.” After an average follow-up of three years, the stem cell transplant had failed in three out of 52 patients (6 percent), compared with 30 of 50 (60 percent) in the control group. The report also stated that individuals who received the HSCT experienced a reduction in disability, while individuals in the drug group experienced worsening symptoms.
In February 2018, the results of a study using autologous mesenchymal stem cell-derived neural progenitors (MSC-NPs) were published in EBioMedicine. The study took place at Tisch MS Research Center of New York, where they found evidence that a stem cell-based treatment may reverse disability in progressive multiple sclerosis (MS). This type of stem cell is derived from the patient’s own bone marrow, and with this form of stem cell transplant, chemotherapy is not used to reset the immune system. Instead, these cells are returned to the individual via intrathecal (into the spinal canal) injection, with the aim to potentially repair damaged nerves and improve function.
Twenty individuals with progressive MS took part in this study. As determined by improvements on the expanded disability status scale (EDSS), the participants demonstrated an improvement in disability. In addition, 70 percent of the participants saw an improvement in muscle strength, and 50 percent experienced improved bladder function. The report also states that all 20 participants completed the 60 scheduled treatments without any serious adverse effects; minor adverse events included transient fever and mild headaches.
MSAA’s Chief Medical Consultant Dr. Jack Burks explains, “Each year we learn of new trials with stem cells and many successful patient stories. The fact that many scientists from many countries are working together is fabulous. I am excited with these pilot studies, however, some deaths occur – too often without appropriate medical caution. We need to find more uniform scientific protocols that reduce adverse events – and we are getting there. While I consider it to be very promising research, stem cell transplant should not be considered as the standard first line of care.”
Siponimod Reduces Disability Progression in SPMS
In March 2018, Novartis announced the results of the Phase III Expand study, which compared the safety and effectiveness of siponimod to placebo in patients with secondary-progressive multiple sclerosis (SPMS). Siponimod is an investigational, selective modulator of specific subtypes of the S1P receptor, which prevents certain damaging immune-system cells from entering the central nervous system and leads to anti-inflammatory effects. It also has the potential to modulate damaging cell activity and reduce the loss of neurological function.
Taken orally once per day, siponimod showed significant reductions in the risk for disability at three and six months, and also reduced annual relapse rates (in those still experiencing relapses) and disease activity as shown on MRI – including a reduction in brain-volume loss. The Expand study had 1,651 participants from 31 countries, all with SPMS. Novartis plans to submit siponimod in 2018 to the United States Food and Drug Administration (FDA) for approval as the first disease-modifying therapy to delay disability progression in SPMS.
Zinbryta® Withdrawn from Marketplace
On March 2, 2018, Biogen and AbbVie announced the voluntary withdrawal of their MS medication, Zinbryta® (daclizumab), due to new safety concerns. At least 12 cases of inflammatory encephalitis and meningoencephalitis – both forms of brain inflammation – have been reported in Europe. Earlier issues with liver failure have also played a role in the decision. People who are currently taking this medication should contact their healthcare provider as soon as possible to discuss discontinuing the medication and to learn about different treatment options. Anyone with questions or concerns about the withdrawal should contact Biogen’s service center at (800) 456-2255 or their website at www.zinbryta.com. For additional research news items, please visit mymsaa.org.
Written by Susan Wells Courtney, MSAA Senior Writer
Reviewed by Dr. Jack Burks, MSAA Chief Medical Consultant