Bone marrow-formed blood cells called hematopoietic stem cells (HSCs) have been shown to delay disease when transplanted into patients with relapsing-remitting multiple sclerosis (RRMS).
Few studies have focused on autologous hematopoietic stem cell transplantation (HSCT) for the treatment of secondary progressive multiple sclerosis (SPMS), a more advanced stage of the disease. Replace diseased cells.
A new study published online in neurologyThe American Journal of Neurology, the medical journal of the American Academy of Neurology, reports that patients with active SPMS who have undergone stem cell transplantation (either continue to experience relapses of multiple sclerosis or develop new lesions, as seen on MRI). (meaning ) had a slower accumulation of disability than those who did. Taking anti-inflammatory disease-modifying therapy (DMT).
Most people with relapsing-remitting MS are initially diagnosed with relapsing-remitting MS, which is characterized by flares of symptoms followed by periods of remission, but many people with relapsing-remitting MS eventually progress to secondary progressive MS. To do. Rather, it is a steady, slow deterioration of the disease.
Studies Show Promising Results
“Current treatments for secondary progressive multiple sclerosis have modest to small advantages, but stem cell transplantation not only delays disability more than many other multiple sclerosis treatments, but also reduces symptoms. Our results are promising because we found that it may also provide improvement.Study author at the University of Genoa, Italy, and neurology, radiology, and neuroscience at the Icahn School of Medicine, Mount Sinai, New York City study author Mathilde Inglese, M.D., Ph.D., a professor at
A retrospective analysis involved 79 people with active secondary progressive MS who underwent stem cell transplantation and 1,975 people from an Italian MS registry who were treated with MS medications. People in the medication group take anti-inflammatory disease-modifying therapies (DMTs) such as beta-interferon (Avonex, Betaseron), azathioprine (Azason), glatiramer acetate (Copaxone, Glatopa), mitoxantrone (Novantron), and fingolimod (Gilenya). received. , natalizumab (Tysabri), methotrexate (Rheumatrex), teriflunomide (Aubagio), cyclophosphamide (Cytoxan), dimethyl fumarate (Tecfidera), and alemtuzumab (Lemtrada).
Not only does it delay disability, it improves EDSS scores
Disability in this study was measured on the Extended Disability Status Scale (EDSS), a 10-point scale. For example, a score of 6 means that a cane or brace must be used intermittently or on one side whether or not you rest about 100 meters. A score of 6.5 is defined as the need to use a cane or brace on both sides at all times to walk approximately 20 meters without rest.
Five years into the study, researchers found that 62% of those who received stem cell transplants experienced no worsening of their MS impairment compared to 46% of those who took the drug.
At 5 years, those who had stem cell transplants were more likely to see lasting improvement over time, with 19% having less disability than at the start of the study, compared to taking medication. Only 4% did.
At age 10, disability scores decreased by an average of 0.01 points per year in those who received stem cell transplants. This means that the fault has been remedied. On the other hand, the average score of a person taking the drug increased by 0.16 points a year. This means that their disability has worsened.
Larger studies are needed to confirm benefits
“Proportion of AHSCT Patients [autologous hematopoietic stem cell transplants] said Daniel Ontaneda, M.D., Ph.D., of the Mellen Multiple Sclerosis Center at the Cleveland Clinic in Ohio. “However, this data should be obtained with caution as it is all observational and not made in the context of the gold standard of randomized clinical trials. Although we tried to confirm that, some residual confounders may still be present.”
While the findings so far are encouraging, Dr. Inglese, who is also a member of the American Academy of Neurology, says more studies with larger groups of people are needed to confirm the findings. “This finding does not apply to patients with secondary progressive MS who have no evidence of inflammatory disease activity,” she added.
The study did not include people taking the multiple sclerosis drugs siponimod (Mayzent), cladribine (Mavenclad), ocrelizumab (Ocrevus), ofatumumab (Kesimpta), or rituximab (Rituxan). I did.
HSCT is aggressive MS treatment
Christopher Rock, M.D., clinical associate professor of neurology and neuroscience at Stanford University Medical Center in California, said: Stem cell transplantation is an aggressive form of MS treatment and is not without risks, so it is usually considered only when other treatments fail.
“Stem cells are harvested by passing the blood through a machine that separates the blood into its various components, after which the stem cells are collected,” said Dr. Rock, who was not involved in the research. “After the stem cells are saved, patients are given potent chemotherapy drugs that ‘remove’ or ‘knock out’ remaining bone marrow cells, including autoreactive cells thought to cause damage to myelin. . [insulating layer around nerves] in MS.
“The immune system heals by putting stem cells back in. It’s like hitting a reset button,” Lock adds.
MS Society UK warns that chemotherapy can have serious side effects such as hair loss, fever, nausea and infertility. The risk of future infections will also increase. If a person already has a lot of nerve damage due to multiple sclerosis, chemotherapy may do more harm than good. There is a nature.
HSCT is expensive and time consuming, but sometimes very effective
The porting process is also expensive and time consuming. The National MS Society estimates that her average total cost of AHSCT for an inpatient is her $150,000, although costs vary within the United States. Out-of-pocket costs for surgery are generally much lower for patients with insurance.
The association also says that the initial stem cell preparation and collection takes five to 15 days, followed by three weeks of hospitalization to prepare the immune system for the transplant, perform the stem cell transplant, and allow recovery time. increase.
One of the major advantages of AHSCT is long-term control of MS.
“This is clearly a long-acting treatment that may reduce the need for conventional immunomodulatory drugs used in multiple sclerosis,” said Ontaneda, who was not a study contributor. says Dr. “However, some patients show disease activity despite AHSCT.” It’s an area of research.”
