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Bone Marrow Stem Cell Transplant – HSCT


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What is HSCT for multiple sclerosis?

HSCT (Hematopoietic Stem Cell Transplantation) attempts to “reboot” the immune system, which is responsible for damaging the brain and spinal cord in MS. In HSCT for MS, hematopoietic (blood cell-producing) stem cells, which are derived from a person’s own (scientifically referred to as “autologous”) bone marrow or blood, are collected and stored, prior to depleting much of the immune system using chemotherapy drugs. Then the stored hematopoietic stem cells are reintroduced to the body. The new stem cells migrate to the bone marrow and over time reconstitute the immune system. 

Is HSCT an FDA-approved therapy option for people with MS?

The medications and procedures used in HSCT are already approved by the FDA. Publication of the outcomes from well controlled clinical studies of HSCT therapy will encourage greater acceptance and use by the medical community.

What is the idea behind autologous HSCT for MS?

The goal of HSCT is to reset the immune system and stop the inflammation that contributes to active relapsing MS.

​What is involved in the HSCT procedure?

While the general approach is the same, there are different treatment protocols that vary depending on the medical center and doctors performing the procedure. In general, these are some of the steps involved:
  1. A person undergoing HSCT to treat MS is given some form of chemotherapy, usually by infusion in the vein, for up to 10 days (usually as an outpatient) to stimulate the production of bone marrow stem cells and promote their release into the blood. Then some blood is drawn from a vein and the stem cells in the blood are stored for later use.
  2. Then the individual is usually hospitalized, and given a powerful mix of chemotherapies for up to 11 days to kill or suppress immune cells throughout the body.
  3. The stored stem cells are then infused into the bloodstream through a vein.
  4. The individual is usually given medicines such as antibiotics to help combat infection.
  5. The person remains in the hospital for an additional period of time while the immune system begins to rebuild itself. Sometimes individuals are discharged from the hospital in two to four weeks, but this can be longer. In a recently published Canadian study, the hospital stay after transplantation lasted 10 to 160 days, depending on any side effects experienced.
  6. The immune system gradually rebuilds itself within 3 to 6 months.

Read more frequently asked questions about HSCT and MS

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Latest News About HSCT

Ongoing Research in HSCT

Additional research is focusing on figuring out who might benefit from this procedure and how to reduce its risks. HSCT is being investigated in Canada, the United States, Europe and elsewhere. For example:

 • A multicenter phase 3 clinical trial of HSCT (“BEAT-MS”) began in 2020 in Cleveland, Seattle and up to 19 sites across the country. This study is recruiting participants in Cleveland and will begin recruiting at additional sites as they get their paperwork in order. The Society has been engaged with the team planning the trial and plans to help with recruitment as the sites begin screening potential participants. Read more about this trial on the BEAT-MS website

 • A clinical trial is getting underway at medical centers in Denmark, Netherlands, Norway and Sweden. The trial is testing treatment with HSCT compared with alemtuzumab in people with active relapsing-remitting MS. Read more about this trial on 

 • A phase 3 trial at Northwestern Medical in Chicago launched by Dr. Richard Burt to determine the optimal protocol for safety and benefit is ongoing and no longer recruiting participants. According to Northwestern, Dr. Burt will be going on a planned research sabbatical at the end of 2019.
Jennifer Moulson, diagnosed in XXXX

One woman’s journey with HSCT

Latest published scientific results about HSCT

• Researchers from Florence, Italy report on a small, open-label study involving 26 people with moderate to severe secondary progressive MS who underwent HSCT. Most showed signs of clinical or MRI inflammatory activity in the year prior to the procedure. Five years after the procedure, 42% of participants were stable with no further progression of disability that was reduced to 30% 10 years after transplant. No relapses or inflammatory activity occurred on MRI scans after treatment. These results suggest that HSCT might be appropriate in a subgroup of people with SPMS that have significant inflammatory activity as measured by MRI. Further study in larger numbers are needed to understand who among those with secondary progressive MS might benefit from HSCT. Read a summary of the findings or an abstract in The MS Journal.

• In January 2019, an international team of researchers led by Richard K. Burt, MD (MD (going on research sabbatical late 2019 from Northwestern University, Chicago, IL) published results of the first randomized, control trial of bone marrow stem cell transplant (HSCT) in people with aggressive relapsing-remitting MS. They enrolled 110 people whose MS was not controlled by available disease-modifying therapies. Half received immunosuppressant therapy followed by hematopoietic (blood cell-producing) stem transplant. The other half were switched to a different disease-modifying therapy. Significantly fewer people experienced MS progression in the group that underwent HSCT, compared with the group who were switched to a different MS disease-modifying therapy. There were no deaths or life-threatening adverse events in either group. The investigators consider this study to be preliminary and recommend that further research is needed to confirm these findings and to determine longer-term outcomes and safety. Read the summary or read the abstract in JAMA.

• In December 2018, Drs. John Moore, David Ma (St. Vincent’s Hospital, Darlinghurst, NSW, Australia) and colleagues reported results of a small clinical trial of HSCT conducted at a single medical center in Australia. This trial enrolled 35 people with relapsing-remitting MS or secondary progressive MS whose disease had not responded well to disease-modifying medications. There was no control group or blinding; all participants underwent the HSCT procedure. The team reported on results after following participants from 12 to 66 months after transplantation. After 12 months, 82% remained free of relapses, MRI-detected new or enlarging lesions, and progression (called “Event-Free Survival” or EFS). At two years after transplant, 65% of the group had EFS, and at three years 60%. EFS was better in those who had relapsing MS. Of 8 who experienced MS progression after transplantation, 2 had relapsing-remitting MS and 6 had secondary progressive MS. Twelve of thirteen whose disability scores improved after transplantation had relapsing-remitting MS. At this center, which has a long experience with bone marrow transplants, there were no transplant-related deaths. Many experienced complications expected from the chemotherapy cocktail (called “BEAMS”) used to deplete their bone marrow cells in preparation for the transplant. Read a summary or read the abstract in the JNNP.

• In April 2017, researchers in Italy combined and analyzed results from 15 previously published studies of HSCT (Hematopoietic Stem Cell Transplantation) involving 764 people with various forms of MS. They found that overall, the procedure showed a significant benefit against disease activity and progression. Two years after transplantation, about 83% of all participants had not progressed; overall, studies involving more people with relapsing-remitting MS had lower progression rates. The pooled results showed an overall transplant-related mortality rate of 2.1%. There were fewer deaths in later studies as researchers gained more experience with the procedure. Read a summary of more details here or the abstract in Neurology

​• In February 2017, results of an international study were published. The study evaluated long-term outcomes from HSCT in 261 people with different forms of MS. The transplants took place between 1995 and 2006, with a follow-up period of up to 16 years. Several different transplant protocols were followed. After 5 years, 46% still had not experienced any progression or worsening of symptoms, including 73% of those with relapsing MS and 33% of those with secondary progressive MS. Eight deaths (2.8%) occurred within 100 days of the transplant. Most of these occurred during the early development of the procedure; improvements in patient selection and transplant techniques have significantly reduced the mortality. Those with the best outcomes tended to be younger, had relapsing MS, lower accumulation of disability and had used fewer MS therapies prior to the transplant procedure. Additional research is needed to better understand who might benefit from this procedure and how it compares to the benefits of powerful immune-modulating therapies now available. 
Read a summary of the results or the paper in JAMA Neurology

• In February 2017, results were published from a multi-center, 5-year trial called the HALT MS Study. It tested HSCT in 24 people with MS and active relapsing-remitting disease that was not controlled by disease-modifying medications. Results suggest that after five years, 69.2% of participants experienced no new disease activity after the procedure and did not need disease-modifying therapies to control their disease. All participants experienced severe and/or life threatening adverse events. Most of these occurred within the first 30 days after transplant and were related to low white blood cell counts and infections. This trial, which was funded by the National Institutes of Health, is an important addition to research needed to determine whether this approach to stem cell transplantation is safe and effective in people with MS. A larger, phase 3 study is in the process of launching (see"Ongoing Research in HSCT" above).  Read a summary of the results or the paper in Neurology 

• In June 2016 researchers in Canada published results of a long-term HSCT trial involving 24 people with aggressive relapsing-remitting MS whose disease was not controlled with available therapies. Three years after the procedure, 70% remained free of disease activity, with no relapses, no new MRI-detected inflammatory brain lesions, and no signs of progression. None of the surviving participants experienced clinical relapses or required MS disease-modifying therapies to control their disease, and 40% experienced reductions in disability. One participant died and another required intensive hospital care for liver complications. All participants developed fevers, which were frequently associated with infections, and other toxicities. Read more about this study

• In October 2015, researchers at the University of Genoa and other institutions in Italy reported on a small trial of HSCT in seven people with very active relapsing-remitting MS that was not controlled with MS disease-modifying therapy. They underwent a “low-intensity lympho-ablative regimen” in which the immune system was suppressed but not completely depleted before the stem cell transplant as an approach to reducing toxicity. The investigators did MRI scans (for 3 years) and clinical evaluations (for 5 years). They found dramatic reductions of MRI-detected inflammation after the procedure, but did not achieve complete absence of inflammation. After 5 years, two participants remained stable, one significantly improved, and four had mild disease progression. One experienced a relapse after treatment. No severe side effects occurred. The authors conclude that the low-intensity regimen they used was not sufficient to treat aggressive MS. Read an abstract from the paper (Multiple Sclerosis 2015 Oct;21(11):1423-30)
• In January 2015, doctors at Northwestern University published their10-year experience of treating people with HSCT. The report included 123 people with relapsing-remitting MS and 28 with secondary-progressive MS. Their method is “nonmyeloblative” HSCT, in which the immune system is suppressed but not completely depleted before the stem cell transplant. Individuals were followed from 6 months to 5 years, or an average of 2.5 years. The EDSS disability scores improved, compared to pretreatment, by one point or more in 64% of those followed out to year 4. Relapses and MRI-detected disease activity were also reduced. In evaluating which type of individuals benefited from the therapy, the doctors suggested that people with relapsing-remitting MS who had had MS for ten years or less showed improvements in their disability scores, whereas those with secondary-progressive MS or disease duration greater than ten years did not show improvements on their disability scores. They reported no treatment-related deaths or serious infections. ITP (immune-mediated thrombocytopenia), a potentially serious bleeding disorder, developed in 7 people, and thyroid disorders developed in 7 people.  Read a summary of their results or the paper in JAMA (Published online January 20, 2015).

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