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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?

No, HSCT is considered experimental for the treatment of multiple sclerosis.

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 published scientific results about HSCT

• 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. A phase 3 trial of HSCT is now in planning stages. The Society is engaged with the team planning the trial and is encouraging quick action to design and launch the trial.
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 trial is in planning stages. 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).

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:

• An international clinical trial of this procedure, being led by Dr. Richard Burt of Northwestern University in Chicago, is ongoing at Northwestern University, Rush University Medical Center, University of Sao Paulo, Uppsala University and Sheffield Teaching Hospitals NHS Foundation Trust in individuals who have not responded to other disease-modifying therapies. THIS TRIAL IS NOT RECRUITING PARTICIPANTS. Dr. Burt and colleagues recently reported interim results of this ongoing study at a medical meeting.
 • Dr. Burt has recently begun a new phase 3 clinical trial at Northwestern to try to determine the optimal protocol for safety and benefit. Read more about this trial on
• 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 ​

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