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, 10 (1), 68-76

Hematopoietic Stem Cell Therapy for Multiple Sclerosis: Top 10 Lessons Learned


Hematopoietic Stem Cell Therapy for Multiple Sclerosis: Top 10 Lessons Learned

Harold L Atkins et al. Neurotherapeutics.


Reports from more than 600 hematopoietic stem cell transplants (HSCT) have appeared in the medical literature for the last 1 and one-half decades. The patient's own stem cells are harvested and stored temporarily while high doses of chemotherapy and biologics are used to destroy the auto-destructive immune system. The immune system is regenerated from the infused autologous hematopoietic stem cells. Increasing clinical experience has refined patient selection criteria and management in the peri-transplant period leading to a reduction in treatment-related complications. HSCT, when used to treat patients with aggressive highly active multiple sclerosis, can reduce or eliminate ongoing clinical relapses, halt further progression, and reduce the burden of disability in some patients, in the absence of chronic treatment with disease-modifying agents. The top 10 lessons learned from the growing experience using HSCT for the treatment of multiple sclerosis are discussed.


Fig. 1
Fig. 1
Once an appropriate candidate is identified, autologous hematopoietic stem cells (HSC) are collected (a). HSC may be collected directly from the bone marrow through multiple aspirations performed under regional or general anesthesia. Alternatively, HSC may be mobilized from the bone marrow into the circulation using chemotherapy and/or hematopoietic growth factors. HSC are then collected by leukopheresis. The product can be processed to remove contaminating immune cells and them be cryopreserved or it can be cryopreserved without further manipulation. Two to 6 weeks later, the patient may undergo HSCT (b). Chemotherapy with or without immune depleting biologic agents are administered to destroy the immune system. The cryopreserved HSC are rapidly thawed in a water bath and infused intravenously through a central venous catheter. The patient is receives supportive care during the acute phase of the transplant, when potentially the most serious side-effects may occur. Hematopoietic reconstitution generally occurs 10–14 days after HSCT. Immune reconstitution and full recovery from the chemotherapy may take 3 to 6 months

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