Position statement from American Society for Blood and Marrow Transplantation… “AHSCT is an efficacious and safe treatment for active relapsing forms of MS to prevent clinical relapse, magnetic resonance imaging-detectable lesion activity, and worsening disability and to reverse disability without unexpected adverse events. Based on the available evidence, the ASBMT recommends that treatment-refractory relapsing MS with high risk of future disability be considered a “standard of care, clinical evidence available” indication for AHSCT.”

Read the full statement here: https://www.bbmt.org/article/S1083-8791(19)30139-9/fulltext

The long-awaited JAMA publication of Dr. Burt’s latest clinical trial evidences that: “mean EDSS scores decreased (improved) from 3.38 to 2.36 in the HSCT group and increased (worsened) from 3.31 to 3.98 in the DMT group (between-group mean difference, −1.7; 95% CI, −2.03 to −1.29; P< .001). There were no deaths and no patients who received HSCT developed nonhematopoietic grade 4 toxicities (such as myocardial infarction, sepsis, or other disabling or potential life-threatening events” (Burt, Balabanov, Burman, et al., 2019). Translation: at the study’s one-year point, the HSCT patients IMPROVED and the control group (who remained on DMTs) DECLINED on the study measures.

Full citation: Burt RK, Balabanov R, Burman J, et al. Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis: A Randomized Clinical Trial. JAMA. 2019;321(2):165–174. doi:10.1001/jama.2018.18743

For the latest information about how to find HSCT clinical trials, be sure to visit ClinicalTrials.gov

Media

To gain information about HSCT, you can watch this presentation of Dr. Burt being honored with the esteemed Van Bekkum award at the 44th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) in March 2018 held in Lisbon, Portugal.

More information about this latest study can be seen in Dr. Burt’s Day 1 and Day 3 presentations at the Annual Meeting of the European Society for Blood and Marrow Transplantation, March 2018 held in Lisbon, Portugal.

The presentation by Dr. Burt at the Cellular Horizons: How Science, Technology, Information and Communication Will Impact Society conference in Vatican City, April 28-30, 2016, includes testimonial from two patients.

If you know of additional resources not on the list, please share a link or citation through the Contact page.

Scholarly References

Atkins, H.L., Bowman, M., Allan, D., Anstee, G., Arnold, D.L., Bar-Or, A., Bence-Bruckler, I., Birch, P., Bredeson, C., Chen, J., Fergusson, D., Halenny, M., Hamelin, L., Huebsch, L., Hutton, B., Laneuville, P., Lapierre, Y., Lee, H., Martin, L., McDiarmid, S., O’Connor, P., Ramsay, T., Sabloff, M., Walker, L., Freedman, M.S., (2016). Immunoablation and autologous haemopoietic stem-cell transplantation for aggressive multiple sclerosis: A multicentre single-group phase 2 trial. Lancet, 388(10044):576-85.

Bakhuraysah, M.M., Siatskas, C., & Petratos, S., (2016). Hematopoietic stem cell transplantation for multiple sclerosis: Is it a clinical reality? Stem cell research & therapy, 7(12). doi:10.1186/s13287-015-0272-1

Boster, A., Hreha, S., Berger, J.R., Bao, F., Penmesta, R., Tselis, A., Endress, C., Zak, I., Perumal, J. Caon, C., Vazquez, J., Tyler, K.L., Racke, M.K., Millis, S., & Khan, O., (2009). Progressive multifocal leukoencephalopathy and relapsing-remitting multiple sclerosis: A comparative study. Journal of the American Medical Association, Neurology, 66(5):593-599. doi:10.1001/archneurol.2009.31

Burman, J., Iacobaeus, E., Svenningson, A., Lycke, J., Gunnarsson, M., Nilsson, P., Vrethem, M., Fredrikson, S., Martin, C., Sandstedt, A., Uggla, B., Lenhoff, S., Johansson, J., Isaksson, C., Hagglund, H., Carlson, K., & Fagius, J., (2014). Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: The Swedish experience. Journal of Neurological Neurosurgery Psychiatry, 2(25):1-6. doi:10.1136/ jnnp-2013-307207

Burt RK, Balabanov R, Burman J, et al. Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis: A Randomized Clinical Trial. JAMA. 2019;321(2):165–174. doi:10.1001/jama.2018.18743

Burt, R. K., Han, X., Gozdziak, P., Yaung, K., Morgan, A., Clendenan, A. M., … Helenowski, I. (2018). Five year follow-up after autologous peripheral blood hematopoietic stem cell transplantation for refractory, chronic, corticosteroid-dependent systemic lupus erythematosus: effect of conditioning regimen on outcome. Bone Marrow Transplantation, (6), 692.

Burt, R.K., Balabanov, R., Han, X., Sharrack, B., Morgan, A., Quigley, K., Yaung, K., Helenowski, I.B., Jovanovic, B., Spahovic, D., Arnautovic, I., Lee, D.C., Benefield, B.C., Futterer, S., Oliveira, M.C., & Burman, J., (2015). Association of nonmyeloablative hematopoietic stem cell transplantation with neurological disability in patients with relapsing-remitting multiple sclerosis. Journal of the American Medical Association, 313(3):275-284. doi:10.1001/jama.2014.17986

Burt, R. K., Shah, S. J., Dill, K., Grant, T., Gheorghiade, M., Schroeder, J., … Barr, W. (2011). Articles: Autologous non-myeloablative haemopoietic stem-cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomised phase 2 trial. The Lancet, 378, 498–506. https://doi.org/10.1016/S0140-6736(11)60982-3

Burt, R.K., Loh, Y., Cohen, B., Stefosky, D., Balabanov, R., Katsamakis, G., Oyama, Y., Russell, E.J., Stern, J., Muraro, P., Rose, J., Testori, A., Bucha, J., Jovanovic, B., Milanetti, F., Storek, J., Voltareli, J.C., & Burns, W.H., (2009). Autologous non-myeloablative haemopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: A phase I/II study. Lancet, 09(70017-1). doi:10.1016/S1474-4422

Burt, R. K., Cohen, B., Rose, J., Petersen, F., Oyama, Y., Stefoski, D., … McGuirk, J. (2005). Hematopoietic Stem Cell Transplantation for Multiple Sclerosis. Archives of Neurology, 62(6), 860. Retrieved from https://search.ebscohost.com/login.aspx?direct=true&db=edb&AN=17338142&site=eds-live&authtype=sso&custid=s8992667

Burt, R. K., Kallunian, K., Patel, D., Thomas, J., Yeager, A., Traynor, A., … Snowden, J. (2004). The rationale behind autologous autoimmune hematopoietic stem cell transplant conditioning regimens: concerns over the use of total-body irradiation in systemic sclerosis. Bone Marrow Transplantation, 34(9), 745–751. https://doi.org/10.1038/sj.bmt.1704671

Statkute, L., Verda, L., Oyama, Y., Traynor, A., Villa, M., Shook, T., … Burt, R. K. (2007). Mobilization, harvesting and selection of peripheral blood stem cells in patients with autoimmune diseases undergoing autologous hematopoietic stem cell transplantation. Bone Marrow Transplantation, 39(6), 317. Retrieved from https://search.ebscohost.com/login.aspx?direct=true&db=edb&AN=24259891&site=eds-live&authtype=sso&custid=s8992667

Farge, D., Burt, R. K., Oliveira, M.-C., Mousseaux, E., Rovira, M., Marjanovic, Z., … Snowden, J. A. (2017). Cardiopulmonary assessment of patients with systemic sclerosis for hematopoietic stem cell transplantation: recommendations from the European Society for Blood and Marrow Transplantation Autoimmune Diseases Working Party and collaborating partners. Bone Marrow Transplantation, (11). https://doi.org/10.1038/bmt.2017.56

Farge D, Labopin M, Tyndall A, Fassas A, Mancardi GL, Van Laar J, Ouyang J, Kozak T., Moore J., Kötter I., Chesnel V., Marmont, A., Gratwohl, A., & Saccardi, R., (2010). Autologous hematopoietic stem cell transplantation for autoimmune diseases: an observational study on 12 years’ experience from the European Group for Blood and Marrow Transplantation Working Party on Autoimmune Diseases. Haematologica, 95:284-292. doi:10.3324/haematol.2009.013458

Fassas, A., Kimiskidis, V.K., Sakellari, I., Kappinas, K., Anagnostopoulos, A., Tsimourtou, V., Sotirakoglou, K., Kazis, A., (2011). Long-term results of stem cell transplantation for MS: A single-center experiences. Neurology, 2011 Mar 22, 76(12): 1066-70. doi:10.1212/WNL.0b013e318211c537

Hamerschlak, N., Rodrigues, M., Moraes, D. A., Oliveira, M. C., Stracieri, A. S. P. L., Pieroni, F., … Voltarelli, J. C. (2010). Brazilian experience with two conditioning regimens in patients with multiple sclerosis: BEAM/horse ATG and CY/rabbit ATG. Bone Marrow Transplantation, (2), 239. Retrieved from https://search.ebscohost.com/login.aspx?direct=true&db=edsgih&AN=edsgcl.219375350&site=eds-live&authtype=sso&custid=s8992667

Hartung, D.M., Bourdette, D.N., Ahmend, S.M., & Whitham, R.H., (2015). The cost of multiple sclerosis drugs in US and the pharmaceutical industry: Too big to fail? Neurology, 84:2185-92. doi:10.1212/WNL.0000000000001608

Krasulova, E., Trneny, M., Kozak, T., Vackova, B., Pohlreich, D., Kemlink, D., Kobylka, P., Kovarova, I., Lhotakova, P., & Havrdova, E., (2010). High-dose immunoablation with autologous hematopoietic stem cell transplantation in aggressive multiple sclerosis: a single centre 10-year experience. Multiple Sclerosis, 16(6): 685-693. doi:10.1177/1352458510364538

Kurtzke, J.F., (1983). Rating neurologic impairment in multiple sclerosis: An expanded disability status scale (EDSS). Neurology, 33(11): 1444-1452.

Mancardi, G.L., Sormani, M.P., Vuolo, G.L., Gualandi, F., Amato, M.P., Capello, E., Curro, D., Uccelli, A., Bertolotto, A., Gasperini, C., Lugaresi, A., Merelli, E., Meucci, G., Mottie, L., Tola, M.R., Scarpini, E., Repice, A.M., Massacesi, L., Saccardi, R., (2011). Autologous haematopoietic stem cell transplantation with an intermediate intensity conditioning regimen in multiple sclerosis: The Italian multi-centre experience. Multiple Sclerosis Journal, 18(6): 835-842. doi:10.1177/1352458511429320

Nash, R.A., Hutton, G.J., Racke, M.K., Popat, U., Devine, S.M., Griffith, L.M., Muraro, P.A., Openshaw, H., Sayre, P.H., Stuve, O., Arnold, D.L., Spychala, M.E., McConville, K.C., Harris, K.M., Phippard, D., Georges, G.E., Wndes, A., Kraft, & Bowen, J.D., (2015). High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for relapsing-remitting multiple sclerosis: A 3-year interim report. Journal of the American Medical Association, Neurology, 72(2):159-169. doi:10.1001/jamaneurol.2014.3780

Rimel, B.J., (2016). Clinical trial accrual: Obstacles and opportunities. Frontiers in Oncology, 6:103. doi:10.3389/fonc.2016.00103

Rogne, S. (2017). Unethical for neurologists not to offer patients with multiple sclerosis chemotherapy with autologous stem cell support. Tidsskr Nor Legeforen, 20(17 January 2017):1931-2. doi:10.4045/tidsskr.14.1027

Ruiz, M. A., Kaiser, R. L., de Quadros, L. G., Piron-Ruiz, L., PeAaAaAeA, a-A. T., Faria, M. A. G., … Burt, R. K. (2017). Low toxicity and favorable clinical and quality of life impact after non-myeloablative autologous hematopoietic stem cell transplant in Crohn’s disease. BMC Research Notes, (1). https://doi.org/10.1186/s13104-017-2824-1

Saccardi, R., Freedman, M.S., Sormani, M.P., Atkins, H., Farge, D., Griffith, L.M., Kraft, G., Mancardi, G.L., Nash, R., Pasquini, M., Martin, R., Muraro, P.A., (2012). A prospective, randomized, controlled trial of autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: a position paper. Multiple Sclerosis Journal, 18(6): 825-834. doi:10.1177/1352458512438454

Statkute, L., Verda, L., Oyama, Y., Traynor, A., Villa, M., Shook, T., … Burt, R. K. (2007). Mobilization, harvesting and selection of peripheral blood stem cells in patients with autoimmune diseases undergoing autologous hematopoietic stem cell transplantation. Bone Marrow Transplantation, 39(6), 317. Retrieved from https://search.ebscohost.com/login.aspx?direct=true&db=edb&AN=24259891&site=eds-live&authtype=sso&custid=s8992667

Weinshenker, B.G., Bass, B., Rice, G.P., Noseworthy, J., Carriere, W., Baskerville, J., Ebers, G.C., (1989). The natural history of multiple sclerosis: A geographically based study. Brain, Feb.(112):133-146. doi:10.1093/brain/112.1.133

Facilities

Reputable hospitals/clinics that offer HSCT around the world include:

Northwestern Memorial Hospital
Dr. Richard Burt
Division of Immunotherapy and Autoimmune Diseases
Chicago, Illinois USA

Sheffield Teaching Hospitals NHS Foundation Trust
(modeled after Northwestern protocols)
Royal Hallamshire Hospital,
Sheffield, UK

Uppsala University
(modeled after Northwestern protocols)
Uppsala, Sweden

A.A. Maximov Department of Hematology and Cellular Therapy
Dr. Denis Fedorenko
Moscow, Russia

Ottawa General Hospital
Dr. Mark Freedman
Ottawa, Canada

Clinica Ruiz

Dr. M.A. Ruiz
Puebla, Mexico