Interim Clinical Trial Results Suggest 80% of Patients Benefit from Experimental Multiple Sclerosis Stem Cell Treatment

A new study, recently published by The American Medical Associations’ Journal of Neurology, offers a promising ray of hope for those battling the most common form of Multiple Sclerosis[1], a disease in which the immune system eats away at the protective covering of nerves.

The study, sponsored by the National Institute of Health’s National Institute of Allergy and Infectious Diseases (NIAID), reveals that the procedure of depleting and then re-establishing the immune system with stem cells may improve or stabilize a type of early-stage multiple sclerosis called Relapsing-Remitting MS (RRMS), a type of MS which intermittently attacks the central nervous system, damaging nerve fibers and disrupting communication between brain and body.

The goal of this treatment – called HDIT/HCT (high-dose immunosuppressive therapy with autologous hematopoietic cell transplant) – is to “reset” the immune system with a stem cell transplant so that it stops attacking the central nervous system, and could soon be a promising direction for future potential treatment for those with RMMS who fail to respond to conventional immunotherapy.

First, a patient’s hematopoietic stem cells (HSCs)—cells isolated from sources bone marrow or peripheral blood—are collected.[2] This same type of stem cell is also found in cord blood. These stem cells form into blood and immune cells, providing constant maintenance and immune protection through the continuous production of new blood, and have some of the greatest powers of self-renewal of any adult tissue in the body.[3] Once the HSCs are collected, high-dose chemotherapy and other drugs are used to deplete the patient’s immune system. Finally, the patient is infused with his or her own HSCs, which then develop into red and white blood cells to re-establish the patient’s immune system.[4] The use of one’s own stem cells is paramount in order to establish safety and minimize the risk of rejection of a donor’s cells.

Researchers across the U.S. are monitoring 24 volunteers with RRMS for five years following HDIT/HCT treatment. The interim results, three years after HDIT/HCT treatment, show that nearly 80% of trial participants had survived without an increase in disability, relapse of MS symptoms, or new brain lesions. Patients didn’t receive any MS drugs during those three years. Few serious early complications or unexpected side effects occurred. These very promising results will continue to be followed over the next two years, and will hopefully lead to the development of larger clinical trials to evaluate the HDIT/HCT treatment in other people with multiple sclerosis.


[1] Hooper K. Managing Progressive MS. New York, NY. National Multiple Sclerosis Society; 2011.


[3]  (Introduction)


Gina Satta

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Gina Satta is a writer, namer and new mom living in San Francisco. When she isn’t brainstorming brilliant copy, she is either looking for parking, making up new versions of classic nursery rhymes, or eating chocolate. Gina is a paid consultant to CBR.

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