Transplant Promising as MS Therapy

ORLANDO — High-dose immunosuppressant therapy and autologous hematopoietic stem cell transplant (HSCT) induced durable freedom from relapses and MRI lesions in patients with multiple sclerosis, researchers reported here.

At evaluations performed 1 and 2 years after the procedure, patients showed no gadolinium-enhancing lesions on MRI scans, and only six of the 24 patients receiving the transplants experienced relapses, reported Richard A. Nash, MD, of Colorado Blood Cancer Institute in Denver, and colleagues.

In a poster presented at the joint meeting of the Consortium of Multiple Sclerosis Centers and the Americas Committee for Treatment and Research in Multiple Sclerosis, the investigators indicated that overall functional ability and quality of life were improved as well with the transplant procedure.

Immunosuppressant therapy with drugs and dosages high enough to be severely myelosuppressive, followed by HSCT, had been tested previously in patients with advanced, progressive MS, for which there is currently no broadly effective treatment. The results were disappointing, Nash and colleagues indicated, as “many patients continued to lose neurological function.”

But it was unclear whether the lack of effect was because the treatment failed to suppress MS autoimmune activity or because the process of neurodegeneration had become independent of autoimmune mechanisms.

Since the latter seemed like the more likely explanation, Nash and colleagues tested a similar regimen in patients with relapsing-remitting MS, in which autoimmunity is widely believed to be the chief pathological process.

The 25 patients enrolled in the study had a median age of 38 and median duration of disease of 6.4 years. Median score on the Expanded Disability Status Scale (EDSS) was 4.5, indicating moderate disability. About 40% had gadolinium-enhancing lesions at baseline, including five patients with two or more each.

Participants first underwent stem cell mobilization with granulocyte colony-stimulating factor (along with cyclophosphamide in one patient who did not mobilize enough cells with G-GSF alone), with CD34 stem cells then collected.