Scientists Reverse Early MS With Patients' Own Stem Cells

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Scientists Reverse Early MS With Patients' Own Stem Cells

30 Jan 2009

A small trial at a US hospital where patients with early stage MS had

their own immune system stem cells transplanted back into their

bodies appears to have reversed the neurological dysfunction of the

early stages of the disease by causing their immune systems

to " reset " . The scientists said the results should now be confirmed

with a larger, randomized trial.

The trial was the work of researchers from Northwestern University's

Feinberg School of Medicine in Chicago, plus colleagues from other

research centres in and outside the US, and is published early online

in The Lancet Neurology on 30 January; it will appear in the March

print issue.

The patients on the small phase I/II trial experienced improvements

in several areas affected by their MS, including walking, ataxia

(loss of muscle coordination), limb strength, vision, and

incontinence. They continued to improve for 24 months after receiving

the transplants and then stabilized.

MS (Multiple Sclerosis) is an autoimmune disease where the person's

own immune system attacks their central nervous system causing all

kinds of neurological dysfunction such as loss of control over

muscles and loss of ability to take in information through the

senses.

The early stage is called relapsing-remitting MS and the person has

intermittent symptoms from which they partially or fully recover and

then relapse into again. These include visual impairment, fatigue,

sensory problems, limb weakness or paralysis, tremors, lack of

coordination, problems with balance, changes in bowel and bladder,

and psychological changes.

After about 10 to 15 years of relapsing-remitting MS, patients enter

another stage called secondary progressive MS, where symptoms

steadily become worse and irreversible.

Lead researcher on the team, Dr Burt, who works at using

immunotherapy for autoimmune diseases at the Feinberg School said:

" This is the first time we have turned the tide on this disease. "

For the trial, Burt and colleagues recruited 21 patients aged 20 to

53 who had had MS for an average of 5 years. They all had relapsing-

remitting multiple sclerosis that had been treated with interferon

beta for at least 6 months but with no response.

First, they had to destroy the patients' immune system with

chemotherapy, then they injected them with their own stem cells that

had been harvested before the chemo. This seeded a new immune system.

The procedure is called " autologous non-myeloablative haematopoietic

stem-cell transplantion " .

After an average follow-up of three years after receiving their

transplants (which took place between January 2003 and February

2005), 17 patients (81 per cent) improved by at least one point on a

disability scale. And for all patients, the disease had stopped

progressing. Five patients relapsed in the early days, but then

experienced remission after further immunosuppression.

Burt said that they focused on destroying only the immune system part

of the bone marrow and then regenerating it, a procedure that is less

toxic than traditional chemotherapy for cancer.

But amazingly, when the new immune system is created, the patient's

new white blood cells are self-tolerant, as Burt explained:

" In MS the immune system is attacking your brain. "

" After the procedure, it doesn't do that anymore, " he said.

The authors concluded from the trial that this type of stem cell

transplantation in patients with relapsing-remitting MS " reverses

neurological deficits " , and Burt said the results were " promising and

exciting " , but to get real proof, you need a randomized trial, which

he has already launched.

Burt has been working with MS patients for some time; in earlier

research he tried transplanting immune system cells into patients

with late-stage MS but it didn't help them like it did the early

stage patients in this trial.