Multiple Sclerosis Reversed in Patients Taking Own Stem Cells

Jan. 29 (Bloomberg) -- Patients in the early stages of multiple
sclerosis had their disability reversed in a study that used their stem
cells to *reset* their malfunctioning immune system.

All 21 patients in the study at Northwestern University in Chicago had
the *relapsing-remitting* form of the disease that makes their
symptoms alternately flare up and recede. Three years after being
treated, on average, 17 of the patients had improved on tests of their
symptoms, 16 had experienced no relapse and none had deteriorated, the
study found.

*This is the first study to actually show reversal of disability,*
said Richard Burt, an associate professor in the division of
immunotherapy at Northwestern, and the lead author of the study
published today in the British journal, the Lancet. *Some people had
complete disappearance of all symptoms.*

In multiple sclerosis, or MS, a patient*s immune cells attack the
central nervous system, degrading their vision, coordination, balance
and sometimes their cognitive abilities.

The vast majority of patients with this disease are first diagnosed
with the relapsing-remitting form and some progress to more serious
stages. The study included only patients whose flare-ups continued after
being treated with protein-based drugs known as interferons.

Participants had their hematopoietic, or blood-forming, stem cells
extracted before chemotherapy drugs killed immune cells in their bone
marrow. The patients* stem cells were then returned to rebuild their
marrow.

Vision Dimmed

One of the patients was Edwin McClure, now a 24-year-old graduate
student in marketing at Virginia Commonwealth University in Richmond.
McClure was diagnosed with multiple sclerosis as a high school senior in
2002, after his vision dramatically worsened.

*It was like someone had turned down the dimmer switch,* he said in
a telephone interview yesterday. He also suffered from dizziness, poor
balance and fatigue so bad that he*d collapse and sleep for three
hours every day after school.

Over the next few years, McClure was treated with steroids and
interferons. While they controlled the disease for a time, his symptoms
eventually broke through, triggering fresh attacks.

McClure went to Chicago to take part in Burt*s study at the end of
2005, spent a month being treated, and hasn*t needed any drugs since.

*A Blessing*

*It*s a blessing,* he said. *My disease has been halted.*

Even the stress of being in the competitive graduate program -- a
factor known to exacerbate symptoms of multiple sclerosis -- hasn*t
caused a single attack, he said. His balance is better and his vision
hasn*t deteriorated further.

Researchers believe that in the early stage of the disease, the
hyperactive immune cells attack nerve cells. This damages the myelin, an
insulating material that surrounds the axons, long fiber tails that
extend from a neuron and help transmit electrical signals.

*Research has shown it*s critical to stop the inflammation early
and that*s probably the best way to stop neural degeneration and
progression of the disease,* said Patricia O*Looney, vice president
of biomedical research at the National MS Society, in a telephone
interview yesterday.

In previous efforts, Burt and other scientists tried giving bone marrow
stem cells to patients with more advanced disease, with no benefit.

Late-Stage Failure

*I called it a failure,* he said. *When you do it in late-stage
patients, they don*t improve,* probably because the immune cells
have already done their damage.

O*Looney said the results of Burt*s study were promising and should
now be replicated in a larger trial that randomly compares the stem-cell
treatment with existing therapy. Burt is now starting such a trial,
which will recruit 55 patients in the U.S., Canada and Brazil.

If the results of today*s study are borne out in the new one, *I
think we can really change the way this disease is approached,* Burt
said.

To contact the reporter on this story: Rob Waters in San Francisco at
rwaters5@bloomberg.net.
Last Updated: January 29, 2009 18:30 EST
-----------------------------

Stem cell transplant reverses early-stage multiple sclerosis
Public release date: 29-Jan-2009
http://www.eurekalert.org/pub_releases/2009-01/nu-sct012909.php

CHICAGO --- Researchers from Northwestern University's Feinberg School
of Medicine appear to have reversed the neurological dysfunction of
early-stage multiple sclerosis patients by transplanting their own
immune stem cells into their bodies and thereby "resetting" their immune
systems.

"This is the first time we have turned the tide on this disease," said
principal investigator Richard Burt, M.D. chief of immunotherapy for
autoimmune diseases at the Feinberg School. The clinical trial was
performed at Northwestern Memorial Hospital where Burt holds the same
title.

The patients in the small phase I/II trial continued to improve for up
to 24 months after the transplantation procedure and then stabilized.
They experienced improvements in areas in which they had been affected
by multiple sclerosis including walking, ataxia, limb strength, vision
and incontinence. The study will be published online January 30 and in
the March issue of The Lancet Neurology.

Multiple sclerosis (MS) is an autoimmune disease in which the immune
system attacks the central nervous system. In its early stages, the
disease is characterized by intermittent neurological symptoms, called
relapsing-remitting MS. During this time, the person will either fully
or partially recover from the symptoms experienced during the attacks.
Common symptoms are visual problems, fatigue, sensory changes, weakness
or paralysis of limbs, tremors, lack of coordination, poor balance,
bladder or bowel changes and psychological changes.

Within 10 to 15 years after onset of the disease, most patients with
this relapsing-remitting MS progress to a later stage called secondary
progressive multiple sclerosis. In this stage, they experience a steady
worsening of irreversible neurological damage.

The 21 patients in the trial, ages 20 to 53, had relapsing-remitting
multiple sclerosis that had not responded to at least six months of
treatment with interferon beta. The patients had had MS for an average
of five years. After an average follow-up of three years after
transplantation, 17 patients (81 percent) improved by at least one point
on a disability scale. The disease also stabilized in all patients.

In the procedure, Burt and colleagues treated patients with
chemotherapy to destroy their immune system. They then injected the
patients with their own immune stem cells, obtained from the patients'
blood before the chemotherapy, to create a new immune system. The
procedure is called autologous non-myeloablative haematopoietic
stem-cell transplantion.

"We focus on destroying only the immune component of the bone marrow
and then regenerate the immune component, which makes the procedure much
safer and less toxic than traditional chemotherapy for cancer," Burt
said. After the transplantation, the patient's new lymphocytes or immune
cells are self-tolerant and do not attack the immune system.

"In MS the immune system is attacking your brain," Burt said. "After
the procedure, it doesn't do that anymore."

In previous studies, Burt had transplanted immune stem cells into
late-stage MS patients. "It didn't help in the late stages, but when we
treat them in the early stage, they get better and continue to get
better," he said.

"What we did is promising and exiting, but we need to prove it in a
randomized trial," Burt noted. He has launched a randomized national
trial.