BOSTON—Interim results from a small, prelimi- nary study support a new type of treatment for
progressive multiple sclerosis (MS). Results from the
first six people enrolled in the phase I study, which
was designed to enroll 10 people, were presented at
the 69th Annual Meeting of the American Academy of
Neurology. “While these results are very preliminary,
and much more research is needed, we are excited there
were no serious side effects,” said study author Michael
P. Pender, MD, PhD, a Professor and Director of the
Multiple Sclerosis Research Group at the University of
Queensland in Brisbane, Australia.
The study investigated the relationship between MS
and the Epstein-Barr virus (EBV). Previous research has
suggested a role for EBV in MS pathogenesis. The study
involved six people with progressive MS who had moderate to severe disability (ie, Expanded Disability Status
Scale scores between 5.0 and 8.0).
In some people with MS, EBV-infected autoreactive B
cells might accumulate in the CNS because of defective
cytotoxic CD8+ T-cell immunity. Elimination of EBV-infected B cells may reduce the destruction of myelin in MS.
For the study, researchers removed the participants’
own T cells and stimulated them to boost their ability
to recognize and destroy cells infected with EBV. They
then injected participants with infusions of escalating
doses of T cells every two weeks for six weeks. They
followed the patients for 26 weeks to look for evidence
of side effects and possible improvement of symptoms.
Three participants showed symptomatic and objective clinical improvement, starting two to eight weeks
after the first infusion.
“One person with secondary progressive MS showed
striking improvement,” Professor Pender said. This par-
ticipant had normalization of lower extremity tone and
plantar responses for the first time in 16 years. “This par-
ticipant had a significant increase in ambulation from 100
yards with a walker at the start of the study, and over
the previous five years, to three quarters of a mile, and
was now also able to walk
shorter distances with only
one-sided assistance. Lower
leg spasms that had persist-
ed for years resolved.”
Professor Pender said
another participant with
primary progressive MS had
reduced fatigue, increased
productivity, and improved
balance. Another responder
had improved color vision,
visual acuity, and manu-
al dexterity; reduced fatigue; fewer lower extremity
spasms; and less urinary urgency. All three responding
participants had improvements in fatigue and ability to
perform daily activities.
“The best responses were seen in the two people who
received T cells with the highest amount of reactivity to
the EBV,” Professor Pender said. None of the six partici-
pants had serious side effects.
“Much more research needs to be done with larger
numbers of participants to confirm and further evaluate
these findings,” Professor Pender said. “But the results
add to the mounting evidence for a role of the Epstein-
Barr virus infection in MS and set the stage for further
The study was a collaboration between the QIMR
Berghofer Medical Research Institute, Royal Brisbane and
Women’s Hospital, and the University of Queensland.
The study was supported by MS Queensland, MS Re-
search Australia, QIMR Berghofer Medical Research In-
stitute, and Perpetual Trustee Company Limited. NR
—Glenn S. Williams
Pender MP, Csurhes PA, Smith C, et al. Epstein-Barr virus-specific adoptive immunotherapy for progressive multiple sclerosis. Mult Scler.
2014; 20( 11):1541-1544.
Preliminary Study Suggests Possible
New Treatment for Progressive MS
Autologous EBV-specific T cells may be a potential treatment for patients with
Michael P. Pender,