theme this year is the continued progress towards an effective oral treatment for MS. Preliminary results
for the largest trials were presented at the AAN meeting last year.
were 2 presentations on a large Phase III study of fingolimod versus placebo in relapsing MS, which was recently published
in the New England Journal of Medicine. This is a pill given once a day. It has good
effectiveness, reducing relapse rate by 55 to 60%, slowing progression of disability, and reducing MRI activity by 70 to 80%.
The issue with this treatment is going to be side effects, since there have been 2 cases of fatal infections.
Other concerning side effects are eye problems (macular edema) and possibly skin cancers. This drug
is currently under review by the FDA. It is more convenient and probably more effective than our current
standard treatments. But because of the side effects, I will likely use it mostly for people with more
severe MS until we have enough experience with it to decide how risky it is.
study of cladribine versus placebo was also recently published, and there was a supplemental presentation. This
is an oral, immune suppressive medicine. It appears to be very effective, and so far has not had any troubling
side effects. Since it does suppress the immune system, I expect I will be fairly careful using it when
it becomes available.
Teriflunomide is an oral medicine that suppresses the immune system. It is similar to another
medicine already in use for rheumatoid arthritis. We heard the results of a small phase II study (about
120 patients overall, 40 per group) designed to look at the safety of the combination of glatiramer and teriflunomide.
The results were similar to a previous study of teriflunomide combined with interferon. The combination
appeared to be safe, and reduced disease activity on MRI. This medicine is now in phase III studies, and
we should have information on how effective it is in 2011.
was a disappointing report on an oral medication called CDP323. This medicine should do the same thing
as natalizumab (Tysabri), but would be easier to use since it is a daily pill instead of an intravenous infusion.
It might also be safer, since the effects wear off quickly after the drug is stopped. The trial showed very little
benefit at an intermediate time point, and was ended early.
second disappointing report regarded fish oil. This is a dietary supplement containing omega-3 unsaturated
fatty acids, and there was some suggestive evidence that it might be beneficial in MS. It is also thought
to be useful for preventing vascular disease. There were two presentations on a study of fish oil in MS,
which did not show any suggestion of benefit.
were two other fashionable topics.
There were several presentations on chronic cerebrospinal venous insufficiency
(CCSVI). The theory that MS is caused by blockage in the veins that drain blood from the brain was proposed
by Dr. Zamboni in Italy, and has recently gotten a lot of attention. To date, all the publications had
come from Dr. Zamboni’s group, which reported that 100% of MS patients and 0% of controls had CCSVI. Dr.
Zivadinov’s group from Buffalo reported on their studies. In a group of 500 subjects they found that
about 55% of MS patients and 25% of controls had CCSVI. I would like to see the findings of additional
independent groups before making up my mind on this issue. Certainly, any attempts at treatment outside
of a clinical study are premature.
was one report on stem cell therapy. Stem cells were the hot idea for the last few years, until CCSVI pushed
them out of the news. The group in Jerusalem is using mesenchymal stem cells, which probably do not
develop into nerve cells or myelin-producing cells. They take a bone marrow sample, grow up the stem cells,
and then inject them into both the blood and the spinal fluid. They have treated 15 MS patients so far,
and it is still too early to draw any conclusions about effectiveness. They have not found any toxicity.