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Making Sense of MS Research (Transcript)

March 3, 2005 Web Chat with Dr. Ben Thrower

Moderator:

Good evening, everyone, and welcome. This is the first of the web chats scheduled throughout the month of March in honor of National MS Education and Awareness Month. We are pleased that you could join us, and honored to welcome our special guest, Dr. Ben Thrower of the MS Center at Shepherd Center in Atlanta, GA. This evening’s topic will be Making Sense of MS Research. With no further ado, Dr. Ben…

Dr Ben:

Good evening. With the events of the past week involving Tysabri, the importance of clinical research is evident. Clinical research in MS can come in many forms. Ideally, we would have double-blind placebo controlled trials over a long period of time. This may not always be practical however, as new and promising treatments become available. The challenge will be to balance the benefit from new therapies with the risk of any side effects. Tonight we are going to look at research in multiple sclerosis including current and possible future therapies. Rather than going further with any prepared agenda, why don’t we open it up for questions? I’m sure many people have a lot on your mind.

Sam Question:

I want to know about progressive MS research.

Dr Ben: Answer:

Most trials in have focused on relapsing forms of MS. Part of the reason for this is that you can do shorter trials and perhaps show the treatment effect. For example, if a person has six relapses per year and the therapy reduced that by half in one year we may have an effective treatment. In contrast, to show that a drug slows progression of disability, one would need to do trials of perhaps three or four years. But we are seeing research now focusing on progressive forms of MS. An example would be rituxamab. This is a monoclonal antibody that is being targeted at progressive MS. There are other new projects looking at secondary progressive MS as well.

Peggy Question:

Please explain what the blood brain barrier is.

Dr Ben: Answer:

The blood brain barrier is the "door" to the central nervous system. It is fairly selective in what can enter the central nervous system. It is thought the inflammatory white blood cells may enter the brain inappropriately through the blood brain barrier. Dye uptake on MRI into an MS lesion also goes through the blood brain barrier.

Paula Question:

If Rituxamab is a monocolonal antibody, wouldn't have the same risks as Tysabri?

Dr Ben: Answer:

Rituxamab targets a different step in the immune process. The problem with Tysabri may be that it was too effective at keeping all white blood cells out of the central nervous system.

HollyGrace Question:

I read somewhere about an oral ms medication that was set for FDA approval, do you know anything about it?

Dr Ben: Answer:

There are several oral medications in testing at this time, but none that are very close to FDA approval. I would suspect with the recent events with Tysabri that the FDA will be especially careful in examining new medications.

Moderator:

For those just joining us, we are chatting with Dr. Ben Thrower about MS research. You may send your questions for our guest now and at any time throughout the chat. Questions will only be displayed to the audience as they are answered.

Susan Question:

How did the FDA approve Tysabri with such little clinical trial information

Dr Ben: Answer:

It was felt that with the one year data, the benefit and public need for the medication outweighed the risk. This will always be a problem in research where all side effects may not be fully known until the drug has been observed for several years.

HollyGrace Question:

So there are no new ms medications that will be coming out soon?

Dr Ben: Answer:

We are doing research with a number of oral medicines, but to my knowledge nothing is slated for the next six months.

Susan Question:

Most clinical trials for ms therapies only go to 10 years, has any therapies exceeded that time limit

Dr Ben: Answer:

There are no controlled trials that have gone truly longer than five years. After that point, the trials follow people in a non-blinded fashion. This is still very important information. It gives us reassurance that the drug is effective and safe over long periods of time. Copaxone has ten-year data and I've heard that Betaseron may have information for sixteen years presented at one of the next MS meetings.

Paula Question:

Have there been any head to head studies with Copaxone vs. Rebif vs. Betaseron or any combination of the three?

Dr Ben:

There are currently trials examining Rebif vs. Copaxone and a separate trial looking at Copaxone vs. Betaseron vs. a double dose of Betaseron. Past trials have looked at Rebif vs. Avonex and Betaseron vs. Avonex. There is a combination trial looking at Avonex with Copaxone.

Alex Question:

About seven years ago, I was told that a cure was expected within ten years. Do you have any estimate for how long before a cure?

Dr Ben: Answer:

This is always a tough question and probably depends on the definition of a cure. Some of the challenges to truly finding the cure are that MS could be actually different diseases manifesting in a similar fashion. We will understand the genetics of MS better over the next two years and may indeed find out the MS is not truly one disease. That said, a cure could mean finding a treatment that stops the process completely and some people do achieve that goal with our existing therapies. The other part of a cure would be repairing the damage that has already been done. The concept of neural repair has advanced significantly in the past five years. I am always cautious about giving timelines in medicine. It seems that science moves in stops and starts and advances can be unpredictable in their timing.

Susan Question:

In the past, I was on Avonex twice a week but had to stop because insurance wouldn't cover it since there was no information backing it's efficacy. Has there been any research recently done for that

Dr Ben: Answer:

We know that there is a dose/response curve with interferon such as Avonex. Many studies have shown that more frequently dosed interferons tend to be more effective. A simple solution for your problem would be to use a partial dose of Rebif. Rebif is the same molecule as Avonex. It could be dosed twice weekly like you were doing with Avonex and the cost would actually be lower.

Paula Question:

There are many people who did well on Tysabri, and feel that it greatly improved their quality of life. Considering this, do you think Tysabri will return to the market?

Dr Ben: Answer:

No one knows at this point when and if Tysabri will be available. The side effect that resulted in the drug being stopped (PML) is very serious. There are researchers who feel like similar complications could arise and have just not been seen yet. This morning a patient of mine was interviewed for a local news station. This gentleman felt that he had done well after two doses of Tysabri. He had failed to respond to any of our other medications. I truly feel for people like him and I know there are others out there, but I don't know what the likely outcome of this situation will be.

Ricey26 Question:

Are there any studies using higher doses of Rebif?

Dr Ben: Answer:

No studies have looked at higher doses of Rebif yet, but there is a study looking at a higher dose of Betaseron. Betaseron study showed that twice the usual dose was well tolerated and for some people was more effective than the usual dose. I would not be surprised to see a similar study started with Rebif, and I would expect similar results.

HollyGrace Question:

How many research projects are being done within the US?

Dr Ben: Answer:

I'm going to have to guess a little bit but probably well over twenty major projects and hundreds of smaller investigator-driven projects.

Snapper Question:

In you answer to Susan about the partial dose of Rebif, do you mean to take 22mcg twice per week, or to save half the 44 mcg and use the other half and re-inject?

Dr Ben: Answer:

I would recommend 44 mcg twice weekly. I would not recommend doing a partial dose and the giving the other part later.

Ellen Question:

Why money on immune drugs and mot on rebuilding myelin sheath?

Dr Ben: Answer:

Both are important goals. In all honesty, stopping relapses and slowing progression is an easier goal than repair in the central nervous system. It wasn't too long ago that it was felt that repair of the CNS was impossible. Fortunately, that is not felt to be the case now and there are many researchers worldwide who devote their life to this goal. The goal of neural repair is not unique to MS, but also applies to conditions such as Parkinson’s disease, stroke, and spinal cord injury. Answers for our MS community could well come from research in some of these other areas.

Alex Question:

Does Copaxone's effectiveness diminish over time?

Dr Ben: Answer:

There is no indication that Copaxone of the interferons become less effective over time. A small portion of people on interferon therapy may develop neutralizing antibodies. These antibodies could make the interferon less effective, but even this area is controversial.

Susan Question:

Is it true that Switzerland has more therapies currently used than we have here in the US

Dr Ben: Answer:

I don't believe that there are more approved therapies in Switzerland than we have. It does seem that there are sometimes more aggressive "alternative" therapies in Europe than in the US. This really gets back to the whole issue of risk vs. benefit. Sometimes the FDA has been criticized for moving too slowly, but then we have situations like that with Tysabri or even Vioxx that would suggest perhaps we move too quickly.

Susan Question:

For secondary progressive patients, is there any research done for those patients in rehabilitation programs (inpatient and outpatient)

Dr Ben: Answer:

There has been research into rehabilitation in both inpatient and outpatient settings in MS. Some research has looked at progressive forms and some have looked at the effect of rehab on relapse recovery. I believe the rehabilitation in any form is a vital part of our MS therapy program. Aggressive rehabilitation is one of the few therapy modalities that has the potential for improving function.

Snapper Question:

What types of studies are currently enrolling for the ABCR drugs for patients who are already on one of these therapies?

Dr Ben: Answer:

To my knowledge there are no studies that are enrolling patients who are already on an ABCR drug. The Avonex\Copaxone combination trial is looking for people who are on no current therapy. These people will be randomized to either one of the drugs alone or a combination of the two.

Noname Question:

What are your thoughts on combination therapy (ie. Betaseron and cellcept)?

Dr Ben: Answer:

I think combination therapy is going to be an important part of managing MS. To date, most completed trials of combination therapy have been small uncontrolled trials. The combination of an interferon such as Betaseron with an immunosuppressive drug such as Cellcept does make sense. I don't believe that it has been studied in a controlled fashion, but again, it is not an uncommon combination.

HollyGrace Question:

What country is the leader in MS research?

Dr Ben: Answer:

In terms of numbers of clinical trials, the US and Canada probably account for the majority of research. A unique challenge that is arising is that of the ethics of placebo controlled trials. In the US and Canada, the message has been heard in regards to the importance of early and aggressive therapy. How then can we ethically enroll people in a trial where they may potentially be on a placebo? Partially for such reasons, we are seeing many trials being offered in Europe. Some of our European neighbors have healthcare systems that make it difficult to get on medication. The MS research community truly is an international one. Every year information between the countries is exchanged at several different meetings.

Snapper Question:

Is methotrexate used widely in your clinic? Are there any large scale studies showing its benefit in relapsing MS?

Dr Ben: Answer:

We do use some methotrexate, typically as an add-on drug or in people who cannot tolerate some of the injectable medications. Studies have looked at methotrexate by itself and in combination with other drugs. The combination studies showed more promising results.

Test Question:

What is being studied in terms of myelin sheath restoration?
Dr Ben: Answer: Myelin sheath repair strategies have included cell transplants (Schwann cells and olfactory nerve ensheathing cells). Other strategies have looked at natural inhibitors in the CNS of myelin repair. Some research is focusing on inhibiting the inhibitors. Researchers are aware that caution will be needed, as some of these natural factors obviously are there for a reason.

Dr Ben:

A good resource for keeping up to date on neural repair strategies is The Myelin Project. They can be accessed online and they do send out a quarterly research update.

Moderator:

We have just a few more minutes. If you have more questions, now is the time to get them in!

Test Question:

What other resources can you tell us about for MS research?

Dr Ben: Answer:

The National MS Society's website has a list of studies that are recruiting for various projects. The Consortium of MS Centers also provides some of this information but it is a little more geared towards healthcare providers. It doesn't hurt to call MS Centers in your area and speak with the individual research coordinator. They can keep you up to date on what is going on in your area.

Snapper Question:

In a typical drug study, how often does a patient visit the clinic?

Dr Ben: Answer:

It varies depending on the study. For instance in trials involving treatment of spasticity, visits could be as often as every two weeks. More typically, visits would be on an every three months basis. Research visits are usually much longer than a typical clinic visit. The participant will usually see the research nurse, have blood drawn, and see both a treating and a blinded neurologist. Periodic MRIs are usually part of these clinical trials.

Alex Question:

What are the best ways to deal with balance problems associated with MS?

Dr Ben: Answer:

Balance issues are usually best dealt with by a physical therapist. The most common cause for balance trouble would be a decrease in position sense in the feet. While the therapist may not be able to bring that sensation back, they can work on strengthening the legs and coming up with ways of adapting to the sensory loss. One of the big goals of physical therapy is fall prevention as well.

Dr Ben:

Thanks to everyone for participating for not being able to get to everyone's questions. I look forward to being able to do this kind of event again in the future.

Moderator:

That concludes our time for this evening. Thank you for your participation, and a special thanks to Dr. Thrower for taking the time out of his schedule to join us tonight. I hope you all will return for the rest of the events in our series. The room will now be open for general discussion.



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