Saturday, October 22, 2011

Where is the PPMS research at ECTRIMS 2011?


When I wrote that I was coming to ECTRIMS 2011 to report on the most current MS treatment and research on the disease, I got several e-mails and blog comments from people with primary progressive MS (PPMS), who were anxious to hear what treatment was in development for them. My friends, I wish I had better – indeed, any – news to report. At this conference, there was no research presented about developments in treatment of PPMS.

Why the huge disparity in research activity around treatment for relapsing-remitting MS (RRMS) and PPMS? Of course, the first thing that comes to mind is that PPMS treatment has much less potential for huge profit than a successful RRMS drug, due to the fact that only 10 to 15% of people with MS have PPMS, with the vast majority having RRMS.
I won't lie to you – the bottom line is definitely a factor. However, there are other big challenges to running trials to test medications specifically for PPMS, though. Many of these have to do with designing a study that would give any meaningful results. Remembering that virtually every clinical trial is complicated by difficulties in recruitment, follow-up, unexpected side effects, adherence, etc., which makes any clinical trial a huge and expensive undertaking. Even for a motivated group of researchers, any of the characteristics of PPMS as a disease and the population with PPMS make trials even more daunting.
Disability: Many studies require people to be ambulatory (able to walk) in order to be included in a study. This is so that the effect of the study drug on walking ability, energy level and other physical abilities can be studied by having people walk a certain distance within a certain time. In this way, researchers can use scores on the Expanded Disability Status Scale (EDSS) as an outcome measure. Many people with PPMS are no longer ambulatory.
Age: People with PPMS tend to be older, meaning that they often have additional age-related health problems, such as cardiac issues, diabetes, high blood pressure, vision problems and others. This makes it more difficult to determine which disease (MS or one of the other ones) is causing or contributing to disability. It also makes it difficult to determine which symptoms are side effects from the study drug and which are due to underlying health problems. In addition, people with other conditions may need to take other medications, which would exclude them from the study, as interactions between medications would not be known or could interfere with results.

Side Effects: It may be harder for people with PPMS to tolerate side effects from the study drug, both due to MS-related symptoms, other health issues and age.

PPMS Is Unpredictable: PPMS behaves differently in different people — some people experience a very quick progression to disability and others progress very slowly, often experiencing “plateaus” of several months or even years where symptoms remain stable. This variability makes it very difficult for researchers to compare placebo groups to treatment groups.

People whose PPMS progresses very slowly may have to be eliminated from the study so that researchers could figure out if the drug is doing anything (otherwise the study would have to continue for many years). That would leave only the people with quickly-progressing PPMS, though, which would also not be a good test of a drug, as these cases may not be representative of all (or even most) cases of PPMS.

Basic Research is Being Done: All of these points are just to illustrate why research on PPMS treatment is very difficult. However, this does not mean that scientists are ignoring PPMS. There are some studies in very early stages, many still in animal models, exploring treatments that regenerate and rebuild myelin and lost axons, as well as studies on DNA and other biomarkers that will eventually guide treatment.
In addition, researchers are starting to explore different technology, such as triple-dose gadolinium, and more powerful MRI scans to learn more about the effects of PPMS that have been difficult to assess until recently. I would have loved nothing more than to bring news to you about a "breakthrough" in treating PPMS, hopes are that these developments will eventually help in the search for effective therapies for people with PPMS.


  1. The numbers are somewhat skewed as to the amount of progressive MS cases as well, My doctor diagnosed me with PRMS, but documents it as RRMS, so the insurance company will pay for treatments that help me....Tysabri treats relapses, but as soon as insurance see the progressive in front, they decide theres nothing that works for us. So, alot of doctors downgrade the diagnosis so their patients can get some form of treatment, any form

  2. My first written diagnosis was for RRMS that the doctor crossed out on my copy and wrote Primary Progressive by hand. I'm on meds that are usually for RRMS and haven't had any trouble with my insurance.

  3. One doc from a highly regarded University diagnosed me with PPMS, several others are not sure. I am going in monday for lower spinal surgery as my spinal cord is being squashed...praying for a miracle. God help me to be available for my family.

  4. Nani said...

    "My first written diagnosis was for RRMS that the doctor crossed out on my copy and wrote Primary Progressive by hand. I'm on meds that are usually for RRMS and haven't had any trouble with my insurance."

    Sounds like he is doing same as my doc, by documenting you as RRMS, then putting PPMS on your personal copy, hes letting ya know what ya got but passing the RRMS diagnosis to the insurance.

  5. I was diagnosed in May 1995 with PPMS and told by my treating neurologist that I had 2 years. I had just had my 30th birthday and two small children. With no other options open to me (and at the time having no health insurance) I went into human subject’s research at well respected university where I was re diagnosed as chronic progressive MS. The terminology for typing MS has changed a lot over the past 17 years.

    I spent 8 years doing human subjects research as I was once again re diagnosed as secondary chronic progressive MS so I could be in the studies. I did three trials of different medications. One became an FDA approved IM injection for RRMS the other two where failures. I ended up with thyroid cancer as a result of one of the studies.

    In 2007 I was re diagnosed again as RRMS and continued imunomodulating therapy. However: at the end of 2009 my MS took a progressive turn and in January 2011 I was forced to stop working as an executive as my MS is now PRMS and I could no longer handle my duties.

    I am still fully ambulatory and recently began using Ampyra for walking which has been a real blessing.

    I asked several very well known and respected neurologists why there was no real research into the progressive population, as 85% of people with RRMS will become PRMS. I was told just what Ms. Stachowiak said in her article; that there is “no money for drug companies in progressive MS.” It is sad but true, even though almost everyone diagnosed with MS will become progressive at some point in their lives, no one is researching it.

    In the end it all comes down to dollars; and there are no dollars in progressive MS. We can paint all kinds of scenarios of what would make research in humans difficult but the reality is there is currently no trials going for progressive MS and based on my conversations with neurologists in the know……and there won’t be.

    We with PPMS and PRMS are the forgotten, lost, outcasts of the world of MS. It is our jobs to keep fighting as long as we possibly can until the inevitable occurs. I wish I could be more upbeat but Ms. Stachowiak is correct there is nothing to be upbeat about.

  6. I have PPMS. I'm 63 and am taking part in the Novaris fingolmod study. Fingolimod is approved for relapsing forms of MS but not progressive. Novaris wants to see if this drug can help slow down the progression in PPMS. Its already known that it slows down relapses.
    Yes, My Dr told me it has to do with money also. He said that PPMS behaves so differently from the other forms of MS that "it might as well be an entirely different disease." And I understand the drug companies not wanting to spend money on a relative few in the population.
    All we can do is keep taking part in the studies, keep our hopes up and keep moving. Keep walking, keep swimming and keep as active as possible. MS is the ultimate "use it or lose it" disease.
    I would love to communicate with others who have PPMS on email.