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Why I volunteer for research trials (and you should too)

I have Multiple Sclerosis. My life would be a great deal better if I didn’t have a progressive chronic disease. But, dare I say, there is one upside.

Before I go any further, let me be clear: by using ‘upside’ I am not trying to be ‘inspirational’, and I am certainly not glamourising life with a chronic disease. But I am trying to make a point.

I have had some unusual – even remarkable – experiences as a result of engaging with my disease and volunteering for medical research trials. I can’t get rid of MS, but I can work with it. These experiences don’t make up for the downsides (and the downsides are many), but they are great experiences. And I wonder why more people in my situation don’t seem to have them.

In the current trial I volunteer for, I‘ve been turned into an avatar. There is now a stick figure replica of me that moves exactly as I move, based on my height, weight, bone structure, gait and balance.

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In order to create this avatar, my measurements were taken and I was covered in stick on sensors by hard-working post-doc researchers. I then strutted around on a raised platform waving at my digital self (she always waved back) while motion sensors and cameras tracked my every movement. And yes, I admit, I pretended I was filming a CGI movie.

As I went through the exercises and tasks the researchers gave me, my strut turned into a stumble and my balance was, shall we say, less than perfect. Balance and coordination difficulties are two of the many varied symptoms of MS. In this case, my stumbling didn’t matter because the researchers were getting the data they need.

As part of this trial, I’ll be going back for two half days every six months for two years.

People ask me why I give up my time for medical research that may never benefit me.

I am doing this because the current treatments for Relapsing Remitting MS (the type of MS I have) are significantly better than the older generation of treatments. I benefit from these new treatments, and they are only available to me because people with MS gave their time and consent in the past.

When I was a child my neighbour had MS. She was in a wheelchair, and my memories of her are vague. She made me uncomfortable, and I used to ignore her or avoid her. Now, as a woman with the same disease in the 21st century, I am thankful I have access to treatments she did not in the 1980s.

She may have been one of the many people with MS who participated in trials and gave their consent over the last decades in Australia.

Remembering my old neighbour and watching the post-doc researchers collect data that may lead to new treatments or better management options made me feel good.

It also made me think: Why was the post-doc researcher so thankful I signed up? She was so apologetic about the time it would take me. She gave me the impression people say no more than they say yes. Don’t other people volunteer?

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Let me clear, I am refereeing to recognised trials in Australia’s leading universities and hospitals. I am not referring to any form of medical research or experimental treatment available at private clinics abroad.

In this case, participating costs me time. But depending on the trial, involvement can mean many things.

I was part of a double-blind placebo-controlled trial for a year, and my neurologist and I will only find out what drug and dose I took when the trial closes in a few years.

In other trials, I’ve given my consent for my medical data (everything from blood tests to MRIs) to be analysed. I’ve signed up to long-term studies, where my answers to questionnaires will be tracked over decades.

My final contribution to medical research will take place when I die: I’m donating my brain to the Victorian Brain Bank.

I am lucky. I live in Melbourne, a city with a vibrant medical research community. I try to participate in every trial a researcher or specialist asks me to. I think you should too.

The Progress in MS Research Public Lectures

MS research is controversial

Medical research is controversial. And medical research into Multiple Sclerosis tends to be more controversial than most. The stakes are high. Plenty of people gave positive feedback on my last post, and a handful gave what I am going to describe euphemistically as ‘constructive criticism’.

So I am going to let the experts speak for themselves.

Progress in MS Research Public Lecture

The 2015 Progress in MS Research Conference held in Melbourne in October 2015. MS Research Australia has released four public lectures covering the highlights of the conference.

Current and emerging treatment for MS in Australia: An update

Impact of social experience on adult myelin repair

Physical activity and exercise: A lifespan approach to maintain health

Progress in MS Research Conference: Latest news and developments

All lectures can be watched at MS Research Australia.

HSCT is not yet a treatment (although I wish it was)

Caveat: I am neither for nor against HSCT. I have RRMS, and HSCT may very well be in my future. But I am concerned with the recent media coverage, positioning the treatment as a ‘remarkable’ breakthrough that is able to ‘reverse disability’ and lead to a ‘cure’ for MS.

HSCT on BBC Panorama

HSCT (Hematopoietic Stem Cell Transplant) for Multiple Sclerosis hit the news in a big way this week, mainly due to the feature story BBC Panorama ran (you can also take a look at the associated articles here and here). BBC Panorama’s story was then picked up by other outlets, including SBS News in Australia and The Guardian in the United Kingdom.

It is a controversial topic, with all sides vehement in their point of view. I’m a bit nervous wading into the debate.

So, what is HSCT? Hematopoietic Stem Cell Transplant (HSCT) is an aggressive treatment that involves:

  1. Harvesting hematopoietic stem cells from the bone marrow or blood of the patient with MS
  2. Purifying and freezing the harvested stem cells
  3. Administering chemotherapy to the patient to destroy their immune system (which in MS attacks the central nervous system)
  4. Rebooting the patient’s immune system by returning the thawed stem cells through infusion.

I am confused at the coverage HSCT received this week. The coverage (led by BBC but also featured elsewhere) positioned HSCT as a ‘remarkable’ breakthrough that is able to ‘reverse disability’ and may even lead to a ‘cure’ for MS.I have MS. And let me be clear: I get excited when news about MS research comes my way.

In BBC Panorama’s story:

  • About 20 patients have received bone marrow transplants using their own stem cells in a clinical trial at Royal Hallamshire Hospital at Sheffield Teaching Hospitals NHS Foundation Trust over the last three years.
  • Some patients have experienced a degree of disability reversal. Two patients with profoundly positive experiences were interviewed:
    • Steven Storey, who went from needing 24-hour acute care to standing unaided after HSCT and four months of physiotherapy, and
    • Holly Drewry, who after HSCT no longer needs a wheelchair (but it was not stated whether she still needs a walking device).
  • The trial is international, with sites in the United States (Rush University Medical Center and Northwestern University), Sweden (Uppsala University) and Brazil (University of Sao Paulo). BBC Panorama did not report results from these trial locations.

But as a person with RRMS, I want to know what happened to the other 18 in the trial in the United Kingdom. Did they decline the interview? Did HSCT not work for them? Did they experience benefits that did not last? And given the aggressive nature of the therapy, did they experience severe complications? After treatment, do they still deem the risks worth it? And what about results from the other three countries in the trial?

These questions are important. Because despite this positive press by BBC Panorama, neurologists are clear: HSCT is not yet a cure , or even a proven treatment, and may not be effective for all types or stages of MS.

Neurologist Basil Sharrack, who is involved in the trial, stated in The Guardian, ‘[this treatment is only suitable for patients with relapsing remitting multiple sclerosis disease who have had two or more significant relapses in the past 12 months, failed to respond to standard drug treatment and who have had the illness for at least 10 years’.

And Dr Emma Gray, Head of Clinical Trials at the MS Society in the United Kingdom and not directly involved in this particular trial, stated ‘trials have found that while HSCT may be able to stabilise or improve disability in some people with MS, it may not be effective for all types of the condition’.

In Australia, MS Research Australia paints a similar picture. You can read the January 2016 position statement here.

HSCT is complicated. Current trials may prove that it is effective, or they may not. Or they prove HSCT effective for only a specific type or stage of MS. The point is, we don’t yet know. And the recent media coverage simplifies HSCT by suggesting it is a proven and effective treatment for MS.

What needs to happen before HSCT can become a therapy

This study (and possibly others) needs to conclude. This will happen in December 2017, when the results are expected. The results will:

  1. Confirm the type of MS and the stage of MS that HSCT is an effective therapy for (if any). For example, early indications suggest that HSCT may be effective for people with RRMS in the inflammatory stage of the disease. At present, there is little evidence that HSCT has a positive impact on SPMS or PPMS.
  2. Demonstrate the safety of HSCT over the long term, with the risk of each and every individual side effect catalogued and understood.

After the study concludes and the benefits and risks are known, HSCT will still need to be commercialised and the cost determined. I shudder to think how expensive it could be, and whether or not it will be covered by the PBS here in Australia. MS Australia has indicated that if HSCT reaches this stage the organisation will lobby hard for inclusion on the PBS, as they have for other MS treatments.

So my point is simple: if you have MS, don’t get too excited about HSCT just yet. Keep an eye out for more news, and most importantly, for the results of the clinical trials.

If you are interested in the results of international trials, MS Research Australia publishes reviews and updates.

And you can see me on SBS News talking about my personal views on HSCT here.

Living with MS and accepting risky treatment options

MS is a damn awful disease and those of us living with it often have a different sense of risk than those who aren’t. Sometimes we deem the risky side effects of treatment worth it.

For example, some of us take a daily tablet (Gilenya) that may slow our heart or cause liver disease, others among us accept a monthly infusion (Tysabri) that risks a brain infection that can cause death, and many of us regularly accept industrial strength steroids (methyl prednisolone) to minimise relapses. They are just three examples, for the list is long. But those treatments have all gone through internationally accepted clinical trials.

I understand that the risks that come with HSCT – the treatment involves chemotherapy after all – won’t necessarily put many of us off, particularly if our form of MS is debilitating.

While I am currently stable on my current treatment, I am all too aware that I have only had MS for two years and the road ahead of me is long. HSCT or another such aggressive treatment may be in my future. I am open to it. But I won’t decide until I see the results. I want to understand the extent of the risk I am taking.

But any decision about any treatment for MS should involve your neurologist. If your neurologist is hesitant about you undertaking HSCT (or any other therapy), there may be a medical reason why. Get a second opinion. But remember, the potential benefits of HSCT may not be deemed worth it based on your particular type and stage of MS.

I know some people with MS are willing to travel abroad and undergo HSCT outside of a trial setting. I would be interested in hearing from anyone who has taken such a course of action. Remember, I too have MS and I can learn from everyone else’s experiences.

But in addition to the cost, I can’t help but think any treatment outside of a trial and/or clinical setting entails risks additional to the treatment itself, such as inadequate long term support and follow up treatment. And I can only think that is a bad idea.

Still, I think there is hope for those of us with MS, and it has nothing to do with HSCT.

Remember: Other new drugs are coming soon

HSCT is not the first potential MS treatment that has recently made international news and been hailed as a ‘breakthrough’ treatment.

The most recent I have come across is Roche’s Ocrelizumab, which in late 2015 was called a ‘game changer’ by the media (for example, see the Daily Mail here and The Guardian here). This is because in Phase III (i.e. the final stage) of clinical trials, Ocrelizumab was shown to positively impact both RRMS and PPMS.

Let me say that again. In addition to treating RRMS, Ocrelizumab is the first treatment to show positive results for PPMS in final trials. That is a mighty fine development. Because at present there are no recognised treatments for PPMS, one of the most severe forms of MS.

Now that is news I wish BBC Panorama had spread around the world. And it is further news that should give the MS community hope.