What Vimizim means to Angela

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What Does Vimizim Mean to Angela?

As featured in out Autumn 2014 Magazine, Angela describes her experiences with Vimizim, and the drastic effect this drug has had on her life.

I was diagnosed with Morquio Syndrome in the late 1970s at the age of four and grew up in the 1980s and early 1990s when a treatment for Morquio was still a far off hope and not something I expected see within my lifetime.

In 2008 I attended the MPS Society Morquio conference in Northampton where it was announced that enzyme replacement therapy had been developed by BioMarin and that it would soon be ready for human trials. I was in my 30s by this time and so, while I was thrilled that there was now hope of a treatment, I was also very much of the opinion that this would be something that would only benefit young children and future generations. I assumed that for adults like myself, the damage was done so to speak and it would therefore be too late for ERT to have any real effect. I was aware in the following years that clinical trials had begun in children but it was quite a surprise in 2011 when my consultant told me the clinical trial was now going to be extended to include adults and asked if I would be interested in taking part.

Upon reading the consent form I found the prospect of taking part in the clinical trial, at first, rather daunting. It was still an experimental treatment with a number of potential reactions and side effects. I was also very uncertain that I could physically manage the demands of the trial. Living in Leeds and the nearest trial site being Manchester meant I would have to drive myself to Manchester and back every week for infusions. This is a 120 mile round trip, making it a very long day, and in addition to which, I would still have to go to work for the rest of the week. As I was working a five day week at that time, albeit slightly reduced hours, it also meant that I would have the financial burden of further reducing my working hours in order to accommodate the weekly infusions.

It was not an easy decision but my decision to take part in the trial, in the end, came down to two things. Firstly, if I could manage it physically, then I wanted to play a part in the development of a treatment for the benefit of everyone with Morquio. Especially for those who, for whatever reason, were not able to take part in the trial themselves but would benefit from a licensed treatment. Secondly, from a health point of view, I felt I had nothing to lose. My stamina, energy and ability to manage everyday tasks had been reducing for some years, but especially so in the last few years. I had recently had to move to a bungalow due to my increasing inability to manage stairs and despite having reduced my working hours at little each day, I was still struggling physically most days. I therefore decided to go ahead and give it a chance. I felt that if the treatment improved the way I felt then that would be fabulous. Even if it just slowed down the progression of the disease then that still would be a bonus and if it had no benefit at all then at least I could move on knowing that I had tried and not left wondering ‘what if?’

I had my first infusion in February 2012, aware that for the first 24 weeks of the trial I may not be getting the treatment at all but rather just a placebo. My employer was incredibly accommodating, reducing my working hours, allowing me to work a four day week in order that I could have my infusions and giving me paid time off for my assessments. I was incredibly nervous for the first infusion, however, everything went fine. I had no problems or initial reactions to the infusion and so began the weekly trips to Manchester. I was very surprised at how quickly I adapted to making the trip to Manchester every week. It is surprising how something that at first seems so daunting can so quickly become routine.

For the first 7 or 8 weeks of the trial I felt no different. Naturally, I was constantly wondering and speculating as to whether I was on treatment or placebo but as I had felt no change and had no initial reaction to the infusions I was swaying towards placebo. Then, after around 7 or 8 weeks of infusions, I began to realise that I was feeling physically better. Prior to starting the trial I spent a great deal of my time feeling stiff, sluggish and generally very tired. Everyday tasks such as getting showered and ready for work in the morning would often leave me feeling worn out and I was struggling to maintain a balance between being able to work and still having enough energy for a normal social life. Even though I was working slightly reduced hours, I rarely had the energy to do anything in the evenings, going to work was as much as I could manage and most weekends I would need to spend at least one full day just resting at home to recover from the working week.

Approximately 7 or 8 weeks after the infusions had started this began to change. The first thing I noticed was that I wasn’t as tired after getting ready for work in the morning or feeling exhausted at the end of the day when I came home from work. As my energy and stamina increased I found that I was able to do more. I was able to do simple things like go out for a meal or to the cinema after work where before I would often have declined these invitations, being too tired after a day at work. I also found I was able to do more at the weekend, no longer needing to rest as much. Being at work became much easier and more enjoyable too as I began to feel less tired, stiff and sluggish. While these may seem like small improvements, just being able to get on with life and enjoy my leisure time as everyone else does without constantly feeling exhausted has had a very big impact on my quality of life.

The enzyme (Vimizim) is not a cure for Morquio and cannot undo the bone damage that has occurred. I still have a lot of physical limitations and need assistance with many aspects of everyday life. I still have to live with a lot of joint pain and severely restricted mobility but I have found that the pain is a lot easier to deal with when it is not accompanied with the tiredness and stiffness that it previously was. Joint pain that at one time would have resulted in me taking time off work or cancelling arrangements, I now find I am able to deal with and still manage to get on with life.

Two and a half years on I can undoubtedly say that my quality of life as improved as a result of being on this clinical trial and receiving this treatment. Overall, my working week is now much longer than it was prior to starting the trial. I now work and extra hour per day on the 4 days that I do work in addition to leaving home at 6.30 am every Wednesday for a full day round trip to Manchester for my infusion. Yet despite this I still have so much more energy than I ever had previously and manage to take part in many more social activities.

For those of us who are lucky enough to be receiving this treatment, the thought that it could be taken away from us if the NHS does not deem it to be worth the cost is devastating. Despite all of this uncertainty, however, I am still incredibly grateful that I was given the opportunity to take part in this clinical trial and in the two and a half years I have been on the trial there has never been a single occasion when I have regretted my decision to take part in it.

Finally, I could not write about my experience on this trial without mentioning and saying thank you the wonderful and dedicated medical staff who have cared for me at the Manchester Clinical Research Facility. Not only the doctors, nurses, physiotherapists etc who are directly involved with the trial but also those nurses, doctors and other staff on the ward who care for me week in and week out during my infusion visits. It is in no small part due to the kindness, warmth, compassion and humour of all of these people that I have managed to cope so well with both the physical and emotional demands of this trial and to all of them I will always be grateful.