Sarah Long’s Open Letter To David Cameron

Readers, this is Sarah Long’s open letter to David Cameron:

Dear David Cameron

A life worth living with Morquio: A Funding request for Vimizim

Having Vimzim (elosulfase alfa) has not just extended my life, but made it worth living, and now the governance of this country led by yourself will make a decision if this Enzyme Replacement Treatment will be available in the United Kingdom. This decision will ultimately decide whether I will continue living.  I was in the final stages of Morquio (MPSIV) and did not have much longer however I am living proof this drug prolongs life as well as providing a quality of life.  If as it seems increasingly likely the UK will not provide this treatment, it will be my end. I hope you can prove this assumption wrong.

Can you imagine what it is like being locked inside a body, a fog distancing myself from others? Disconnected to an extent between what is going on around me and being an active player in it?  Everyday, struggling to breath, choking on thick mucus, ever amazed at the volume I used to cough up each day.  The pain echoing from the lungs as they struggled to function; my cervical spine and throughout my body feeling like someone has walked up and pulled two strings at the back of the neck tightly. Wondering if my arms, hands or other limbs will be working each day. Reflecting now on my past self I am aware that my body felt like a lead weight compared to now. It was hard work to do things, like wadding through treacle, a strong resistance to achieve anything. Never having refreshed sleep, the oxygen headaches, the fear carers had of me not waking up even though the reality of me sleeping was rare.  Have you ever been asked to leave a theatre because your breathing is too noisy? I have.

Maybe at 44 years old living with Morquio I ask too much to live longer. I can’t imagine what my parents felt when they were told when I was 6 I would live until I was 15 years old. I can’t ask either as my mother died from cancer when I was 18.  This is when I had to become independent. It was her battle with cancer and her attitude to forging teaching roles for women in a very male dominated school (Ampleforth) that inspired me to push myself.  Education was important to my mother and she pushed for me to have one. Initially I was not entitled to state education, the local primary school refused to enrol me, as this was when I stopped growing.  My parents chose a private convent school at 6 I remember well the fear that gripped me when I went there each day. Other kids were not allowed to play with me in case I got hurt. This endorsed rejection meant not only bullying, jeering from children, but also from teachers.  I moved when my parents realised the horror of it, to a prep school although a struggle as they had to pay for it. It also meant painfully for my mother and myself having to leave home at 6 years old for boarding school. My school years were isolating. I remained in residential educational establishments all my life, private then a special college when disabled children were at last provided with some form of education. When I was growing up I remember the frustrations of finding it difficult to walk, watching on the side-lines as I could not keep up with others and it was hard to engage practically with lessons. It was not just my legs that began to fail but also my wrists. I remember being bullied because I could not sharpen a pencil! My pediatrician spotted that I had intelligence and insisted I learnt to type as my writing was going to be one of the first thing to go.  It did. I loved art and drama but as my arms and then voice began to fail I could not continue. Life for children with Morquio is very different now with touch screens, laptops and tablets.

I call myself a special school survivor because it was a difficult place to reside, particularly since many pupils died and people used to wonder if I was going to live through each year.  I rebelled at school by studying, as this was counter-cultural! I got an undergraduate place at the University of Bath, and I had access to the independent living fund so was able to cope living and making my own choices in the world. I worked for 15 years before my health began to make me withdraw more and more from working. This was a major frustration. My most significant deterioration was my breathing, as my lungs got restricted, my immune system began to fail and slowly my world got smaller, I saw friends less and less, my ambitions and drive got squashed. I did however pass my Masters of Research with distinction, hoping I could somehow undertake freelance research work; I then got offered a funded Phd.  My world however seemed to drift away from me shortly after as it became a struggle to function, I did not sleep and was diagnosed with hyperventilation during the day and hypoventilation at night. It was like someone just pulled out the power plug during the day without warning! I did not sleep more that 2 hours at any given time. I was in what they term the final stages.

It was not a difficult decision to join the BioMarin trial for Vimizim and it felt a real privilege to be asked to give back to others with my impairment; I started treatment on the 29^th February 2012. I am utterly amazed how Vimizim (elosulfase alfa) has benefited me I would not have been able to draft this if I had not the treatment!  I only had one infection at the beginning of my participation, with Vimizim attributed to my extremely quick recovery.  It only took four weeks to recover from pneumonia, previously pre-Vimizim it took nine months. Over the past two and a half years I have been free of respiratory infections, and although my support workers have aliments and I spent time at the university I have not picked things up!  I sleep for over four hours a night, that is so refreshing compared to before. Although still reliant on a nebuliser four hourly, I don’t need the oxygen.  My clinical evidence validates the Vimizim research data as my oxygen and carbon dioxide levels have stabilised particularly during my sleep.  I have my voice back, and my body feels so much lighter than it has for twenty years.  I was so unaware of how I had slipped into this world of “heaviness” although I feel lighter, I have not had my full physical strength return, after all my joints were too damaged and many of them have collapsed. The pain however, has become less and so much easier to manage.  I am able to cope with my independent living scheme taking on the employer role saving the local authority money and dealing with the now imposed changes. Previously I would get very anxious about these things particularly given I have no family support. My PhD supervisors noticed my written work had improved significantly probably because my oxygen levels were better and it looks like I could of finished my thesis by the beginning of next year. I heard on the Monday before NICE published its finding I have an academic article accepted for publication ‘the intrinsic quality of life’. I am also able to drive my van further, and spent last weekend in Devon with friends. I was beginning to get active and be back in the community.

It is terrifying that my last treatment is tomorrow having benefited from BioMarin generous compassionate provision, and there still is no solution or decision in the UK. It has to be a testimony that the treatment is a success given that having a drip every week (it has to be every week) for 4-5hours is a tough regime, but you go through it as you know you (or your child) benefit from it. It is surprising that over 30 other countries are providing the treatment and the UK is not, ironic since I will without it be trapped in this country unable to fly to seek it elsewhere. As an academic and former civil servant at local and national government levels it’s troubling that there is seemingly a lack of accountability. Along with the worrying fact that rare impairments’ groups are having their treatments evaluated by instruments designed for mass (larger population) treatments. As you know families with rare diseases have very different experiences of health care services compared to other larger impairment groups. Humanity is pluralistic by nature and we rare folk are part of it! So what now, for me? It feels like a death sentence will I live or die, this is what you are deciding.

The faces behind the label ‘Morquio’ are not just children there are also adults, as medical advances take place the kids of today may grow up to and experience my dependent state that I ended up in. My experiences demonstrate that this treatment enables regression of the disease, and has stripped away many of the restrictions Morquio imposes. To support these claims I can provide witness statements from friends, assistants and clinical evaluations.  There can be no doubt that this drug does prolong life. Contradicting NICE conclusions. I am concerned as I read NICE related documents that they have come with a predetermined agenda masking their potential lack of knowledge of living with Morquio. The applied matrix evaluation tools overwhelm our small population that have Morquio, so as a consequence our lived realities are not captured within their conclusions. Many of the questions they want answers for would need longitudinal studies; of course being a rare impairment means there is a lack of knowledge e.g. peer articles and any study and/or evaluation is always a learning curve. They have failed to authenticate the realities of the aging process aligned with the degenerative experiences for people with Morquio who survive into adulthood both physiologically and psychologically.

Access to Vimizim is a matter of life and death – I personally beg you as someone you are likely let die over this, not to let the young people experience what I have. Although you are aware of your own fragility you also worry about how your family and friends will cope. Without Vimizim I can make that academic assumption that I will slip back into isolation, suffer the poor consequences of having a bad immune system. It is unknown what will happen as we were told to have it every week, and my body craves it, I am dependent on the drug. I had dreams again, ambitions, but this is now most likely to be taken from me if NHS England vote no to interim provision this week, and NICE rejects it in October. Now more care will be needed as I slip back to that dark place I was trapped in before, solitude you probably cannot imagine. It was wonderful being able to laugh again, reflect on academic challenges, be ambitious, and begin to live.  I only wish I lived in a society that thought my future was an investment, now I find myself dehumanised by the processes in place, a non-person.  I am left desperate, imploring you to intervene and provide this innovative and effective treatment. Vimizim creates a future full of opportunities, a stability that means less dependency on the health care services and promotes overall wellbeing.

Please support the funding of this treatment, not only is Vimizim innovative, the first cases tried here in the UK, this is a legacy that you can lead to change a lifetime.

Yours sincerely

Sarah A. Long BSc(hons) DipSW MRes