The pain of waiting for a cure

Medical researchIn the wake of the debate on so-called ‘three-parent babies’ another conversation emerged on the issue of how long it can sometimes take for the legal permissions to be granted to enable new technologies to save lives. This week on The Today Programme a woman was interviewed whose son suffers from a blood disorder which leaves him exhausted much of the time. In a bid to find a bone marrow match for her son she had gone through 14 cycles of IVF, sadly not successfully. Because the law did not allow her to do this in the UK she had had to pay to have the treatment in Canada. She is now beyond the age when she can conceive in any way, so that hope for her son has gone. (Though thankfully gene therapy is now a possibility on the horizon for her son and those with the same condition).

I felt for this lady. I related strongly to her pain, seeing the hope of a cure slip away, and feeling that legal limitations were partly to blame. Our son also has a life-limiting illness. At 5 months he was diagnosed with cystic fibrosis – he is now 16. He is doing OK, living a pretty normal life, taking GCSEs etc. – but he has a lung function of 50% of that of his peers. Before he was diagnosed with CF he was falsely diagnosed with a mitochondrial disorder. We lived with that diagnosis for about a month until it was disproved. It was made very clear to us then that he would not have lived very long. Mitochondrial disorders are horrendous and a technology to enable those carrying genetic abnormalities in their mitochondrial DNA to have children free from these diseases is a huge breakthrough. At the time, to be told our son had CF and not this other disease was, ironically, a huge relief.

We also wait in the hope of something like a cure for our son. Gene therapy is the great hope for people suffering from CF. When our son was diagnosed the consultant, offering what hope he could, said that he thought that it could be a real possibility within the next 10 years. His prediction is 6 years out, and counting. And so we wait, and hope that it will not be too late for technology, and the law, to provide what we long for. Our son was last year invited to be part of the clinical trials that are being run to test the new gene therapies that are on the horizon. Unfortunately a long-standing infection that we have since got on top of prevented him being part of the trials. And so we wait whilst those trials take place on other CF sufferers and trust that at some point in the future it may make a difference for him.

The question is how do we wait? Do we wait passively? Leaving everything to the experts, after all they know best don’t they, and hope for the call or the email or the word from our consultant to say the time has come. Or do we press and fight and persuade, make a campaign of our lives for the sake of our son. I guess both extremes of this spectrum can be unhelpful, one is so passive as to seem uncaring. The other can simply overwhelm us and make the disease our living, breathing, every day concern to the exclusion of all else.

A healthy kind of waiting is somewhere in the middle – we are prepared to fight for the medical technology that might benefit our son but we also have to live our lives and not allow this disease to be our identity as a family. And we also have to trust that those researching new treatments, those conducting clinical trials, and those creating a safe legal framework in which they can take place, are working in our best interests. It is good to question and hold to account those working in these fields to ask to what extent these processes might be safely accelerated. But once we begin to put undue pressure on them for the sake of those we love we start to threaten the careful balance between the individual and the common good which these processes are designed to protect.

From the 16 years of taking my son to clinic and sitting down with the consultant each time to discuss things I have also grown to appreciate the close relational way in which the medical world works. Our consultant knows us, she knows the background to our family and our lives, she has known our son for 8 years. Whilst not a friend, our consultant is part of the warp and weft of the tapestry of relationships that make life what it is for us. She is also ongoing contact with other consultants in the UK and across the globe, many of whom are at the forefront of medical research into the disease. We have grown to appreciate hugely the degree of care and concern for our son as an individual amongst thousands of other CF sufferers. So if we begin to assert a kind of right for our son to have the kind of treatments sooner than is in realistically possible, what looks like rightful care and concern, actually threatens the relationships that have built upon which our son’s future health depends.

Trust, of those who care for our son’s present health and his future health, is a fundamental part of the system of medical advancement. Asserting an individual’s right to have treatments that are in development, or demanding undue acceleration in the careful process of research and trial of new treatments begins to undermine this trust. It says ‘we don’t think you care enough’. It says ‘you should be caring for my needs over and above the needs of the wider community’. Both assertions damage the relationships between sufferers, their families and those who care and fight for them on their behalf against life-limiting diseases. Both assertions potentially damage the delicate balance of relationships that are at the heart of the process of medical research and advance.