Peter was invited to participate in a drug research trial for patisiran, a now-approved medicine for the rare disease amyloidosis. It happens when a protein called amyloid builds up inside the body, potentially affecting organs such as the heart and kidneys.
In 2006, close to the age of 60, I semi-retired from stressful professional life as senior partner of a medium sized accounting firm. Three years later, I developed atrial fibrillation accompanied by breathlessness and a feeling of weakness for the first time. I was referred to a consultant who diagnosed early signs of heart failure and accompanying high blood pressure, which were treated successfully with ‘traditional’ controlling medicines.
I took steps to manage my health through diet, exercise, and reduction of stress to create a stable pattern of life at a slower pace. This endured for almost ten years. I met my consultant at least annually until he decided that his original diagnosis might have missed something, because the nature of the progression of my heart failure did not conform to the expected pattern. He suspected it might be amyloidosis, which was only known as a very rare illness at the time.
In 2021, the diagnosis was formally confirmed at the National Amyloidosis Centre (NAC) at the Royal Free Hospital. I was relieved that a diagnosis had been made. But this was countered by the news that there is no cure for amyloidosis, and that there are very few treatments for it.
The NAC contacted me a few days later and invited me to put myself forward to participate in a drug trial researching a possible treatment for amyloidosis – in particular, the drug patisiran, which is designed to suppress the body’s production of the ‘offending’ amyloid proteins so as to slow the progress of the disease.
Taking part in the trial
The research was conducted by Trials 4 Us, part of Richmond Pharmacology (RP) in Southwark. In February 2021, I attended RP for interview and tests as to my suitability to participate. I had blood testing, normal vitals (blood pressure, temperature, weight, medication review, etc) taken, and an interview with a specialist physician. These procedures sound simple but were conducted and recorded with great care and respect – the label ‘laboratory rat’ could not be applied.
I would join a small cohort of participants, all of whom would be at similar stages of the advancement of the disease. I was advised that the trial would run for two years with the possibility of a further two-year extension. During the first year, one half of the cohort would be given a placebo and neither patients nor any staff would know which persons would be receiving the drug.
The care was exemplary and every test conducted by the nursing staff was checked or witnessed (sometimes both) and time recorded to the second
On the day of my first dosing visit, I was collected by a smart black London cab and left home on time at 06:30. The cabbies are regular drivers for RP as well as working all over London. Their knowledge of the needs of RP participants is as extensive and helpful as their knowledge of travel in London. They are part of the reception team at RP and go out of their way to ensure a comfortable trip.
I attended Richmond Pharmacology every three weeks for a full day to receive a dose of the trial drug by infusion via cannula in the arm. This would be preceded by a review of my vital signs and a preliminary infusion of a protective pre-medication dose administered to suppress any later reaction to the infusion of the drug.
After dosing, all injection sites were examined and photographed. During the ensuing three days I regularly felt bloated and gained and then lost weight – I also felt a bit high and then low – possibly caused by the steroids. All this was reported and recorded during the medical reviews.
This was my routine for two years and was followed by a further 2-year extension, that has led to patisiran’s acceptance by NICE.
The care was exemplary and every test conducted by the nursing staff was checked or witnessed (sometimes both) and time recorded to the second.
'A life that is full'
During those years, there is no doubt that my condition stabilised – not cured but effectively treated. There was improvement but this may have been as much due to my reform in my personal care, activity, and diet. The new drug Tafamidis that targets existing amyloid deposits was added to my daily medications about 6 months before the end of the course and continues to the present day.
As the programme concluded, I was offered a cohort participant place for research into a successor drug vutrisiran – administered quarterly by injection and with no pre-med requirement. A much simpler and far quicker visit regime. I am now well into the second year of this course.
[This] has given me a future that I would not have expected or been able to enjoy
I am able to say that I am physically stronger and healthier than I was at the start of the process, over 5 years ago. The suppression of the progress of the amyloidosis and the stability that this has brought about has enabled me to maintain sufficient fitness to enjoy a regular and near normal life. Perhaps not the same ‘normal’ as everyone else, but a life that is full with family and friends and has given me a future that I would not have expected or been able to enjoy.
To anyone embarking on such a research project: it is worth thinking long and hard about what to expect and what may be gained by participation. In many cases stability alone might be a success. But it is important to realise that every participant has a part to play – one should not just sit back and wait for something to happen, nor should the dosing be treated as quasi private medical care.
The developer is seldom mentioned but deserves to be thanked, too. And I cannot overlook the endless support that I have enjoyed from my ever-optimistic wife.
Richmond Pharmacology have been exemplary in my case – professional, kind, generous and friendly. It is probably true to say that life is a progressive and terminal illness – in many ways, thanks to them, I have been given the time to fully prepare and come to terms with it without fear.