I was diagnosed with PD in 2003 at 43, which was identified by a process of elimination, apparently there was no clear test for PD at the time. The clearest symptom of which I was aware was micrographia (hand writing becomes very small and spidery), which prompted me to seek medical advice. I can probably recognise symptoms from around my mid-thirties, at diagnosis it is likely that you have lost 50-80% of the dopamine producing cells. There are people diagnosed much younger than I was, it is not solely an ‘older’ person’s disease and it affects people in different ways.
I was fortunate enough to have a good employer-based occupational health team so despite the attempts of the GP and private consultant to keep me poorly informed, I made contact with the Bristol branch of PUK. I met up with 3 or 4 people with young/early onset PD. We formed a group specifically for ourselves and people in a similar position; still working, young families etc. who couldn’t spare the time to get heavily involved with the branch, and partly did not want to ‘face’ a room full of people that could be the future. We mainly arranged fund raising and social events, Karen Rose became quite inspirational as our leader, one of the first to appear on national TV.
I migrated to lead the local early onset, or YPN group as it became, joining the PUK Bristol Branch committee. I also became involved with PPI group for the neurology dept in the North Bristol Trust of the NHS. The PPI group became part of the MOVE-HiT (Health integration team) of Bristol Health Partners, and I now chair this group and sit on the executive board of the HiT.
I continued working for c. ten more years, before I took retirement, as the symptoms became progressively difficult to deal with, (muscular rigidity is my main symptom, little or no tremor, anxiety (depression?) and had total dependence on medication, which I take every three hours.
The GDNF Trial
Through the association with the branch and the neurology team I became aware of the GDNF trial. It was at the forefront of research in to finding a cure for PD and sponsored by PUK and the Cure Parkinson’s Trust, of which the founder, Tom Isaacs, was on the trial. Early days I thought, lets watch this one, so towards the end of the recruitment for the trial I signed on. After a fairly rigorous assessment of health, memory, cognitive ability, reaction times, etc. Alan Whone and Lucy Mooney were great and said ‘you are an ideal candidate and you don’t need to do this’. Too late, I was tail-end Charlie for most of the next 18 months of a double- blind test.
The operation to install the deployment kit in my brain. A port on the outside, secured to the skull and requiring daily cleaning, with catheters which crossing the protective blood/brain barrier, deliver a minute amount of ‘liquid’ to the two putamen, the dopamine producing areas of the brain. The path of the 4 catheters is plotted and steered via a robotic Renishaw CMM to miss anything vital. Prior to my operation Steven Gill, the creator of the delivery mechanism, also manufactured by Renishaw, (and which is also being used to deliver different medication to other parts of the brain in different cases e.g. otherwise inaccessible tumours) came to me in the pre-op area at Southmead and said, “we have the installation right now, the BBC are in, can they film your op for training purposes!”
Not first on my list of priorities when I came to in the neuro ward at Southmead, with a turban of bandages on my head, and the mother of all headaches. After several rounds with the nursing staff over pain killers, ownership of my medication and a debate as to whether I could be released over Easter weekend I eventually left the building. Much that the neuro ward nursing staff could learn from the GDNF trial nurses.
In time the staples were removed and I was ready to go.
So 18 months of monthly infusions via the port connected to four ‘syringes’, a bit like something out of the MATRIX, if anyone asks! 18 months of alternate off and on meds video assessment interspersed with MRI scans, blood samples, reaction tests etc. and reams and reams of paper work. 18 months of sitting in the arm chair for 3 hours every month with one or two recognised faces amongst the recipients but getting to know the trial nurses who were just fab. I recall Alan dropping in to fit the connector to the port (just to keep his hand in I think), and being relieved of the duty by the nurses when he was trying too hard.
The worst, but possibly most evident test, is the DAT scan of the putamen at the beginning and end of the trial. An off-meds taxi journey to Cardiff, radioactive tracers, and lie absolutely still for 45 mins whilst we play Beatles music at you; not fun. In the end it turned out to be worth the effort!
During the first 9 months 50% received the placebo and 50% the GDNF, only the pharmacist knows who. In the second 9 months we all received GDNF. After 9 months I noticed the increase in my energy levels and was able to reduce some of my medication which helps minimise side effects, such as dyskinesia.
My progress with GDNF was noticeable and not just to me. My partner Caryl relates that, apart from the cats food bowl was always full of to overflowing, I was able, off meds, to get to and from the taxi taking me for assessment and infusions, unaided. At the beginning of the trial I needed help, and a wheel chair ride at Southmead.
In the meantime I had become an arm chair sports enthusiast for rugby, cricket, golf, F1 etc. and I probably went through a period of depression. As I had always played some form of sport I knew I was missing something as I was not active, but I didn’t know what it was.; I had played club standard/regional rugby, hockey, volleyball, squash (not cricket – the only time I played it was in a friendly where I handed the female umpire my box as it kept falling out of my shorts!)
…and then I was introduced to Nordic Walking, great for everything head to toe……. and as a result of the GDNF infusion I got it all, never been so fit or had so much energy. I NW’d the 2016 Bristol 10k in 1hr 22 mins passing a fair number of runners. Still attend classes twice a week with Bristol NW which is the biggest group in the UK, with new starters joining all the time.
I had the energy to develop a weekly non-contact boxing class (I have only hit the coach once!) at the Broad Plain gym and support the introduction of PD warrior classes which consist of a variety of high speed, large amplitude movements.
Then the results were in and the hard work began. The volumes of paper data meant it took a long time to process the data. The results took even longer time to resolve, Pfizer were rumoured to be considering whether to sponsor a much larger trial but pulled out (phuck Pfizer, thanks Vicky!) ……….and then Alan had to present the results to his peers and based on the test hypothesis set in the UPDRS, it was shown to be a failure. The documentary shows how the attendees were just dumbstruck and could not believe it.
We had an overall group feedback in June at the Bristol Hotel, where we met others for the first time and recognised that we all thought the GDNF was beneficial despite the results. The ceo of Medgenesis had committed $10m of his own money; he wasn’t about to give up. We hoped that the documentary would soon be aired to keep the ball rolling, however it could not be released until the technical papers had been published (BBC2 two part documentary planned for June 2016/7 was continuously delayed to Spring 2019)
There was great disappointment in the group; Alan had to keep rewriting the reports to satisfy the publications and demonstrate a positive outcome despite the results. Eventually Alan was accepted by a forward-looking publication that recognised the success of the biological results in the growth of cells in the putamen. In my case approx. a 25% – 30% increase in dopamine producing cells, identified by the DAT scan.
My last infusion of GDNF was January 2016; since then I have had to increase my meds beyond the stage before the trial., I couldn’t make it to the front door let alone to a taxi when I am off, and it is becoming increasingly difficult to maintain the exercise classes, both mentally and physically. The cat goes hungry.
There is a bright spark however that would not die, and in hope and adversity we regrouped and began to campaign to support involvement in the next trial providing the benefit of our experience for a further trial. Our support has led us to conversation with PUK (unheard of) in our potential role and influencing the archaic assessment of the results which does not consider the biological effects and the views of the recipients… and for some of us, the possibility of obtaining further GDNF. A forward thinking, small, agile organisation has evolved and phase 3a is on the horizon. We travel in hope that we could obtain GDNF on compassionate grounds and we work hard to keep that spark alive.