I’ve recently been diagnosed with Parkinson’s. If you were me – which research project or trial would you volunteer for?
This question is being asked by a woman in – oh, her early thirties? She’s near the front so it’s a bit difficult to see from my chair at the back. Although, being by a window, I do have an amazing view of the misty city’s domes and spires. From the ninth floor of the Bentley Institute building, we are looking down on St Paul’s. We’d spent a little time before the meeting started picking out landmarks and talking with one of the staff. ActorLaddie asked if they had a roof garden. No, apparently they had a domed roof. So not the best shape for a roof garden. Unless, I suppose, a hanging one.
So that you don’t burst with suspense, I’m going to tell you now that the answer to the woman’s question was, in essence, it depends.
I think that it’s a very positive sign about the progress being made in research that this young woman not only knows that there are things happening, exciting things happening, which are going to defeat Parkinson’s but also that there is so much going on, she might actually have a choice of projects.
Range was the key thought resonating through the Cure Parkinson’s Trust research meeting last week (11/4/18).
In his introduction, Dr Richard Wyse had pointed out that there would be more than twenty different clinical trials taking place by next year, in over eighty hospitals. So a great range of studies.
But Parkinson’s itself is a condition with a considerable range of symptoms, effects, challenges, outcomes. There’s an immense difference between the Parkinson’s of, say, someone diagnosed with a tremor in their twenties and someone diagnosed with stiffness, memory and balance problems in their late seventies.
So the key challenge for researchers is getting the right patient involved with the right trial. For example, suppose a drug is tried with ten patients and found to have a positive effect on the five younger but not on the five older. Without looking carefully at the range of patients, a misleading conclusion might be drawn that, on average, the drug was not effective.
So a range of volunteers is needed to support the range of research programmes and Richard urged patients to get involved.
Professor Huw Morris gave an optimistic talk about his work focusing on the biology of PD and particularly genetics. It’s a complicated subject, and I can’t hope to do it justice in a summary: the whole talk will be available soon through the CPD website, I believe. Some of the points which stood out for me were:
- An underlining of the range of PD experience: in some people the condition seems very stable, perhaps not changing much over twenty years; in others it may move rapidly from the start.
- So, if you’re asked about a whole load of symptoms you don’t have, this doesn’t mean that you will necessarily get these symptoms. It’s just a reflection of the variation among people with PD.
- Only a very small percentage of PD is inherited: less than 5%. But beyond this, there are genetic variations which make people much more or a little more susceptible to developing the disease.
- Learning more about the biochemistry and genetics behind the various manifestations of PD will facilitate getting the right patients on the right trials.
- Also, this knowledge might make it possible in the future to pick up and address PD pre-symptomatically.
- Key phrase: precision medicine.
Professor Roger Barker asked the question “Is PD one disease or several?” His area of work is looking at large groups of people – and we’re talking, all of Cambridgeshire – who either have or might have PD; collecting and analysing information about those people and monitoring their progress over years. He’s done this with two separate cohorts, fifteen years apart. He’s then gone on to compare results.
Again, there was lots of information to take in but well worth a look once it’s online. The points I jotted down were:
- Having PD does not affect the rate of mortality. That is, PD doesn’t kill you: you die with and not from
- The dominant factor governing progression paths appears to be age at diagnosis. In his most recent study, the average age at diagnosis was 69 (though 59 in the USA – I have no idea why). The older you are at diagnosis, the faster the condition tends to progress, in general. Though, of course, not for everyone.
- Knowing more about PD and the direction it is likely to take means that research can use smaller, more focused, cohorts. Maybe, even, without the need for a control group. If the PD doesn’t progress along the predicted path, something is having an impact.
- Roger considered Parkinson’s to be one ‘disease-process’ which can be entered at different points, and – like cancer – can take more benign or more malignant forms, requiring different treatments.
As a post-script, Roger also told us a little about the work going on around taking a sliver of cells from the skin and turning these into neurons producing dopamine. This is yet to be tried on humans in the UK but the lab work is looking promising. Not a cure as such but a possible relief to symptoms while a cure is being found.
After the keynote speakers, there came the chance for more questions. Then refreshments and networking.
The second half of the afternoon looked at the Fatigue in PD, which was defined as not being the same as ordinary tiredness, apathy, lack of sleep but, rather, when you have “the urge to horizontalise.” Aka, a bit of a lie down. This is now my new favourite phrase and I expect to use it frequently.
This was the first Cure Parkinson’s Trust Research Event that I’ve attended since co-founder Tom Isaacs died last summer. But his spirit was very much there, his name frequently mentioned and his legacy bursting with life.
Btw, the title has no relation to this blog. But it made me laugh. Thanks LegoBoy