BMJ Research Forum - giving a voice to patients

Can Parkinson’s 'catch a wave'?

I’m a newcomer to the medical research and technology scene - just about everything I write about here involves me trying to understand a new subject, whether that is the byzantine structure of the NHS or how gene therapy works. So I was very flattered to be asked to moderate a panel on Parkinson’s research at the British Medical Journal’s inaugural research forum.

Picture: BMJ

My involvement and the presence on the panel of two people plugged into the Parkinson’s community - as well as two leading researchers - seemed to send a clear message that the voice of the patient has to play a bigger role in framing research.

And the scene was set for our discussion by the forum’s opening speaker Sir Chris Whitty who delivered a no-holds barred critique of the state of medical research and the interaction between scientists and policymakers. First, England’s Chief Medical Officer flattered his audience - their work was transformational - then he laid into them.

Too much research was like primary school children playing football, charging after the ball “where it is now rather than thinking about where's it going to be in the future.”

Researchers were not good at addressing multimorbidity, people with several conditions at once. (As someone with an ocular melanoma as well as Parkinson’s I nodded along to that.)

This meant there was a great need for a multidisciplinary approach but too many researchers were arrogant and dismissive about disciplines they didn’t understand such as the social sciences. (I could imagine my wife, a leading economist, nodding along to this.)

Researchers, those who funded them, and journals like the BMJ which published them, were focusing all their firepower on areas such as cardiovascular disease, leaving mental health and other less “exciting” conditions out in the cold.

And while the two big drivers of ill health were age and deprivation, most research took place in North Oxford and North London, not Eastbourne or Blackpool.

We kept on returning to Sir Chris’s themes in our panel on Parkinson’s. The two researchers outlined some promising developments in the long hunt for a treatment which can slow or stop the disease, rather than just mask the symptoms as drugs like Sinemet have done for sixty years.

Professor Steve Gentleman explained how the Parkinson’s brain bank he runs was advancing understanding of how the disease progresses through the brain so that drugs might be found which could halt that process. And reflecting Sir Chris’s point about too narrow a focus in research he said Parkinson’s was a disease so varied in its symptoms and with so many co-morbidities that his work was “all about multidiscipliniarity .”

Professor Steve Gentleman runs the Parkinson’s Brain Bank

Professor Tom Foltynie enthused about the Phase 3 trial of the diabetes drug Exenatide, which had shown promising signs in earlier tests that it could slow the progress of Parkinson’s. The trial was designed to include a wide and diverse range of people - “we're trying to look for the treatment that may help the larger population of patients with Parkinson's.”

But Juliet Tizzard from Parkinson’s UK and Natasha Ratcliffe from Couch, a health engagement agency, said getting a diverse range of patients involved in research was still a work in progress. One key lesson, said Juliet, was that people who did get involved in clinical trials needed to be kept informed at all stages:”We are entirely reliant on participants for research to progress. Then if you take part and you don't hear what the outcome was of that research, which we know people are really interested in, you're not very willing to take part again.” 

Natasha Ratcliffe talked about the challenge of getting underrepresented communities involved in trials:”We are not going to be able to do that if we're just going to ask people to come to us and work in the current system. If we're really serious about that, we need to think how can we do things differently and to meet people where they're at.”

But she stressed that the investment in getting a wide range of people involved from the beginning was worthwhile - a single amendment in the protocol of a trial once it was underway could cost as much as half a million pounds: “We know there's evidence that involving patients in the process, pre phase two trials, pre phase three trials, can reduce the amount of amendments that are needed.”

I wanted to know whether patients could influence the kind of research that was funded. Drug trials are expensive but presumably far more glamorous for both researchers and funders than investigations into areas such as exercise and diet which people with Parkinson’s know can help manage the disease.

Tom Foltynie, who along with his drug research is still an active clinician, said it was important not to give people false hope about the imminent arrival of a wonder drug: “You want to be honest with people and you want to first empower them, tell them what they can do for themselves, in terms of diet and an exercise regime, which is really important and shown to be useful, slowing down disease progression.”

“I think people affected by Parkinson's have been integral to get to where we are now and getting funding allocated so that we can have strong evidence for the role of exercise,” said Natasha Ratcliffe. “People with the condition have been saying for many years ‘this works, this improves my condition.’”

Earlier, Sir Chris Whitty had told the audience of the importance of “catching a wave”, recognising the moment when policymakers and funders suddenly take an interest in a medical condition and making the most of the brief window of opportunity.

Maybe the Parkinson’s wave is about to arrive - certainly there are plenty of brilliant researchers working on promising treatments. There is also a. vibrant, well informed and realistic community of “Parkies” eager to help shape that research. Let’s hope that together we can ride that wave when it comes.