Parkinson's - hope in a headline

This time it really could be a breakthrough

Hope at breakfast

As I sat eating breakfast at 07.15 on Thursday there was a ping from my phone as an email arrived. It was from one of my Parkinson’s friends and contained a link to a Financial Times story.

The online headline read “Quest for pill to stall Parkinson’s disease takes step forward.” Now the FT is a very serious newspaper and this was a pretty sober take on an encouraging drug trial - not exactly the “Miracle Cure!” line you might get in a tabloid. (Though the newspaper headline - “Pill offers hope for Parkinson’s victims” - was a little more straightforward.)

But the fact that my friend had chosen to email me at such an early hour shows two things about we “Parkies” - we are desperate for some positive news about our condition and we tend to rise early because we are poor sleepers.

The drug which has got our hopes up goes by the rather utilitarian name DNL201 and aims to suppress excessive levels of an enzyme, LRRK2. This enzyme can damage a kind of waste disposal system in the brain and that in turn can harm the production of dopamine, a lack of which is at the heart of Parkinson’s.

The phase 1 trial of DNL201 showed that it was safe and it did reduce the enzyme levels, though the study was not designed to measure any reduction in Parkinson’s symptoms. There will now be a phase 2 trial, not of this drug but of a related one DNL151 with 640 people in the early stages of Parkinson’s.

Although the article by the FT’s excellent science editor Clive Cookson mentioned the hope that the drug might slow, halt or reverse the progress of the disease - the Holy Grail which has evaded researchers thus far - I came away from reading it slightly dissatisfied. After all, there have been so many “breakthroughs” reported over the last year or so. I suspected that we were still at a very early stage with these experimental drugs, that it could take many years to complete the trials - and then the results might prove inconclusive.

So I picked up the phone to Simon Stott, deputy research director at Cure Parkinson’s, one of the people quoted in the article. I’d expected him to be cautious and downbeat, confirming that there was a long way to go.

Not a bit of it, he seemed genuinely excited. He explained that, unlike the repurposed diabetes treatments I had heard about at the Cure Parkinson’s research day back in May, DNL201 and DNL151 were something completely different: “These are a brand new class of drugs, and that's why it's exciting.”

It had all started with the discovery of a mutation in a gene which could be linked to Parkinson’s and there had then been ten years of biology research before anything was tried on patients. In other words, we were already a fair way down the track. The phase 2 trial should be completed by 2025 and if it was successful - still quite a big if - then the American drug regulator the FDA could approve it for clinical use by 2026. (Back in January Simon wrote a long blogpost about the science behind what are known as LRRK2 inhibitors.)

So there are real reasons to be cheerful about progress towards halting Parkinson’s, and Simon Stott thinks there has been too much negativity for too long: “I get a little bit uppity when people say it’s been 200 years and there's been no progress. What they fail to appreciate is, if you look at all the research publications on Parkinson's, 80% of it's been done in the last twenty years.”

Let’s hear it then for all the researchers now attacking this, the world’s fastest growing neurological condition, from every angle.

On that note, I went on a day trip to Exeter this week to enrol on a clinical study about Parkinson’s, genetics and sleep disruption. Or to give its full name:

“Study on the effects of single nucleotide polymorphisms in -4 (AQP4) gene on the clinical phenotype in patients with idiopathic and familial Parkinson’s disease.”

More on that soon….when I’ve worked out what it all means.