The great ice hockey player Wayne Gretzky once said “A good hockey player plays where the puck is. A great hockey player plays where the puck is going to be” (the original quote actually came from his father, Walter).
At the start of each year, it is a useful practise to layout what is planned for the next 12 months. This can help us better anticipate where ‘the puck’ will be, and allow us to prepare for things further ahead.
2017 was an incredible year for Parkinson’s research, and there is a lot already in place to suggest that 2018 is going to be just as good (if not better).
In this post, we will lay out what we can expect over the next 12 months with regards to the Parkinson’s-related clinical trials research of new therapies.
Many readers will be familiar with the name Warren Buffett.
The charming, folksy “Oracle of Omaha” is one of the wealthiest men in the world. And he is well known for his witticisms about investing, business and life in general.
He regularly provides great one liners like:
“We look for three things [in good business leaders]: intelligence, energy, and integrity. If they don’t have the latter, then you should hope they don’t have the first two either. If someone doesn’t have integrity, then you want them to be dumb and lazy”
“Work for an organisation of people you admire, because it will turn you on. I always worry about people who say, ‘I’m going to do this for ten years; and if I really don’t like it very much, then I’ll do something else….’ That’s a little like saving up sex for your old age. Not a very good idea”
“Choosing your heroes is very important. Associate well, marry up and hope you find someone who doesn’t mind marrying down. It was a huge help to me”
Mr Buffett is wise and a very likeable chap.
Few people, however, are familiar with his business partner, Charlie Munger. And Charlie is my favourite of the pair.
A college drop-out who served as a meteorologist in the US Army Air Corps, before going to Harvard to study Law. At age 31, he met Warren at a dinner and convinced him to go into investing. And the rest is history.
When it comes to the one lines, Charlie gets to the point colder and quicker than Warren. And it was Charlie who provided one particular pearl that I hold on to:
The secret to happiness is to lower your expectations.
Easy to say for a billionaire, do I hear you mutter?
Charlie is not for a moment suggesting that one should not be ambitious. Rather he is simply saying aim for the stars, but lower your expectations so that if you only reach the moon you won’t be disappointed. It would still be an amazing achievement.
I have taken Mr Munger’s quote one step further: I have no expectations.
My cup is completely empty. Thus, whenever anything happens, it’s magic! This particular strategy has served me well in scientific research where (if we are honest) few things ever seem to meet expectations.
Now, having said all this: I do have some expectations for 2018
They are based on things that we know are coming. For example:
- Two new countries will emerge in the form of New Caledonia and Bougainville as they both hold independence referendums from France and Papua New Guinea.
- The FIFA football world cup will be held in Russia
- NASA will deploy the James Webb Space Telescope (which will be more powerful than the Hubble telescope)
- The results of the phase II GDNF clinical trial from Bristol will be published (see below).
In this post we will outline the things we can expect from Parkinson’s clinical research in 2018 :
I am going to frame this discussion around a common theme that I had in many of my public talks last year with regards to what a ‘cure for Parkinson’s’ will look like, for example:
That theme was: any ‘cure for Parkinson’s’ is going to require three components:
- A disease halting mechanism
- A neuroprotective agent
- Some form of cell replacement therapy
In this post we will explore each of these components individually and discuss what we should learn in 2018. Let’s look at the first component of any “Cure for Parkinson’s”:
1. A disease halting mechanism
Parkinson’s is a progressive neurodegenerative condition. Thus, the first and most critical component of any ‘cure’ for Parkinson’s involves a treatment that will slow down/halt that progression of the condition.
This can be done either directly or indirectly.
The direct approach
You can think of the direct approach in military terms as a full frontal assault on the enemy. Going straight at the condition and trying to punch a hole through those enemy lines, which gets at the heart of the problem. A direct approach in halting Parkinson’s disease, however, requires a fundamental understanding of how the condition is actually progressing.
And if we are completely honest, we “are not there yet”. We do not know the mechanisms that underly the gradual progress of this condition. But fear not.
As Prof George Church recently pointed out in an excellent interview, “we developed effective smallpox prevention back when we had close to zero understanding of virology and immunology”.
Prof George Church. Source: Biospace
Within the Parkinson’s field, we do have some good theories about how the condition may be spreading in the brain and they are worth testing while new ideas are being investigated.
One of those theories is the idea that a toxic form of the Parkinson’s-associated protein, alpha synuclein, could be being passed from cell to cell, and as it is absorbed by each new healthy cell it starts causing trouble which results in clustering of protein (leading to the appearance of Lewy bodies).
And there is some evidence for this as individuals who received cell transplants back in the 1990s have since passed away and their brains have been examined post-mortem. One very interesting finding from those brains is that some of the cells in the transplants up to 10% in one case – have Lewy bodies in them. This suggests that condition is being passed on from the Parkinson’s affected brain to the healthy transplanted cells in some way. Researchers have proposed that the alpha synuclein protein may be the guilty party in this process.
1.PD Nilotinib, which is being conducted at Georgetown University in Washington DC (Click here for the more details about this study). This study involves 75 participants and will involve two parts:In the first part of the study, one third of the participants receiving a low dose (150mg) of Nilotinib, another third receiving a higher dose (300mg) of Nilotinib and the final third will receive a placebo drug (a drug that has no bioactive effect to act as a control against the other two groups). The outcomes will be assessed clinically at six and 12 months by investigators who are blind to the treatment of each subject. These results will be compared to clinical assessments made at the start of the trial.In the second part of the study, there will be a one-year open-label extension trial, in which all participants will be randomised given either the low dose (150mg) or high dose (300mg) of Nilotinib. This extension is planned to start upon the completion of the first part (the placebo-controlled trial) to evaluate Nilotinib’s long-term effects.
- NILO-PD, which is being conducted at the Feinburg School of Medicine at Northwestern University (Click here for the more details about this study). This study will involve two groups (or cohorts) of participants.The treatment will firstly be given to a first cohort of 75 people everyday for six months, during which the participants will be closely monitored. Following the treatment phase there will be an 8 week follow up period (during which all treatments are stopped), in order to determine Nilotinib’s safety and tolerability. Trial participants will be tested on their motor and cognitive abilities, and they will give sampled (i.e., blood and spinal fluid) for biological testing. All of these tests will provide the investigators with data with which to evaluate the potential impact of Nilotinib on Parkinson’s symptoms and the disease progression.If the drug is shown to be safe in this first cohort, a second trial is planned to be immediate initiated which would test Nilotinib in a second cohort of 60 people with early Parkinson’s disease. These subjects would be given the highest tolerated daily dose of Niltotinib (based on the results of the first study) or a placebo for 12 months. The same measurements (eg. biological samples) would be applied to this study as well.
Click here to read an SoPD post about Nilotinib and these clinical trials. Both of these studies are expected to provide results in 2020.
To review articles that says click on as well as see more information, please go to this:
https://scienceofparkinsons.com/2018/01/07/2018/
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