Today – 27th February, 2019 – the long-awaited results of the Phase II GDNF clinical trial were published.
GDNF (or glial cell line-derived neurotrophic factor) is a protein that our bodies naturally produce to nurture and support cells. Extensive preclinical research suggested that this protein was particularly supportive of dopamine neurons – a group of cells in the brain that are affected by Parkinson’s.
The results of the Phase II clinical trial suggest that the treatment was having an effect in the brain (based on imaging data), but the clinic-based methods of assessment indicated no significant effect between the treatment and placebo groups.
In today’s post we will look at what GDNF is, review the previous research on the protein, discuss the results of the latest study, and look at what happens next.
And be warned this is going to be a long post!
Boulder, Colorado. Source: Rps
It all began way back in 1991.
George H. W. Bush was half way into his presidency, a rock band called Nirvana released their second album (‘Nevermind’), Michael Jordan and the Chicago Bulls rolled over the LA Lakers to win the NBA championship, and Arnold Schwarzenegger’s ‘Terminator 2’ was the top grossing movie of the year.
But in the city of Boulder (Colorado), a discovery was being made that would change Parkinson’s research forever.
In 1991, Dr Leu-Fen Lin and Dr Frank Collins – both research scientists at a small biotech company called Synergen, isolated a protein that they called glial cell-derived neurotrophic factor, or GDNF.
And in 1993, they shared their discovery with the world in this publication:
Title: GDNF: a glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons.
Authors: Lin LF, Doherty DH, Lile JD, Bektesh S, Collins F.
Journal: Science, 1993 May 21;260(5111):1130-2.
For the uninitiated among you, when future historians write the full history of Parkinson’s, there will be no greater saga than GDNF.
In fact, in the full history of medicine, there are few experimental treatments that people get more excited, divided, impassioned and evangelical than GDNF.
This ‘wonder drug’ has been on a rollercoaster ride of a journey.
What exactly is GDNF?
In this post, I will address a question that I get asked a lot: What would you do if you were diagnosed with Parkinson’s today?
Before we start, please understand that there is no secret magical silver bullet to be discussed in the following text. Such a thing does not exist, and anyone offering such should be treated with caution.
Rather, in this post I will spell out some ideas (or a plan of attack) of what I would consider doing if I was confronted with a diagnosis today and how I would approach the situation.
An email I received this week:
Love the website. I think you are amazing and I love your dreamy eyes and perfect hair.
[ok, I may be exaggerating just a little bit here]
Given everything that you have read about Parkinson’s, what would you do if you were diagnosed with Parkinson’s today?
I get this kind of correspondence a lot, and you will hopefully understand that I am very reluctant to give advice on this matter, primarily for two important reasons:
- I am not a clinician. I am a former research scientist who worked on Parkinson’s for 15 years (and now help co-ordinate the research at the Cure Parkinson’s Trust). But I am not in a position to be giving medical/life advice.
- Even if I was a clinician, it would be rather unethical for me to offer any advice over the internet, not being unaware of the personal medical history/circumstances in each case.
While I understand that the question being asked in the email is a very human question to ask – particularly when one is initially faced with the daunting diagnosis of a condition like Parkinson’s – this is not an email that I like to receive.
I am by nature a person who is keen to help others, but in this particular situation I simply can’t.
During the last week of September, the Van Andel institute and the Cure Parkinson’s Trust held their annual Parkinson’s research meetings in Grand Rapids, Michigan.
The meetings – the Linked Clinical Trials meeting, Grand Challenges in Parkinson’s, and Rallying to the Challenge – provided an opportunity for members of the Parkinson’s community (both researchers and advocates) to come together, share research/knowledge/experience, and discuss what needs to be done.
I attended the meetings this year for the first time.
In today’s post, I thought I would provide some feedback and share some of my thoughts on the meetings.
Jay Van Andel (left) and Rich DeVos. Source: Amwayconnections
The history of Amway is an interesting story.
One of ambition, determination, and a refusal to give up.
It begins with the two founders – Jay Van Andel and Rich DeVos – trying and failing to get seven different businesses off the ground before they eventually founded the multi-level marketing company that we know of as Amway.
One aspect of the story that many people do not know, however, is that for a decade before he passed away in 2004, Jay Van Andel lived with Parkinson’s.
In a recent post we discussed the results of the Exenatide clinical trial from last year, and looked at some further analysis of the data, which hinted at the possibility that the drug may be having additional benefits (Click here to read that post).
The researchers behind the Exenatide study have now published the results of a second deep dive into the data and found something potentially very interesting and useful: they may have identified certain characteristics of those participants in the study who responded the best to the drug.
The researchers are quick to point out that this type of post hoc analysis is only conducted for the purpose of generating hypotheses, but it will be interesting to determine if this is finding is validated in further clinical investigations of Exenatide.
In today’s post, we will review the new finding and discuss what they could potentially mean.
Tom Isaacs. Source: GrannyButtons
For most of the Parkinson’s community, Tom Isaacs requires no introduction.
In 1996 – at just 27-years of age – the London-based surveyor was diagnosed with Parkinson’s. After dealing with the initial shock of it all, Tom embraced his situation and became a committed, (utterly) tireless activist. He first walked the entire coastline of the UK to raise money and awareness for Parkinson’s. His book, “Shake well before use“, discusses that trip and adapting to life with Parkinson’s. It is a fantastic read.
And upon returning from his epic walk, he (along with three others) founded and set up the Cure Parkinson’s Trust.
It is pretty safe to say that beyond Michael J Fox and Muhammad Ali, Tom was one of the most impactful members of the Parkinson’s community on marshaling scientific research efforts to find a cure for Parkinson’s.
And he did it all with style and humour:
This was a video of Tom in 2009, talking about life with Parkinson’s:
His passing last year was a terrible loss to the community, and this month, the European Journal of Neuroscience has a special tribute edition dedicated to the memory of Tom.
One of the research reports in that issue involves a study that was very close to Tom’s heart: The Exenatide study.
And the report provides some very interesting new results based on re-analysis of the results of the clinical study.
What does the new report say?