REVIEW: The GDNF trial
The BBC documentary entitled The Parkinson's Drug Trial: A Miracle Cure?, a two-part series, captures what a group of parkinsed people in the UK endure to be cured of their Parkinson's Disease (PD). The GDNF trial documents the invasive surgery in all its goriness. I have a copy of the complete documentary on my PVR, but have been unable to locate it again, bar online snippets.
In 2013, neurologist and chief investigator in the GDNF trial, Dr Allan Whone, acknowledged that in the field of PD research they had "good symptom improving therapies that work for some of the people some of the time...". Again, this further reinforces the notion of the 'conundrum' in the L-dopa Conundrum article referred to in my previous post. Around 2017, Dr Whone and the team proceeded with the GDNF trial involving 41 volunteers undergoing GDNF infusion.
GDNF, an abbreviation of glial cell line-derived neurotropic factor, is a protein produced in the brain and promotes the growth of brain cells including those lost as a result of PD. The GDNF, discovered in the 1950's and first trialed in the 1990's, would be introduced via tiny, catheter-like mechanisms surgically inserted into the brain and then accessed via an external port drilled into the skull. The monthly infusions of either GDNF or placebo would be via this port into the volunteers' surgical implants that, over a two-day period, would be dispensed to the appropriate area inside the brain.
In 2019 Dr Whone (open link and scroll down for his YouTube lecture) presented the procedure as well as the results of the trial. Unfortunately, volunteers' results (this site also has a link to the Journal article) of the clinical trial had fallen below the required threshold. As this had not complied with what had been scientifically determined, the trial was to be discontinued. Consequently, the pharmaceutics company withdrew its support, much to the immense disappointment of the volunteers. It is interesting to note that the trial result images also showed that GDNF, with varying degrees of significance, appeared to have revived brain cells damaged by PD. Sadly, this form of evidence was insufficient for that trial to have continued.
There were times that I cringed, seeing how parkinsed people were treated during the documentary. GDNF volunteers' levels of anxiety - visibly that of Tom Isaacs - appear to have been raised when in hospital rather than being lowered. Here, I refer to certain triggers for parkinsed people that exacerbate anxiety and accompanying tremors.
Both the movie Awakenings and The Parkinson's Drug Trial: A Miracle Cure? are depressing, real life accounts of experiments and possible cures. In both instances, one can conclude that medical science and pharmaceutics companies invest enormous amounts of time and resources to achieve their goals. This is especially cruel for the GDNF trial because one has to assume that all volunteers, seduced by the possibility of a cure for PD, will now spend the rest of their lives with these implants (I hope I'm mistaken here!).
Also one may deduce, in the case of PD, that science marches on in search of new funding, new collaboration and new journal articles. Sadly, in the centre of the GDNF trial are the brave 41 who pinned their future on hope and faith but will remain parkinsed: flotsam at the edge of the world of PD and science.
Next, I will examine how I started to adapt to my growing affliction. In the words of Peter Quince in Shakespeare's A Midsummer Night's Dream I should say: "Bless thee [Ari], thou art translated...".
GDNF, an abbreviation of glial cell line-derived neurotropic factor, is a protein produced in the brain and promotes the growth of brain cells including those lost as a result of PD. The GDNF, discovered in the 1950's and first trialed in the 1990's, would be introduced via tiny, catheter-like mechanisms surgically inserted into the brain and then accessed via an external port drilled into the skull. The monthly infusions of either GDNF or placebo would be via this port into the volunteers' surgical implants that, over a two-day period, would be dispensed to the appropriate area inside the brain.
In 2019 Dr Whone (open link and scroll down for his YouTube lecture) presented the procedure as well as the results of the trial. Unfortunately, volunteers' results (this site also has a link to the Journal article) of the clinical trial had fallen below the required threshold. As this had not complied with what had been scientifically determined, the trial was to be discontinued. Consequently, the pharmaceutics company withdrew its support, much to the immense disappointment of the volunteers. It is interesting to note that the trial result images also showed that GDNF, with varying degrees of significance, appeared to have revived brain cells damaged by PD. Sadly, this form of evidence was insufficient for that trial to have continued.
Reflection
The reality check for a viewer who has not been parkinsed is the impact of the documentary's in-depth recording of the lives of a small sample of the 41 volunteers. The documentary goes a long way to bridging the gaps between merely reading about PD and actually seeing its debilitations manifest in real life. One bears witness, first-hand, to what the intruder is capable of doing when allowed.
There were times that I cringed, seeing how parkinsed people were treated during the documentary. GDNF volunteers' levels of anxiety - visibly that of Tom Isaacs - appear to have been raised when in hospital rather than being lowered. Here, I refer to certain triggers for parkinsed people that exacerbate anxiety and accompanying tremors.
Both the movie Awakenings and The Parkinson's Drug Trial: A Miracle Cure? are depressing, real life accounts of experiments and possible cures. In both instances, one can conclude that medical science and pharmaceutics companies invest enormous amounts of time and resources to achieve their goals. This is especially cruel for the GDNF trial because one has to assume that all volunteers, seduced by the possibility of a cure for PD, will now spend the rest of their lives with these implants (I hope I'm mistaken here!).
Also one may deduce, in the case of PD, that science marches on in search of new funding, new collaboration and new journal articles. Sadly, in the centre of the GDNF trial are the brave 41 who pinned their future on hope and faith but will remain parkinsed: flotsam at the edge of the world of PD and science.
Next, I will examine how I started to adapt to my growing affliction. In the words of Peter Quince in Shakespeare's A Midsummer Night's Dream I should say: "Bless thee [Ari], thou art translated...".
No comments:
Post a Comment